Patti's People

Patti Peeples of the The Peeples Collaborative speaks with leaders in the field of real-world evidence, health economics, health technology assessment, artificial intelligence, patient engagement and health equity.

On this page, you can explore the interview series guest-by-guest, or by focusing on specific topic areas.

Browse by People Browse by Topic

You can also catch up on all episodes from Patti's People on Spotify and other podcast providers, or stream the latest episode below.

If you would like to nominate some to be one of Patti's People, please get in touch here.

---

About Patti 

Dr Patti Peeples is an accomplished Healthcare Consultant and Health Economist who partners with senior management and boards of directors for business expansion, strategic planning and improved operations.  As President of The Peeples Collaborative, Dr Peeples brings experience in for-profit start-ups, scaling, acquisitions, and product line extensions. Previously, she served as Vice President of Evidence and Content at Scientist.com, the world’s largest global sourcing marketplace for scientific research services, while also leading profitable marketing services expansion for the company. Dr Peeples was the Founder and CEO of HealthEconomics.Com, a global digital community of healthcare value, evidence and market access leaders acquired by Scientist.com. Widely published in the peer review literature and a frequent speaker and corporate educator, Dr Peeples serves on multiple journal editorial boards as well as facilitated graduate education through a decade of service on the Board of Grants for the American Foundation for Pharmaceutical Education. Dr Peeples holds a PhD in Health Economics from the University of the Sciences, an MS in Pharmaceutical Marketing, and is a registered pharmacist.

---

Browse by People

---

Browse by Topic

Select a topic of interest below to browsr Patti's People content by topic and question.

Select Topic Artificial Intelligence Health Economics Health Equity Health Policy Health Technology Assessment Inflation Reduction Act Market Access Outcomes Research Patient Engagement Real-World Data/Real-World Evidence Regulation StakeholderEngagement Value Assessment

Artificial Intelligence

• Blythe Adamson: How are you utilizing LLMs to scale real-world data curation?
• Marc Berger: Can AI be used to deduce whether or not a data source is real or potentially fraudulent?
  "We're going to need some breakthroughs in technology, and whether that turns out to be quantum computing or something else, I don't know. But we're going to have to upgrade our hardware in order to be able to actually explore the true power of machine learning and artificial intelligence."
• Marc Berger: How receptive is our medical community to information derived from AI/ML; what hurdles are there, and what do we need to do facilitate?
  "The skepticism is perfectly natural and appropriate because what comes out of AI and machine learning right now is a black box, we know what the predictions are, but we don't know why... as data continues to improve which it is, and as analytic approaches continue to improve, which they are, I think we'll see greater and greater use of this over time."
• Marc Berger: What were some of the key takeaways from ISPOR 2023?
  "Everybody's gaga about AI and ML... The question is, how do we use it intelligently and not get ahead of our skis?"
• Jagpreet Chhatwal: What could we do better in HTA for the US and worldwide?
• Sarah Emond: How is ICER using AI, and assessing its role in value assessment?
• Racheael Fleurence: Put AI into context with respect to transformative potential. Identify the critical areas of opportunity and ones that you’ve moved down the list. Why?
• Racheael Fleurence: Can you identify the more advanced HTAs with respect to AI acceptance and usage, and what do researchers need to do better or differently to align with this intention?
• Racheael Fleurence: Biases in AI are a significant concern. Describe 1 or 2 major concerns and explain how our field is addressing this bias?
• Rich Gliklich: We are not really ‘there’, where patients recognize the benefits of RWE. What would ‘there’ be?
  "The impact of AI... if you can measure outcomes, they'll ultimately be able to predict them for individual patients. That's the Holy Grail."
  
• Rich Gliklich: How is AI changing the industry from research and treatment perspectives?
  "Artificial intelligence is enabling us to extract more from clinical records. Another area that we're really excited about is something we call outcome estimation...we can also reduce the burden on the patient if we're able to use AI and aggregate some of the other measures without forcing them to complete long, long outcome scales."
• Rich Gliklich: Are there any unexpected consequences of using AI in this field?
  "There are a couple of areas that we really do need to watch out for. One is bias... if the data are biased, you're going to see biases in the AI models. And the way around that is to have as broad a data set as possible."
• Peter Neumann: How could AI tools be used for better value assessments?
  "I think the promise is that this is a tool that will help our lives. And as you said, I think, at the end of the day, we're still going to have to interpret this information... we will have to make judgments and interpretations of this information, so that won't go away."
• Eberechukwu Onukwugha: As AI scales, how can we ensure underrepresented regions aren’t left behind – and what’s your approach to bridging that gap?
• Laura Pizzi: Now that the initial hype around AI has settled, what are the 2–3 key areas ISPOR is focusing on to guide the conversation?
• David Thompson: How do you see AI impacting HEOR and RWE now and in the future?
  "There is a movement towards what's being referred to as living HTA, which is the setting up of an HTA submission in such a way that it is updated periodically on a pre-specified basis, such that any new literature coming out can quickly be integrated, whether that's into the SLR or to the meta analysis... it's an area that just begs for AI involvement."
• Jeff Trotter: What are you most excited about in the whole field of healthcare?
  "Technology is going to allow us to do things that weren't dreamed of 10 years ago."
  
• Jeff Trotter (2025): Is observational research declining due to the rise of secondary real-world data and AI, or is it poised for a resurgence thanks to its flexibility?
• Jeremy Rassen: How are you applying your AI background to data analytics in your current work?

---

Health Economics

• Blythe Adamson: The use of real-world data from various countries has sparked concerns about external validity or transportability, leading to a preference for local data. How have you tackled this?
• Blythe Adamson: Why are SDoH and racial/ethnic inequities not a guiding factor in HEOR research?
• Blythe Adamson: What can you tell us about the recent ISPOR SUITABILITY checklist for EHRs for HTA use on data delineation and data fitness for purpose, and what do you think it will accomplish?
• Kirsten Axelsen: What, if anything, should health economists and outcomes researchers be saying about issues like biosimilars potentially disappearing and China’s rise?
• Marc Berger: If you were to give advice to a biopharma manufacturer on how to organize or set up operational centers to maximize RWE data availability, application and impact, what advice would you give to them?
  "The companies that are going to put more investment into knowledge management are going to be the ones who are going to be more likely still be around in 15 years. And the ones who don't are likely going to be forgotten in history."
• Marc Berger: If we have this conversation in 5 years, what do you think we’ll be discussing as the major issues in value, evidence and access for the pharma industry?
  "I am not Nostradamus, but I do know that the current marketing model - the prevalent marketing model that is the engine for the economic model of pharmaceutical companies, which is to come out with blockbuster drugs and charge as much as you can for them to fund every other drug that doesn't make it to the market. I'm not sure that is going to be sustainable."
• Jagpreet Chhatwal: How would you respond to the claim that HEOR analysis has contributed to rising drug prices, reduced affordability, and increased disparities?
• Jagpreet Chhatwal: Why don’t health economists have more impact on drug development, on healthcare access?
• Natalie Douglas and Ragan Hart: What is the big picture investment arena for real-world evidence, HEOR, and pricing/acesss, data tech companies?
  "I think we still have the patient as the last thing that we are thinking about... when it comes to patient-centered outcomes, patient-centered research, patients-centered healthcare, that's something where we can grow...That's something which we can learn from value-based healthcare, how you put patients at the center of the design of your process of care. I think that's something which also in our field from an industry perspective, from a health economics perspective, we have to do more."
• Natalie Douglas and Ragan Hart: What are the funding avenues available to founders and when should they be considering these options?
  "Do your own diligence on know your corporate with regard to what are the strategic priorities of the corporate over the next five years and how does your device or solution fit into that. Or if it doesn't fit into that, totally OK. There's a different set of corporate VC constituents that absolutely could be value add."
• Natalie Douglas and Ragan Hart: How do founders know “it’s time” to seek outside funding, and what research should a founder do before identifying investment channels?
  "How does your opportunity map to the size of the fund and checks that they can write? And then, further to that, how are you receiving value from their point of view? And so you start from - I mean, in the age of information right now, there are innumerable lists out there that folks have organically put together in terms of spreadsheets."
• Natalie Douglas and Ragan Hart: After a founder has identified potential investors, how do you make the first connection so that it sets you up for future successful conversation?
  "We get to see the future every day with how other founders are pitching us. And so, with that said, your version of the future is going to be different than how we understand it. And we need you to set the context in a simple, consumable manner from a narrative point of view versus immediately jumping into the nuance and differentiation of what you're building."
• Natalie Douglas and Ragan Hart: What makes a good pitch, and who should be involved in developing the pitch? What are common features of a failed pitch or blind spots?
  "You have to think about the audience that you're talking to and what their level of understanding might be... do your research. What do they know about this space? What are the models are similar? You're going to need to pitch your deck to the audience that you're speaking to, bearing in mind you've done your research. And hopefully, you've got a short list of the best possible options. But I think avoiding jargon might-- is the most obvious thing, and, also, not being the only person speaking."
• Natalie Douglas and Ragan Hart: You both have led and served on Boards. When is the time to form a Board, does the make-up of the Board differ, and what are some of the challenges once you have a Board?
• Natalie Douglas and Ragan Hart: What should a founder do with a “NO”? How can they turn that “NO“ into a better next pitch?
• Natalie Douglas and Ragan Hart: Let’s put the shoe on the other foot. From an investor standpoint, what pain points are you dealing with that you wish founders would consider so that the two parties can work in the most mutually agreeable and successful way?
• Natalie Douglas and Ragan Hart: What does due diligence look like, and what should founders be prepared for? Does this differ by geographic location and if so, how?
• Natalie Douglas and Ragan Hart: Founding a company, expanding reach, scaling for growth, are necessities that many founders/CEOs have little experience in, and often the investors are negotiating sticky situations. Let’s talk about some of these challenges.
• Rich Gliklich: If there was an area of data and its applications that has the most room for improvement, what would that be?
  "A lot of work on the real-world data and evidence side has been on the field of oncology...But if you look at where the greatest costs are in the US health care system...it's in chronic diseases...so chronic disease is an area that I think is underrepresented currently in uses of real-world data."
• Jens Grueger: What changes do you see occurring as a result of how manufacturers are pursuing HTA and price approval?
  "Having 27 reviews of the very same evidence is a huge waste. It's duplication and triplication for that, and that's probably one of the reasons why the average time from marketing authorization to actual availability to patients in Europe is 517 days, that's 1 and 1/2 years. That is totally unacceptable. And so what we are now going to see from January 2025... it will be much faster."
• Jens Grueger: You mentioned that many people fear that there will be several base-case scenarios, the so-called PICOs. Please explain this and the implications.
  "The amount of data that has to be submitted for some of the national procedures, it's huge, thousands of pages. Not as much as what we have for regulatory approval, but much more than what companies were able to submit to CMS for their price negotiations. So it will be a significant kind of new process for manufacturers."
• Jens Grueger: Does Medicare have a ‘value framework?’ How will that be actualized in a way that can be used by manufacturers in development? What does CMS need to share/publicly state, will they, and if not, what can we impute?
  "I think that's the billion dollar question at the moment, because at the moment, we can only speculate. We can only say that the majority of data points that were requested are more financial data points in terms of the cost of production and investments that have been made for that... So far, what we have seen is that CMS exactly follows the letters of the law and that's how they selected the drug."
• Mark McClellan: What is that one problem you grapple with that, if you had an unlimited budget, staff and time, you’d really like to solve?
  "If there is one common denominator for all of our work, it's the big gap that persists between what our medical technologies and know how are capable of, and what we actually seem to be delivering in practice."
• Mark McClellan: What would you advise a pharmaceutical company to be working on right now?
  "I'd encourage organizations to think about some of the features of this law that may actually help them out in important ways. For example, the law includes the biggest expansion of Medicare Part D since that program was created back when I was at CMS, in particular, new strong protection against so-called catastrophic drug expenditures."
• Peter Neumann: What are the concerns and controversies around the new QALYs threshold of $100k–$150k, and when should it be used?
  "There's long-standing debates about what the cost-effectiveness threshold should be... should it be higher for treatments for more severe diseases, for example, should it be lower for treatments for mild diseases, should it vary by other dimensions that we might think of, rare diseases versus more common diseases, and so forth?"
• Peter Neumann: What do you think is the perfect use case of QALYs, given all of the controversies?
  "It can be used in decisions around how we might price a new drug that brings real health benefits but is very expensive, and it helps us think about what might be an appropriate price."
• Peter Neumann: What are some of the intended and unintended consequences of the Inflation Reduction Act, and what will you be watching for?
  "We might expect pharmaceutical companies have new incentives in terms of their development programs. They now will have stronger incentives to develop biologics or small-molecule drugs because those biologics will have more time in the US market before they're subject to negotiations. There are incentives to bring orphan drugs to the market, as long as they only have a single indication."
• Peter Neumann: What do you anticipate will be the patient impact if pharma starts to front-load the development of orphan drugs on areas with the larger populations?
  "There are always trade-offs with new legislation. The trade-off here is we'll forego some innovation, and in return we're going to get lower drug prices, we're going to get some savings to the federal government, and we're going to lower the out-of-pocket spending for Medicare beneficiaries."
• Peter Neumann: What will be the key differences between ‘IRA negotiation’ vs ‘traditional HTA negotiation’ regarding pricing?
  "The big one is traditional HTA is typically done at the product's launch... In the Medicare negotiations, those will take place many years after the product is launched. So we will have a lot more information about these products, particularly from real-world data. And I think that's going to be a key difference."
• Peter Neumann: Regarding your book, ‘The Right Price: A Value-Based Prescription for Drug Cost’. What surprised you as you were pulling this book together?
  "Most people have insurance for their drugs. So we need to think hard about the step of what goes into the benefit package, and not simply a patient who has a disease and what the value is of the drug to them, but the patient who has insurance but doesn't have the disease, but might have the disease one day and the value of the drug to them, because we're all in a way better off when these drugs get developed even if we don't have that disease, but we might one day."
• Peter Neumann: If you were to advise an insurance company, what messages might you feed to them?
  "We should aim in the right direction - try to get value-based prices and try to lower co-pays and cost-sharing drugs that people really need, and healthcare that people really need, not just drugs."
• Peter Neumann: The Value Initiative: what does this group bring to the table, and how will we know if it is contributing what it intends?
  "I think, if we really want to get better value for our health spend in this country, we need to do more. That's the purpose of that initiative."
• Peter Neumann: If you were to advise medical schools and universities, what should they be adding to their curriculum to train students to be better-prepared for the environment they will be practicing in?
  "We have to really think hard about all the things they're already doing, and does that mean displacing something that is also important... Having said that, I think it would be very helpful for medical students to have some exposure to economics and decision science, to think hard about trade-offs in society that we make and we will always be forced to make."
• Peter Neumann: In 5 years, what will be front-and-center?
  "Has the population health improved in five years. And in what areas? And has it been improving equitably across populations? Those are really, at the end of the day, the critical questions."
• Melvin 'Skip' Olson: Transforming pharmaceutical discovery to accelerate drug development has been discussed for decades. What sets your perspective apart, and what makes your proposals unique?
  
• Laura Pizzi: What is ISPOR’s vision for 2030?
• Laura Pizzi: HEOR and Market Access have grown into distinct but connected fields. How is ISPOR working to improve their efficiency and alignment?
• Sam Roberts: Do you feel that there is a Halo effect on NICE decisions, and, if so, do you consider this in your decision-making process?
  "There is some evidence that other HTA bodies and payers do pay attention to NICE... We are here to serve the taxpayers and public of the country that we're in. So we're obsessed about the fact that our methods reflect their preferences. We're obsessed that we do everything in a rigorous and transparent and independent way, that we're not unduly influenced by factors that we shouldn't be."
• Sam Roberts: What keeps you up at night?
  "The thing that keeps me up at night is these high-price medicines for high prevalent conditions where there is a big deal- a great deal of uncertainty in both the service delivery model and the cost."
• Sam Roberts: NICE has a 5-year plan. What is it?
  "Already we are one of the biggest medicines, devices, diagnostics, and digital and guideline creators in the world. So firstly, and most importantly, is let's keep that magic... Keep the magic of today, but add relevance, timeliness, usability, and focus on impact."
• Paul Scuffham: What do you see as the real purpose of health economics today?
• Jason Shafrin: You’ve written on value frameworks and outcomes for reimbursement. If you could redesign HTA from scratch, what would you toss and what would you keep?
• David Thompson: You’ve written an interesting blog on LinkedIn summarizing the RWE Market and its segmentation, which garnered a lot of feedback. What was your reason for writing this, and what feedback did you get? Why does this matter, and to whom?
  "I came up with eight different segments... And the point of the exercise was to say, OK, you want to be big in the RWE market, how are you going to go about doing that?"
• David Thompson: How do you see AI impacting HEOR and RWE now and in the future?
  "There is a movement towards what's being referred to as living HTA, which is the setting up of an HTA submission in such a way that it is updated periodically on a pre-specified basis, such that any new literature coming out can quickly be integrated, whether that's into the SLR or to the meta analysis... it's an area that just begs for AI involvement."
• David Thompson: Can you tell us about some of the critical issues in which you are engaging, and what are you hoping to achieve?
  "There's expectation that ISPOR is going to be a major player on the global stage as relates to some of the most important aspects of health promotion, not just in the value realm but to use the tools and techniques of HEOR to contribute elsewhere. So it's going to be something fun to watch."
• Kimberly Westrich: How does ICER’s framework inform payer decision-making, which aspects are most important to payers, and how has ICER evolved the framework?
  "Coverage and reimbursement, price negotiations, what we consistently heard is, if there is a value-based price available from ICER, it's going to be used in price negotiations."
• Melanie Whittington: How does traditional cost-effectiveness modeling differ from risk-adjusted NPV models, and do you see a hybrid approach emerging? Who might lead that shift?
  
• Melanie Whittington: What advice would you offer to women starting out in health economics today?

---

Health Equity

• Jagpreet Chhatwal: How would you respond to the claim that HEOR analysis has contributed to rising drug prices, reduced affordability, and increased disparities?
• Nancy A. Dreyer: There is a lot of discussion around diversity and inclusion in clinical trials, secondary data sources, etc. How much of a concern is this, from a drug approval and post-approval RWE standpoint?
  "It's an important issue. We know that there are ethnic differences in responses to treatments. We know that even within ethnicities, there are differential responses. And we don't exactly know why... you need to understand the natural history of the disease, the unmet needs. And that's where you start with real-world evidence."
• Rich Gliklich: Are there any unexpected consequences of using AI in this field?
  "There are a couple of areas that we really do need to watch out for. One is bias... if the data are biased, you're going to see biases in the AI models. And the way around that is to have as broad a data set as possible."
• Jens Grueger: Where does health equity fit into HTA decision-making?
  "Health equity is something that starts in schools, that starts how we educate our children, how we feed our children, how we provide values to our children and go broadly for that. So that is not within the scope of what the pharma company can do, but the pharma company can certainly help in particular diagnostic companies. I think we have a large opportunity at the early stages to understand the differential risk between different populations and make sure that we are consistent."
• Jagpreet Chhatwal: Why don’t health economists have more impact on drug development, on healthcare access?
• Chris Hendriksz: What are the unique issues around rare diseases in Africa that sets it apart from other continents or populations? How are you tackling these?
• Chris Hendriksz: What ethical challenges do you face in rare diseases, and how are these issues unique to Africa yet relevant globally?
• Mark McClellan: What is that one problem you grapple with that, if you had an unlimited budget, staff and time, you’d really like to solve?
  "If there is one common denominator for all of our work, it's the big gap that persists between what our medical technologies and know how are capable of, and what we actually seem to be delivering in practice."
• Mark McClellan: How important is the patient perspective in this process?
  "I would feel better if we were further along than we are in routinely capturing and studying those patient perspectives. And I'd also like to see... your primary care, provider group, and other physicians and health systems are paid at least in part based on getting a better patient experience and getting better patient outcomes."
• Peter Neumann: Has FDA expedited review worked to the benefit of American society, and what risk have we absorbed in exchange for access to more health gains?
  "We want more innovation and we're willing to take some risks. The idea that the QALY gains seem to be higher, I think, is a good signal that maybe we're doing this in a way that makes sense."
• Peter Neumann: In 5 years, what will be front-and-center?
  "Has the population health improved in five years. And in what areas? And has it been improving equitably across populations? Those are really, at the end of the day, the critical questions."
• Eberechukwu Onukwugha: As AI scales, how can we ensure underrepresented regions aren’t left behind – and what’s your approach to bridging that gap?
• Sam Roberts: Tell us how NICE has evolved its methods and processes
  "So in addition to the usual consultation, when we change methods that are reflecting what society values, we do quite a deep piece of listening deliberative work with the society we serve, which is in England, to understand whether they think the decisions that we are making or the weight that we're giving to certain things is reflective of what they value."
• Sam Roberts: What are we learning more and better from patients today, compared with 10 years ago?
  "I think all of us involved in health economics and outcomes research need to focus much more on pithy summaries, plain English language description of concepts, et cetera... patients tell us a lot when they speak to us about a specific medicine. But what they've told us is something we can use again and again and again. For example, we could use it in a guideline. We could use it in selecting a topic for a digital therapeutic. So they've said to us, we want you to use our advice, and multiple times. Don't just use it on one medicine."
• Sam Roberts: If we speak again in 2033, what are we going to be talking about?
  "The problem we should have solved is around multimorbidity, and the problem we're going to be talking about is health inequalities."
• Jeff Trotter: What have you learned that patients can offer us?
  "Patients are just fundamentally important... I think the power of patient advocacy has grown over the last decade such that the patient voice really needs to be heard and is valued... I think it's absolutely essential that the patient perspective be part of labeling."
  
• Melanie Whittington: As health equity gains attention in economic evaluation, how is HEOR adapting – and where is it still falling short?

---

Health Policy

• Blythe Adamson: How is Flatiron engaging with CMS and other stakeholders on the Inflation Reduction Act?
• Kirsten Axelsen: You’ve said predicting this administration’s moves is impossible. What tools help you turn that chaos into strategy?
• Marc Berger: What are the major issues our industry is grappling with today that will change how we conduct research and utilize RWE?
  "The regulators are evolving and how they're using real-world evidence, and their stance about how it can inform labeling is changing."
• Marc Berger: What specific clarifications would be helpful from the US FDA so manufacturers and researchers can have clear guideposts?
  "I applaud the FDA because they want to inspire and promote innovation, particularly for diseases that are terrible and don't have good treatments. On the other hand, we also want to be mindful about how we do that, and make sure we're getting the information as rapidly as possible to assure us that the benefit-risk profiles of these drugs are what we think they are."
  
• Marc Berger: If you were to advise the FDA, what one or two things would you recommend they ‘plant a flag in the sand’ for now?
  "The FDA is a cautious agency and appropriately so... But I think that they could be much clearer on the elements that they are considering and/or the things that they are requiring."
• Sarah Emond: With CMS now negotiating drug prices under the IRA, how is ICER positioning itself?
• Ramiro Gilardino: How do MFN and reference pricing work, and how do they differ in purpose and impact?
• Jens Grueger: What are the cross-border implications of the EU HTA legislation with the US IRA?
  "On an asset by asset basis, the business cases for the investment into the late stage development, they will look different, and they will kind of look different by indication, they will look different by population that I'm targeting, whether it's a younger population or an older population I'm targeting and whether it's small or large molecule."
• Jens Grueger: Are there any unintended consequences that you predict as a result of this legislation, from an overall launch perspective, pursuit of multiple indications, and for how portfolios are managed?
  "The majority of the products that have been selected based on the gross Medicare expenditures are primary care products... where I think there will be some disappointment on behalf of CMS, and the legislator that the expected savings will not be realized. We'll realize that these products are already very significantly discounted and there are limits for the maximum fair price, depending on how long the products will be on the market."
• Jens Grueger: Does Medicare have a ‘value framework?’ How will that be actualized in a way that can be used by manufacturers in development? What does CMS need to share/publicly state, will they, and if not, what can we impute?
  "I think that's the billion dollar question at the moment, because at the moment, we can only speculate. We can only say that the majority of data points that were requested are more financial data points in terms of the cost of production and investments that have been made for that... So far, what we have seen is that CMS exactly follows the letters of the law and that's how they selected the drug."
• Chris Hendriksz: Let’s talk about rare disease drug pricing. What challenges do you face, and what unique solutions have you proposed to Pharma, NGOs, and policymakers?
• Mark McClellan: Can you tell us a bit about the diversity in skillset and background that you have assembled at Duke-Margolis?
  "My own background from spending a lot of time in government is that these health care reform problems require maybe not all hands on deck, but certainly all fresh perspectives, all stakeholder perspectives possible."
  
• Mark McClellan: Lately, class action suits have been brought against the IRA; what do you see as the impact of these on the roll out of the IRA and its implementation?
  "Well, they're definitely going to be part of the implementation process for, I think, at least a few years to come, maybe longer as these multiple challenges work their way through the courts. And I wouldn't be surprised if there are some areas where there might be differences in views across judges, eventually making it to the Supreme Court."
• Mark McClellan: What are three issues that the CDC needs to get right to regain the trust of the American public?
  "Number one for CDC right now is regaining broad public trust by demonstrating that coming out of COVID, it can do more to address the public health challenges facing the nation." 
• Peter Neumann: You, with colleagues, advocated for a US Institute of HTA. What were you proposing that’s not being addressed by ICER?
  "So part of what's missing is more funding for the non-drug space. I think there are also other activities that a new body, a new HTA organization, ideally a public one, could do - work on methodological development, for example... it would be better to have an independent public HTA body, which could exist and probably should exist alongside ICER. There's no reason for ICER to go away. But this body would think about somewhat different areas of focus - services, procedures, diagnostics, devices, methods, thinking about activities that are not being done by ICER."
• Peter Neumann: Has FDA expedited review worked to the benefit of American society, and what risk have we absorbed in exchange for access to more health gains?
  "We want more innovation and we're willing to take some risks. The idea that the QALY gains seem to be higher, I think, is a good signal that maybe we're doing this in a way that makes sense."
• Peter Neumann: If you were to advise medical schools and universities, what should they be adding to their curriculum to train students to be better-prepared for the environment they will be practicing in?
  "We have to really think hard about all the things they're already doing, and does that mean displacing something that is also important... Having said that, I think it would be very helpful for medical students to have some exposure to economics and decision science, to think hard about trade-offs in society that we make and we will always be forced to make."
• Peter Neumann: In 5 years, what will be front-and-center?
  "Has the population health improved in five years. And in what areas? And has it been improving equitably across populations? Those are really, at the end of the day, the critical questions."
• Laura Pizzi: What is ISPOR’s vision for 2030?
• Emily Reuben: From a policy standpoint, you took on the disease stages of Duchenne’s and made them more granular and introduced a Transitional Stage; do you believe that all progressive rare diseases should receive this kind of review?
• Sam Roberts: Tell us how NICE has evolved its methods and processes
  "So in addition to the usual consultation, when we change methods that are reflecting what society values, we do quite a deep piece of listening deliberative work with the society we serve, which is in England, to understand whether they think the decisions that we are making or the weight that we're giving to certain things is reflective of what they value."
• Sam Roberts: What are we learning more and better from patients today, compared with 10 years ago?
  "I think all of us involved in health economics and outcomes research need to focus much more on pithy summaries, plain English language description of concepts, et cetera... patients tell us a lot when they speak to us about a specific medicine. But what they've told us is something we can use again and again and again. For example, we could use it in a guideline. We could use it in selecting a topic for a digital therapeutic. So they've said to us, we want you to use our advice, and multiple times. Don't just use it on one medicine."
• Sam Roberts: What is your opinion on the US Inflation Reduction Act, and how it may (or may not) affect globally HTA decisions?
  "I think for those of us engaging in HTA internationally, the really interesting question is, how are you going to decide the price?"
• Sam Roberts: Will NICE pay any attention to decisions that are made by CMS?
  "Our decision points are at completely different times. So the NICE decision point is that the point of medicine launch, whereas the CMS decision, my understanding is it's 10 to 12 years after launch."
• Sam Roberts: Do you feel that there is a Halo effect on NICE decisions, and, if so, do you consider this in your decision-making process?
  "There is some evidence that other HTA bodies and payers do pay attention to NICE... We are here to serve the taxpayers and public of the country that we're in. So we're obsessed about the fact that our methods reflect their preferences. We're obsessed that we do everything in a rigorous and transparent and independent way, that we're not unduly influenced by factors that we shouldn't be."
• Sam Roberts: What keeps you up at night?
  "The thing that keeps me up at night is these high-price medicines for high prevalent conditions where there is a big deal- a great deal of uncertainty in both the service delivery model and the cost."
• Sam Roberts: NICE has a 5-year plan. What is it?
  "Already we are one of the biggest medicines, devices, diagnostics, and digital and guideline creators in the world. So firstly, and most importantly, is let's keep that magic... Keep the magic of today, but add relevance, timeliness, usability, and focus on impact."
• Sam Roberts: If we speak again in 2033, what are we going to be talking about?
  "The problem we should have solved is around multimorbidity, and the problem we're going to be talking about is health inequalities."
• Jason Shafrin: What’s one policy or regulation you believe is well-intentioned, but has led to poor health or economic outcomes?
• Jeff Trotter: What is on your wish list to attain more widespread utilization of RWE to change decision-making?
  "I think more and more organizations are comfortable that there is a place for real world evidence and real world data within the regulatory submission process, and that's pretty radical." 
• Jeff Trotter: What have you learned that patients can offer us?
  "Patients are just fundamentally important... I think the power of patient advocacy has grown over the last decade such that the patient voice really needs to be heard and is valued... I think it's absolutely essential that the patient perspective be part of labeling."
• Jeff Trotter (2025): What happens when society starts to reject facts?
• Kimberly Westrich: Why are the available tools for value assessment (e.g., ICER’s) not more impactful?
  "They are impactful, but they're not as impactful as we're typically led to believe... it's only one piece of a much broader set of information that payers continue to consider, and it's rarely available for most of the decisions that payers are thinking about."
• Kimberly Westrich: How does ICER’s framework inform payer decision-making, which aspects are most important to payers, and how has ICER evolved the framework?
  "Coverage and reimbursement, price negotiations, what we consistently heard is, if there is a value-based price available from ICER, it's going to be used in price negotiations."
• Kimberly Westrich: Regarding cell and gene therapies, you just published a new analysis that explores ICER’s adaptations for valuing potential cures. What have you learned through this analysis?
  "Gene therapies are potentially transformative. One administration potentially bringing a lifetime of value cure to patients, diseases that we previously thought were incurable - really, really exciting. But it introduces some real challenges to value assessment. Two of those challenges are timing and uncertainty."
• Kimberly Westrich: How is ICER’s work informing or influencing the CMS approach to Medicare drug price negotiations?
  "What has shifted in the framing for the CMS assessment, and this is also new framing in ICER's updated value assessment framework, is that the equal value life year is now front and center. So while reports have historically included both, and going forward, they'll continue to use both, the evLYG, Equal Value Life Year Gained, is front and center in the CMS report."
• Kimberly Westrich: With CMS moving into evidence assessment under IRA, there is talk of CMS being perceived as a national HTA body. What are your thoughts on that?
  "70% of US payers responding to our survey said, yes, we should have a national value assessment body... Payers were interested, are interested in having a national value assessment body."

---

Health Technology Assessment

• Blythe Adamson: What can you tell us about the recent ISPOR SUITABILITY checklist for EHRs for HTA use on data delineation and data fitness for purpose, and what do you think it will accomplish?
• Jagpreet Chhatwal: What could we do better in HTA for the US and worldwide?
• Sarah Emond: What is the goal of ICER’s HEMA collaboration with NICE and CDA?
• Sarah Emond: With CMS now negotiating drug prices under the IRA, how is ICER positioning itself?
• Racheael Fleurence: Can you identify the more advanced HTAs with respect to AI acceptance and usage, and what do researchers need to do better or differently to align with this intention?
• Jens Grueger: What changes do you see occurring as a result of how manufacturers are pursuing HTA and price approval?
  "Having 27 reviews of the very same evidence is a huge waste. It's duplication and triplication for that, and that's probably one of the reasons why the average time from marketing authorization to actual availability to patients in Europe is 517 days, that's 1 and 1/2 years. That is totally unacceptable. And so what we are now going to see from January 2025... it will be much faster."
• Jens Grueger: You mentioned that many people fear that there will be several base-case scenarios, the so-called PICOs. Please explain this and the implications.
  "The amount of data that has to be submitted for some of the national procedures, it's huge, thousands of pages. Not as much as what we have for regulatory approval, but much more than what companies were able to submit to CMS for their price negotiations. So it will be a significant kind of new process for manufacturers."
• Jens Grueger: What are the cross-border implications of the EU HTA legislation with the US IRA?
  "On an asset by asset basis, the business cases for the investment into the late stage development, they will look different, and they will kind of look different by indication, they will look different by population that I'm targeting, whether it's a younger population or an older population I'm targeting and whether it's small or large molecule."
• Jens Grueger: Where does health equity fit into HTA decision-making?
  "Health equity is something that starts in schools, that starts how we educate our children, how we feed our children, how we provide values to our children and go broadly for that. So that is not within the scope of what the pharma company can do, but the pharma company can certainly help in particular diagnostic companies. I think we have a large opportunity at the early stages to understand the differential risk between different populations and make sure that we are consistent."
• Kristi Martin: If you were designing a US HTA framework today, what would you build differently to make it work in practice?
• Peter Neumann: What will be the key differences between ‘IRA negotiation’ vs ‘traditional HTA negotiation’ regarding pricing?
  "The big one is traditional HTA is typically done at the product's launch... In the Medicare negotiations, those will take place many years after the product is launched. So we will have a lot more information about these products, particularly from real-world data. And I think that's going to be a key difference."
• Peter Neumann: You, with colleagues, advocated for a US Institute of HTA. What were you proposing that’s not being addressed by ICER?
  "So part of what's missing is more funding for the non-drug space. I think there are also other activities that a new body, a new HTA organization, ideally a public one, could do - work on methodological development, for example... it would be better to have an independent public HTA body, which could exist and probably should exist alongside ICER. There's no reason for ICER to go away. But this body would think about somewhat different areas of focus - services, procedures, diagnostics, devices, methods, thinking about activities that are not being done by ICER."
• Laura Pizzi: What is ISPOR’s vision for 2030?
• Emily Reuben: How did your group engage directly with NICE and NHS around Project Hercules?
• Emily Reuben: NICE is opening their process more to patients; have you sat on decision-making committees at the HTA level or what is your organization’s role at this level?
• Sam Roberts: What is your opinion on the US Inflation Reduction Act, and how it may (or may not) affect globally HTA decisions?
  "I think for those of us engaging in HTA internationally, the really interesting question is, how are you going to decide the price?"
• David Thompson: How do you see AI impacting HEOR and RWE now and in the future?
  "There is a movement towards what's being referred to as living HTA, which is the setting up of an HTA submission in such a way that it is updated periodically on a pre-specified basis, such that any new literature coming out can quickly be integrated, whether that's into the SLR or to the meta analysis... it's an area that just begs for AI involvement."
• Walter Toro: Have patient aspects always been of interest in HTAs?
• Walter Toro: What is considered patient involvement in HTA?
• Walter Toro: Are there differences in how patient participation is presented in countries with HTAs? Can they be categorized based on the degree of development of this participation?
• Walter Toro: You mentioned the new guideline for patient involvement in the Joint Clinical Assessment. How will this impact patient involvement?
• Walter Toro: You explained that patient involvement includes both research into patient aspects and patient participation in HTA. What are the expected outcomes from each, and what are the specific differences?
• Walter Toro: It seems there is a need to adapt HTA methodologies to better incorporate patient involvement and address emerging healthcare challenges. What changes in HTA and patient involvement do you foresee in the future?
• Walter Toro: What is necessary to continue improving patient involvement in HTA?
• Kimberly Westrich: With CMS moving into evidence assessment under IRA, there is talk of CMS being perceived as a national HTA body. What are your thoughts on that?
  "70% of US payers responding to our survey said, yes, we should have a national value assessment body... Payers were interested, are interested in having a national value assessment body."

---

Inflation Reduction Act

• Blythe Adamson: How is Flatiron engaging with CMS and other stakeholders on the Inflation Reduction Act?
• Sarah Emond: With CMS now negotiating drug prices under the IRA, how is ICER positioning itself?
• Ramiro Gilardino: How do MFN and reference pricing work, and how do they differ in purpose and impact?
• Jens Grueger: What are the cross-border implications of the EU HTA legislation with the US IRA?
  "On an asset by asset basis, the business cases for the investment into the late stage development, they will look different, and they will kind of look different by indication, they will look different by population that I'm targeting, whether it's a younger population or an older population I'm targeting and whether it's small or large molecule."
• Mark McClellan: What is going to be the impact of the Inflation Reduction Act on real-world evidence?
  "People should view the IRA implementation as a stepwise process. If I was at CMS right now, I would focus on getting the most important basics right. Can we make sure we don't make a big mistake in the law?"
  
• Mark McClellan: What would you advise a pharmaceutical company to be working on right now?
  "I'd encourage organizations to think about some of the features of this law that may actually help them out in important ways. For example, the law includes the biggest expansion of Medicare Part D since that program was created back when I was at CMS, in particular, new strong protection against so-called catastrophic drug expenditures."
• Peter Neumann: What are some of the intended and unintended consequences of the Inflation Reduction Act, and what will you be watching for?
  "We might expect pharmaceutical companies have new incentives in terms of their development programs. They now will have stronger incentives to develop biologics or small-molecule drugs because those biologics will have more time in the US market before they're subject to negotiations. There are incentives to bring orphan drugs to the market, as long as they only have a single indication."
• Peter Neumann: What do you anticipate will be the patient impact if pharma starts to front-load the development of orphan drugs on areas with the larger populations?
  "There are always trade-offs with new legislation. The trade-off here is we'll forego some innovation, and in return we're going to get lower drug prices, we're going to get some savings to the federal government, and we're going to lower the out-of-pocket spending for Medicare beneficiaries."
• Peter Neumann: What will be the key differences between ‘IRA negotiation’ vs ‘traditional HTA negotiation’ regarding pricing?
  "The big one is traditional HTA is typically done at the product's launch... In the Medicare negotiations, those will take place many years after the product is launched. So we will have a lot more information about these products, particularly from real-world data. And I think that's going to be a key difference."
• Sam Roberts: What is your opinion on the US Inflation Reduction Act, and how it may (or may not) affect globally HTA decisions?
  "I think for those of us engaging in HTA internationally, the really interesting question is, how are you going to decide the price?"
• Sam Roberts: Will NICE pay any attention to decisions that are made by CMS?
  "Our decision points are at completely different times. So the NICE decision point is that the point of medicine launch, whereas the CMS decision, my understanding is it's 10 to 12 years after launch."
• Melanie Whittington: With policies like the IRA taking shape, are we striking the right balance between cost control and long-term innovation, or are new incentives needed?

---

Market Access

• Marc Berger: If we have this conversation in 5 years, what do you think we’ll be discussing as the major issues in value, evidence and access for the pharma industry?
  "I am not Nostradamus, but I do know that the current marketing model - the prevalent marketing model that is the engine for the economic model of pharmaceutical companies, which is to come out with blockbuster drugs and charge as much as you can for them to fund every other drug that doesn't make it to the market. I'm not sure that is going to be sustainable."
• Jagpreet Chhatwal: Why don’t health economists have more impact on drug development, on healthcare access?
• Natalie Douglas and Ragan Hart: What is the big picture investment arena for real-world evidence, HEOR, and pricing/acesss, data tech companies?
  "I think we still have the patient as the last thing that we are thinking about... when it comes to patient-centered outcomes, patient-centered research, patients-centered healthcare, that's something where we can grow...That's something which we can learn from value-based healthcare, how you put patients at the center of the design of your process of care. I think that's something which also in our field from an industry perspective, from a health economics perspective, we have to do more."
• Natalie Douglas and Ragan Hart: What are the funding avenues available to founders and when should they be considering these options?
  "Do your own diligence on know your corporate with regard to what are the strategic priorities of the corporate over the next five years and how does your device or solution fit into that. Or if it doesn't fit into that, totally OK. There's a different set of corporate VC constituents that absolutely could be value add."
• Sarah Emond: With CMS now negotiating drug prices under the IRA, how is ICER positioning itself?
• Ramiro Gilardino: How do MFN and reference pricing work, and how do they differ in purpose and impact?
• Jens Grueger: You mentioned that many people fear that there will be several base-case scenarios, the so-called PICOs. Please explain this and the implications.
  "The amount of data that has to be submitted for some of the national procedures, it's huge, thousands of pages. Not as much as what we have for regulatory approval, but much more than what companies were able to submit to CMS for their price negotiations. So it will be a significant kind of new process for manufacturers."
• Jens Grueger: What are the cross-border implications of the EU HTA legislation with the US IRA?
  "On an asset by asset basis, the business cases for the investment into the late stage development, they will look different, and they will kind of look different by indication, they will look different by population that I'm targeting, whether it's a younger population or an older population I'm targeting and whether it's small or large molecule."
• Mark McClellan: What would you advise a pharmaceutical company to be working on right now?
  "I'd encourage organizations to think about some of the features of this law that may actually help them out in important ways. For example, the law includes the biggest expansion of Medicare Part D since that program was created back when I was at CMS, in particular, new strong protection against so-called catastrophic drug expenditures."
• Peter Neumann: If you were to advise an insurance company, what messages might you feed to them?
  "We should aim in the right direction - try to get value-based prices and try to lower co-pays and cost-sharing drugs that people really need, and healthcare that people really need, not just drugs."
• Peter Neumann: The Value Initiative: what does this group bring to the table, and how will we know if it is contributing what it intends?
  "I think, if we really want to get better value for our health spend in this country, we need to do more. That's the purpose of that initiative."
• Laura Pizzi: What is ISPOR’s vision for 2030?
• Laura Pizzi: HEOR and Market Access have grown into distinct but connected fields. How is ISPOR working to improve their efficiency and alignment?
• Emily Reuben: How did your group engage directly with NICE and NHS around Project Hercules?
• Emily Reuben: NICE is opening their process more to patients; have you sat on decision-making committees at the HTA level or what is your organization’s role at this level?
• Sam Roberts: What keeps you up at night?
  "The thing that keeps me up at night is these high-price medicines for high prevalent conditions where there is a big deal- a great deal of uncertainty in both the service delivery model and the cost."
• Walter Toro: Have patient aspects always been of interest in HTAs?
• Walter Toro: What is considered patient involvement in HTA?
• Walter Toro: Are there differences in how patient participation is presented in countries with HTAs? Can they be categorized based on the degree of development of this participation?
• Walter Toro: You mentioned the new guideline for patient involvement in the Joint Clinical Assessment. How will this impact patient involvement?
• Walter Toro: You explained that patient involvement includes both research into patient aspects and patient participation in HTA. What are the expected outcomes from each, and what are the specific differences?
• Walter Toro: It seems there is a need to adapt HTA methodologies to better incorporate patient involvement and address emerging healthcare challenges. What changes in HTA and patient involvement do you foresee in the future?
• Walter Toro: What is necessary to continue improving patient involvement in HTA?

---

Outcomes Research

• Blythe Adamson: Why are SDoH and racial/ethnic inequities not a guiding factor in HEOR research?
• Natalie Douglas and Ragan Hart: What is the big picture investment arena for real-world evidence, HEOR, and pricing/acesss, data tech companies?
  "I think we still have the patient as the last thing that we are thinking about... when it comes to patient-centered outcomes, patient-centered research, patients-centered healthcare, that's something where we can grow...That's something which we can learn from value-based healthcare, how you put patients at the center of the design of your process of care. I think that's something which also in our field from an industry perspective, from a health economics perspective, we have to do more."
• Rich Gliklich: We are not really ‘there’, where patients recognize the benefits of RWE. What would ‘there’ be?
  "The impact of AI... if you can measure outcomes, they'll ultimately be able to predict them for individual patients. That's the Holy Grail."
• Rich Gliklich: How is AI changing the industry from research and treatment perspectives?
  "Artificial intelligence is enabling us to extract more from clinical records. Another area that we're really excited about is something we call outcome estimation...we can also reduce the burden on the patient if we're able to use AI and aggregate some of the other measures without forcing them to complete long, long outcome scales."
• Mark McClellan: What is going to be the impact of the Inflation Reduction Act on real-world evidence?
  "People should view the IRA implementation as a stepwise process. If I was at CMS right now, I would focus on getting the most important basics right. Can we make sure we don't make a big mistake in the law?"
• Laura Pizzi: What is ISPOR’s vision for 2030?
• Laura Pizzi: HEOR and Market Access have grown into distinct but connected fields. How is ISPOR working to improve their efficiency and alignment?
• David Thompson: How do you see AI impacting HEOR and RWE now and in the future?
  "There is a movement towards what's being referred to as living HTA, which is the setting up of an HTA submission in such a way that it is updated periodically on a pre-specified basis, such that any new literature coming out can quickly be integrated, whether that's into the SLR or to the meta analysis... it's an area that just begs for AI involvement."
• David Thompson: Can you tell us about some of the critical issues in which you are engaging, and what are you hoping to achieve?
  "There's expectation that ISPOR is going to be a major player on the global stage as relates to some of the most important aspects of health promotion, not just in the value realm but to use the tools and techniques of HEOR to contribute elsewhere. So it's going to be something fun to watch."
• Jeff Trotter: If you were to list one area that RWE has been used and really improved patient outcomes, what would that be?
  "Being used in the real world with patients that would have been excluded perhaps from clinical trials, it's a vital importance... Clinicians only see what's in their practice, and, frankly, not all that systematically either these days."

---

Patient Engagement

• Nancy A. Dreyer: You had significant experience in COVID-related real-world research while at IQVIA. What learnings came from this?
  "We're starting to appreciate how much we can learn from community reporters without the interface of a clinician, which was so important during the pandemic, when all our healthcare workers were just exhausted from working around the clock. And the question is, how can we not further burden them?"
• Rich Gliklich: We are not really ‘there’, where patients recognize the benefits of RWE. What would ‘there’ be?
  "The impact of AI... if you can measure outcomes, they'll ultimately be able to predict them for individual patients. That's the Holy Grail."
• Chris Hendriksz: What are the unique issues around rare diseases in Africa that sets it apart from other continents or populations? How are you tackling these?
• Mark McClellan: How important is the patient perspective in this process?
  "I would feel better if we were further along than we are in routinely capturing and studying those patient perspectives. And I'd also like to see... your primary care, provider group, and other physicians and health systems are paid at least in part based on getting a better patient experience and getting better patient outcomes."
  
• Mark McClellan: What are three issues that the CDC needs to get right to regain the trust of the American public?
  "Number one for CDC right now is regaining broad public trust by demonstrating that coming out of COVID, it can do more to address the public health challenges facing the nation." 
• Peter Neumann: What would you advise pharma companies to be prepared for with respect to increased demand and opportunity for RWE?
  "Pharmaceutical companies already do a lot of work in the real-world data space. I think they'll need to double down on it and prepare for these negotiations, marshaling all of the real-world evidence, as well as all the trial evidence. I think there will be a place for marshaling other kinds of data that we typically may not see as much of patient-centered data, perhaps some surveys, and the degree to which patients might prefer a product because of its better dosing schedule or tolerability or things like that."
• Eberechukwu Onukwugha: Has a single patient story ever changed your perspective more than large datasets? How should research methods account for that?
• Emily Reuben: Tell us about the genesis of your involvement in Duchenne’s Muscular Dystrophy and how did you move into patient advocacy?
• Emily Reuben: How did your group engage directly with NICE and NHS around Project Hercules?
• Emily Reuben: NICE is opening their process more to patients; have you sat on decision-making committees at the HTA level or what is your organization’s role at this level?
• Emily Reuben: What are we missing about patient centricity and what do you wish we did better?
• Sam Roberts: What are we learning more and better from patients today, compared with 10 years ago?
  "I think all of us involved in health economics and outcomes research need to focus much more on pithy summaries, plain English language description of concepts, et cetera... patients tell us a lot when they speak to us about a specific medicine. But what they've told us is something we can use again and again and again. For example, we could use it in a guideline. We could use it in selecting a topic for a digital therapeutic. So they've said to us, we want you to use our advice, and multiple times. Don't just use it on one medicine."
• Walter Toro: Have patient aspects always been of interest in HTAs?
• Walter Toro: What is considered patient involvement in HTA?
• Walter Toro: Are there differences in how patient participation is presented in countries with HTAs? Can they be categorized based on the degree of development of this participation?
• Walter Toro: You mentioned the new guideline for patient involvement in the Joint Clinical Assessment. How will this impact patient involvement?
• Walter Toro: You explained that patient involvement includes both research into patient aspects and patient participation in HTA. What are the expected outcomes from each, and what are the specific differences?
• Walter Toro: Regarding research, what are the barriers and enablers to patient-based evidence?
• Walter Toro: What methodologies exist for measuring what matters to patients?
• Walter Toro: It seems there is a need to adapt HTA methodologies to better incorporate patient involvement and address emerging healthcare challenges. What changes in HTA and patient involvement do you foresee in the future?
• Walter Toro: What is necessary to continue improving patient involvement in HTA?
• Jeff Trotter: What have you learned that patients can offer us?
  "Patients are just fundamentally important... I think the power of patient advocacy has grown over the last decade such that the patient voice really needs to be heard and is valued... I think it's absolutely essential that the patient perspective be part of labeling."
• Jeff Trotter: If you were to list one area that RWE has been used and really improved patient outcomes, what would that be?
  "Being used in the real world with patients that would have been excluded perhaps from clinical trials, it's a vital importance... Clinicians only see what's in their practice, and, frankly, not all that systematically either these days."

---

Real-World Data/Real-World Evidence

• Blythe Adamson: How is Flatiron engaging with CMS and other stakeholders on the Inflation Reduction Act?
• Blythe Adamson: The use of real-world data from various countries has sparked concerns about external validity or transportability, leading to a preference for local data. How have you tackled this?
• Blythe Adamson: Why are SDoH and racial/ethnic inequities not a guiding factor in HEOR research?
• Marc Berger: What are the major issues our industry is grappling with today that will change how we conduct research and utilize RWE?
  "The regulators are evolving and how they're using real-world evidence, and their stance about how it can inform labeling is changing."
• Marc Berger: To demonstrate trust, one needs to be fully transparent. Where might that information be held, and who would manage it?
  "When I advise people about what they should be thinking about if they're going to submit a real-world evidence to a regulator, I tell them there are three elements they have to pay really close attention to. One is good study hygiene. The second is rigorous study design. And the third is rigorous analytics plan."
  
• Marc Berger: Regarding trying to align RWE methodology with an RCT, what’s the downside? Does it risk the design defining the objective rather than the objective defining the design?
  "That will also be context dependent. It will depend on the question at hand... I don't think we need randomized controlled evidence in every case to convince ourselves that a new therapeutic works. Certainly, that has been the case historically."
• Marc Berger: In the case of tacrolimus for patients receiving lung transplantation, what motivated the FDA to agree that the RWE was sufficient and an RCT not required?
  "Well, it checked all of the boxes... First of all, it was a known data source... They had experience in using it before... It wasn't a sample of patients in the US. It was all of the patients in the US. So there's no question about was it representative of the population that it was deemed necessary to treat... it was unethical to do an RCT... Everything lined up so that the FDA could say yes, we believe that there is substantial evidence that tacrolimus works in lung cancer."
• Marc Berger: What specific clarifications would be helpful from the US FDA so manufacturers and researchers can have clear guideposts?
  "I applaud the FDA because they want to inspire and promote innovation, particularly for diseases that are terrible and don't have good treatments. On the other hand, we also want to be mindful about how we do that, and make sure we're getting the information as rapidly as possible to assure us that the benefit-risk profiles of these drugs are what we think they are."
• Marc Berger: If you were to give advice to a biopharma manufacturer on how to organize or set up operational centers to maximize RWE data availability, application and impact, what advice would you give to them?
  "The companies that are going to put more investment into knowledge management are going to be the ones who are going to be more likely still be around in 15 years. And the ones who don't are likely going to be forgotten in history."
  
• Natalie Douglas and Ragan Hart: What is the big picture investment arena for real-world evidence, HEOR, and pricing/acesss, data tech companies?
  "I think we still have the patient as the last thing that we are thinking about... when it comes to patient-centered outcomes, patient-centered research, patients-centered healthcare, that's something where we can grow...That's something which we can learn from value-based healthcare, how you put patients at the center of the design of your process of care. I think that's something which also in our field from an industry perspective, from a health economics perspective, we have to do more."
• Nancy A. Dreyer: If you were to sum up the field of RWE as it stands today, what are the top issues you are grappling with as a research epidemiologist?
  "The thing that's top of mind for every researcher who wants to use real-world evidence or biopharmaceutical company or medical device manufacturer who wants to submit real-world data for regulator decision-making or for payers, everybody wants to know, how can you trust the quality of the data?"
  
• Nancy A. Dreyer: How do you ascertain quality of data, and how are FDA and other regulatory agencies responding to these issues?
  "There has been a spate of guidance documents all around the world... you have to have a solution that fits your country's needs."
• Nancy A. Dreyer: You had significant experience in COVID-related real-world research while at IQVIA. What learnings came from this?
  "We're starting to appreciate how much we can learn from community reporters without the interface of a clinician, which was so important during the pandemic, when all our healthcare workers were just exhausted from working around the clock. And the question is, how can we not further burden them?"
• Nancy A. Dreyer: There is a lot of discussion around diversity and inclusion in clinical trials, secondary data sources, etc. How much of a concern is this, from a drug approval and post-approval RWE standpoint?
  "It's an important issue. We know that there are ethnic differences in responses to treatments. We know that even within ethnicities, there are differential responses. And we don't exactly know why... you need to understand the natural history of the disease, the unmet needs. And that's where you start with real-world evidence."
• Nancy A. Dreyer: Are there any other additional trends that are affecting RWE in terms of collection analysis and use?
  "I think the next big development is record linkage... I think my concern or my guess is that some of the things that may come to light as we start to go through particularly electronic health records is, the notes may not be as informative as we thought."
• Rich Gliklich: Can you cast a critical eye on the state of RWE, and tell us from your perspective what are we doing well, and what needs to get better?
  "For the researcher real-world evidence has opened the door to a far greater understanding of what works for who, and at what cost in actual practice... For the regulator... real-world evidence is a bit like a wild horse that needs to be tamed."
• Rich Gliklich: If there was an area of data and its applications that has the most room for improvement, what would that be?
  "A lot of work on the real-world data and evidence side has been on the field of oncology...But if you look at where the greatest costs are in the US health care system...it's in chronic diseases...so chronic disease is an area that I think is underrepresented currently in uses of real-world data."
• Rich Gliklich: As there is more intent to integrate evidence across the full drug development continuum, are you seeing more alignment with your client organizations in terms of their real-world development strategies?
  "So to me, using different data sources in a fit for purpose way...As to where different life sciences companies are on the maturation curve for using real-world data across the product life cycle and across departments, if you've seen one pharma company, you've seen one pharma company."
• Mark McClellan: What is going to be the impact of the Inflation Reduction Act on real-world evidence?
  "People should view the IRA implementation as a stepwise process. If I was at CMS right now, I would focus on getting the most important basics right. Can we make sure we don't make a big mistake in the law?"
• Peter Neumann: What will be the key differences between ‘IRA negotiation’ vs ‘traditional HTA negotiation’ regarding pricing?
  "The big one is traditional HTA is typically done at the product's launch... In the Medicare negotiations, those will take place many years after the product is launched. So we will have a lot more information about these products, particularly from real-world data. And I think that's going to be a key difference."
• Peter Neumann: What would you advise pharma companies to be prepared for with respect to increased demand and opportunity for RWE?
  "Pharmaceutical companies already do a lot of work in the real-world data space. I think they'll need to double down on it and prepare for these negotiations, marshaling all of the real-world evidence, as well as all the trial evidence. I think there will be a place for marshaling other kinds of data that we typically may not see as much of patient-centered data, perhaps some surveys, and the degree to which patients might prefer a product because of its better dosing schedule or tolerability or things like that."
• Laura Pizzi: What is ISPOR’s vision for 2030?
• David Thompson: You’ve written an interesting blog on LinkedIn summarizing the RWE Market and its segmentation, which garnered a lot of feedback. What was your reason for writing this, and what feedback did you get? Why does this matter, and to whom?
  "I came up with eight different segments... And the point of the exercise was to say, OK, you want to be big in the RWE market, how are you going to go about doing that?"
• David Thompson: You’ve published a commentary in Value in Health entitled, “Replication of Randomized Controlled Trials Using Real-World Data: What Could Go Wrong?” In it, you call into question the appropriateness of using RCT findings as a standard by which RWE should be judged, especially by the FDA. What is the fundamental issue here? What in fact could go wrong?
  "I don't think it's really a matter of RCT data versus RWE or RWD data. What I think it is is, to what extent do we want regulatory bodies to be comfortable with non-randomized data?"
• David Thompson: You note that, “the train has left the station with RCT replication.” What are the unintended consequences in, say, 5 years if this is the position adopted by FDA and industry?
  "You cannot expect to just put RWE in your package and think that's going to be OK if you haven't gotten the study design pre-approved from your counterparts at FDA or EMA or what have you."
• David Thompson: Another one of your pet projects is a survey of researcher pain points when using RWD sources to generate RWE. Can you tell us more about this?
  "There is tremendous variation from one study to the next in terms of the things - very fundamental things about how researchers will identify a patient population, how they will separate patients into different treatment groups, how they will identify relevant comorbidities and disease severity markers of interest, and then, of course, even the outcomes of care."
• David Thompson: How do you see AI impacting HEOR and RWE now and in the future?
  "There is a movement towards what's being referred to as living HTA, which is the setting up of an HTA submission in such a way that it is updated periodically on a pre-specified basis, such that any new literature coming out can quickly be integrated, whether that's into the SLR or to the meta analysis... it's an area that just begs for AI involvement."
• Jeff Trotter: What are the biggest changes that our industry is grappling with right now?
  "Education is really critical, I think methodologies are improving for how we generate data, how we access data, how we generate evidence... what the ROI of RWE is, is something that I think we need."
  
• Jeff Trotter: How are biopharma organizations changing operationally to better deploy RWE?
  "To be able to really optimize real-world evidence from the perspective of a pharma company or a biotech company or even a device company, we've really got to bridge important gaps... between the traditional land masses of clinical development and commercialization."
• Jeff Trotter: If you were to advise a company on building an RWE Center of Excellence, what skillsets should be in there?
  "I think it's got to start with an individual that is very comfortable on that full continuum, understanding the clinical trials, understanding the traditional way of gathering evidence for regulatory submissions and also very comfortable and fluid in how data can be used and how evidence can be generated for different stakeholders."
  
• Jeff Trotter: What is on your wish list to attain more widespread utilization of RWE to change decision-making?
  "I think more and more organizations are comfortable that there is a place for real world evidence and real world data within the regulatory submission process, and that's pretty radical." 
  
• Jeff Trotter: Tell me about your experiences in interacting with clients and FDA in early discussions to support approvals.
  "Early discussions, informed discussions, making sure the people that are doing the discussing really understand the nuances... it goes back to education and ensuring that we're saying the right things... I think terminology and appropriate lexicon really helps ensure that the parties on both sides understand that you get it."
• Jeff Trotter: What do you wish that you had known about RWE that you know now?
  "I wish I had taken more psychology courses... we're challenging people's comfort zones... [they] now have to figure out what's all this real-world evidence stuff... This is a new world and we've really got to ensure that we're being as gentle and as adaptable as possible."
  
• Jeff Trotter: If you were to list one area that RWE has been used and really improved patient outcomes, what would that be?
  "Being used in the real world with patients that would have been excluded perhaps from clinical trials, it's a vital importance... Clinicians only see what's in their practice, and, frankly, not all that systematically either these days."
  
• Jeff Trotter (2025): Since our last conversation, what shifts in the RWE landscape have caught your attention? 
• Jeff Trotter (2025): The ROI of RWE is getting clearer, but are we asking the right questions about value?
  
• Jeff Trotter (2025): What’s one RWE project that exceeded expectations?
• Jeff Trotter (2025): Is observational research declining due to the rise of secondary real-world data and AI, or is it poised for a resurgence thanks to its flexibility?
• Jeremy Rassen: Do you think we've overreached in using RWD, and, if so, in what areas?
  

---

Regulation

• Marc Berger: What are the major issues our industry is grappling with today that will change how we conduct research and utilize RWE?
  "The regulators are evolving and how they're using real-world evidence, and their stance about how it can inform labeling is changing."
• Marc Berger: Regarding trying to align RWE methodology with an RCT, what’s the downside? Does it risk the design defining the objective rather than the objective defining the design?
  "That will also be context dependent. It will depend on the question at hand... I don't think we need randomized controlled evidence in every case to convince ourselves that a new therapeutic works. Certainly, that has been the case historically."
• Marc Berger: In the case of tacrolimus for patients receiving lung transplantation, what motivated the FDA to agree that the RWE was sufficient and an RCT not required?
  "Well, it checked all of the boxes... First of all, it was a known data source... They had experience in using it before... It wasn't a sample of patients in the US. It was all of the patients in the US. So there's no question about was it representative of the population that it was deemed necessary to treat... it was unethical to do an RCT... Everything lined up so that the FDA could say yes, we believe that there is substantial evidence that tacrolimus works in lung cancer."
• Marc Berger: What specific clarifications would be helpful from the US FDA so manufacturers and researchers can have clear guideposts?
  "I applaud the FDA because they want to inspire and promote innovation, particularly for diseases that are terrible and don't have good treatments. On the other hand, we also want to be mindful about how we do that, and make sure we're getting the information as rapidly as possible to assure us that the benefit-risk profiles of these drugs are what we think they are."
• Marc Berger: If you were to advise the FDA, what one or two things would you recommend they ‘plant a flag in the sand’ for now?
  "The FDA is a cautious agency and appropriately so... But I think that they could be much clearer on the elements that they are considering and/or the things that they are requiring."
• Nancy A. Dreyer: How do you ascertain quality of data, and how are FDA and other regulatory agencies responding to these issues?
  "There has been a spate of guidance documents all around the world... you have to have a solution that fits your country's needs."
• Rich Gliklich: Can you cast a critical eye on the state of RWE, and tell us from your perspective what are we doing well, and what needs to get better?
  "For the researcher real-world evidence has opened the door to a far greater understanding of what works for who, and at what cost in actual practice... For the regulator... real-world evidence is a bit like a wild horse that needs to be tamed."
• Rich Gliklich: How do you assess data for fit-for-purpose, and what is the future of data aggregation utility?
  "I think fit-for-purpose has to be, has the data been acquired in a way that yields a high-quality dataset for the particular question? Does it have sufficient and representative population sample? Can you understand and mitigate the biases? Is it the least costly approach to address the question most completely as the FDA would ask? ...in the end, if you're collecting clinical data, it's all about the outcomes, and that's what we want to focus on."
• Jens Grueger: What changes do you see occurring as a result of how manufacturers are pursuing HTA and price approval?
  "Having 27 reviews of the very same evidence is a huge waste. It's duplication and triplication for that, and that's probably one of the reasons why the average time from marketing authorization to actual availability to patients in Europe is 517 days, that's 1 and 1/2 years. That is totally unacceptable. And so what we are now going to see from January 2025... it will be much faster."
• Mark McClellan: What would you advise a pharmaceutical company to be working on right now?
  "I'd encourage organizations to think about some of the features of this law that may actually help them out in important ways. For example, the law includes the biggest expansion of Medicare Part D since that program was created back when I was at CMS, in particular, new strong protection against so-called catastrophic drug expenditures."
• Mark McClellan: Lately, class action suits have been brought against the IRA; what do you see as the impact of these on the roll out of the IRA and its implementation?
  "Well, they're definitely going to be part of the implementation process for, I think, at least a few years to come, maybe longer as these multiple challenges work their way through the courts. And I wouldn't be surprised if there are some areas where there might be differences in views across judges, eventually making it to the Supreme Court."
  
• Peter Neumann: Has FDA expedited review worked to the benefit of American society, and what risk have we absorbed in exchange for access to more health gains?
  "We want more innovation and we're willing to take some risks. The idea that the QALY gains seem to be higher, I think, is a good signal that maybe we're doing this in a way that makes sense."
• David Thompson: You’ve published a commentary in Value in Health entitled, “Replication of Randomized Controlled Trials Using Real-World Data: What Could Go Wrong?” In it, you call into question the appropriateness of using RCT findings as a standard by which RWE should be judged, especially by the FDA. What is the fundamental issue here? What in fact could go wrong?
  "I don't think it's really a matter of RCT data versus RWE or RWD data. What I think it is is, to what extent do we want regulatory bodies to be comfortable with non-randomized data?"
• David Thompson: You note that, “the train has left the station with RCT replication.” What are the unintended consequences in, say, 5 years if this is the position adopted by FDA and industry?
  "You cannot expect to just put RWE in your package and think that's going to be OK if you haven't gotten the study design pre-approved from your counterparts at FDA or EMA or what have you."
• David Thompson: Another one of your pet projects is a survey of researcher pain points when using RWD sources to generate RWE. Can you tell us more about this?
  "There is tremendous variation from one study to the next in terms of the things - very fundamental things about how researchers will identify a patient population, how they will separate patients into different treatment groups, how they will identify relevant comorbidities and disease severity markers of interest, and then, of course, even the outcomes of care."

---

Stakeholder Engagement

• Blythe Adamson: How is Flatiron engaging with CMS and other stakeholders on the Inflation Reduction Act?
• Marc Berger: As a peer reviewer, how will it ever be possible to access all of the information needed to deduce whether the data source, statistical/analytics approaches etc are appropriate?
  "We're going to have to find a way to make it manageable that every time you review a study, you don't have to go back to square one to ask the question, whether is this a legitimate data set? Has it been curated in an appropriate way? What are the quality metrics that let me in general how much missingness, or how much out of range day there was, and how much continuity of the data there is? We're going to have to find a way to shortcut that, and that solution will have to come out of discussions in the public sphere."
• Mark McClellan: Can you tell us a bit about the diversity in skillset and background that you have assembled at Duke-Margolis?
  "My own background from spending a lot of time in government is that these health care reform problems require maybe not all hands on deck, but certainly all fresh perspectives, all stakeholder perspectives possible."
• Laura Pizzi: What is ISPOR’s vision for 2030?
• Jeff Trotter: How are biopharma organizations changing operationally to better deploy RWE?
  "To be able to really optimize real-world evidence from the perspective of a pharma company or a biotech company or even a device company, we've really got to bridge important gaps... between the traditional land masses of clinical development and commercialization."
• Jeff Trotter: If you were to advise a company on building an RWE Center of Excellence, what skillsets should be in there?
  "I think it's got to start with an individual that is very comfortable on that full continuum, understanding the clinical trials, understanding the traditional way of gathering evidence for regulatory submissions and also very comfortable and fluid in how data can be used and how evidence can be generated for different stakeholders."
• Jeff Trotter: Tell me about your experiences in interacting with clients and FDA in early discussions to support approvals.
  "Early discussions, informed discussions, making sure the people that are doing the discussing really understand the nuances... it goes back to education and ensuring that we're saying the right things... I think terminology and appropriate lexicon really helps ensure that the parties on both sides understand that you get it."
  
• Jeff Trotter (2025): What happens when society starts to reject facts?
• Kimberly Westrich: What other stakeholder groups are not being considered?
  "The voices that we don't see, but I do believe they are having an opportunity to comment, are our payers... we're not seeing that transparency and visibility around what payers want from ICER's framework in the public sphere."

---

Value Assessment

• Blythe Adamson: How is Flatiron engaging with CMS and other stakeholders on the Inflation Reduction Act?
• Marc Berger: If we have this conversation in 5 years, what do you think we’ll be discussing as the major issues in value, evidence and access for the pharma industry?
  "I am not Nostradamus, but I do know that the current marketing model - the prevalent marketing model that is the engine for the economic model of pharmaceutical companies, which is to come out with blockbuster drugs and charge as much as you can for them to fund every other drug that doesn't make it to the market. I'm not sure that is going to be sustainable."
• Jagpreet Chhatwal: How would you respond to the claim that HEOR analysis has contributed to rising drug prices, reduced affordability, and increased disparities?
• Sarah Emond: ICER has been a disruptor in the value assessment space. Where do you see it heading over the next five years?
• Sarah Emond: What is the goal of ICER’s HEMA collaboration with NICE and CDA?
• Sarah Emond: What do you see as ICER’s biggest strategic blind spot, and how are you addressing it?
• Sarah Emond: With CMS now negotiating drug prices under the IRA, how is ICER positioning itself?
• Sarah Emond: How is ICER using AI, and assessing its role in value assessment?
• Ramiro Gilardino: How do MFN and reference pricing work, and how do they differ in purpose and impact?
• Jens Grueger: Does Medicare have a ‘value framework?’ How will that be actualized in a way that can be used by manufacturers in development? What does CMS need to share/publicly state, will they, and if not, what can we impute?
  "I think that's the billion dollar question at the moment, because at the moment, we can only speculate. We can only say that the majority of data points that were requested are more financial data points in terms of the cost of production and investments that have been made for that... So far, what we have seen is that CMS exactly follows the letters of the law and that's how they selected the drug."
• Jens Grueger: You’ve written on IEPs. What are the biggest barriers to implementation, and advancing this idea from the “theoretical’ to a standard practice?
  "The starting point is that we have to recognize what is a medicine that we bring to market. The medicine comes in the form of a tablet or an infusion, but in the end, it's a piece of knowledge that we're making available, knowledge of how to treat a certain disease"
• Chris Hendriksz: Let’s talk about rare disease drug pricing. What challenges do you face, and what unique solutions have you proposed to Pharma, NGOs, and policymakers?
• Peter Neumann: What are the concerns and controversies around the new QALYs threshold of $100k–$150k, and when should it be used?
  "There's long-standing debates about what the cost-effectiveness threshold should be... should it be higher for treatments for more severe diseases, for example, should it be lower for treatments for mild diseases, should it vary by other dimensions that we might think of, rare diseases versus more common diseases, and so forth?"
• Peter Neumann: What do you think is the perfect use case of QALYs, given all of the controversies?
  "It can be used in decisions around how we might price a new drug that brings real health benefits but is very expensive, and it helps us think about what might be an appropriate price."
• Peter Neumann: Regarding your book, ‘The Right Price: A Value-Based Prescription for Drug Cost’. What surprised you as you were pulling this book together?
  "Most people have insurance for their drugs. So we need to think hard about the step of what goes into the benefit package, and not simply a patient who has a disease and what the value is of the drug to them, but the patient who has insurance but doesn't have the disease, but might have the disease one day and the value of the drug to them, because we're all in a way better off when these drugs get developed even if we don't have that disease, but we might one day."
• Peter Neumann: If you were to advise an insurance company, what messages might you feed to them?
  "We should aim in the right direction - try to get value-based prices and try to lower co-pays and cost-sharing drugs that people really need, and healthcare that people really need, not just drugs."
• Peter Neumann: The Value Initiative: what does this group bring to the table, and how will we know if it is contributing what it intends?
  "I think, if we really want to get better value for our health spend in this country, we need to do more. That's the purpose of that initiative."
• Peter Neumann: How could AI tools be used for better value assessments?
  "I think the promise is that this is a tool that will help our lives. And as you said, I think, at the end of the day, we're still going to have to interpret this information... we will have to make judgments and interpretations of this information, so that won't go away."
• Melvin 'Skip' Olson: Is what you're talking about a new way of value assessment, or is it implementation science?
• Laura Pizzi: What is ISPOR’s vision for 2030?
• Paul Scuffham: Amid geopolitical and economic pressures, how do you see global pricing and value frameworks evolving – and what might this mean for patient access?
• Jason Shafrin: You’ve written on value frameworks and outcomes for reimbursement. If you could redesign HTA from scratch, what would you toss and what would you keep?
• Kimberly Westrich: Why are the available tools for value assessment (e.g., ICER’s) not more impactful?
  "They are impactful, but they're not as impactful as we're typically led to believe... it's only one piece of a much broader set of information that payers continue to consider, and it's rarely available for most of the decisions that payers are thinking about."
• Kimberly Westrich: How does ICER’s framework inform payer decision-making, which aspects are most important to payers, and how has ICER evolved the framework?
  "Coverage and reimbursement, price negotiations, what we consistently heard is, if there is a value-based price available from ICER, it's going to be used in price negotiations."
• Kimberly Westrich: Regarding cell and gene therapies, you just published a new analysis that explores ICER’s adaptations for valuing potential cures. What have you learned through this analysis?
  "Gene therapies are potentially transformative. One administration potentially bringing a lifetime of value cure to patients, diseases that we previously thought were incurable - really, really exciting. But it introduces some real challenges to value assessment. Two of those challenges are timing and uncertainty."
• Kimberly Westrich: How is ICER’s work informing or influencing the CMS approach to Medicare drug price negotiations?
  "What has shifted in the framing for the CMS assessment, and this is also new framing in ICER's updated value assessment framework, is that the equal value life year is now front and center. So while reports have historically included both, and going forward, they'll continue to use both, the evLYG, Equal Value Life Year Gained, is front and center in the CMS report."
• Kimberly Westrich: With CMS moving into evidence assessment under IRA, there is talk of CMS being perceived as a national HTA body. What are your thoughts on that?
  "70% of US payers responding to our survey said, yes, we should have a national value assessment body... Payers were interested, are interested in having a national value assessment body."
• Melanie Whittington: How does traditional cost-effectiveness modeling differ from risk-adjusted NPV models, and do you see a hybrid approach emerging? Who might lead that shift?
• Melanie Whittington: If you could redesign the value framework, what elements do you think are missing – and why?