Overcoming barriers to cell and gene therapy adoption with real-world evidence
This Deep Dive examines insights from a recent ISPOR webinar hosted by the PPD Evidera Solutions team at Thermo Fisher Scientific, exploring how real-world data and evidence can address the evidentiary, operational, access and uptake challenges shaping the future of cell and gene therapies.
Cell and gene therapies (CGTs) have evolved from a pioneering scientific concept into a clinical reality, reshaping expectations for what modern medicine can achieve. Once considered experimental, they now offer considerable hope of curing or transforming outcomes for patients with rare and previously untreatable diseases. Yet their journey from development to widespread adoption is far from straightforward, requiring new approaches to evidence generation, regulatory assessment, and reimbursement to ensure these advances translate into real-world patient benefit.
That was the message at a recent ISPOR webinar hosted by the Evidera Solutions team of Thermo Fisher Scientific titled “Unlocking success: Overcoming cell & gene therapy challenges with real-world evidence.” Moderated by Karen Krewson, panelists included Martin Parkinson, Amanda Pulfer, Heather Gelhorn, and Pedro Saramago Gonçalves from the PPD Evidera Solutions team of Thermo Fisher, joined by guest speaker Richard Diaz, Associate Director of Medicines Evaluation (Rare Diseases) at the National Institute for Health and Care Excellence (NICE).
They explored how real-world data (RWD) and real-world evidence (RWE) can be integrated within a comprehensive and holistic regulatory and access strategy to help address the practical hurdles that threaten to inhibit CGT adoption. The discussion unfolded around three core themes: the opportunities that CGTs offer, the challenges in value demonstration, and how RWE can bridge the gaps between innovation, evidence generation, and patient access.
In this Deep Dive, we bring together the panel’s perspectives to highlight where opportunities lie, where uncertainty persists and how RWE can help bridge the gap.
About the speakers
Pedro Saramago Goncalves, MSc, PhD
Research Scientist, Consulting, PPD Evidera Health Economics and Market Access, Thermo Fisher Scientific
Pedro has over 18 years of experience in the economic evaluation of health and care interventions, including drugs, devices, procedures, diagnostic tests and long-term care. Pedro's interests are related to the development and application of statistical, econometric and decision analysis methods in all components of HTA, including statistical analysis of primary data, evidence synthesis, decision modeling methods and value of further research analysis. Pedro is a member of the NICE Technology Appraisal Committee C and, until recently, was a member of the Executive Commission of Health Technology Assessment at INFARMED in Portugal. Pedro has an Honorary Fellowship with the Centre for Health Economics, University of York. Pedro holds an MSc and PhD in Health Economics from the University of York, and an MSc in Applied Mathematics from the University of Lisbon.
Martin Parkinson, MRPharmS
Executive Director, Consulting, PPD Evidera Health Economics and Market Access, Thermo Fisher Scientific
Martin is a pharmacist with more than 30 years’ experience in government, industry, and life sciences consulting. His interests include the theoretical and practical issues of outcomes-based contracting for medicines, HTA methods and approaches, healthcare and medicines policy, and innovative pharmaceutical commercial and medical models. He has a wide range of experience including redesigning commercial organization functions such as market access capability assessment and builds, payer and funding flows, pharmaceutical pricing. At Evidera he has led many projects over the last 5 years preparing companies and medicines for the new Joint Clinical Assessment.
Amanda Pulfer, BA
Executive Director, Integrated Solutions, RWE, PPD Evidera Real-World Data & Scientific Solutions, Thermo Fisher Scientific
Amanda is responsible for the development of integrated RWD and RWE client solutions including studies using diverse data designs and innovative data sources. She has more than 15 years' experience in the consulting industry, providing RWD and RWE consultancy services and designing research programs balancing client needs with scientific rigor. Previously, Amanda worked for more than 10 years in commercial roles in biopharma; responsible for life-cycle management of brands through launch to maturity. She has experience in multiple therapy areas including oncology (hematological malignancies and solid tumors), rare diseases, multiple sclerosis, cardiometabolic disease, renal medicine, immunology, infectious disease and addiction. Amanda completed her BA at Cambridge University, UK, specializing in molecular cell biology, and has a postgraduate diploma in marketing from the Chartered Institute of Marketing,
Heather Gelhorn, PhD
Vice President, Science, PPD Evidera Patient-Centered Research, Thermo Fisher Scientific
Dr Gelhorn has more than 20 years of experience in designing and conducting health outcomes research in a variety of therapeutic areas (e.g., oncology, respiratory, endocrinology, rheumatology, sexual health, mental health, and urology). Dr Gelhorn’s experience includes PRO instrument development and validation, and patient preference studies. Trained in a variety of statistical methods including structural equation modeling, multivariate techniques, longitudinal data analysis, and item response theory (IRT), Dr Gelhorn’s interests include the application of advanced statistical methodology to practical problems in the health sciences.
Richard Diaz, BS
Associate Director – Medicines Evaluation, Rare Diseases, NICE
Richard is responsible for leading and managing a portfolio of health technology evaluations working with the Highly Specialised Technologies committee at NICE. He has more than 17 years of experience at NICE developing guidance products and supporting technology appraisals with a focus on rare diseases, HTA methodology and commissioning. Previously, Richard worked at the Healthcare Commission. Richard completed his SB degree at MIT, specializing in molecular biology, and has a law degree.
Sponsorship for this Deep Dive was provided by Thermo Fisher Scientific.