Celebrating 10 years of the Journal of Comparative Effectiveness Research

When JCER launched in January 2012, the term ‘comparative effectiveness research’ (CER) was a relatively new one. As stated in the Foreword to the launch issue [1], written by founding Senior Editors Sheldon Greenfield and Eugene Rich, “Around the globe, healthcare decision-makers are facing an intense need for new and different types of evidence to guide them.” Research in both CER and the wider field of health economics and outcomes research (HEOR) has progressed rapidly in the intervening years, and more than ever, decision makers require robust, trustworthy and nuanced information to guide treatment approvals and health policy.

Greenfield and Rich also highlighted the demand for more patient-centered care in their article, and of course the Patient-Centered Outcomes Research Institute (PCORI) was established by Congress in 2010. PCORI’s remit is to “work with … healthcare stakeholders to identify critical research questions and answer them through comparative clinical effectiveness research, or CER, focusing on outcomes important to patients,” and JCER has been delighted to publish many studies that have received funding from PCORI.

Although the field of CER and HEOR has progressed tremendously over the years, certain topics have been, and continue to be, of great interest to researchers in the field. Table 1 highlights the top 10 articles published in the journal since launch (based on download numbers), and many of these articles continue to be read and cited years after first being published.

The most read article published to date in JCER is a white paper from Hampson et al. [2]. Perhaps unsurprising, as this article discusses the challenges involved in applying traditional drug approval and funding policies to innovative treatments like gene therapy, a crucial and continuing area of work and debate. The paper, which brings together authors from the Office of Health Economics (UK) and the Institute for Clinical & Economic Review (MA, USA), goes on to discuss potential solutions, and present policy recommendations.

Patient and stakeholder engagement has been a key topic for the journal over the past decade, and Esmail et al.’s perspective article discusses the all-important topic of moving theory into practice, highlighting the need to continually assess the success and impact of engagement activities [3].

Of course, as well as overarching themes relating to CER policies and methodology, the journal has also prided itself on focusing on disease-specific research, and how CER can complement randomized clinical trial data. In their review article, Allan et al. discussed four recently approved direct oral anticoagulants, and the use of propensity score matching and inverse probability of treatment weighting to look at the differences between them, to help understanding of their use in the treatment of atrial fibrillation [4].

“The high interest in our paper reflects shifting attitudes around real-world evidence from a nice to have to an important pillar of healthcare decision making and recognition of the value of propensity score-based methods in robustly addressing patient selection biases when estimating and comparing treatment effects. Propensity score-based methods have been pivotal in the field of atrial fibrillation research comparing real-world effectiveness and safety of direct oral anticoagulants. An emerging novel use of such methods is in external control arm designs which leverage real-world data to act as comparators to single-arm clinical trials. This approach may prove to be particularly useful for comparative effectiveness research in rare diseases.” Victoria Allan (Bristol-Myers Squibb, UK).

A similar theme is addressed in the next article on the list, a review article from Németh et al. [5] that discusses the use of multicriteria decision analysis (MCDA), with a focus on the advantages and disadvantages of various weighting methods and the implications of this for decision making in low- and middle-income countries.

“MCDA methodology has been gaining popularity over the course of the last few years. Decision makers and researchers have sought out new ways for decision support even before the COVID-19 pandemic. While the literature on the theoretical background of this topic has already been quite extensive, we wished to include some practical considerations in our paper as well. In addition to this, we gave a special focus to lower income countries, as these are the nations where making erroneous policy decisions generally have more severe consequences compared to more affluent countries.” Bertalan Németh (Syreon Research Institute, Hungary).

Comparing different drug treatments in specific indications is a cornerstone of CER, and the next two articles to feature in the top ten do exactly that. In their research article, Davies et al. describe their work using single-arm trial and real-world data to compare alectinib versus ceritinib in non-small-cell lung cancer patients [6]. And in their systematic review, Caroff et al. examine 11 studies to compare the use of tetrabenazine or valbenazine for the treatment of tardive dyskinesia [7].

Editorial articles give authors a chance to share their thoughts on a particular hot topic of the moment; in their piece from 2014, Schaaf and Case-Smith discussed sensory interventions for children with autism spectrum disorders, explaining the different interventions practiced and evidence of their efficacy [8], and in a further editorial piece, Olson discusses the importance of pharmaceutical companies developing an integrated, cross-department approach to evidence generation [9].

“My article on integrated evidence generation has been so well read because it puts a tangible framework around a topic that readers have come to realize is invaluable. Since writing the article, integrated evidence has moved from being a 'nice to have' idea, to a fundamental way of working to better address the holistic evidence needs of key healthcare stakeholders.” Melvin 'Skip' Olson (Novartis, Switzerland).

According to the National Academy of Medicine, a learning healthcare system is one that “generates and applies the best evidence for the collaborative healthcare choices of each patient and provider; drives the process of discovery as a natural outgrowth of patient care; and ensures innovation, quality, safety and value in healthcare”. In their perspective article, Mullins et al. discuss the challenges of implementing this, along with a vision for a learning healthcare community [10].

“I am thrilled to hear that our 2018 article is one of the journal’s most-read articles. I believe its popularity highlights a priority area for health services researchers worldwide: active and continuous stakeholder engagement to improve the quality and value of health care within a community. Our work at the University of Maryland Baltimore was originally funded through the US Agency for Healthcare Research and Quality’s (AHRQ) Infrastructure Development Program in Patient-Centered Outcomes Research (PCOR). With over 7000 downloads of the article, it is clear that AHRQ’s investment in PCOR a decade ago has helped to facilitate exponential gains in digital infrastructure, clinical decision support and patient-centered care. It is our hope that healthcare administrators and health services researchers continue to thoughtfully consider assessment, planning and implementation frameworks for sustainable community-academic partnerships, sustainability that requires collaboration, communication, resource sharing and trustworthiness between partners." Hillary A Edwards (University of Maryland School of Pharmacy, MD, USA).

The final article in the top ten is also the most recently published; and unsurprisingly discusses the most important healthcare emergency of modern times, the COVID-19 pandemic. In their review article published in November 2020, Rafiullah and Siddiqui discuss the use of corticosteroids in the treatment of patients [11].

As always in the January Foreword, I also want to mention some of our more recent highly read content (Table 2), as this always provides an interesting snapshot of the field today. Along with exciting research across several fields of clinical research, I particularly wanted to highlight the Clinical Trial Protocol article from Thorndike et al., which describes the aims and study design of the Digital Real-world Evidence trial for Adults with insomnia treated via Mobile (DREAM), looking at a prescription digital therapeutic for the delivery of cognitive behavioral therapy [12]. Telehealth is an important and growing field in healthcare delivery, and studies such as this will be crucial to ensure this is delivered effectively in the future.

The year 2021 also saw the introduction of a new feature to the journal, the Industry Update, which is being written by regular author Sreeram Ramagopalan and his colleague Alex Simpson (F Hoffmann-La Roche, Switzerland). The piece provides a round-up of developments in real-world evidence relating to health technology assessment, and I look forward to further pieces from Ramagopalan and Simpson in 2022 [22–24].

As mentioned above, patient engagement continues to be a key topic of discussion in JCER and the healthcare community as a whole. An important feature of this is working with patients and patient advocates throughout the treatment development pathway, and crucial to this is ensuring the results of published research is accessible to all involved. It is also vital the results of research are integrated effectively into the clinical care of patients. As such, Future Science Group has been delighted to lead the way with the introduction of standalone Plain Language Summary of Publication articles, conveying the results of scientific research in language that is accessible to nonspecialists. The first such article was published in our sister journal Future Oncology in 2020 [25], and I look forward to featuring these articles in JCER in the future, alongside the lay abstracts many of our articles already include.

As many readers know, JCER is proud to partner with website The Evidence Base [26]. The site continues to publish an array of content, freely available to all members, on the topics of CER, HEOR and real-world evidence. And on the theme of patient engagement, the site is currently planning an exciting Ask the Experts feature in which a panel of experts will explore the benefits of patient engagement for RWD/E, the importance of the market and regulators, alongside some key challenges and events affecting patient engagement.

Once again, many thanks to all our Editorial Board members, authors and reviewers who have made publication of JCER possible over the last 10 years. I look forward to seeing what comes next for the journal!

L Dormer is an employee of Future Medicine Ltd. The author has no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

No writing assistance was utilized in the production of this manuscript.