Editor’s highlights from the Journal of Comparative Effectiveness Research – January 2024
In this monthly column, I share some of my Editor’s highlights from the latest issue of the Journal of Comparative Effectiveness Research, partner journal of The Evidence Base.
Firstly, I would like to wish a Happy New Year to all our readers – I hope you had a fun and festive holiday period and 2024 has started well for you.
The new year also brings a new volume of the Journal of Comparative Effectiveness Research (JCER), and the first issue is now online (Volume 13, Issue 1). This issue includes the latest health technology assessment (HTA) Industry Update from Bray and Ramagopalan; a Methodology article addressing a key challenge with state transition models; and Research Articles including one assessing the management of major depressive disorder, and another examining gastrointestinal manifestations in the rare neurodevelopmental disorder Rett syndrome. Read more on my Editor’s highlights below.
R WE ready for reimbursement? A round up of developments in real-world evidence relating to health technology assessment: part 14
In their latest HTA Industry Update, Bray and Ramagopalan (Lane, Clark & Peacock, UK) discuss a recent FDA publication regarding definitions of real-world data (RWD) and evidence (RWE), along with target trial emulation, the 1-year anniversary of the National Institute of Health and Care Excellence (NICE) RWE framework, and a NICE/Flatiron Health publication on the utility of US RWD for UK HTA. You can read more insights from Ramagopalan on this update on The Evidence Base here.
Improved estimation of overall survival and progression-free survival for state transition modeling
A key challenge of state transition models is their difficulty in achieving a satisfactory fit to the observed within-trial endpoints (such as overall survival and progression-free survival). This can have implications for long-term extrapolations and, therefore, for medical decision making as well. In this Methodology article from Wigfield et al. (Cytel, The Netherlands), the authors define a novel estimation approach that enables improved fits to modeled end points.
Real-world assessment of treatment inertia in the management of patients treated for major depressive disorder in the USA
Major depressive disorder (MDD) affects approximately 17.3 million adults in the USA. In new research from Nelson et al. (University of California San Francisco, CA, USA), the authors assess the frequency of treatment inertia (where some people do not respond to initial treatment but continue to use the same medication) among adult patients with MDD treated in a real-world setting.
Gastrointestinal manifestations in pediatric and adult patients with Rett syndrome: an analysis of US claims and physician survey data
Rett syndrome (RTT) is a rare and severe neurodevelopmental disorder that almost exclusively affects girls and women. Gastrointestinal manifestations are common in patients with RTT and add to patient burden and caregiver concerns. This new study from May et al. (Acadia Pharmaceuticals Inc., CA, USA) describes their prevalence, and associated physician perspectives and medical costs in pediatric and adult patients. On The Evidence Base, our first Spotlight of 2024 explores ATMPs, Orphan Drugs & Rare Diseases, covering the many ways in which our industry is supporting development in this important area. You can read our curated content here.
In addition to my Editor’s highlights, you can read the full January issue of JCER here, where all the articles are freely available to read. I look forward to sharing more content with you throughout 2024, and to working with all our Editorial Board members, authors, reviewers and readers. Happy New Year!
