CER Daily Newsfeed

The Comparative Effectiveness Research Daily Newsfeed®, known for short as the CER Daily Newsfeed®, offers the latest news, research and related information on comparative effectiveness research, real-world data and evidence, value assessment and other important health care topics. 

News from Thursday, September 22, 2022

Articles

How Life Sciences Companies Can Strengthen Regulatory Submissions With External Control Arms

(9/21, Meg Richards, PhD, Applied Clinical Trials) comments “...RWD data—because they were created for a different purpose than biopharmaceutical research—are by nature messier than clinical trial data. Some outcomes are captured less frequently or not at all; concepts of interest may be recorded in a non-standardized way or exist only in physician notes rather than in a coded field...This ‘messiness’ of an RWD-informed ECA can introduce some error in the estimation of treatment effect, but if the effect size is large enough, the difference (favorable or unfavorable) will manifest. This makes an ECA a useful option for new and promising treatments but not for those likely to demonstrate only a modest benefit. Moving forward, ECAs are likely to play a larger role in assessing benefits and risks for treatments that receive accelerated approval or in diverse patients who were not included in pivotal clinical trials.” Full

 

Using RWE in Rare Disease Drug Development: Effective Innovations with Historical Controls

(9/22, William Maier, MD, PhD, European Pharmaceutical Review) comments “...RWE from rare diseases is available from a wide variety of sources for use as an HC in rare disease drug development. HCs are used in drug approvals, HTAs and in describing the impact of new therapies in different clinical settings for rare diseases. As a result, the utility of HCs based on RWE should be evaluated for potential applications to increase speed of product approval, reimbursement and clinical practice adoption.” Full

 

Are $3M Gene Therapies the New Normal? Experts Say Bluebird Will Be a Test Case for a Market Ready for Prime Time

(9/21, Zachary Brennan, Endpoints News) reports “...Mark Trusheim, strategic director of NEWDIGS at the Center for Biomedical System Design at Tufts Medical Center, told Endpoints that he expects about 64 product-indication approvals for cell and gene therapies through 2030, with about half of those in this durable gene therapy space that can command these multimillion-dollar list prices. ‘Moving forward, each new product will be judged on the specific benefits its evidence demonstrates,’ Trusheim noted. ‘That said, more such list prices before discounts and outcomes contracts results would seem likely in the future.’” Full

 

UK: Merck Bags NICE Recommendation for Renal Cell Carcinoma Adjuvant Treatment

(9/22, Emily Kimber, PMLiVE) reports “Merck & Co...has announced that Keytruda (pembrolizumab) has been recommended by the National Institute for Health and Care Excellence as an option for the adjuvant treatment of renal cell carcinoma (RCC) at increased risk of recurrence after nephrectomy, with or without metastatic lesion resection, in adults. The decision marks the first time patients with RCC are able to access immunotherapy at an earlier stage in their treatment pathway.” Full

Press Releases

Two Popular Diabetes Drugs Outperformed Others in Large Clinical Trial

(9/21/ National Institutes of Health Press Release) “In a large clinical trial that directly compared four drugs commonly used to treat type 2 diabetes, researchers found that insulin glargine and liraglutide performed the best of four medications approved by the U.S. Food and Drug Administration to maintain blood glucose levels in the recommended range. Blood glucose management is a key component of keeping people with type 2 diabetes healthy. All four medications evaluated were added to treatment with metformin, which is the first-line drug to treat type 2 diabetes. The trial was funded by the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), part of the National Institutes of Health.” Full

 

GAO Makes Appointments to PCORI Governing Board, New Chair and Vice Chair Named

(9/22, Government Accountability Office Press Release) “...The newly appointed members are Chris Boone, Ph.D., Vice President, Global Head of Health Economics and Outcomes Research, AbbVie; Ryan Bradley, ND, MPH, Director of Research and Senior Investigator, National University of Natural Medicine; Zoher Ghogawala, MD, FACS, Professor, Department of Neurosurgery at Tufts University School of Medicine and Chairman, Department of Neurosurgery at Lahey Hospital & Medical Center; Debbie Peikes, Ph.D., MPA, Vice President, Humana Healthcare Research; Kimberly Richardson, Founder, Black Cancer Collaborative; and Christopher L. White, Esq., General Counsel and Chief Policy Officer, Advanced Medical Technology Association (AdvaMed).” Full

Journals

After Metformin — Next Steps for Type 2 Diabetes with Low Cardiovascular Risk

Lars Rydén, M.D., and Eberhard Standl, M.D., Ph.D.

September 22, 2022, The New England Journal of Medicine

NEJM

Events

Webinar: Keeping Pace with RWE: Innovative Approaches in Patient Access and Medical Affairs

October 18 - 19, 2022

9:00AM - 12:00PM EDT

IQVIA