News from Thursday, September 14, 2023
Articles
US FDA Commissioner Pushes Real-World Evidence To Guide Use Of Alzheimer’s, Obesity Drugs
(9/13, Kate Rawson, Pink Sheet) reports “...Califf has been particularly interested in using real-world evidence in the post-approval setting, which he described as a ‘system in shambles.’ He reiterated that ‘we're not talking about the fundamental structure of drug and device approval. I think that works pretty well. We are talking about what happens after that, where FDA is only part of the system.’ Califf reiterated his view that other countries have a longer life expectancy and a better quality of life (he mentioned Singapore, specifically), ‘because they have organized approaches to information.’ Israel uses real-time electronic health data, he noted, and the UK National Health Service enables a streamlined approach in which clinical trials are integrated directly into clinical care, ‘in other words, real-word evidence.’” Subscription Required
Pushing Both Sides of the Drug Pricing Aisle
(9/14, Asher Mullard, Nature Reviews Drug Discovery: An Audience With) reports “Few topics are as controversial in healthcare as drug pricing. As prices edge ever higher, budgets are stretched ever thinner. And while some patients and clinicians clamor for new and better therapeutics, others question the value of the drugs that are reaching the market. The Institute for Clinical and Economic Review...has leaned into this tension, developing systematic and transparent methods to assess the cost-effectiveness of new treatments. With ICER’s founding President Steve Pearson now stepping down, President-elect Sarah Emond is ready to take up the medical mantle.” Subscription Required
England's NICE Backs Pfizer's Vydura as Treatment for Acute Migraines
(9/13, Kevin Dunleavy, Fierce Pharma) reports “...This time, NICE still stopped short of recommending Vydura for primary use. According to the draft guidance, rimegepant is endorsed for adults who have had unsuccessful results with at least two triptan medicines. Vydura also is suggested for use by those who can't take triptans and had unsuccessful results with nonsteroidal anti-inflammatory drugs (NSAIDs) or the beta blocker paracetamol. ‘NICE understands that rimegepant could eventually be used in primary care but should initially be started in secondary care,’ the regulator said...” Full
Press Releases
ICER Publishes Evidence Report on Gene Therapy for Metachromatic Leukodystrophy
(9/14, ICER Press Release) “The Institute for Clinical and Economic Review today posted its revised Evidence Report assessing the comparative clinical effectiveness and value of atidarsagene autotemcel (“arsa-cel”, Orchard Therapeutics) for metachromatic leukodystrophy (MLD).” Full
Researchers Will Use PCORnet® Resources To Explore Whether Cancer Screening Practices Change After Weight Loss Surgery
(9/13, PCORnet Press Release) “...Can weight loss associated with bariatric surgery reduce the risk of some cancers? Maybe, according to a recent meta-analysis of several key studies, which found that people with obesity who had bariatric surgery had a substantially lower risk of developing five types of solid tumors. But it remains unclear if the factors driving the risk reductions might be physical, behavioral, or both. Now, a new study funded by the National Cancer Institute will use PCORnet resources to understand if one of those factors is a change in adherence to recommended preventative cancer screenings after bariatric surgery.” Full