CER Daily Newsfeed

Accelerated Approval Is Delivering for Patients

(4/16, National Pharmaceutical Council: E.V.I.dently Today) comments “...A recent NPC analysis found that since 1992, 60% of [Accelerated Approval] drug applications have converted to full approval, 14% have been withdrawn following postmarketing review, and 26% remain in the confirmatory trial phase...The AA program lowers barriers to developing new treatment options in areas of high unmet need. NPC research found that diseases targeted by AA drugs impose a greater economic burden and reflect more severe disease characteristics than non-AA conditions, supporting concerns that delaying coverage of AA treatments would disproportionately impact patients with high unmet medical need.” Full

US FDA's Accelerated Approval for Drugs Needs More Transparency, Says Research Firm

(4/16, Sneha S K, Reuters) reports “...The National Pharmaceutical Council...said that despite the challenges, ‘research shows the pathway is largely working as intended.’ It added that accelerated approvals deliver value to patients, with the overall benefits outweighing risks.” Full

FDA’s Shift Signals a New Era for Data-Driven Drug Development

(4/16, David Lazerson, Fabio Lievano, MD, PhD, PharmExec) comments “...[C]linical trials remain the standard for regulatory approval, yet it is increasingly difficult to adequately populate the studies to fully represent patient populations. As a result, there are cases where drugs have been approved based on trials involving small and fragmented cohorts, regulators having been forced to accept them due to a lack of alternatives. The FDA’s shift towards real-world evidence reflects a genuine effort to address this longstanding problem. Rather than lowering standards, it is raising them back to previous levels, while expanding the availability of tools that can meet them. The FDA is not making approvals easier. It is removing the excuses. Drug developers can now access better data and so are expected to use it.” Full

Drug Pricing Plan Risks Innovation and Patients

(4/15, John Stanford, RealClearHealth) comments "...Importing price controls means importing the mindset that produces them. Many OECD countries evaluate new medicines through bureaucratic cost-effectiveness formulas that try to put a price on life -- and discriminate against people with disabilities or rare or terminal conditions. When treatments are deemed ‘too expensive’ under these formulas, these countries' centralized health systems delay or deny access to them -- even when they represent the only hope for patients." Full

New PCORnet® Playbook Case Studies: Patient-Centered Health Research in Action

(4/16, PCORnet Blog) comments “...The new PCORnet® Playbook Case Studies module highlights real-world examples of how PCORnet® Study teams have: Used the PCORnet® Common Data Model to quickly identify potential participants, assess study outcomes, and confirm the generalizability of their findings. Collaborated with the research-ready community of PCORnet to design studies that reflect patient perspectives and support broader dissemination. Leveraged research expertise within the network to conduct multi-site studies and accelerate their research to deliver fast, trustworthy answers.” Full