CER Daily Newsfeed

Bayer Challenges J&J’s Real-World Data Analysis For Erleada Survival Advantage Claims

(2/24, Sue Sutter, Pink Sheet) reports “...J&J’s assertion of a 51% reduced risk of death with the prostate cancer treatment apalutamide compared with Bayer’s darolutamide in an observational study is unsubstantiated and the analysis fails to meet the FDA’s rigorous standards, Bayer said in a federal lawsuit.” Subscription Required

PCORnet® Research Project Laid the Foundation for Studies Using Patient-Reported Outcomes to Improve the Health and Health Care of Patients

(2/24, PCORnet Blog) comments “...Led by Justin Bachmann, MD, MPH, at Vanderbilt University Medical Center (VUMC), the study has had a lasting impact – expanding the use of patient-reported outcome measures (PROMs) and improving care across treatment areas. It also laid the foundation for Bachmann’s study published in 2025 in the Journal of the American College of Cardiology. In a recent Q&A session, Dr. Bachmann reflected on how the rapid-cycle research project inspired him and his team to develop the Vanderbilt Patient-Reported Outcomes Measurement System (VPROMS) – a system that leverages data insights from more than 3 million PROMs collected in routine clinical practice to drive meaningful improvements in care.” Full

Real-World Evidence (RWE) in Pharma: Use Cases

(2/25, Roman Bevz, Avenga Blog) comments “...Real-world studies that measure the efficacy of a treatment as it is used in everyday clinical settings, employing comparative effectiveness research (CER), provide evidence to evaluate how well the therapy performs compared to other therapies. The type of information that CER generates provides regulators with insights that help fill gaps typically left by traditional clinical trials, particularly with respect to long-term outcomes, diverse populations, and safety after the product is used in a broader range of patients.” Full

America Needs to Address Our Health Care Crisis Head-On

(2/24, Rep. Andy Harris, M.D. (R-MD), and Dr. Rene Desmarais, The Washington Times) comments “...We should improve interoperability of electric health records (EHR), use social media and digital messaging to enhance health maintenance, screening and literacy and smooth communication among all those involved in patient care delivery. EHRs contain enormous amounts of data that should be used to conduct virtual clinical trials and comparative effectiveness research, develop better quality metrics and screening tools, improve diagnostic accuracy and increase the yield of public health research.” Full

FDA’s Ultra-Rare Disease Framework Faces Funding, Infrastructure Test, Stakeholders Say

(2/24, Maaisha Osman, Inside Health Policy) reports “...While federal law defines a rare disease as one affecting fewer than 200,000 people in the United States, [FDA acting director Tracy Beth Høeg] said FDA ‘was very careful to leave it open-ended’ to avoid creating what she described as an arbitrary cutoff between rare and ultra-rare conditions. Instead, eligibility would hinge on whether a therapy meets the elements of the proposed plausible mechanism framework -- including a well-defined underlying biology, a targeted intervention and evidence that treatment alters the disease course beyond what would be expected based on natural history. Beth also pointed to prior approvals supported by real-world evidence rather than randomized controlled trials...” Subscription Required

PhRMA, BIO Allege GLOBE, GUARD Models Illegal

(2/24, Gabrielle Wanneh, Inside Health Policy) reports “...‘GLOBE/GUARD presumes that foreign government drug prices are appropriate reference points, but these governments artificially suppress prices through controls and discriminatory valuation methods like QALYs, which devalue the lives of seniors, persons with disabilities, and the chronically ill. Whether relying on the QALY or other standards of comparative- and cost-effectiveness analysis, these systems result in restricted access to medicines compared to the U.S., where patients enjoy broader and faster access to innovative treatments,’ PhRMA says in its comment letters.” Subscription Required

FDA Proposes Plausible Mechanism Framework to Support Approval of Individualized Therapies for Ultra-Rare Diseases

(2/25, Katie McCool, The Evidence Base) reports “...The agency has recently stated that one adequate and well-controlled clinical investigation, supported by confirmatory evidence, will generally be sufficient for marketing authorization, formally moving away from the long-standing expectation of two pivotal trials. In late 2025, it also confirmed that it will consider real-world evidence derived from de-identified real-world data sources, including patient registries, insurance claims data, and electronic health records. Viewed together, these developments signal a more integrated model of evidence generation in which regulatory-grade RWD are positioned not as ancillary, but as central to confirmatory evidence, external control development, and strengthened post-approval oversight, particularly in contexts where conventional trial designs are not feasible.” Full

Exploring New Models That Encourage Primary Care ACO Participation

(2/25, David Raths, Healthcare Innovation) reports “...Aisha Pittman, M.P.H., senior vice president of government affairs at the National Association of ACOs, spoke about what NAACOS is hoping to learn from the Primary Care Flex model and how that might help shape the future of the MSSP. ‘What I'm hoping to learn from it is how total cost of care and outcomes can improve with shifting the downstream payment mechanisms, focusing first on primary care. I think PC Flex reflects that.’” Full