News from Tuesday, September 2, 2025
Articles
Towards Standardized Quality Management for Secondary Data Research
(9/2, The Evidence Base) reports “As real-world evidence (RWE) takes on a growing role in regulatory, clinical, and policy decision-making, ensuring the quality of studies using secondary data sources is increasingly critical. In this Peek Behind the Poster, we speak with Dr. Susan Oliveria (Vice President and Global Head of Epidemiology and Scientific Affairs, PPD Observational Studies, Thermo Fisher Scientific) about the ISPE 2025 poster, ‘Considerations for developing a quality management system: real-world evidence utilizing secondary data.’ The discussion explores the rationale for the assessment, methodological insights, and next steps in advancing quality frameworks for RWE.” Full
New Real-World Evidence Confirms Bempedoic Acid’s Efficacy in Lowering LDL-C in German Patients
(9/1, GlobalData) reports “...These real-world results align with previous clinical trial outcomes, confirming that bempedoic acid delivers substantial LDL-C reduction across diverse patient groups. Notably, its LDL-C-lowering effect persists in those already receiving other lipid-lowering treatments, highlighting the additive benefit of bempedoic acid.” Full
Press Releases
Institute for Clinical and Economic Review Publishes Final Evidence Report on Treatments for Spinal Muscular Atrophy
(9/2, ICER Press Release) “The Institute for Clinical and Economic Review (ICER) today released a Final Evidence Report assessing the comparative effectiveness of apitegromab (Scholar Rock Holdings), as well as the disease-modifying therapies nusinersen (Spinraza®, Biogen), onasemnogene abeparvovec-xioi (Zolgensma®, Novartis), and risdiplam (Evrysdi®, Genentech) for spinal muscular atrophy (SMA). Apitegromab is an investigational new drug seeking FDA approval for improving motor function in patients with SMA.” Full
Journals
Real-World Viral Effectiveness of Two-Drug Versus Three-Drug Regimens for HIV in Taiwan: Impact of Adherence and Treatment Selection
Tien-Heng Ku, et al.
August 27, 2025, Journal of Microbiology, Immunology and Infection
Early Impact of the Inflation Reduction Act on Small Molecule vs Biologic Post-Approval Oncology Trials
Hanke Zheng, Julie A Patterson, Jonathan D Campbell
August 28, 2025, Health Affairs Scholar
How Time is Accounted for when Modeling Pharmaceutical Impacts: Investment vs. Assessment Perspectives
Melanie D Whittington, PhD, Louis P Garrison, PhD, John Michael O’Brien, PharmD, MPH, Jonathan D Campbell, PhD
August 30, 2025, Health Affairs Scholar
The Impact of the Inflation Reduction Act’s Drug Price Negotiation Program on Incentives for Clinical Development of New Drugs
Robert J. Nordyke PhD, Julie A. Patterson PhD, James Motyka PharmD, Benjamin March MBA, Noam Y. Kirson PhD, Genia Long MPP
September 1, 2025, Value in Health
Comparative Effectiveness of Alternative Initial Doses of Opioid Agonist Treatment for Individuals with Opioid Use Disorder: A Protocol for a Retrospective Population-Based Study Using Target Trial Emulation in British Columbia, Canada
Ruyu Yan, et al.
September 1, 2025, BMJ Open
Instrumental Variable Approaches for Estimating Time-Varying Treatment Effects in Comparative Effectiveness Research
Daniel Tompsett, et al.
September 2, 2025, BMC Medical Research Methodology