CER Daily Newsfeed

Value Viewpoint: August 15, 2025

(8/15, Kimberly Westrich, LinkedIn) comments “Last week, a new article in Health Affairs proposed a framework for incorporating health technology assessment (HTA) methods into the Medicare Drug Price Negotiation Program (DPNP)...About their framework, the authors say: ‘…our proposal contains specific approaches for expanding patient and public engagement across multiple steps in the negotiation process. Moreover, it combines clinical, economic, and other information to inform a Maximum Fair Price offer based on weightings for a decision-making process that is transparent and replicable.’” Full

Kids Feel Less Pain With Underused Kidney Stone Treatment

(8/19, Brittany Vargas, Medscape) reports “Shockwave lithotripsy (SWL) for pediatric patients leads to less pain and fewer abnormal urinary symptoms after recovery than the widely used ureteroscopy (URS) procedure, according to research published in JAMA Network Open.” Full

Reinstated Prasad Signals Flexibility On RCTs For Rare Disease Drugs

(8/18, Maaisha Osman, Inside Health Policy) reports “FDA biologics chief Vinay Prasad, recently reinstated after a brief exit, said Thursday that randomized clinical trials (RCTs) are not always required for rare disease drug approval, signaling a potential shift in how he approaches therapies for rare diseases now that he is back at the agency. In the past, Prasad has called for stricter testing standards, pushing for RCTs with clear clinical outcomes even for some rare disease therapies. FDA’s approval announcement for a new rare disease immunotherapy includes a statement from Prasad that randomized clinical trials are not always necessary to approve therapies, a likely attempt to reassure advocates of unfettered access to rare disease treatments whose criticisms, including in the Wall Street Journal, likely played a role in Prasad’s short-lived exit from the agency.” Subscription Required

Duke-Margolis 2025 RWE Policy Series: Strengthening Global Alignment and Sharing Lessons from Regulatory Applications of Real-World Evidence

(8/19, Katie McCool, The Evidence Base) reports “At the Duke-Margolis Institute for Health Policy’s State of Real-World Evidence Policy 2025 conference, two sessions examined international real-world evidence (RWE) regulatory harmonization and practical lessons from regulatory use cases. Speakers discussed evolving guidance, cross-jurisdictional alignment, data quality, and transparency, drawing on examples from Japan, global ICH initiatives, and post-market and externally controlled studies.” Full