News from Thursday, May 15, 2025
Articles
ISPOR 2025 Daily Round-Ups: Day 1
(5/14, Joanne Walker, Katie McCool, Laura Dormer, The Evidence Base) reports “...Jon Campbell (National Pharmaceutical Council) discussed method sharing, emphasizing that HTA relies not only on rigorous science but also on contextual judgment and transparency. He highlighted HEMA’s work to define best practices in HTA methodology and urged stakeholders to recognize how values and funding sources shape assessments. He concluded that HTA methods should be seen as tools not rules and must be applied with nuance.” Full
Exclusive: Datavant to Acquire Real-World Data Specialist Aetion, Doubling Life Sciences Team
(5/15, Andrew Dunn, Endpoints News) reports “The health tech company Datavant is acquiring Aetion, a rival in the real-world data space, Endpoints News exclusively reports. Datavant on Thursday plans to announce the deal, which will double the size of its life sciences team to roughly 400 employees.” Subscription Required
Most Favored Nation Policy 'Lazy, Misguided,' But US Price Reforms Politically 'Inevitable'
(5/15, Cathy Kelly, Pink Sheet) reports “...[University of Washington professor Sean Sullivan] said the policy is lazy because ‘importing prices from other countries that have been determined through value assessments done by those countries’ careful evaluations of drugs, applying social constructs … should not ‘replace the hard work we should do as a country [on] those same value assessments.’ And it is ‘misguided because the view that other countries will raise their prices if we lower ours is naive and it shows a real lack of understanding of how other countries do [health technology assessments] and make decisions,’ Sullivan added.” Subscription Required
RFK Jr. Open To Working With Sanders On 'Most Favored Nation' Bill
(5/14, Maaisha Osman, Inside Health Policy) reports “...Sanders' plan to introduce a new bill on MFN policy comes after President Donald Trump signed an executive order Monday (May 12) that gives drug makers 30 days to voluntarily bring U.S. prices down to those of other developed countries or face formal regulatory action by the administration requiring them to do so...Kennedy responded that he supports the concept and is willing to work with Sanders on the upcoming legislation.” Subscription Required
Managed Access Agreement Fails To Help BioMarin’s Brineura Secure Routine Reimbursement In England
(5/15, Francesca Bruce, Pink Sheet) reports “...BioMarin’s ultra-rare disease drug Brineura has breached cost-effectiveness thresholds for highly specialized treatments in England and looks set to be denied routine funding on the National Health Service.” Subscription Required
Press Releases
New Studies Recommend Inclusion of Dynamic Inputs in Cost-Effectiveness Analyses to Better Reflect Drug Value Over Time
(5/13, National Pharmaceutical Council Press Release) “Two new studies published in Value in Health underscore the importance of incorporating dynamic pricing and other evolving inputs into cost-effectiveness analyses (CEAs) to more accurately represent the value of prescription drugs across their lifecycles. ‘CEAs are comprised of both science and judgment – which is why they are a tool, not a rule,’ said NPC Chief Science Officer Jon Campbell, PhD, who was an author on both studies. ‘This research supports more useful tools that better reflect market realities.’” Full
Deal Agreed for Continued Access to Life-Changing Treatment for Rare Inherited Disease
(5/14, NICE Press Release) “We are consulting on draft guidance for cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 (CLN2), a type of Batten disease. Our draft guidance does not, at present, recommend the treatment’s use for future patients with this rare, life limiting disease due to its high price and the limited evidence of long-term effectiveness.” Full
Journals
Comparative Effectiveness of Sodium-Glucose Cotransporter-2 Inhibitors Versus Glucagon-Like Peptide-1 Receptor Agonists in Reducing Cardiovascular Events in Patients With Type 2 Diabetes
Hashmat Ullah Khan, et al.
May 14, 2025, Cureus
Evaluation of HTA Frameworks for In Vivo Diagnostics: Assessing Methodological Gaps and Implications for Market Access
Dr. Ricardo De la Fuente, PhD, et al.
May 15, 2025, Value in Health
Reports
What Proportion of the Total Value Generated by New Drugs Accrues to Manufacturers? Review of Empirical Estimates of Producer Surplus
May 14, 2025
National Pharmaceutical Council
Ongoing Accelerated Approval Trends and What It Means for Patients
May 14, 2025
National Pharmaceutical Council
Are Existing Descriptions of Unmet Medical Need Fit for Purpose in the CMS Drug Negotiation Program Context?
May 14, 2025
National Pharmaceutical Council
Early Signals of the IRA on Orphan Drugs
May 15, 2025