News from Tuesday, September 9, 2025
Articles
Stagnation in Reimbursement Keeps Biomedical Innovation From Reaching All Patients, COA Panel Says
(9/9, Mary Caffrey, The American Journal of Managed Care) reports “...[National Pharmaceutical Council president and CEO John O’Brien, PharmD, MBH] highlighted brand-new research, which featured 3 authors from [NPC], that showed 340B accounted for a 2% increase in spending among the Affordable Care Act exchange population from 2018 to 2022, with significant effects on employers and state and local governments...O’Brien raised alarms about the Inflation Reduction Act (IRA), which he said will generate savings for up to 3% of patients who will see reduced out-of-pocket costs. But he said the IRA is already slowing innovation on rare diseases and is creating the potential for duplicate discounting in 340B. More troubling, he said, are cases where drugs that have maximum fair prices must compete with those in the same class that can still offer rebates.” Full
PALOMA-2 Trial Supports SC Amivantamab Q4W as a Patient-Centered Alternative to IV Therapy
(9/8, Alana Hippensteele, MA, The American Journal of Managed Care) reports “...Amivantamab (Rybrevant; Janssen Biotech), a bispecific antibody targeting EGFR and MET, has already demonstrated efficacy in combination with the third-generation EGFR tyrosine kinase inhibitor (TKI) lazertinib (Lazcluze; Janssen Biotech). Intravenous (IV) administration of this regimen every 2 weeks (Q2W) was shown in the phase 3 MARIPOSA trial (NCT04487080) to significantly prolong progression-free survival (PFS) compared with osimertinib (Tagrisso; AstraZeneca), establishing it as a frontline standard for EGFR-mutated NSCLC.” Full
Press Releases
77% of Investors Say IRA's "Pill Penalty" Disincentivizes Small Molecule Investments
(9/9, National Pharmaceutical Council Press Release) “...‘Inflation Reduction Act Impact on Pharmaceutical Investment: Insights From Investor Interviews’ demonstrates early signals that the IRA and its Medicare Drug Price Negotiation Program (DPNP) is already influencing investment strategy and decisions in the life sciences — particularly for small molecule drugs and indication sequencing. The study is authored by UW’s William J. Canestaro, PhD, MSc, and NPC’s Julie A. Patterson, PharmD, PhD, and Jon D. Campbell, PhD. ‘Though the IRA has been law for more than three years, direct measurement of the law’s impact on investment strategy and decisions remains limited,’ said Dr. Patterson, NPC Senior Director of Research. ‘Investors decide daily which companies to fund, technologies to license, and programs to prioritize. An interview-based approach gives us a well-rounded view of the IRA’s effect on real-time investment choices with downstream impact on patient access to new medicines.’” Full
PCORI Awards New Patient-Centered CER to Support Informed Health Care Decisions
(9/9, PCORI Press Release) “...‘Patient-centered comparative clinical effectiveness research provides patients and those who care for them with the evidence they need to make more informed health care decisions and manage their health more effectively,’ said PCORI Executive Director Nakela L. Cook, M.D., MPH. ‘These newly awarded studies will generate valuable information for individuals and families facing various health issues that affect many adults and children nationwide.’” Full
Institute for Clinical and Economic Review Publishes Evidence Report on Treatment for Non-Cystic Fibrosis Bronchiectasis
(9/8, ICER Press Release) “...‘NCFB is an incredibly burdensome condition with impacts that go well beyond its respiratory effects and include social, emotional, and productivity challenges,’ said ICER’s Chief Scientific Officer and Director of Health Technology Assessment Methods and Engagement, Dan Ollendorf, PhD, MPH. ‘ICER’s analyses found that brensocatib, the first medication to be specifically indicated for NCFB, reduces the rate of pulmonary exacerbations and may also somewhat slow the rate of deterioration in lung function. Despite these benefits, however, we found that brensocatib’s expected discounted price was far out of alignment with the modest clinical improvements demonstrated in clinical studies. Pricing policy that balances innovation signals while addressing affordability and value will be an important topic at the public meeting.’” Full
Institute for Clinical and Economic Review Releases Draft Evidence Report on Treatments for Obesity
(9/9, ICER Press Release) “The Institute for Clinical and Economic Review (ICER) today released a Draft Evidence Report assessing the comparative clinical effectiveness and value of semaglutide (Wegovy®) (Novo Nordisk) and tirzepatide (Zepbound®) (Eli Lilly and Company) for obesity management. ICER is also assessing how these treatments affect additional obesity-related outcomes.” Full
OSF Approved for Multi-Million-Dollar Award to Improve Hypertension Care
(9/9, OSF HealthCare Press Release) “OSF HealthCare has been awarded $2.5 million from the Patient-Centered Outcomes Research Institute (PCORI) to advance how high blood pressure (hypertension) is managed...Building on the success of a recently completed capacity building project also funded by PCORI, this project will improve hypertension management by implementing findings from a PCORI-funded comparative clinical effectiveness research (CER) study showing the comparative clinical effectiveness of clinic and telehealth-based models of hypertension care.” Full
Journals
Comparative Effectiveness of Direct Oral Anticoagulants and Warfarin on Venous Thromboembolism in Cancer Patients
Hye-In Jung, et al.
September 2025, Cancer Medicine
Comparative Effectiveness of Valoctocogene Roxaparvovec and Efanesoctocog Alfa in the Treatment of Severe Hemophilia A: A Matching-Adjusted Indirect Comparison of Bleeding Frequency
Thomas G Douglas, et al.
September 9, 2025, Advances in Therapy