In this issue: HHS announcement on MFN, early IRA signals on orphan drugs, 340B and premium growth, and more.
National Pharmaceutical Council
Health policy research on value, evidence, innovation & access for patients.
Welcome to NPC This Week! We hope you'll join us each week for a look-ahead at the policy, research, and industry conversations that matter to the future of biopharmaceutical innovation. The DMs are open if you have suggestions — and please share with your network. - Michael Pratt, Devon Bortz, and Haley McKeefer
New From NPC
Early Signals of the IRA on Orphan Drugs: In a new Policy & Evidence Brief on the ”Early Signals of the IRA on Orphan Drugs,” NPC analyzed patterns of orphan drug designations over time to understand how the Inflation Reduction Act (IRA) may be impacting drug development.
Key Finding: Following the IRA’s passage, the percentage of drugs with a first orphan designation that later received a second designation decreased by 48.0% (12.1% pre-IRA to 6.3% post-IRA).
More Recent NPC Research:
Ongoing Accelerated Approval Trends and What It Means for Patients
Are Commercial Insurance Premiums Associated With the 340B Drug Pricing Program?
Impacts of the Inflation Reduction Act on 2025 Formulary Coverage in Medicare Part D Plans
ISPOR 2025 Recaps: NPC Chief Strategy Officer Kimberly Westrich shared highlights from ISPOR’s annual meeting in special editions of "Value Viewpoint" — read the issues from May 12, 13, 14, and 15.
Industry News
HHS, CMS Set Most Favored Nation Pricing Targets: This morning, HHS issued a press release with more information about how it planned to set MFN pricing targets as part of its implementation of the May 12 executive order (EO). STAT, Endpoints News, and Fierce Pharma report on more details.
New COVID-19 Vaccination Approval Limits: Trump administration officials announced today that approval for seasonal COVID-19 shots will be limited to seniors and high-risk individuals. FDA leaders Drs. Vinay Prasad and Marty Makary published an editorial in The New England Journal of Medicine (NEJM Group) establishing new requirements for vaccine manufacturers to conduct additional studies of COVID-19 vaccine efficacy for children and healthy adults.
Federal Court Ruling on 340B Rebate Case: Bloomberg Law’s Nyah Phengsitthy reports on the ruling from the U.S. District Court for the District of Columbia in the lawsuit related to rebates in the 340B program.
Measuring Insurer Exits After the Part D Redesign: A new JAMA study found that, compared to prior years, more Part D beneficiaries were affected by their insurer exiting the marketplace after the passage of the IRA; 2.9 million beneficiaries lost their Part D insurer in 2024.
HHS Solicits Comments on "10-to-1" Order: HHS and the FDA are seeking public input on regulations, rules, or guidelines that should be eliminated in accordance with President Trump’s EO to withdraw 10 federal regulations for each new regulation issued.
ICYMI
Unchecked Growth of 340B Program: AJMC - The American Journal of Managed Care published a Q&A with MMIT (Managed Markets Insight & Technology)’s Dinesh Kabaleeswaran on the unchecked growth of the 340B program and the need for reforms, citing NPC research on 340B profit margins in wealthier areas.
Go Deeper: In a Policy & Evidence Brief, NPC researchers analyzed the growth of the 340B program and explored how future policy efforts that increase program integrity, compliance, and transparency are needed to ensure the program's original intent is restored.
Potential Changes to PDUFA: During the May 16 Food and Drug Law Institute annual meeting, Grace Graham, the FDA Deputy Commissioner for Policy, Legislation, and International Affairs, shared that the FDA is considering restructuring the Prescription Drug User Fee Act (PDUFA) program. "It is worth exploring whether and how restructuring and simplifying the user fee program may help rebuild trust in the FDA," she noted. Endpoints News shares more insights.
Cuban’s Cost Plus Drugs, Rutgers Join New Federal Partnership: In an initiative spearheaded by HHS's Administration for Strategic Preparedness and Response (ASPR), academic and private sector organizations are partnering with the agency for a project called "Equip-A-Pharma." The program is intended to improve U.S. manufacturing for essential medicines.
Senate Leaders Call for PBM Reform: Sen. Chuck Grassley, R-Iowa, suggested Congress could move to advance PBM reform again later this summer, saying "Americans are fed up … they’re eager for Congress to act to put a stop to shady PBM practices" during a Senate Judiciary Committee hearing.
State Action: A new Arkansas law bans PBMs from owning pharmacies — and would prevent Arkansas residents from receiving out-of-state care and prescription deliveries. Pharmacist and Milliman consultant Marc Guieb said the law, which is intended to go into effect on January 1, 2026, "would change the entire landscape of how PBM contracts are structured."
Value Viewpoint: In Friday’s issue of "Value Viewpoint," Ms. Westrich reviewed ICER’s Final Evidence Report for a retinitis pigmentosa treatment and a Health Affairs Forefront commentary on a holistic approach to quality measures in value-based care.