Wrapping Up 2025 Legislation and Kicking Off 2026 With New Health Policy Priorities
The ISPOR Policy Brief offers concise insights into emerging developments in the global health policy space that shape access, innovation, and affordability. Each installment spotlights timely issues with relevance for the health economics and outcomes research (HEOR) community and beyond, which provides readers with a rapid overview of how policy shifts are influencing global markets and stakeholders.
Ana Amaris, MD, MPH, Director, Health Policy Initiatives, ISPOR, Lawrenceville, NJ, USA
Overview: This issue examines a series of health policy developments that emerged in December 2025, including the announcement of new most-favored-nation (MFN) pricing agreements with drug manufacturers, the release of proposed Medicare and Medicaid models aimed at operationalizing international reference pricing, and expert perspectives on whether recent Medicare negotiated prices reflect value for money. The brief also highlights international policy updates, including major reform of pharmaceutical rules in the European Union and a new drug price registry in China. Also, our Health Policy 2026 Forecast highlights 5 trends to watch this year.
US Updates & Perspectives
Most Favored Nation (MFN) Agreements
By mid-January, the Trump administration had reached MFN drug-pricing agreements with 16 companies. The 9 agreements announced on December 19 brought the total number of participating manufacturers to 14 at year’s end; 2 subsequent agreements were finalized on January 8 and January 12. The agreements commit major pharmaceutical companies to align US prices with the lowest prices paid in other developed countries and extend MFN pricing access to all state Medicaid programs. The deals apply to drugs used to treat a wide range of high-cost and chronic conditions, such as high cholesterol, type II diabetes, HIV, certain cancers, influenza, hepatitis C, asthma, multiple sclerosis, and atherosclerosis. In addition to Medicaid provisions, the agreements include commitments to offer discounted prices to patients purchasing medicines directly through TrumpRx and to repatriate additional foreign revenues generated under international pricing reforms.
New Pricing Models
The Centers for Medicare & Medicaid Services (CMS) released proposed rules for 2 mandatory Medicare drug pricing models: the Global Benchmark for Efficient Drug Pricing (GLOBE) for Part B and the Guarding US Medicare Against Rising Drug Costs (GUARD) for Part D. These initiatives are part of a broader push to integrate MFN pricing strategies within federal programs. These models would tie manufacturer rebates under existing inflation rebate frameworks to international pricing benchmarks drawn from a basket of economically similar countries, and if finalized could begin testing in select beneficiary populations in 2026. These proposals are currently open for public comments until February 23, 2026. The proposals discuss a third, voluntary Medicaid initiative known as the Generating Cost Reductions for US Medicaid (GENEROUS) model, which would use supplemental MFN rebates negotiated with states.
Experts’ Perspectives on Medicare Negotiated Prices
A detailed analysis published in Health Affairs Forefront by Neumann et al examined whether Medicare’s Initial Price Applicability Year 2027 negotiated drug prices align with value for money, concluding that the relationship between maximum fair prices and value-based prices varies widely across drugs. Drawing on published cost-effectiveness analyses, the authors found that some negotiated prices appear higher than value-based benchmarks, others are broadly aligned, and many fall below estimated values. This variability highlights the absence of a transparent and predictable framework for incorporating value into price negotiations. While the Inflation Reduction Act restricts the explicit use of quality-adjusted life years, the analysis by Neumann et al underscores CMS’s broad discretion in weighing clinical benefit, unmet need, and cost considerations, and raises questions about whether current negotiations are optimizing long-term innovation incentives and efficient resource allocation. Conclusions call for greater clarity on how “value” is operationalized in Medicare price negotiations as the program matures.
Updates From Regulators
On December 19, the US Food and Drug Administration (FDA) awarded national priority vouchers to 2 investigational products, bringing the total number of products recognized under the Commissioner’s National Priority Voucher (CNPV) pilot program to 18 since the program was established in June 2025. The FDA previously provided updates on CNPVs that were awarded in October and November 2025. According to FDA Commissioner Marty Makary, the awards reflect the agency’s intent to proactively identify potentially transformative therapies and accelerate regulatory engagement. The CNPV pilot aims to shorten review timelines and enhance early interaction with sponsors for products addressing major public health priorities, unmet medical needs, domestic manufacturing, or affordability.
The Great Healthcare Plan
On January 15, 2026, President Trump unveiled The Great Healthcare Plan, a broad framework aimed at lowering drug prices and insurance premiums by increasing pricing transparency, redirecting government subsidies into direct payments for consumers, and proposing codification of the current MFN pricing deals, while seeking greater accountability from insurers and providers in how costs and coverage are communicated to patients.
International Updates
On December 10, the European Commission announced a political agreement between the European Council and Parliament on a major reform of the EU’s pharmaceutical legislation. The reform aims to improve patient access, strengthen medicine supply security, and boost global competitiveness of the EU pharmaceutical sector through faster and more flexible regulatory pathways, stronger incentives for innovation, clarified rules to support timely generic entry, and enhanced EU-wide mechanisms to prevent and manage medicine shortages.
In late 2025, China introduced the China Drug Registry (CDR), a drug price registration system designed to support the global expansion of domestic manufacturers while reducing international reference-pricing risks for multinational companies. The CDR records self-reported transaction prices from China’s private market and existing public procurement prices, allowing manufacturers to register higher, market-based prices without direct government intervention. Under the oversight of the National Healthcare Security Administration, 9 companies, including Roche Switzerland and BeOne Medicines (formerly BeiGene), have registered products through the CDR.
