Pricing Reforms, Regulatory Shifts, and Global Market Reactions to Most Favored Nation

The ISPOR Policy Brief offers concise insights into emerging developments in the global health policy space that shape access, innovation, and affordability. Each monthly 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 March issue highlights several policy developments shaping the early months of 2026: initiatives affecting pharmaceutical pricing in the United States, regulatory changes related to evidence requirements for drug approval, and early international responses to US pricing policies. Together, these developments illustrate how governments and regulators are reassessing approaches to affordability, access, and innovation.

US Updates & Perspectives

ISPOR Responds to GLOBE and GUARD

ISPOR submitted official commentary to the Centers for Medicare & Medicaid Services on the proposed GLOBE and GUARD models. These proposals signal a significant shift in US drug pricing policy, with implications for international reference pricing, methodological transparency, and the role of value in reimbursement decisions.

In our response, ISPOR emphasized the importance of methodological rigor, clarity in international price comparisons, and careful consideration of health system differences, fiscal contexts, and innovation incentives. The response underscored ISPOR’s position that drug pricing reforms should be grounded in transparent, evidence-based approaches that support patient access while sustaining innovation.

The commentary reflects input from ISPOR’s Health Policy leadership and members with expertise in health economics, outcomes research, and pricing policy.

Read ISPOR’s full response here.

Debate Emerges Over Codifying US Most-Favored-Nation Drug Pricing Policy

The Trump administration has urged Congress to make its Most-Favored-Nation (MFN) drug pricing agreements with pharmaceutical manufacturers permanent through legislation, arguing that congressional action is needed to ensure the policy continues beyond the current administration. The agreements aim to reduce US drug prices by linking them more closely to prices paid in other high-income countries.

However, the support from Congress remains uncertain. Some Republican lawmakers and free-market groups have expressed concerns that a formalized policy could resemble government price controls, while industry stakeholders have opposed turning voluntary agreements into binding legislation.

TrumpRx Platform Shows Limited Early Impact

One month after its launch, the Trump administration’s drug discount platform, TrumpRx, appears to have limited scope and uncertain impact. The website lists only a small number of medicines (44 as of March 5th), and data on patient use and savings have not yet been publicly released.

The platform relies on voluntary agreements between the administration and pharmaceutical manufacturers to offer discounted prices on selected medicines. Analysts note that while the initiative could provide savings for some patients, its broader impact on overall drug spending will depend on whether more manufacturers and products are added to the program.

FDA Signals Shift Away From 2-Study Requirement for Drug Approvals

The US Food and Drug Administration (FDA) plans to move away from its long-standing expectation that new drugs be supported by 2 clinical trials. Under the new approach, the agency’s default position will allow approval based on a single pivotal study supported by additional evidence.

FDA leadership argues that advances in science and trial design allow regulators to determine effectiveness without requiring duplicate trials. The change is part of a broader effort to speed up drug development and regulatory review.

Single-trial approvals are already common for treatments targeting rare or life-threatening diseases. The new policy could expand this approach to drugs for more common conditions. Experts note that the impact will depend on how the FDA applies the new standard in practice.

International Updates

Nordic Countries Assess Potential Impact of US MFN Drug Pricing Policy

Denmark and Sweden are evaluating the potential effects of the United States’ MFN drug pricing policy, which aims to link US medicine prices to the lowest list price among comparable Organization for Economic Cooperation and Development countries. Both governments have launched analyses to assess possible implications for their pharmaceutical pricing systems and access to medicines.

Authorities in both countries note that the policy implementation remains unclear, and no concrete effects on drug access have been observed so far. However, policy makers and industry stakeholders have raised concerns that MFN could influence launch strategies for new medicines or reduce confidential price discounts for payers.

Recent developments in Denmark have further deepened these concerns. Amgen announced in February 2026 that it would withdraw its cholesterol-lowering medicine Repatha, (evolocumab), from the Danish market, citing changes in global pricing conditions. Some analysts have suggested that policies linking international prices could increase the risk that companies might delay launches or withdraw access to medicines in smaller-market MFN reference nations like Denmark to prevent negotiated discounts from affecting larger markets such as the United States.

Germany Warns MFN Uncertainty May Delay Drug Launches

Concerns are emerging in Germany that uncertainty around the MFN drug pricing policy could begin to affect the launch of new medicines. In a recent interview with Ärzte Zeitung, VFA President Han Steutel noted that some companies are delaying or reconsidering product launches in Germany while they wait for greater clarity on how US pricing policies may evolve.

The issue comes amid broader debate about the financial sustainability of Germany’s statutory health insurance system. A government-appointed Health Finance Commission is expected to present initial proposals by March 2026 to address rising health expenditures. Analysts note that additional pricing pressure, combined with uncertainty around international pricing policies, could increase the risk of delayed access to innovative medicines in the German market.