From Policy to Practice: PBM Reform, TrumpRx, and Global Drug Development

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.

From Policy to Practice: PBM Reform, TrumpRx, and Global Drug Development

Ana Amaris, MD, MPH, Director, Health Policy Initiatives, ISPOR, Lawrenceville, NJ, USA

This issue examines several health policy dynamics shaping the early months of 2026. It highlights newly enacted reforms to pharmacy benefit manager (PBM) practices in Medicare Part D, recent updates to the Medicare Drug Price Negotiation Program, and the launch of the TrumpRx platform alongside early reactions to its implementation. The brief also outlines how ISPOR is assessing stakeholder feedback on emerging pricing models and explores how China’s growing appeal as a site for clinical trials provides a global context for these US policy shifts. Together, these developments offer insight into the evolving landscape of cost containment, market sustainability, and pharmaceutical innovation in the United States.

US Updates & Perspectives

PBM Reform Is Now Law

A federal spending bill, signed into law on February 3, introduced major reforms to PBM practices in Medicare Part D. The law aims to increase price transparency and reduce incentives that reward higher drug list prices.

The new law requires PBMs to move away from rebate-based compensation and, instead, be paid through flat administrative fees. It also mandates that 100% of manufacturer rebates, fees, and other payments be passed through to plan sponsors. The Centers for Medicare & Medicaid Services (CMS) is responsible for enforcement and may impose financial penalties for noncompliance.

In addition, the law expands reporting requirements, allowing CMS to track drug spending, rebate flows, spread pricing, and formulary decisions, and to establish standards for “reasonable” PBM contract terms.

Stakeholder reactions have underscored the significance of the reform. Rob Abbott, ISPOR’s Chief Executive Officer, welcomed the legislation as a positive step toward making healthcare more accessible and affordable. He noted the reforms aim to facilitate transparency, enhanced oversight of PBM compensation, limits on spread pricing, and stronger protections for pharmacy reimbursement and network participation across Medicare and the commercial market. At the same time, he emphasized that PBM reform is not a “silver bullet” for drug pricing or access challenges and applauded the bipartisan collaboration needed to make these reforms a reality. 

CMS Negotiations

CMS announced the selection of 15 high-cost prescription drugs for the third cycle of the Medicare Drug Price Negotiation Program, including, for the first time, drugs covered under Medicare Part B. CMS also identified one previously negotiated drug for renegotiation. Negotiations will occur in 2026, with negotiated prices scheduled to take effect on January 1, 2028. The selected drugs represent the highest spending products across Medicare Parts B and D, accounting for approximately $27 billion in Medicare drug spending. Manufacturers with drugs selected for the third negotiation cycle have until February 28, 2026, to confirm their participation in the negotiation process.

TrumpRx Launches Following Legal Delays

After being delayed due to legal concerns, the Trump Administration announced on February 5th the launch of TrumpRx.gov, a federal platform intended to provide consumers with direct access to lower prices on selected high-cost, brand-name prescription drugs. The platform is based on most-favored-nation (MFN) pricing agreements negotiated with participating manufacturers, with the stated goal of aligning US prices with those in other developed countries.

At launch, TrumpRx included roughly 40 brand-name drugs from a limited number of manufacturers, with a focus on high-spend medicines such as GLP-1 therapies. The platform allows consumers to purchase medications directly through manufacturer websites or, in some cases, to access coupons redeemable at participating pharmacies. TrumpRx is primarily targeted at patients facing high out-of-pocket costs outside traditional insurance coverage, with additional products expected to be added on a rolling basis as new agreements are finalized.

Early reactions to TrumpRx have raised questions about whether the platform will meaningfully reduce drug costs for most Americans. Experts argue that the platform is primarily designed for cash-paying, uninsured, or underinsured patients, offering limited benefits to most individuals who obtain prescriptions through insurance and rely on negotiated rates and out-of-pocket protections. Critics further caution that shifting drug purchases outside insurance may weaken collective bargaining mechanisms and disproportionately benefit manufacturers by expanding direct-to-consumer cash markets. Analysts have also noted that several branded drugs listed on TrumpRx have significantly cheaper generic alternatives available, raising concerns about whether the platform consistently directs patients toward the lowest-cost options.

ISPOR Engagement

As drug pricing policy continues to evolve across legislative, regulatory, and administrative channels, stakeholder engagement will play a critical role in shaping durable solutions. In this context, ISPOR gathered member feedback on the proposed GLOBE and GUARD drug payment models to provide a formal response to the Federal Register, with the goal of contributing evidence-based perspectives to ongoing federal deliberations. Comments submitted to the Federal Register will be shared in a future issue of the Policy Brief.

International Updates

Global Shifts in Drug Development

Amid persistent pressures on US drug pricing and innovation, structural cost differences in drug development are increasingly shaping global competitive dynamics. Clinical trials in China have lower operational costs, more streamlined regulatory processes, and larger patient populations than in Europe and the United States, which keeps overall costs low and allows for faster execution. In 2025, China contributed roughly 39% of global cancer drug trials, outpacing US trial volume and accelerating the pace of clinical evidence generation. These financial and operational advantages have contributed to a growing number of licensing deals and strategic partnerships between Western pharmaceutical companies and Chinese innovators, expanding China’s role in early-stage drug development.

AstraZeneca’s recently announced $15 billion expansion of manufacturing and research and development in China reflects a long-standing strategy to leverage the country’s speed in early human testing, early stage venture investment, and licensing partnerships. In 2025, nearly one-third of the company’s disclosed biotech deals involved China-based firms, an example of how global manufacturers are reallocating drug development risk toward regions that offer faster and more cost-efficient pathways to clinical proof-of-concept.