US Drug Policy Reforms and Global Policy Reactions
Policy Briefs offer concise insights into emerging developments in the global health policy space that shape access, innovation, and affordability. Each Policy Brief will spotlight timely issues with relevance for the health economics and outcomes research (HEOR) community and beyond. The goal is to provide readers with a rapid overview of how policy shifts are influencing global markets and stakeholders. The Briefs present an overview of the issue and its implications for the HEOR field, followed by concise summaries that reflect key stakeholder perspectives.
US Drug Policy Reforms and Global Policy Reactions
Ana Amaris, MD, MPH, Director, Health Policy Initiatives, ISPOR, Lawrenceville, NJ, USA
Overview: This column focuses on the evolving landscape of US pharmaceutical policy following the government’s recent agreements with some drug manufacturers and the results from the second round of Medicare Drug Price Negotiation (MDPN) under the Inflation Reduction Act (IRA). Occurring amid the 43 day-long government shutdown that began on October 1, 2025, these developments introduce new layers of complexity to an already dynamic policy environment.
The column also examines emerging international reactions and global stakeholder perspectives on the potential implications of these US policy shifts for access, regulation, and market stability.
US Stakeholder Perspectives
Academia and Research
An article described the deal between US President Donald Trump and Pfizer, part of the administration’s ongoing “Most Favored Nation” (MFN) effort to align US drug prices with those in other high-income countries, as largely symbolic. The direct-to-consumer “TrumpRx” platform, expected to launch in 2026, is seen as unlikely to make drugs more affordable, since insured patients already benefit from negotiated prices and uninsured individuals would still face high out-of-pocket costs. Experts caution that linking US prices to international benchmarks could prompt higher prices or restricted market access abroad, reinforcing calls for domestic value-based pricing reforms.
Private Sector/Innovators
Cost Plus Drugs, the online pharmacy founded by Mark Cuban, will participate in the TrumpRx drug-price transparency platform, sharing real-time access to its pricing database so users can compare costs across direct-to-consumer websites. The partnership aligns with the administration’s goal of expanding access to upfront pricing and bypassing traditional intermediaries. Cuban, who has long criticized pharmacy benefit managers (PBMs) for inflating US drug costs, said participation will boost Cost Plus’s visibility and potentially lower prices as volumes grow. His comments at the HLTH 2025 conference reflected cautious optimism, noting that while TrumpRx could improve transparency, PBMs have yet to feel major disruption.
Industry
On November 6, 2025, the White House announced new agreements with Novo Nordisk and Lilly that will sharply reduce the prices of several leading GLP-1 medicines. Under the deal, Ozempic pricing will drop from $1000 to $350 per month and Wegovy from $1,350 to $350, while Zepbound and Orforglipron will be priced at $346 per month through the new TrumpRx platform, to be launched in 2026. The agreements also reduce Medicare prices for these treatments to $245 per month with a $50 patient copay and extend $35 monthly pricing for insulin products such as Novo Nordisk’s NovoLog and Tresiba. In exchange, the companies will guarantee MFN pricing for future products, repatriate additional foreign earnings, and grant state Medicaid programs access to MFN prices. The administration frames these measures as a “historic reduction” in drug costs aimed at expanding affordability and reshaping US pricing norms.
Payers and PBMs
The Centers for Medicare and Medicaid Services (CMS) announced in late November the second round of price negotiations under the IRA, securing 38%–85% reductions for 15 high-expenditure medicines, with new prices taking effect in 2027. CMS estimates these cuts will reduce Medicare’s net spending on the selected products by 44% compared to 2024, generating approximately $12 billion in savings and lowering beneficiary out-of-pocket spending by $685 million. Recent analyses suggest that, although the reductions seem substantial, many manufacturers already provide significant rebates in Part D, meaning the differences from net prices are more limited based on the estimated prices reported in Value in Health. CMS will select 15 additional drugs for negotiation in 2026 and up to 20 annually thereafter, signaling a continued expansion of Medicare price setting.
Cigna’s Evernorth division announced a rebate-free, pass-through model for Express Scripts, shifting away from traditional PBM rebates toward greater transparency and upfront discounts. The model, which will roll out across all plans by 2028, aims to lower brand-name drug costs by about 30%, expand real-time price comparisons for patients, and introduce a cost-plus reimbursement system for pharmacies beginning in 2026.
Regulatory
The FDA has accepted Sanofi’s Tzield for expedited review under the Commissioner’s National Priority Voucher pilot program, a new pathway aimed at accelerating access to therapies that address high unmet medical needs. If approved, Tzield would be the first disease-modifying treatment to delay the progression of stage III type 1 diabetes in both adults and children. The FDA’s evolving fast-track mechanisms, including the Commissioner’s National Priority Voucher program and accelerated approval programs, are intended to balance innovation with access, even amid operational constraints linked to the 43-day government shutdown.
International Perspectives
The United States and the United Kingdom announced an agreement in principle on pharmaceutical pricing as part of the new US–UK Economic Prosperity Deal, aimed at addressing perceived imbalances in bilateral pharmaceutical trade. Under the arrangement, the United Kingdom will increase the net prices paid by the National Health Service (NHS) for new innovative medicines by 25% and limit the use of broad portfolio-wide rebates under schemes such as the Voluntary Scheme for Branded Medicines Pricing, Access, and Growth, with repayment rates expected to fall to 15% in 2026 and remain at or below that level thereafter. In return, the United States will exempt UK-origin pharmaceuticals, ingredients, and medical technologies from Section 232 tariffs, refrain from initiating Section 301 investigations into UK pricing practices during the current administration, and support access to emerging pharmaceutical innovations for UK patients. US officials describe the agreement as part of a broader effort to foster more “fair” international contributions to pharmaceutical innovation and to ensure that American consumers no longer shoulder a disproportionate share of global drug costs, while also reinforcing US–UK cooperation in life-sciences investment and manufacturing.
In Asia, Japan’s pharmaceutical market is emerging as one of the first to feel the effects of US MFN drug pricing policies, given their inclusion among the 8 countries named as pricing comparators. Industry leaders warn that unless Japan reforms its current pricing approach, which already reduces drug prices annually, the country could face declining investment and limited access to new medicines. A recent survey of multinational pharmaceutical companies operating in Japan found that MFN is influencing both global pricing and research and development strategies, with several firms delaying launches or adjusting development plans.
These results highlight Japan’s vulnerability to ripple effects from US pricing reforms and underscore the need for renewed dialogue between the Japanese government and industry on sustaining innovation and access.
In Europe, the United Kingdom is reportedly preparing to raise the cost-effectiveness threshold used by the National Institute for Health and Care Excellence by around 25%, from the current £20,000–£30,000 per quality-adjusted life year, in response to pressures related to US drug-pricing reforms and threatened tariffs on pharmaceuticals. This move is seen as part of efforts to strengthen the United Kingdom’s life-sciences sector, maintain the country’s attractiveness as a launch market, and address industry concerns that the longstanding threshold has become misaligned with inflation and innovation. The proposal has drawn commentaries from health economists who caution that raising the threshold could increase cost pressures on the NHS and affect population-health trade-offs.
