We write as patients, people with disabilities, caregivers, and the organizations that represent us – people who live every day with serious, chronic, and rare diseases, and who depend on continued medical progress to survive and to thrive. We are urging you to oppose Most Favored Nation (MFN) drug pricing policies that would import foreign prices set using system known to delay and deny patient access, and to instead advance patient-centered solutions that can lower costs without sacrificing American innovation.

For decades, the United States has built an internationally respected research enterprise by empowering expert peer review, encouraging collaboration across institutions and disciplines, and providing researchers with the stability needed to pursue difficult scientific questions whose answers may take years to emerge. This system is not perfect and appropriately has built-in levers during the annual appropriations process that allow for accountability, but what we must not overlook is that it has produced extraordinary benefits for patients, taxpayers, and the American economy. The proposed rule would fundamentally alter that model.

As a coalition of biotechnology and pharmaceutical innovators, patient advocates, investors, and physicians working at the forefront of science and medicine, we write at this pivotal moment to recommend a leader who can secure and strengthen the U.S. Food and Drug Administration: Dr. Richard Pazdur.

As we indicated in our November 20th letter, No Patient Left Behind (NPLB)'s coalition of biotech investors, innovators, researchers, physicians, and patient advocates is deeply concerned about the direction of the FDA. We further wish to highlight and emphasize the issues raised in the December 3, 2025 Perspective published in the New England Journal of Medicine by twelve FDA commissioner predecessors.

US biotech innovators and investors value FDA prioritizing the recruitment and retention of clinical, scientific and regulatory specialists who offer innovators the benefit of experienced, case-specific guidance and problem-solving. This institutional expertise is especially important to small, early-stage, and pre-commercial US biopharma entrepreneurs, who account for the majority of new drug and biologic applications submitted to the FDA.

Our modern world needs a modern FDA with the capacity to accelerate access to safe, effective treatments while sustaining U.S. leadership in biotechnology. PDUFA VIII offers a historic opportunity to make the drug review program faster, more consistent, and more transparent by prioritizing core review activities, modernizing processes, strengthening accountability, and embracing patient-centered science.

American patients and taxpayers disproportionately fund the global R&D ecosystem that enables life- saving medical advances, while wealthy countries impose price controls that underpay by as much as 60% relative to their economic capacity. Adopting the Most Favored Nation (MFN) approach for price setting undermines U.S. leadership in medical innovation, hurts patients at home and abroad, and also impairs global progress against disease.

US biomedical innovators, the investors that support them, and the patients who depend on continued medical progress rely on the US Food and Drug Administration’s guidance and rigor to navigate policy and medical standards and to approve new medicines. The agency’s reorganization must preserve the institutional knowledge and core functionality that makes the FDA the world’s leading regulatory body.

When other countries pay less for drugs, they are free-riding on American innovation and our willingness to pay for new treatments. Forcing manufacturers to charge the same price that these other countries do would backfire. Instead, policymakers should use trade negotiations to pressure other wealthy countries to pay their fair share. No Patient Left Behind wrote to the Office of the United States Trade urging an end to unfair trade practices in drug pricing.

No Patient Left Behind wrote to the Office of the United States Trade Representative regarding the 2025 Special 301 Review, urging the agency to use its tariff and market access tools to force OECD countries to pay their fair share for medical innovation and end the free-riding of American biomedical innovation.

The independence and autonomy of the Food and Drug Administration (FDA) is a key part of ensuring that the biotech innovation ecosystem can continue to thrive. As an agency, it is the gold standard globally for determining risk-reward benefit in therapies for patients, and the world looks to it to lead the way. The people at the FDA facilitate the medical progress we all enjoy, and their work is a service to the American public.

There is an assault on the foundation of U.S. science. Recent actions—including arbitrarily restricting scientists' ability to speak and travel and stalling the dispersal of previously awarded NIH and NSF funds—are more than bureaucratic disruptions. They are an assault on the foundation of biomedical and technological progress.

No Patient Left Behind (NPLB) wrote to the Centers for Medicare and Medicaid services (CMS) urging the agency to allow Medicare to cover anti-obesity medications (AOMs) and to strengthen patient protection “guardrails” through better oversight of Medicare Advantage Part D (MAPD) plans.

Too many Americans cannot afford their out-of-pocket costs (deductibles, copays, or coinsurance) in addition to their premiums. In some cases, this is not because their employer does not want to offer first-dollar, pre-deductible coverage with low or no copays, but because IRS regulations require that, to offer a Health Savings Accounts (HSA), self-insured employers must ensure that their workers pay unnecessarily high out-of-pocket costs. We ask that the Treasury department remove a barrier to such coverage by making a slight regulatory change that would enable self and fully insured companies to continue to offer HSAs and offer first-dollar coverage of medicines with low/no copays if they so choose.

No Patient Left Behind wrote to the Centers for Medicare & Medicaid Services (CMS) asking the agency to adopt rules that ensure that patients—not health plans or PBMs—see savings from the implementation of Medicare price setting.

CBO’s ability to correctly model investor decision-making is vital to our country’s ability to establish policies that achieve lasting biomedical affordability and continued innovation. In support of CBO’s efforts to improve its model, this letter emphasizes a number of economic and financial first principles, notably that investment is incentivized by expected returns based on discounted profits, not revenue, and adjusted for expected dilution from financings.

Under the drug pricing provisions of the Inflation Reduction Act, small molecule treatments will face Medicare “negotiation” (price setting) that makes brand medicines functionally generic just 9 years after FDA approval. This policy will unwisely skew investment towards harder to manufacture biologics and away from small molecule treatments for diseases of aging. Without both kinds of medicines, we will all be worse off.

No Patient Left Behind (NPLB) wrote to the Centers for Medicare and Medicaid services (CMS) urging fixes to its plan for implementation of the Inflation Reduction Act. NPLB suggested fixes that will lower out-of-pocket costs for beneficiaries and protect small molecule innovation.

400+ biotech and pharma executives voice support for the FDA's indispensable role in regulating new medicines. The letter warns that judicial orders undermining the decisions of the FDA would destroy the basic infrastructure necessary for continued medical innovation.

No Patient Left Behind (NPLB) wrote to the Centers for Medicare and Medicaid services (CMS) warning that the exemptions of the Inflation Reduction Act fail to provide adequate protections for orphan drugs and other small molecule treatments.