On April 9, 2026, CCPA hosted “Drug Pricing Reform: What Patients Need to Know,” a webinar moderated by CCPA CEO Liz Helms. The panel featured Sue Peschin and Adina Lasser of the Alliance for Aging Research; Sara van Geertruyden of the Partnership to Improve Patient Care; Jay Greissing of BioMarin; and rare disease patient advocate Dominick Spatafora. Together, the panelists offered patients, caregivers, and advocates a 360-degree view of today’s drug pricing landscape and what it means for access, innovation, and patient care.
Liz Helms set the stage by providing a background of the frameworks currently shaping drug pricing, including the Inflation Reduction Act (IRA); the Trump Administration’s Most Favored Nation (MFN) executive order linking U.S. prices to international benchmarks; and two new mandatory CMS models: GLOBE (Global Benchmark for Efficient Drug Pricing) and GUARD (Guarding U.S. Medicare Against Rising Drug Costs). Keeping up with the pace of change is itself a challenge, she noted, and the goal of the webinar was to help the patient community make sense of what’s at stake.
Diving deeper, panelist Sue Peschin assessed what the IRA’s Medicare Drug Price Negotiation Program has meant in practice. Because CMS targeted the highest-volume drugs to maximize savings for the Medicare program itself, not patients, only about 11% of beneficiaries on the 10 negotiated drugs are expected to see a cost reduction. Yet, plans are responding with formulary changes, tier shifts, and increased prior authorization that push costs back onto patients. Peschin acknowledged some wins within the IRA – the first-ever Part D out-of-pocket cap and the Medicare Prescription Payment Plan – but cautioned that MFN would severely harm future innovation, and that better paths exist to address the U.S.-international pricing gap.
BioMarin’s Jay Greissing further explained how price-setting policies like MFN shape where investment capital flows in the biopharmaceutical industry. He cited a recent Incubate Coalition survey finding that roughly 90% of venture capital and institutional investors would reduce biopharmaceutical investment if MFN-style price controls advance. For rare diseases, the stakes are especially high, as Medicaid is the primary payer for 30–50% of rare disease patients, and when early revenue projections shrink, programs may never launch at all, which means fewer potential cures coming to market.
The risk to research and development is especially concerning for Neuropathy Action Foundation’s Dominick Spatafora, a rare disease patient living with multifocal motor neuropathy (MMN). He spoke from personal experience about the physical, emotional, and financial burden of chronic illness and what federal and state policymakers consistently get wrong. A family of five with the same allergy condition may each need a different medication – and drug pricing policy needs to account for that. “Treat me as an individual with multifocal motor neuropathy,” he said, “not a statistic in the population.”
Adina Lasser then walked attendees through the GLOBE and GUARD “models,” which were announced by the Trump Administration in December 2025 through the CMS Innovation Center. Though labeled as “demonstration models,” they are effectively mandatory as manufacturers have no choice but to participate. The models prioritize government savings rather than improved patient affordability, and would impact approximately 25% of Medicare fee-for-service beneficiaries across both Part B and Part D. Framing these models as simply “matching what other countries pay” obscures what’s really happening: importing the rationing systems those countries use to keep prices low. The IRA’s track record proves this point – only 11% of beneficiaries are seeing actual savings, while premiums, coinsurance, and utilization management (prior authorization, step therapy, etc.) have increased. CCPA and many partner organizations have called on CMS to withdraw both proposals.
Sara van Geertruyden further explained how rationing systems like Quality-Adjusted Life Years (QALYs) are dangerous for all patients. Under the QALY framework used by the Institute for Clinical and Economic Review (ICER), a person living with a condition like multiple sclerosis may be assigned a value of 0.5, meaning their life is considered worth half as much as a fully healthy person’s life in any given year.
QALYs are inherently discriminatory: they devalue treatments for people with disabilities and perpetuate stereotypes. The QALY was developed in the UK specifically as a rationing tool, and other countries use it to restrict access to essential treatments. Medicare law already bars the use of QALYs in coverage and reimbursement decisions, yet MFN-style international reference pricing would bring in rationing through the back door. She highlighted the importance of the Protecting Healthcare for All Patients Act, which would extend this prohibition to other federal programs. The bill passed the House in the last Congress.
As far as next steps, several panelists flagged the urgent need to continue educating members of Congress about preventing Most Favored Nation from being enshrined in law – through stand-alone legislation or the budget reconciliation process. Reconciliation requires only a simple Senate majority, making this a real risk.
Key Takeaways
- The distinction between government savings and patient savings matters. Price-setting programs are primarily designed to reduce what Medicare pays, not what patients pay at the pharmacy counter.
- Every patient is an individual. Population-based cost measures that ignore individual genetics, conditions, and treatment histories put real people at risk. For patients who have had claims denied or delayed, it’s imperative to appeal – resubmitted appeals have a much higher success rate.
- Importing international pricing means importing rationing. Other countries pay less for drugs because they cover fewer of them, restrict access, and wait years longer for approvals.
- Innovation is not inevitable. When R&D investment becomes unpredictable, new programs – especially for rare diseases – may never get started. That directly results in fewer new treatments and potential cures coming to market.
- Patient voices change outcomes. Personal stories and direct engagement with policymakers remain among the most powerful tools advocates have.
For more information and resources, visit chroniccarealliance.org. You can view the complete webinar recording here.