At AMCP Nexus 2025, leaders explored how the health care system can evolve from “listening to patients” to formally integrating patient experience into payer decision-making. The session highlighted new federal mandates requiring greater patient participation in Medicaid and commercial health plan governance—marking a pivotal moment in the movement toward patient-centered access and coverage decisions.

A New Era for Patient Engagement in Coverage Policy

Jamie Sullivan, MPH, Vice President of Policy at the EveryLife Foundation for Rare Diseases, opened the session by framing rare disease as both a public health and economic crisis. With more than 10,000 rare diseases—95% lacking US Food and Drug Administration (FDA)-approved treatments—families bear the majority of the nearly $1 trillion annual economic burden through indirect and nonmedical costs. Sullivan underscored that while a decade of progress in patient-focused drug development (PFDD) has advanced regulatory engagement, similar patient-centered approaches must now extend into the payer and access ecosystem.

Two new Centers for Medicare & Medicaid Services (CMS) rules are driving this shift. The first mandates that state Medicaid agencies establish Beneficiary Advisory Councils, ensuring patient and family perspectives influence policy implementation. The second requires that exchange health plans include at least 1 patient representative on their Pharmacy and Therapeutics (P&T) committees beginning January 1. Sullivan emphasized that these regulations—if implemented meaningfully—can transform patient engagement from a compliance exercise into a mechanism for sustained learning and improvement across the health care landscape.

Building a Culture of Inclusion

Jennifer S. Graff, PharmD, presented practical lessons drawn from payer interviews, patient organizations, and state Medicaid leaders. She stressed that successful implementation begins with culture: senior-level sponsorship, designated points of contact, and a commitment to trust-building are essential to integrating patient voices effectively.

Graff outlined key steps for plans preparing to onboard patient representatives to pharmacy and therapeutics (P&T) committees. Clarifying the role, recruiting intentionally, and equipping both patients and committee members for success were identified as central to meaningful engagement. Compensation, logistical accessibility, and caregiver support must also be considered. She cautioned that simply placing a patient in the room is insufficient—true engagement requires role definition, support, and an environment conducive to open dialogue.

She encouraged payers to think beyond regulatory compliance and consider how patient perspectives can inform utilization management, care coordination, and value-based contract design. “We can only build progress at the speed of trust,” she noted, emphasizing that cultural readiness often determines whether engagement efforts succeed or stall.

Lessons from the Field: Friedreich’s Ataxia and the Fight for Access

Brigid Brennan, JD, representing the Friedreich’s Ataxia Research Alliance (FARA), provided a firsthand account of patient advocacy in action following the approval of omaveloxolone, the first FDA-approved therapy for Friedreich’s ataxia (FA). Brennan, both an advocate and mother to a son with FA, described the extensive role her community played in advancing research and ensuring the patient voice was present throughout the drug’s development and regulatory review.

Yet, as Brennan detailed, FDA approval did not automatically translate into access. Her organization engaged in 11 Medicaid hearings to address restrictive prior authorization criteria that excluded many patients who could benefit from treatment. Criteria such as mobility requirements or heart function thresholds reflected outdated clinical trial designs rather than real-world clinical need. FARA’s testimony helped states adjust coverage policies to better align with patient realities, though barriers remain in some jurisdictions.

Brennan’s account illuminated the practical disconnect between research, regulation, and reimbursement—and the critical need for payer engagement with patient communities to ensure policies reflect lived experience rather than procedural convenience. She urged payers to recognize that patient organizations bring not anecdotes, but robust data and insights capable of improving both the fairness and efficiency of access decisions.

Sustaining Partnership Through Training and Evaluation

As the session concluded, panelists returned to the question of sustainability. Graff and Sullivan stressed that training and continued partnership between patient groups and payers will be crucial as these new regulations take effect. Evaluation processes—such as post-meeting surveys and annual reviews—can help refine how patient insights are used to inform decisions and improve committee function over time.

The EveryLife Foundation has developed a series of toolkits and resources to guide both patients and payers through these transitions, offering frameworks for onboarding, engagement, and evaluation. Ultimately, the speakers agreed that embedding patient experience into access policy is not a one-time adjustment but a long-term cultural evolution that will require collaboration, transparency, and shared learning.

Reference

Brennan B, Graff JS, Sullivan J. Rare Expertise. Rare Data. Real Impact: Best Practices for Embedding Patient Experience Data from Medicaid Beneficiary Advisory Councils and Learnings for Managed Care Pharmacy. Presented at: AMCP Nexus 2025; October 27-30; National Harbor, MD.