Advocacy Does Not Stop at Approval: RAAP Amplifies Patient Voices on Battling Real-World Barriers to Access

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Michael Eging, Executive and Founder, Rare Access Action Project, Washington, DC, USA

Founded to tackle one of the most persistent blind spots in rare disease policy, the Rare Access Action Project (RAAP) focuses on what happens after US Food and Drug Administration (FDA) approval, when therapies meet the realities of coverage, reimbursement, and real-world access. Led by Executive and Founder Michael Eging, RAAP brings together patient organizations, life sciences leaders, and policy stakeholders to address the structural barriers that can delay or deny treatment to patients who have already waited years for a diagnosis. Shaped by lived experience and grounded in policy expertise, RAAP has become a critical voice in reframing access not as a cost problem, but as the final and decisive step in turning rare disease innovation into patient impact.

“If policy makers are willing to bring in real-world data, the patient voice, and a willingness to compromise, we can arrive at solutions that genuinely benefit patients. That is ultimately the benchmark that should matter.”— Michael Eging

PharmaBoardroom: Could you start by introducing RAAP—its mission, positioning, and current policy priorities?

Michael Eging: RAAP is a nonprofit coalition bringing together patient organizations, life sciences stakeholders, and other partners to address access challenges patients face after FDA approval. While many organizations focus on preapproval issues—which are significant—our work concentrates on what happens once a product is approved and patients need real-world access. We focus on state and federal payment and access issues, including Medicare, Medicaid, and other programs.

RAAP began in 2016 as an ad hoc coalition and became a nonprofit in 2018. It started as a grassroots effort with people coming together around shared needs. My involvement is deeply personal: my father died of a rare disease while I was in college. Being able to apply my experience in life sciences to tackle the same questions my family faced—how to access care, what treatments exist, and what a diagnosis truly means—has been particularly meaningful.

That lived experience is reflected across the RAAP team. Many of our staff members have been personally affected by rare disease. We bring not only professional expertise, but a personal understanding of the challenges patients and families face.

PB: You mentioned that FDA approval is just the first step. What are the biggest disconnects between approval and patient access?

ME: The largest disconnect is that much of the healthcare system is structurally designed to say “no.” Rare disease therapies require tremendous effort to move from discovery through phases 1, 2, and 3, often with phase 4 commitments. That takes enormous risk, investment, and collaboration. Yet once a therapy receives FDA approval, the response often shifts. Patients face prior authorizations, step therapy, and lengthy formulary reviews. Some therapies can take 18 to 36 months, or longer, to gain access through Medicaid programs.

The dissonance is stark. We invest to demonstrate life-changing benefits, but the postapproval system prioritizes cost containment over access.

PB: How does the fragmentation of the US payer system, across commercial insurance, Medicare, and Medicaid, affect patient access compared with more centralized European systems?

ME: Many rare disease therapies approved in the United States never launch in other markets. Patient populations are extremely small, and navigating the regulatory and access frameworks outside the United States is uncertain. Even in European single-payer systems, innovation does not always translate into access.

For patients in the United States, navigating Medicaid, Medicare, and commercial insurance is complex. Each program has different requirements, and patients often need substantial support. RAAP connects patients with these resources while advocating to address policy barriers.

I saw this first-hand during the launch of a rare pediatric epilepsy therapy. FDA approval was achieved, and children could finally access the treatment. But as we navigated Medicaid, in one state the therapy was routed through an ophthalmology pharmacy and therapeutics (P&T) committee. There were no rare disease specialists involved. The result was a burdensome prior authorization process that discouraged clinicians and families.

These disconnects are common. Some advocacy organizations have strong voices, but many lack resources to sustain advocacy or even alert patients to barriers they will face.

PB: Given the expertise gaps on review committees and the insurance and policy barriers you described, what policy priorities is RAAP focusing on today?

ME: Our priorities focus on removing postapproval barriers. One example is to model legislation requiring states to embed patients and rare disease specialists in decision making. Beyond rare disease advisory councils, this legislation would require P&T committees, drug utilization review boards, or prescription drug affordability boards to include a patient, a patient advocate, and a physician with relevant rare disease expertise as a voting member whenever a rare-indicated therapy is under review.

By integrating patients and clinicians into these discussions, we connect scientific and regulatory understanding with lived experience. The result is better-informed coverage decisions and a smoother patient journey beyond FDA approval.

PB: Does patient-led development translate into patient involvement in access decisions?

ME: At RAAP, patients are genuinely at the table. We have a Patient Engagement Caucus that is patient-led, and our state and federal committees are cochaired by life sciences and patient advocates. Many issues originate from the patient caucus and are elevated through that structure.

We support patients through education—webinars, white papers, and outreach. We also bring them to policy maker meetings so they can discuss gaps and potential solutions.

By integrating patients and clinicians into these discussions, we connect scientific and regulatory understanding with lived experience. The result is better-informed coverage decisions and a smoother patient journey beyond FDA approval.

PB: The Orphan Drug Act (ODA) is foundational for rare disease. How does it function in today’s environment?

My father died of a rare disease without a treatment. Today, 40-plus years later, the landscape is different. There are therapies even for my father’s cancer. A family friend lived nearly 18 months beyond diagnosis, compared with my father’s 6 months. That is real progress.

The ODA provided the foundation, but accelerated approval pathways, surrogate endpoints, streamlined clinical programs, and pediatric rare disease vouchers have transformed the development landscape. They allow developers to ask if pursuing a therapy for 200 patients can both impact lives and generate a return on investment.

There are proposals to restrict Medicaid reimbursement for accelerated approval therapies, arguing that evidence is insufficient. In rare disease, particularly ultra-rare conditions, traditional trials may be impossible. These tools exist to deliver life-changing treatments.

I was involved in launching a gene therapy for spinal muscular atrophy. Historically, many children died before age 2. Today, some are attending kindergarten. This demonstrates that early patient engagement, combined with thoughtful policy and access planning, doesn’t just accelerate approval; it ensures therapies actually reach the patients who need them. Going forward, additional tools, including artificial intelligence-enabled modeling and smarter priority review mechanisms, can further accelerate discovery and improve both outcomes and access.

PB: How far have outcomes-based and risk-sharing payment models progressed in rare disease, and what would it take to scale them meaningfully in the United States?

We operate in a 21st-century system built largely in the 1990s and early 2000s, with remnants from the 1970s and 1980s. That system struggles to absorb personalized therapies.

For rare disease, we need models that stabilize the marketplace and ensure patients can access therapies at FDA approval. Value-based pricing and payment over time, particularly for cell and gene therapies, are increasingly important.

I am especially interested in Medicaid reinsurance and secondary reinsurance markets to stabilize costs. Arizona offers an example. Since the 1990s, the state has led in risk pooling and innovative financing. Rare disease is like a genetic lightning strike, but it is insurable—much like catastrophic events for which insurance models already exist. We need systems that provide the right coverage at the right time.

Delays can be devastating. Imagine launching a gene therapy and Medicaid takes 24–36 months to decide on coverage. Infants may age out of the label before treatment. Rare disease patients cannot wait. Sustainable access models are critical to ensure research, development, and FDA approvals translate into real patient impact.

Rare disease is like a genetic lightning strike, but it is insurable—much like catastrophic events for which insurance models already exist. We need systems that provide the right coverage at the right time.

PB: To what extent do state-by-state differences in Medicaid coverage create access barriers?

ME: If you have seen one Medicaid program, you have seen one Medicaid program. States operate under different waivers, payment models, coverage criteria, and administrative rules, which creates significant variation in how care is accessed and reimbursed.

Many rare disease patients, particularly pediatric patients who are more likely to rely on Medicaid, must travel across state lines to access centers of excellence. When they do, reimbursement rates, coverage criteria, prior authorization requirements, and drug acquisition costs often differ. That creates an administrative burden for providers and significant exhaustion for patients and families.

There are ongoing discussions about aligning eligibility and access criteria across states, but today the system remains a patchwork. That fragmentation is a material barrier to access and a major source of burden for the rare disease community.

If you have invested years of scientific effort, human capital, and patient participation to bring a therapy to market, it makes no sense for access to become the next battleground without the patient voice present.

PB: What other topics need to be further emphasized?

One area I would emphasize is the growing uncertainty patients face as a result of recent policy initiatives. Between the Inflation Reduction Act, proposed fixes in Congress, state-level price control mechanisms, and most-favored nation pricing, we are seeing real instability in coverage. A patient who has access today may not have access tomorrow.

We need to step back and re-orient the discussion around long-term solutions that provide certainty of coverage. If patients are placed at the center of these conversations, it is possible to lower out-of-pocket costs while maintaining a viable marketplace for plans and manufacturers and managing risk in rare disease coverage.

There are constructive paths forward, but they require collaboration rather than political point-scoring. If policy makers are willing to bring in real-world data, the patient voice, and a willingness to compromise, we can arrive at solutions that genuinely benefit patients. That is ultimately the benchmark that should matter.

PB: What is your final message?

ME: Pharmaceutical executives already know rare disease patients well through development programs, clinical trials, and FDA engagement. These patients are informed, articulate, and deeply invested in their therapies.

Once a drug is approved, I would strongly encourage companies to support and empower those patients to stay involved. Advocacy does not stop at approval. If you have invested years of scientific effort, human capital, and patient participation to bring a therapy to market, it makes no sense for access to become the next battleground without the patient voice present.

These patients are ready to engage. They understand the therapy, its impact and the stakes. Linking them with organizations such as RAAP and others working on access and policy ensures that the people most affected are part of the conversation. That is how we make sure the innovation your teams deliver actually reaches the patients it was designed to help.