Rare Disease Access at Risk: What Biopharma Leaders Need to Know About the 2025 Medicaid Overhaul
Medicaid, covering roughly 1 in 5 Americans, is the largest payer for people with complex, chronic, and rare conditions. Rare diseases affect an estimated 25–30 million people in the United States, and a substantial share of this population depends on Medicaid for high‑cost therapies and diagnostics.
In July 2025, Congress passed the One Big Beautiful Big Act (OBBBA), a sweeping tax-and-spending law that implements a historic Medicaid overhaul and significant updates to the Medicare Drug Negotiation Program. These changes represent the most consequential restructuring of public coverage programs since the Affordable Care Act, with more extensive verification and other bureaucratic requirements for Medicaid recipients to be imposed by the end of December 2026.
Independent and advocacy analyses warn of large coverage losses, higher patient cost exposure, and new administrative frictions, particularly for low-income adults who depend on Medicaid for specialty care and rare disease therapies. As those with rare diseases often require continuous therapies and close monitoring, the impact of cost and coverage changes is likely to be magnified.
Policy Overview: Key Medicaid Changes in the OBBBA
The One Big Beautiful Bill Act introduces three structural Medicaid reforms with direct implications for rare disease patients and the manufacturers that serve them.
More Frequent Work and Eligibility Verifications
Starting as early as January 2027, US adults must document at least 80 hours per month of work, job training, community service, or part-time school enrollment to remain Medicaid eligible (unless they meet an exemption).
This policy penalizes Medicaid beneficiaries who are unemployed, struggling to secure stable work, or unable to access reliable transportation. Subsequently, this shift will impact more than 2.6 million adults with disabilities who do not receive supplemental security income (SSI) or social security disability insurance (SSDI) and face significant challenges working due to their health conditions.
Recent research indicates that eligibility verifications and confirmation of employment status have not demonstrated an increase in employment rates, but an increase in overhead costs and coverage losses, even among eligible beneficiaries.
Mandatory State Cost-Sharing Requirements
Medicaid expansion adults—low-income adults covered through the ACA expansion rather than disability-based Medicaid—are now subject to new cost-sharing requirements, with states required to impose charges of up to approximately $35 per service. For chronically ill or rare disease patients who rely on frequent specialty visits, labs, and diagnostics, these out-of-pocket costs can pile up quickly, increasing the risk of delayed or skipped care.
Consequences of Reduced Medicaid Compliance
Compliance and administrative barriers don’t just inconvenience patients; they have measurable clinical and economic consequences for people with rare and chronic diseases and for the health system as a whole.
For rare disease patients who rely on continuous specialty care and high-touch treatment plans, these barriers can quickly translate into avoidable disease progression, new comorbidities, and poor quality of life. The burden is not evenly shared: administrative hurdles and rising out-of-pocket costs disproportionately affect low-income patients, people of color, and adults with disabilities. These groups already face documented inequities in rare disease access. As Medicaid requirements become more stringent, these policies risk widening disparities, increasing downstream costs, and placing additional strain on providers, hospitals, and patient-support programs.
Safeguarding Patient Access
To effectively navigate the Medicaid overhaul introduced by the OBBBA, rare disease manufacturers will need to prioritize safeguarding patient access amid rising coverage instability. Achieving this will require a more proactive access strategy: one that strengthens patient retention and coverage support, deepens engagement with patient advocacy organizations, and builds Medicaid-specific bridge and continuity programs.
Strengthen Patient Retention and Coverage Support
As Medicaid eligibility becomes more fragile, manufacturers need to expand traditional patient-support programs into a coverage-stability infrastructure. This includes:
- Hands-on documentation and verification support to help patients complete new eligibility checks, a known driver of coverage losses even among qualified enrollees.
- Automated redetermination reminders delivered through text, email, and patient portals to reduce avoidable lapses.
- Cost-sharing support targeted to the most critical services, especially for patients with complex care needs who may now face new or increased OOP responsibilities.
- Partnerships with social-service and community-health organizations, which are positioned to help patients navigate transportation barriers, administrative complexity, and fluctuating employment.
Strengthening these resources not only maintains therapy adherence but also protects clinical outcomes in patient populations that already face substantial diagnostic and treatment hurdles.
Advocacy organizations will be essential in mitigating confusion during Medicaid’s transition. Biopharma companies can collaborate with these groups to ensure patients receive consistent, accurate guidance on:
- Eligibility and exemption criteria, including which rare disease patients qualify for work-requirement exceptions.
- Documentation requirements and timelines, which are expected to become more frequent and more complex under OBBBA.
- Continuity-of-coverage resources, such as community-based help centers, re-enrollment support, and manufacturer assistance programs.
Co-created toolkits, webinars, and patient-friendly FAQs can reduce misinformation and help minimize coverage churn, particularly for individuals with rare, chronic, or debilitating conditions who struggle with administrative tasks.
Build Medicaid-Specific Bridge and Continuity Programs
Given the heightened likelihood of temporary coverage gaps, manufacturers should redesign bridge programs to address Medicaid-specific friction points. Evolved programs could include:
- Longer-duration safety nets that cover patients throughout verification cycles, not just during commercial insurance transitions.
- Integrated re-enrollment navigation, pairing free-drug or copay support with hands-on help completing Medicaid paperwork.
- Customized support for rare disease journeys, such as long diagnostic pathways, recurring specialty diagnostics, and high-cost therapies where any disruption poses significant health risks.
By stabilizing therapy access during administrative outages or eligibility reviews, biopharma companies can prevent treatment abandonment and protect long-term patient outcomes.
The Inflection Point
The Medicaid overhaul within the One Big Beautiful Bill Act represents a pivotal inflection point for rare disease care. For rare disease manufacturers, succeeding in this new environment will require moving quickly to reinforce patient-access infrastructure. Doing so will require a coordinated strategy across three critical areas: patient retention and coverage support, patient advocacy engagement, and Medicaid-specific bridge and continuity programs.
To continue the conversation on navigating these changes and strengthening patient access, please contact Yakir Siegal, Managing Director, or Sam Rieger, Consultant, at Asymmetry Group.
References:
Alliance for Health Policy. (2024). Rare Disease Policy Gaps and Opportunities for 2025 and Beyond. https://www.allhealthpolicy.org/news/the-alliance-for-health-policy-releases-insights-report-highlighting-rare-disease-policy-gaps-and-opportunities-for-2025-and-beyond
Center for American Progress. (2025). The truth about the One Big Beautiful Bill Act’s cuts to Medicaid and Medicare. AmericanProgress. https://www.americanprogress.org/article/the-truth-about-the-one-big-beautiful-bill-acts-cuts-to-medicaid-and-medicare/
CNBC. (2025, July 8). Medicaid cuts in Trump’s megabill will hit some drugmakers. CNBC. https://www.cnbc.com/2025/07/08/healthy-returns-medicaid-cuts-in-trumps-megabill-will-hit-some-drugmakers.html
EveryLife Foundation for Rare Diseases. (2025). Why is Medicaid Critical for the Rare Disease Community? EveryLife Foundation. https://everylifefoundation.org/why-is-medicaid-critical-for-the-rare-disease-community/
Georgetown Center for Children and Families. (2025, June 26). One Big Beautiful Bill Act: Winners and losers in the Medicaid provisions. https://ccf.georgetown.edu/2025/06/26/one-big-beautiful-bill-act-winners-and-losers-in-the-medicaid-provisions/
Johns Hopkins Bloomberg School of Public Health. (2025). The changes coming to the ACA, Medicaid, and Medicare. https://publichealth.jhu.edu/2025/the-changes-coming-to-the-aca-medicaid-and-medicare
Morgan Lewis. (2025, July). Orphan drugs: Big breaks — the quiet carve-out in the One Big Beautiful Bill Act. Morgan Lewis ASPrescribed. https://www.morganlewis.com/blogs/asprescribed/2025/07/orphan-drugs-big-breaks-the-quiet-carve-out-in-the-one-big-beautiful-bill-act
NPR. (2025, July 2). Senate Republicans’ tax bill and Medicaid health-care effects. NPR. https://www.npr.org/sections/shots-health-news/2025/07/02/nx-s1-5453870/senate-republicans-tax-bill-medicaid-health-care
PwC Health Industries. (2025). Impact of OBBBA on U.S. health system. PwC. https://www.pwc.com/us/en/industries/health-industries/library/impact-of-obbba-on-us-health-system.html
Roberts, E. T., Kwon, Y., Hames, A. G., McWilliams, J. M., Ayanian, J. Z., & Tipirneni, R. (2023, June). Racial and Ethnic Disparities in Health Care Use and Access Associated With Loss of Medicaid Supplemental Insurance Eligibility Above the Federal Poverty Level. JAMA Internal Medicine. https://pubmed.ncbi.nlm.nih.gov/37036727/