Rare And Relentless
Rare Disease Policy Tracker: FDA & CMS
This living tracker documents official U.S. federal policies, guidance documents, frameworks, and payment models that affect rare and ultra-rare disease research, regulation, and access. It preserves history so progress—or the lack of it—cannot be rewritten over time.
Why This Tracker Exists
Rare disease policy rarely fails loudly. It fails quietly—through frameworks that are optional, guidance that is unevenly applied, and programs that exist on paper but never reach patients. This tracker distinguishes between what is announced, what is implemented, and what remains theoretical.
FDA — Development & Regulation
How evidence is evaluated and approvals are decidedClarifies how FDA may evaluate effectiveness when traditional trials are infeasible in very small populations.
Official program pageCentral library of rare-disease guidance documents used by sponsors and reviewers.
Official hubEncourages adaptive designs and flexibility for cellular and gene therapy trials in very small populations.
Official guidanceSignals FDA’s public rare-disease priorities and themes (not a binding guidance).
Official meeting pageTranslation Gap
Where regulatory flexibility, biomarkers, and small-N evidence often fail to translate into coverage, reimbursement, or timely patient access.
CMS — Coverage & Payment
How approved therapies are paid for—or delayedOutcomes-based agreements to reduce Medicaid budget shock for high-cost gene therapies (state participation optional).
Official model pageDefines outcome measures, payment mechanics, and manufacturer accountability.
Official FAQsCare coordination and outcomes-based approaches (not rare-specific, but potentially precedent-setting).
Official model pageCMS guidance and policy documents impacting market dynamics, including rare-disease incentive discussions.
Official program hubUpdate Log
- January 2026 — Tracker published; CGT FAQs noted as updated January 2026.
- April 2026 — (planned) Quarterly review.
- July 2026 — (planned) Quarterly review.
- October 2026 — (planned) Quarterly review.
