Back to DashboardEst. $45-80M additional cost per drug$680M annual revenue (43% of NovaPharma total)$50M small business ESOP formation fund
Rare Disease Foundation of America
Rare DiseaseGene TherapyOrphan Drugs
12 provisions tracked
3 positive
5 negative
1 conflict
NegativeSec. FDA-2026-AR · FDA Regulatory Reform
FDA Accelerated Approval Reform — Post-Market Evidence Requirements
2 RDFA member therapies with accelerated approval face tighter timelines. Combined with CMS pricing = devastating for rare disease R&D.
ACTION: Deploy patient advocacy coalition to emphasize rare disease impact in public comments
ConflictSec. CMS-2027-03 · CMS Negotiation + FDA Accelerated Approval Reform
GeneVault Therapy — Rare Disease Gene Therapy
RDFA members advocate for patient access AND affordable pricing — internal tension between member companies and patient groups.
ACTION: Navigate dual mandate: protect manufacturer R&D economics while maintaining patient access advocacy
IndirectSec. IRA-ESOP · Legislative — Tax/Employment
Retirement Security Enhancement Act — ESOP Expansion Impact on Health Sector
Indirect benefit if ESOP structures help rare disease companies retain specialized R&D talent.