RepurpOS

Right to Try Compendium

Unmet medical needs: 106 diseases without FDA-approved disease-modifying therapy where right-to-try access frameworks may apply.

Two Scopes of Right-to-Try Eligibility

Both criteria qualify diseases for right-to-try access, but with different breadth:

Narrower Scope: "Zero Approved Agents"

Diseases with absolutely no approved drugs in any class, even for symptom management. Most restrictive eligibility.

Examples:

  • Mastocytosis with KIT D816V (no treatments)
  • Sjögren's Syndrome (no treatments)
Count: 3 diseases

Broader Scope: "No Indication-Specific Approval" ✓ (Legal Standard)

Diseases with generic symptom-management drugs, but NO disease-modifying therapy specifically approved for THAT disease. This is the legal definition under federal and state right-to-try laws.

Examples:

  • ME/CFS: Has antidepressants for depression → but not for ME/CFS recovery
  • Long COVID: Has anticoagulants for blood clots → but not for post-viral syndrome
  • Alzheimer's: 233 approved drugs → zero disease-modifying therapies for Alzheimer's cognitive decline
Count: 30 diseases (THIS compendium)
Legal Standard: 21 U.S.C. § 360bbb-0a (federal) and Montana SB 535 both require lack of "adequate approved therapy" for the specific disease. A drug approved for hypertension doesn't count as therapy for ME/CFS eligibility, even though it's Phase 4. Right-to-try is about having no disease-modifying therapy for THIS indication.

Related static tools: Right-to-Try laws by US state · Procedure access arbitrage map · Gene therapy mapper

Compendium: 106 Compliant Diseases Without Disease-Modifying Therapy

Sorted by most promising investigational candidates (Phase 3 agents first). Critical/life-threatening diseases flagged.

Right-to-Try Registry. Legal frameworks: 21 U.S.C. § 360bbb-0a (federal) and Montana SB 535. Data: Open Targets, ClinicalTrials.gov, OSMF curation. Not medical or legal advice.