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.