The Orphan Diseases Market: Why 7,000 Rare Diseases Remain Untreated
Objective
Analyze the market failure preventing profitable development of treatments for 7,000+ rare diseases affecting 400M people
Methodology
Comparative market analysis of rare vs. common disease development economics. Analysis of current policy solutions (orphan drug acts, grants, philanthropy) and their limitations. Modeling of alternative financing mechanisms.
Findings
Rare disease drug development costs $1-3B with patient populations averaging 5K-50K globally. At $100K/patient/year, total addressable market ($500M-5B) barely covers development costs, creating fundamental market failure. Orphan drug acts have yielded only 400-500 treatments in 50 years. Outcome-verified pull funds can restructure incentives by decoupling investor returns from patient payment capacity.
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Evaluation Scores
Data Sources
Pharmaceutical Research & Manufacturers Association (PRMA) rare disease survey
Reliability: 95%
