Further Volatility Ahead
2026 will also likely bring further volatility across pricing and regulatory policy, if the scale and speed of 2025’s changes are anything to go by. Policies such as the FDA Commissioner’s National Priority Review Vouchers, or guidance around treatment of CAR-T cell therapies, remain incomplete or opaque. This uncertainty has led some investors to steer clear of complex, high-risk modalities in favor of tried-and-tested mechanisms in more widespread, better-understood conditions.
Biohaven’s pivot from spinal muscular atrophy (SMA) to obesity, though science driven, fits the mood: the biotech was testing its myostatin-targeting drug taldefgrobep alfa in SMA, but Phase 3 results were uncompelling. Now it is doubling down on the drug’s potential in obesity, where it hopes to uncover muscle-preserving benefits that will differentiate it from GLP-1 agonists. Biohaven CEO Vlad Coric warned of a “dire time” for rare diseases patients in a February 2026 Fierce Biotech interview, given the spate of FDA rejections, including of Biohaven’s own troriluzole in SCA. The company is appealing.
Payers now face reimbursement claims for multiple high-priced gene- and cell-therapies.
Orphan drugs have been less exposed to pricing pressure than mainstream therapies. This is changing, though, as the number of orphan drugs continues to rise. Payers now face reimbursement claims for multiple high-priced gene- and cell-therapies, addressing conditions suffered, collectively, by many more patients. Sickle cell disease, for instance, affects over 100,000 Americans. The US Centers for Medicare and Medicaid Services’ (CMS’) CGT Access Model in early 2024 introduced a common outcomes-based agreement framework for Medicaid and the sponsors of two SCD gene therapies, Vertex / CRISPR’s Casgevy and Bluebird bio’s Lyfgenia. These one-time therapies cost over $2 million each; the program is designed to provide rebates if outcomes fall short and may in future include other cell or gene therapies.
China’s rise could add further pricing pressure on orphans sold in the West, if China-licensed molecules result in more crowding and competition. China itself has introduced incentives for rare diseases R&D, including priority review and some market exclusivity, but there is no formal definition of “orphan drug”, and coverage of such drugs is rare7. Other Asian countries including Taiwan, South Korea and Japan have established orphan drug development incentives and access policies.
Renewed investor interest in biopharma, after a multiyear downturn, may mitigate pricing and policy uncertainty around orphan drugs. But those questions could just as likely send investors flocking to what they know: tried and tested modalities in much bigger markets.
Join our expert panel as they take a closer look at the data from this report, and discuss the forces shaping the orphan drugs market in 2026.
How orphan drug sales and pipelines are evolving amid uncertainty
Which companies and franchises are driving rare disease growth, and which are losing momentum
The impact of recent US policy shifts on orphan drug development, including the Inflation Reduction Act
What portfolio teams and investors must prioritize to compete for value in rare diseases in 2026 and beyond
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Analyst and author of the Evaluate Orphan Drugs Report
Manager, Content Strategy, Evaluate
