Why FDA Approval Is No Longer Enough for Rare Disease Access

US rare disease developers must redesign evidence strategy to serve payers, not just regulators, NORD's 2026 Symposium finds

11 May 2026

Fewer than 5% of the more than 7,000 identified rare diseases have an approved treatment in the United States. That figure captures a familiar failure of ambition. A less familiar one sits just behind it: drugs that do win approval often fail to reach the patients who need them anyway.

At the second annual NORD Rare Disease Scientific Symposium, held in Arlington, Virginia this April, the diagnosis was delivered plainly. Ravi Pathak of Takeda put it directly: evidence is built for regulators, not payers. The problem is structural. Drug developers design clinical trials to satisfy the Food and Drug Administration. Once approval is secured, they discover that insurers and government programmes want something else entirely: proof of real-world value, affordability, and impact on outcomes beyond those captured in a trial.

Danny Yeh of Aesera reinforced the point: access decisions require proof of value, affordability, and real-world impact. Without that proof, payers hesitate. Approval becomes a milestone that changes little.

The gap is widening at an inconvenient moment. Orphan drugs, designed for small patient populations, now account for close to half of all new FDA approvals. That concentration has drawn scrutiny. American government programmes have begun negotiating prices for some orphan therapies, and tighter payer controls are spreading. A developer who treats regulatory approval as the finish line is increasingly likely to find that the race continues beyond it, with different rules and a more sceptical audience.

Annica Wayman, Deputy Director of the National Centre for Advancing Translational Sciences at the National Institutes of Health, set an institutional ambition at the symposium: raising the share of rare diseases with an approved treatment from 5% to 25%. That is a plausible goal only if the incentive to develop treatments is matched by a realistic path to patient access.

The prescription is not complicated, though it requires discipline early. From the first stages of development, payer requirements must shape how trials are designed, what data is collected, and how value is measured. Access, in other words, must be treated as a design constraint rather than a problem to solve after the fact. For over 30 million Americans still without a treatment option, the cost of that delayed realisation is borne most directly.