Experts call for modernizing rare disease evidence models using FDA plausible mechanism framework

A recent paper in The Journal of Clinical Investigation argues that the evidentiary approach for rare diseases needs updating.

The authors suggest applying the FDA’s plausible mechanism framework, which would allow more reliance on causal inference methods to generate evidence when patient numbers are limited.

They contend that such changes could speed development and regulatory review of therapies for rare conditions, though implementation will require collaboration among regulators, researchers and sponsors.