New G-BA methodology update shifts evidence expectations for rare disease

Germany's G-BA has updated its benefit assessment methodology for orphan drugs — tightening comparator standards and raising the bar for endpoint acceptance.

The G-BA has published final updated methodology guidance for benefit assessments of orphan medicinal products. The revision tightens the definition of an appropriate comparator and introduces stricter requirements for patient-relevant endpoints in rare disease dossiers, effective for submissions filed from 1 October 2026.

Sponsors with dossiers currently in preparation should review their comparator selection rationale and endpoint hierarchy against the new guidance before submission. HEORvia's dossier builder has been updated to reflect the revised G-BA templates. For a full analysis of the methodology changes and their impact on your portfolio, use HEORvia's reimbursement tracker.