April 28, 2015
The Accelerated Approval pathway serves a vital role for the development of treatments for diseases with high unmet medical needs. An article published in the Orphanet Journal of Rare Diseases describes a scientific framework to assess biomarker endpoints with defined sets of supporting data. This more structured approach will enhance the development of novel orphan drugs for rare diseases that are currently without adequate treatment. Assessment is based on the opinions of experts in drug development and rare disease patient groups. The authors suggest the following recommendations for the development of orphan drugs using the Accelerated Approval pathway and to qualify biomarkers as primary endpoints:
-Establish regulatory rationale for accelerated approval access in rare disease programmes
-Implement a biomarker qualification request process
-Propose a scientific framework to qualify biomarkers as surrogate primary endpoints
Although this article mainly describes examples from the FDA, they are also relevant to sponsors applying for marketing authorisation in other world regions.