Rare Disease Workshop 5: Post Marketing Confirmatory Studies in Rare Diseases. Susan Boynton, VP, Global Regulatory Affairs, Shire Mary O'Donovan, Senior Director, Regulatory Affairs & Policy, Biomarin. Accelerated Approval: Requirements Under §314.510.
Susan Boynton, VP, Global Regulatory Affairs, Shire
Mary O'Donovan, Senior Director, Regulatory Affairs & Policy, Biomarin
“Approval ….subject to the requirement that the applicant study the drug further, to verify and describe its clinical benefit
where there is uncertainty as to the relation of the surrogate endpoint to clinical benefit, or of the observed clinical benefit to ultimate outcome.
Post marketing studies would usually be studies already underway.
When required to be conducted, such studies must also be adequate and well-controlled.
The applicant shall carry out any such studies with due diligence.”
What additional sources of long term robust data could be considered to support adequate and well controlled standard?
What other alternative sources of data could be used to obtain adequate and well controlled confirmatory data?
Slow progressing but debilitating diseases – how long is acceptable to wait to get confirmation?
How to prevent discontinuations from long term studies once product placed on market?
When is it best to start discussion around AA post marketing confirmatory study design and feasibility?
How do we address the commercial challenge of long term studies. Concept of a "Disease Monitoring Program” (DMP) encompassing registry/natural history /long-term extension study in one (Emil Kakkis)