Developing Standards for Breakthrough Therapy Designation in Oncology
Cet article propose des critères détaillés précisant l'appellation "Breakthrough Therapy" mise en place par l'autorité réglementaire américaine pour accélérer le processus d'autorisation de mise sur le marché de nouveaux médicaments ou traitements ayant démontré une réponse spectaculaire dans des essais de phase précoce
In July 2012, Congress passed the Food and Drug Administration Safety and Innovation Act (FDASIA). The Advancing Breakthrough Therapies for Patients Act was incorporated into a Title of FDASIA to expedite clinical development of new, potential "breakthrough" drugs or treatments that show dramatic responses in early phase studies. Using this regulatory pathway, once a promising new drug candidate is designated as a "Breakthrough Therapy", the FDA and sponsor would collaborate to determine the best path forward to abbreviate the traditional three-phase approach to drug development. The breakthrough legislation requires that an FDA Guidance be drafted that details specific requirements of the bill to aid FDA in implementing requirements of the Act. In this article, we have proposed criteria to define a product as a Breakthrough Therapy, and discussed critical components of the development process that would require flexibility in order to enable expedited development of a Breakthrough Therapy.
Clinical Cancer Research , résumé, 2013