Masterstudiengang "Drug Regulatory Affairs"

Master-Thesis

FDASIA "breakthrough" drug therapies – a real regulatory breakthrough for innovations? ***

Silvia Gonzalez (Abschlußjahr: 2013)

Summary
Language: English
The "Breakthrough therapy" (BT) designation constitutes a new expedited development and review tool, introduced with the Food and Drug Administration Safety and Innovation Act (FDASIA) in July 2012. It alludes to innovative drugs for serious conditions, where early clinical data show the potential of substantial superiority over available treatments.
Evaluating the FDASIA law, the new draft Guidance for Industry on "Expedited Programs for Serious Conditions" and information available on the FDA website, the qualifying criteria, designation procedure, incentives and dossier requirements are outlined. Analyzing companies’ press releases and trade press, the drugs for which BT designations have been granted and announced within the first year after the FDASIA are examined.
The BT designation focuses on accelerating the development phase and shall complement the expedited programs Fast track, Priority review and Accelerated approval, which are already well-established in the US. Their characteristics are presented and opposed to the new BT designation.
Furthermore, the tools to enable an early market access in the US are compared to the programs established in the European Union, showing that there are currently no tools available in the EU that foster the development phase of drugs, comparable to the BT- or Fast track designation.
The BT designation reveals to be a unique tool, offering some exceptional features like involvement of senior FDA managers, designation of a cross-disciplinary project lead and close cooperation to design an efficient clinical development program, tailored to the drug. – The latter paves the way for smaller, shorter and more flexible clinical trial designs, potentially cutting off years from clinical development. However, since so far most of the BT designated drugs are already far ahead in clinical development, the full implications on the clinical development will need to show in the future for drugs that are still early in development at time of designation.
Nevertheless, published experiences prove that a BT designation does already make a big difference. Very close FDA cooperation is lived for BT designated drugs: There is frequent and interactive exchange throughout the drug development and the FDA is proactive in providing timely and tailored advice. Eventually, this helped to put Breakthrough therapies a big step ahead of the planned development schedule, already.
Thus, the question raised in the title can be answered in the affirmative:
FDASIA’s BT designation does constitute a real regulatory breakthrough for innovations.
However, the Breakthrough therapy designation so far constitutes a US-specific tool. And since today most drugs are developed globally, this bears the risk that the abbreviated clinical trials will not suffice to support an approval in other countries.
The Breakthrough therapy initiative in the US therefore strongly calls for the adoption of comparable approaches in the other ICH regions, to enable a global development strategy.
Pages: 61, Annexes: 9 pages