As the wave of highly diverse cell/gene/tissue therapies under development continues to build globally, establishing a viable and mutually agreed upon classification system has become a top regulatory priority. While it is clear that the expanding array of cell/gene/tissue therapies will play an increasingly central role in the medical arena and in the portfolios of pharmaceutical companies, it is not as clear how regulators will cope with the unique manufacturing and control challenges they raise.
The response to Europe’s reflection paper on classifying advanced therapy medicinal products (ATMPs) underscores both the challenging nature and high importance of the definitions in creating effective regulatory pathways for these emerging products.
Released for consultation in mid-2014, the EMA received over 160 pages of comments on the reflection paper from 60 stakeholders. The reflection paper, in turn, grew out of the challenges Europe has been facing in trying to refine its ATMP regulatory processes in the context of legislative and administrative constraints. The products are pushing up against the boundaries of the existing processes and forcing EMA to assess what changes are needed at both the legislative and regulatory levels to accommodate them.
At the CASSS CMC Strategy Forum in Japan in December, INFARMED (Portugal) Senior Assessor Margarida Ferreira, who is playing a key role on the EMA’s Committee on Advanced Therapies (CAT), shed valuable light on “the European landscape for regenerative medicine” and how the regulatory process there is evolving.
The CAT reports to EMA’s Committee for Human Medicinal Products (CHMP) and is tasked with assessing advanced therapies, providing recommendations to the CHMP, and following scientific developments in the field. As such, it has important suasion in determining Europe’s approach in the ATMP arena. Ferreira also serves as the advanced therapies expert on EMA’s Biologics Working Party (BWP). Ferreira’s presentation set the stage for a provocative discussion on the similarities and differences in regulation and classification for advanced therapies between Western and Asian countries, and the barriers to and benefits of harmonization. The issues raised by autologous vs. allogeneic products and differing interpretations of how the respective risks should be handled was a focal point (see IPQ March 26, 2015).