BIO Comments on WHO White Paper: Regulation of Cell and Gene Therapy Products
Today, BIO submitted comments to the World Health Organization (WHO) regarding its revised, draft white paper on the regulation of Cell and Gene Therapy Products (CGTPs). As stated previously, BIO emphasized that this paper represents a major milestone and will be the first global regulatory guidance on these innovative therapeutics. In response to the new and extensively revised draft, BIO expressed appreciation and support for this very important and thoughtful document. In particular, the paper provides valuable terminology, which is important given the current global inconsistencies in nomenclature and definitions. BIO also stated support the utilization of a risk-based approach for the proper regulatory oversight of ATMPs and HCT, as is described in the paper. However, BIO suggested there could be challenges when determining the level of regulatory oversight commensurate with product risk, given the paper’s recommendations for basing this assessment on the extent to which cells and tissues are manipulated, as this measure could be somewhat subjective. Therefore, BIO recommended that the paper maintain clarity for when the therapeutic will be for heterologous use, so as to avoid ambiguity. Furthermore, BIO requested additional clarity on how the regulatory reliance mechanisms laid out in the paper will address the challenges that are discussed. Lastly, BIO stated that as the ATMP space continues to rapidly change, it will be crucial for regulators to assess when new issues which need regulatory guidance arise and to quickly provide guidance to ensure clear recommendations.