BIO Comments on WHO Considerations on Regulatory Convergence of Cell and Gene Therapy Products
On Thursday, January 27th, BIO submitted comments in response to the World Health Organization’s (WHO) recently published white paper on the regulation of Cell and Gene Therapy Products (CGTPs). First and foremost, BIO acknowledged that the publication of this white paper is a major milestone in the global recognition and regulation of such products. The paper, while only an initial step in driving regulatory convergence, achieves the appropriate balance in providing key principles for the “state of the art”, without being overly prescriptive. In the comments submitted, BIO expressed agreement that the development and adoption of harmonized regulatory requirements based on sound science and risk-based principles is important to ensure that safe and effective CGTPs are accessible to all patients in a timely manner. Additionally, BIO emphasized the importance of industry-regulator dialogue throughout the product lifecycle and urged WHO, along with other regulatory authorities, to carefully consider the small patient population enrolled in clinical trials for regenerative medicine products as well as the adverse impact duplicative import/export release testing for advanced therapy medicinal products (ATMPs) could have on these patients. Furthermore, BIO noted that future guidelines could contemplate the utility of waivers or other flexible tools in assuring a balanced and risk-based approach. In addition to a number of redline edits, lastly, BIO suggested that the paper could be strengthened by introducing terminology early-on and by grouping definitions and categories together.