On the 10th of November, Dr Laurie Smaldone Alsup and Dr Steffen Thirstrup participated in a webinar – Accelerated Approval strategies for Emerging Biotech, which was a collaboration between NDA and The Conference Forum.
The webinar took the form of a panel discussion where Laurie and Steffen, together with Julie Dixon (Vice President, Head of Global Regulatory Affairs, Oncology, Takeda) and Richard Malamut (Chief Medical Officer & EVP, Collegium Pharmaceutical, Inc), discussed the objectives and reality of accelerating the path to approval.
Accelerated approvals is an important regulatory pathway for indications where it is challenging to demonstrate a clinical endpoint, in which the only option to get the drug on the market time is to use a validated surrogate endpoint. Although there might be concerns around how and by when the data attained with surrogate endpoints should be confirmed, the value of the pathway for unmet needs in small patient groups could not be emphasised enough.
Using the ODAC public hearing previously this year as a catalyst, the panel discussion highlighted the importance of appropriate endpoint measures, adequate numbers of patients and post-approval commitment. Nevertheless, even if the effect might not be statistically significant, for some patients it might be the only option. To watch the recording of the webinar, click the button below:
For further reading about the Accelerated Approval pathway and the ODAC public hearing: