Utilization of medical RWD/ RWE for regulatory purpose

On August 4, 2023, we held the 2nd Academia Clinical Development Seminar online, inviting Dr. Hideki Oi from National Center of Neurology and Psychiatry (NCNP).
He has been involved in the formation of Academic Research Organization (ARO) functions, operational support for clinical research and investigator-initiated clinical trials, and the establishment, operation, and management of patient registries at NCNP.
In this seminar, based on his extensive experience, he spoke about the utilization of medical real-world data and real-world evidence (hereafter referred to as RWD/ RWE) for regulatory purpose.

First of all, he explained about the environment surrounding RWD/ RWE in medicine.
He said that in the field of RWD/ RWE, both in the pre and post-marketing phases of pharmaceuticals, medical devices, and regenerative medicine products (hereinafter referred to as "pharmaceuticals, etc."), we are now facing a major transformation. In the U.S., the "21st Century Act" addressed the issue of expediting regulatory approval by streamlining clinical trials for pharmaceuticals, etc., and there have been cases for expansion of indications via RWD/ RWE.
He also said that in Japan, the regulatory notifications such as　 "Conditional Early Approval System for Drugs" and the “Basic principles on Utilization of Registry for Applications” have been issued, and the infrastructure is being developed, and there are examples of pharmaceutical companies implementing patient registry data in their new drug applications (NDA) as a strategy.

He explained that the timing of the use of RWD/ RWE could include the use as an external control for the NDA on rare diseases, expansion of indications, and post-marketing surveillance (PMS). However, he said that there are some issues to be addressed, such as ensuring the reliability of clinical data and ethical considerations such as how to obtain informed consent.

Next, he said that NCNP's efforts to utilize patient registry data for regulatory purposes.
Regarding Viltolarsen, he said that the drug was approved in Japan under the "Conditional Accelerated Approval System" and that the post-marketing long-term efficacy for the removal of conditions was to be confirmed using data from the NCNP's patient registry (Remudy-DMD) and that the Risk Monitoring Plan included a survey using Remudy-DMD.

He also mentioned that one of the challenges in utilizing the registry data for the PMS is the risk of not being able to secure a sufficient number of patients for evaluation due to the adjustment of patient's demographics and confounding factors, but he would like to establish a scheme whereby academia and companies can work together to improve the accuracy of safety information.

In this seminar, he gave a useful lecture on the utilization of RWD/ RWE for regulatory purpose. The seminar ended successfully with a lively Q&A session and many participants.

The next seminar will be held on Friday, October 6, 2023, with Prof. Hiroyuki Tanishiro from Osaka Dental University, Translational Research Institute for Medical Innovation.
We look forward to seeing you at the next meeting.