Japan’s 10 year experience in regulating clinical access for unapproved and unproven cell therapies: A model for other markets?
Wednesday, May 29, 2024 08:00 AM - 09:00 AM
Room 201
Roundtable
Organized by: ISCT APAC Industry Committee
Moderator(s)
Panelists
Established 10 years ago, understand key learnings from the Japanese experience using the ASRM pathway to regulate clinical access to research-stage unproven therapies for patients in need and how this differs from the PMDA process.
The panel will touch on key learnings highlighting the benefits, challenges, what has worked, and what improvements are needed, to strive to provide patients government-regulated, clinician-accountable access to CGT/RM unapproved / unproven therapies in their time of need.
Key Learning Objectives
Moderator(s)
- William (Bill) Milligan, BSc, Steminent Biotherapeutics, Inc., Canada
- Kunihiko Suzuki, MBA, FIRM (Forum for Innovative Regenerative Medicine), Japan
Panelists
- Shinichi Noda, PhD, Pharmaceuticals and Medical Devices Agency (PMDA), Japan
- Satoshi Sobajima, MD, PhD, Sobajima Clinic, Japan
- Yoshimi Yashiro, PhD, Fujita Health University/Keio University, Japan
Established 10 years ago, understand key learnings from the Japanese experience using the ASRM pathway to regulate clinical access to research-stage unproven therapies for patients in need and how this differs from the PMDA process.
The panel will touch on key learnings highlighting the benefits, challenges, what has worked, and what improvements are needed, to strive to provide patients government-regulated, clinician-accountable access to CGT/RM unapproved / unproven therapies in their time of need.
Key Learning Objectives
- Should patients in need, who have no other treatment options, have access to cutting-edged CGT/RM treatments before therapeutic proof of concept (unproven) or market approval (unapproved), if the government's Ministry of Health can regulate safe, clinician-accountable access to these safe, novel CGT/RM treatments?
- Without clear government regulations, medical practices have spontaneously evolved in most countries/regions in the world to offer these unproven / unapproved therapies to patients willing to pay. For patients in immediate need, holding off until approval could represent a five to ten year wait for these promising treatment options. Can there be a safer, better regulated approach?
- Discuss and debate the 10Y experience of the ASRM (Act on Safety of Regenerative Medicine 2014-2024) regulatory framework as one approach for clinicians to formally apply to the Japan Ministry of Health for access to safe, but unapproved/unproven cell therapies to treat a patient in their time of need.
