PHP10 - Orphan Drug Policy and Access Dynamics in Japan

Abstract

To understand the evolving patient access and coverage for orphan drugs in Japan. The study employed a pragmatic literature, industry, and policy review to determine the impact of national orphan drug policies such as priority review and accelerated approval of breakthrough treatments on Japanese reimbursement of orphan gene therapies, stem cell therapies, and PD-1/L1 inhibitors. Japan has facilitated orphan drugs’ market entry by prioritizing their market authorization process despite limited clinical data at launch. Orphan drugs may be approved in Japan with a published Phase II trial if the manufacturer agrees to conduct a future post-marketing authorization study; however, achieving regulatory approval may be challenging without inclusion of Japanese patients in clinical studies. Following global accelerated pathways to bring innovative products to patients more quickly, Japan has initiated its own system in 2015. Sakigake designation grants fast-tracked regulatory review and extension of the re-examination period; notably, orphan drugs already have extended 10-year pricing re-assessment timelines. Sakigake products need to meet criteria such as having a novel mechanism of action, addressing high unmet medical needs, or being commercialized in Japan before any other market. Six drugs have been designated so far - five by Japanese sponsors (including: DS-5141b – Duchenne; aducanumab – Biogen Japan; Nivolumab – Ono), and one by a multinational sponsor (Merck’s Keytruda); 3/6 target indications with notoriously high unmet need. Orphan drugs, including those under the Sakigake system, can benefit from the Chuikyo’s (pricing body) premium allowances for orphan and innovative products. Increasingly, orphan drugs are prioritized and given concessions during marketing authorization review, and they are eligible for pricing premiums due to their innovative mechanisms of action or their therapeutic area. If MHLW clinical trial criteria is met, the new Sakigake system looks to further facilitate and incentivize patient access of prospective orphan drugs.