[PMDA's Initiatives to Encourage the New Drug Development for Rare Cancers]

Rare cancers, estimated to account for 15% of all cancers, have many unmet medical needs, but at the same time, developing treatments for these cancers is fraught with difficulties. PMDA's efforts to promote and support the development of treatments for rare cancers include the development consultation to meet the needs of emerging biopharma(EBP), promotion of development through multi-regional clinical trials, and development support for investigator-initiated clinical trials. In addition, the development environment for rare cancers and rare subtypes has been improved through the revision of clinical evaluation guidelines, consultation programs and guidance publication for the utilization of RWDs, and the accumulation of tissue-agnostic cancer drug approvals. Regarding the Orphan Drug Designation System, about one-third of the 267 drugs designated during the 15-year period from 2004-2018 were anti-cancer drugs. Looking at the approvals of anti-cancer drugs during the past 10 years(2013-2022), 26%(53/203)of drugs for solid tumors and 60%(62/104)of drugs for hematologic tumors received orphan drug designation. Regulatory measures that support the development for rare cancers include not only the Orphan Drug Designation but also the Conditional Accelerated Approval, which facilitates the development of drugs for diseases where timely conduct of a confirmatory clinical trial is difficult, and the Sakigake Designation that allows the accelerated approval of innovative new drugs. PMDA will continue its efforts to promote drug development in response to changes of the circumstances surrounding drug development as well as developer's need and will make further efforts to disseminate information of those measures both domestically and internationally.