Table 2.

Current challenges related to the benefit-risk assessment for pharmaceuticals in Japan

Challenges	Recently adopted strategies by the government	Unsolved problems
Health hazards related to pharmaceuticals (“Yakugai” in Japanese)	• I ncreasing staff for safety monitoring at the PMDA • Reinforcement of risk communication and management	• Possible increase of the health hazards risk caused by the reduction of the drug lag • Conservative risk assessment to avoid risks as far as possible
Drug lag	• Increasing staff for regulatory review at the PMDA • Promotion of global clinical trials • Decreasing regulatory review time • Accelerated conditional approval (only for regenerative medicine) • Early access to unapproved drugs (only in selected hospitals) • Establishment of the Japan AMED	• Propriety of a mandatory demand for Japanese patients data in clinical trials • Delayed launch of domestic clinical trials • Delayed submission of new drug applications • Early access to novel pharmaceuticals especially for patients with life-threatening diseases • Limited competition through uniform drug pricing under the universal health coverage • Prohibition of a mixed medical treatment in the public insurance
Vaccination policy	• Increased number of vaccine coverage in the routine immunization program	• Vaccine lag • Effective provision of vaccination for unimmunized persons • More transparent, scientifically based policy-making process
Clinical study misconduct	• Strict regulation implementation for post-marketing clinical study including long-term data preservation • Increased transparency concerning conflict of interest	• Effective, prompt, and fair investigation against misconduct allegations • Financial recovery against unnecessary defrayment • Overreliance on funding from pharmaceutical companies • Stagnation of study activities due to excessive regulation

Abbreviations: PMDA, Pharmaceutical and Medical Devices Agency; AMED, Japan Agency for Medical Research and Development.