Table 2 Administrative incentives

Orphan drug designation

Designated in Japan when all three conditions (basically less than 50,000 patients, medical necessity, and possibility to be developed in Japan) are met

Pioneer drug designation Sakigake

With the goal of providing patients with the earliest available state-of-the-art therapeutic agents, drugs that are developed in Japan earlier than in the rest of the world, have expected significant efficacy, and are registered in the early clinical stage receive priority handling in consultation and review related to regulatory approval

Public knowledge-based application

A system for drugs is already approved in Japan to provide approval without having conducted some or all clinical trials in Japan, if there is a scientific basis like approval in a foreign country, experience of use, or a rationale-providing paper

Recommendation in the “Review Meeting of Unapproved Drugs and Off-label Drugs with High Medical Needs”

For drug indications approved in Europe and the USA but not in Japan, drug use requested by academic societies can be approved if the meeting body recognizes the necessity and recommends the drug for approval

Public knowledge-based application after pre-evaluation

Products that have been submitted for public knowledge-based application after being evaluated by the abovementioned committee

Priority review

A system that prioritizes the review of drugs for approval that meet certain standards

Pre-evaluation

Preliminary evaluation based on available data on quality, nonclinical (toxicology, pharmacology, and pharmacokinetics), and clinical studies (phase 1, 2, and 3) in the pre-submission development stage. This consultation system precedes the approval review process. As it allows the extraction and resolution of issues at each development stage before the application, the approval review period is shortened

Conditional early approval system

A drug can be approved if it targets a serious disease, unavailability of effective therapy for the disease, the number of patients is small, and it is difficult or takes a long time to conduct a clinical trial in Japan. A certain level of efficacy and safety without confirmatory studies available at the time of application has to be ascertained, and the drug is approved on the condition that post marketing studies reaffirm the efficacy and safety

Drugs for a specific purpose

Designated by the Ministry of Health, Labor and Welfare based on the assessments of the “Pharmaceutical Affairs and Food Sanitation Council” if the demand for the use is not markedly met or the need for medical treatment is particularly high. It aims to contribute to the promotion of research and development of drugs for which medical needs are not markedly met, such as the lack of established doses and administration routes for children

COVID- 19-related priority review

A now terminated system for priority review of drugs, quasi-drugs, medical devices, in vitro diagnostics, and regenerative medicine for COVID- 19 infections or related symptoms

Special approval

An early approval system for drugs which are available in the US or EU and required urgent use to prevent the spread of diseases that may seriously affect the lives and health of the public and for which no other appropriate method is available

Mandatory post-marketing surveillance

Some new drugs are approved on the condition that post-marketing surveillance is conducted in all patients. It is often required when limited clinical trial data are available at the time of approval such as for orphan drugs or drugs for which concern exists about the occurrence of serious adverse events