Expedited Review Systems for Pharmaceutical Products in Japan, the U.S., and Europe

A Summary of Expedited Review Systems for Pharmaceuticals.

Please note: Machine translated. Please refer to the original Japanese for accuracy. For overseas firms, doing business in Japan typically requires Japanese-speaking staff or local support.

＠K.Kamitani

Comparison of Expedited Approval and Review Programs

Regulatory Function	Japan (MHLW/PMDA)	U.S. (FDA)	European Union (EMA)
Early & Intensive Development Support	Sakigake Designation System	Breakthrough Therapy Designation	PRIME Scheme
Shortened Final Review Period	Priority Review	Priority Review	Accelerated Assessment
Approval Based on Surrogate Endpoints	Conditional Early Approval System	Accelerated Approval	Conditional Marketing Authorisation (CMA)
Response to Public Health Crises	Emergency Approval System	Emergency Use Authorization (EUA), etc.	CMA in Public Health Emergencies
Incentives for Rare Diseases	Orphan Drug Designation System	Orphan Drug Designation	Orphan Medicinal Product Designation

＠K.Kamitani

Sakigake Designation System (Japan)

This system targets innovative new drugs developed ahead of the rest of the world. By designating them in the early stages of development, it provides preferential treatment in consultations and reviews related to pharmaceutical approval. The target review period is 6 months, and it also serves as a national strategy to deliver Japanese innovation to patients worldwide as quickly as possible.

Reference Material:
About the Sakigake Designation System, Pharmaceuticals and Medical Devices Agency (PMDA)
https://www.pmda.go.jp/review-services/drug-reviews/0002.html

＠K.Kamitani

Priority Review (Japan)

This is a broader system for shortening the review period for drugs with high medical necessity. It applies to orphan drugs and drugs that are clearly superior in efficacy and safety compared to existing treatments for serious diseases. The target total review period is 9 months, compared to the standard 12 months for regular items.

Reference Material:
Consultation System for Priority Review, Pharmaceuticals and Medical Devices Agency (PMDA)
https://www.pmda.go.jp/review-services/f2f-pre/consultations/0009.html

＠K.Kamitani

Conditional Early Approval System (Japan)

For drugs where conducting confirmatory clinical trials is difficult due to a small number of patients, this system grants early approval based on exploratory clinical trial data, on the condition that efficacy and safety are reconfirmed after marketing. It is equivalent to the U.S. Accelerated Approval and the EU's CMA.

Reference Material:
About the Conditional Early Approval System, Pharmaceuticals and Medical Devices Agency (PMDA)
https://www.pmda.go.jp/review-services/drug-reviews/0045.html

＠K.Kamitani

Fast Track & Breakthrough Therapy (U.S.)

Fast Track Designation: A process to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. It allows for more frequent communication with the FDA and enables a "rolling review," where parts of the marketing application can be submitted before the complete application is ready.

Breakthrough Therapy Designation: This requires a higher standard than Fast Track. It is for a drug that treats a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy. It includes all Fast Track features, plus intensive guidance and organizational commitment from the FDA.

Reference Material:
Fast Track, Breakthrough Therapy, Accelerated Approval, Priority Review, U.S. Food and Drug Administration (FDA)
https://www.fda.gov/patients/learn-about-drug-and-device-approvals/fast-track-breakthrough-therapy-accelerated-approval-priority-review

＠K.Kamitani

Accelerated Approval & Priority Review (U.S.)

Accelerated Approval: This pathway allows for earlier approval of drugs that treat serious conditions, and that fill an unmet medical need based on a surrogate endpoint. A post-marketing confirmatory trial is required to verify the anticipated clinical benefit.

Priority Review Designation: This designation directs overall attention and resources to the evaluation of applications for drugs that, if approved, would be significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions. The review period is shortened from the standard 10 months to 6 months.

Reference Material:
Fast Track, Breakthrough Therapy, Accelerated Approval, Priority Review, U.S. Food and Drug Administration (FDA)
https://www.fda.gov/patients/learn-about-drug-and-device-approvals/fast-track-breakthrough-therapy-accelerated-approval-priority-review

＠K.Kamitani

PRIME (PRIority MEdicines) Scheme (Europe)

This scheme provides enhanced scientific and regulatory support from an early stage for medicines that have the potential to address unmet medical needs. It aims to optimize development plans and generate robust data, and also encourages early dialogue with Health Technology Assessment (HTA) bodies responsible for pricing and reimbursement after regulatory approval.

Reference Material:
PRIME: priority medicines, European Medicines Agency (EMA)
https://www.ema.europa.eu/en/human-regulatory-overview/research-and-development/prime-priority-medicines

＠K.Kamitani

Accelerated Assessment (Europe)

This is a procedural tool to reduce the review period for a marketing authorisation application from the standard 210 days to a maximum of 150 days. It is for medicines of major public health interest, particularly from the point of view of therapeutic innovation. Medicines that have received a PRIME designation are likely to be eligible for Accelerated Assessment.

Reference Material:
Accelerated assessment, European Medicines Agency (EMA)
https://www.ema.europa.eu/en/human-regulatory-overview/marketing-authorisation/accelerated-assessment

＠K.Kamitani

Conditional Marketing Authorisation (CMA) (Europe)

This is a mechanism to approve a medicine on the basis of less comprehensive data than normally required, where the benefit of immediate availability of the medicine outweighs the risk inherent in the fact that additional data are still required. The authorisation is valid for one year and is subject to legally binding obligations ('specific obligations') to provide comprehensive clinical data post-authorisation.

Reference Material:
Conditional marketing authorisation, European Medicines Agency (EMA)
https://www.ema.europa.eu/en/human-regulatory-overview/marketing-authorisation/conditional-marketing-authorisation

＠K.Kamitani