Abstract
Several expedited regulatory review projects for innovative drugs and regenerative medical products have been developed in the US, the EU, and Japan. Each regulatory agency has elaborated an original regulatory framework and adopted regulatory projects developed by the other regulatory agencies. For example, the Food and Drug Administration (FDA) first developed the breakthrough therapy designation, and then the Pharmaceuticals and Medical Devices Agency (PMDA) and European Medicines Agency (EMA) introduced the Sakigake designation and the priority medicines (PRIME) designation, respectively. In addition, the necessity of the product being first development in Japan is the original feature of the Sakigake designation, while actively supporting the development of advanced-therapy medicinal products (ATMPs) by academia or small/medium-sized sponsors is the original feature of the PRIME; these particular features are different from the breakthrough therapy designation in the US. In this review article, flexible and expedited review processes for new drugs, and cell and gene therapies in the US, the EU, and Japan are described. Moreover, all the drugs and regenerative medical products that were granted conditional approval or Sakigake designation in Japan are listed and analyzed herein.
Highlights
In this review article, flexible and expedited review processes for new drugs and cell and gene therapies in the US, the EU, and Japan are described
This may be granted if all of the following requirements are met: (1) No standard therapy exists or superior clinical usefulness can be demonstrated as compared with the existing products in terms of quality of life of the patients, efficacy, or safety; (2) it is applicable to serious diseases; (3) it is difficult or it would take too long to conduct a confirmatory study; (4) exploratory clinical studies can show the efficacy and safety of the drug; and (5) surveillance or clinical studies must be conducted as a post-marketing requirement [34] (Table 4)
Requirements for the regenerative medicine advanced therapy (RMAT) designation are as follows: (1) The drug is a regenerative medicine therapy, which is defined as a cell therapy, therapeutic tissue engineering product, human cell and tissue product, or any combination product using such therapies or products; (2) it is intended to treat, modify, reverse, or cure a serious condition; and (3) preliminary clinical evidence indicates that the regenerative medicine therapy has the potential to address unmet medical needs for the condition
Summary
Flexible and expedited review processes for new drugs and cell and gene therapies in the US, the EU, and Japan are described. In the US, the Food and Drug Administration (FDA) reviews marketing authorization applications and approves medical devices, drugs, and regenerative medical products. The centralized authorization procedure is compulsory for drugs for acquired immunodeficiency syndrome, diabetes, cancer, immune dysfunctions, and neurodegenerative diseases, as well as biosimilar products, advanced-therapy medicinal products (ATMPs), genetically engineered products, and orphan drugs [3,4,5]. Packaging inserts and review reports (public assessment reports) of approved drugs and ATMPs as well as withdrawn applications are listed on the EMA website [8]. The offices of new drugs I–V, the office of vaccine and blood products, and the office of cellular and tissue-based products deal with new drugs and regenerative medical products [11]. Approved products, including drugs and regenerative medical products in Japan, are listed on the PMDA website [12,13]
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