The Current Status of Sakigake Designation in Japan, PRIME in the European Union, and Breakthrough Therapy Designation in the United States

Abstract

Following the Breakthrough Therapy Designation system in 2012 in the United States, the Sakigake Designation was introduced in 2015 in Japan, and PRIME (PRIority MEdicines) was started in 2016 in the European Union. Each system aims at giving patients better access to innovative drugs and regenerative medicine products by providing product developers with generous regulatory and scientific support from an early development stage. So far, the designation systems have operated independently in each region, and no products with the same indication have been designated commonly under the 3 designation systems. However, no designation system excludes a product designated under another system, which allows the possibility of an applicant to seek all 3 designations; this may happen in the near future. Therefore, an understanding of the current situation under each designation system will contribute to effective operation of each system as well as identification of further collaborative activities between the European Medicines Agency; Japan's Ministry of Health, Labour and Welfare/Pharmaceuticals and Medical Devices Agency; and the United States Food and Drug Administration. Such collaborations can be successful because these organizations have already established a close relationship through international activities such as the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) and the International Coalition of Medicines Regulatory Authorities (ICMRA).

Evaluation of Therapeutics for Severely Debilitating or Life‐Threatening Diseases or Conditions: Defining Scope to Enable Global Guidance Development

“…Severely debilitating or life‐threatening diseases or conditions (SDLTs) are those which cause major irreversible morbidity and/or in which the likelihood of death is high despite available therapies. National and regional expedited regulatory pathways have emerged to accelerate mid‐stage to late‐stage drug development and approval for serious conditions with high unmet need regardless of therapeutic area . In the case of advanced cancer, a streamlined nonclinical testing paradigm was agreed upon at an international level (ICH (International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use) S9 and the associated Q&A) to support rapid clinical development .…”

“…National and regional expedited regulatory pathways have emerged to accelerate mid-stage to late-stage drug development and approval for serious conditions with high unmet need regardless of therapeutic area. [2][3][4][5][6][7][8] In the case of advanced cancer, a streamlined nonclinical testing paradigm was agreed upon at an international level (ICH (International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use) S9 and the associated Q&A) to support rapid clinical development. 9 However, in the case of nononcology SDLTs, even those presenting benefit vs. risk considerations similar to those for advanced cancers, global clarity and alignment regarding regulatory flexibility in the nonclinical and early clinical development space is lacking to support timely and efficient development of therapies to treat SDLTs across therapeutic areas.…”

Biased by design? Clinical trials and patient benefit in oncology

“…However, evidence suggests that uptake by academia is low and, therefore, efforts directed specifically at independent/academic investigators may be required to improve on this [29]. Similar initiatives to PRIME exist elsewhere, such as the breakthrough therapy designation program in the USA and the Sakigake Designation in Japan [33]. Increased early contacts and joint scientific advice with both regulators and HTA bodies should also be encouraged to identify the best comparator in clinical trials and facilitate the choice of primary end points that deliver added therapeutic value, which would ultimately accelerate access to new treatment options for cancer patients [34,35].…”

Global Regulatory Differences for Gene‐ and Cell‐Based Therapies: Consequences and Implications for Patient Access and Therapeutic Innovation

“…In the EU, GCT developers benefit from a reduced fee for scientific advice . Furthermore, PRIME (EU), Sakigake (Japan), and Breakthrough Therapy Designation (US) all enhance opportunities for interaction between regulators and developers. Many of the designated investigational products under these pathways are GCTs; however, eligibility criteria do not overlap entirely between jurisdictions …”