Designing regulatory strategies for accelerating regenerative products innovation and patient access in Taiwan

Abstract

Background & Aim Regenerative products are characterized by their heterogeneity, complexity, and higher risk associated with their use compared to conventional chemical or biological drugs. In addition, the higher R&D cost of Regenerative Products requires a sponsor to have a significant amount of funding in order to reach the desired therapeutic/marketing goals. Up to now, Taiwan Food and Drug Association(TFDA) has not yet approved any regenerative product application, but numerous clinical trials are currently ongoing and it is expected that regenerative products will present great opportunities for treating unmet medical needs based on results shown in recent clinical trials. Designing a regulatory strategy for accelerating the marketing of regenerative products and helping patients gain access to regenerative products or medical treatments is an important goal of TFDA. Methods, Results & Conclusion The management of regenerative medical products is separated into two categories that include “medical practices” and “products” in the late 2018. For management of medical practices based on expected efficacies and accumulated safety profiles of international clinical trials, Taiwanese Ministry of Health and Welfare the (MHOW) chose 6 cell types (Table 1), which were considered as low risk, and allowed the medical institutions to submit medical practices applications based on the usage of the six cell types. In Taiwan, treatment plans and cell quality documents must be submitted and approved by MOHW before the medical practices are performed. During the same period that regenerative medical practices were permitted, TFDA also published “Regenerative Medicinal Product Management Act (Draft)” for managing regenerative products. This act allows conditional and time- limited approvals after confirmation of safety and preliminary efficacy data. Based on the risk and benefit evaluation, TFDA could restrict the use of the conditional approved regenerative products by the approaches of appointing medical Institutions or physicians. Licenses for conditional approval of regenerative products are valid for 5 years and sponsors needs to submit the results of the confirmatory trials. Establishment of the two legislations for regulating regenerative medical practices and regenerative products are expected to shorten the time it takes for regenerative medical products to reach market and to provide access for patients in need in the shortest amount of time.