[Press Release, April 20, 2020 / unofficial translation] Pre-announcement of Legislation (4/21 to 5/31): Proposals for the Establishment of Legislation of Lower Statute of “ACT ON THE SAFETY OF AND SUPPORT FOR ADVANCED REGENERATIVE MEDICINE AND ADVANCED BIOPHARMACEUTICALS” - Preparing concrete procedures and details for the implementation of whole life-cycle safety management for advanced biopharmaceuticals and clinical research on advanced regenerative medicine - □ The Ministry of Food and Drug Safety (Minister Lee Eui-Kyung) and the Ministry of Health and Welfare (Minister Park Neung-Hu) stated that they will make a pre-announcement of legislation from April 21st (Thr) to June 1st (Mon) for proposals for legislation of lower statute of “ACT ON THE SAFETY OF AND SUPPORT FOR ADVANCED REGENERATIVE MEDICINE AND BIOPHARMACEUTICALS” (hereinafter referred to as the “Advanced Regenerative-Bio Act”). ○ The Advanced Regenerative-Bio Act has significant importance since it provided new treatment opportunities for desperate patients suffering from rare and incurable diseases under the national responsibility and, - laid a foundation for a whole life-cycle safety management system of advanced regenerative medicine and advanced biopharmaceuticals utilizing stem cells and technical development in the fast growing market of regenerative medicine. * (Advanced regenerative medicine) Medical technology to treat, replace or regenerate damaged tissues and organs using human cells, etc. * (Advanced biopharmaceuticals) Biopharmaceuticals (cell therapy products, gene therapy products, tissue engineering products, etc.) made from living cells, tissues, or genes. ○ This enactment proposals specify details delegated in the “Advanced Regenerative-Bio Act,” which is set to come into force this coming August 28th, for the designation of medical institutions for review, approval and implementation of clinical research on advanced regenerative medicine, quick commercialization of advanced biopharmaceuticals and the expansion of a whole life-cycle safety system. □ The main contents of the pre-announcement of legislation proposal are as follows. ≪ A Proposal for an Enforcement Decree (presidential decree) of the Advanced Regenerative-Bio Act ≫ [1] The Scope of advanced regenerative medicine, etc (Article 2 to 4 of the Proposal) ○ The range of advanced regenerative medicine is divided into four categories based on the treatment methods (cell therapy, gene therapy, tissue engineering therapy, and fusion therapy), and - Present the clinical research risk level (high, medium, low) standards*, but final decisions are made by a review committee* * Roughly categorized based on whether collected cells are of the same type or different types from one’s own body or someone else’s ▶ (Advanced regenerative medicine clinical research, Article 2 of the Act, etc.) Advanced regenerative medicine treatments are possible under the nation’s safety management authority, such as thorough examination of a nation-affiliated review committee regarding urgency, safety, and effectiveness, with the consent of the patient and the responsibility of a doctor as prerequisites ▶ (Advanced regenerative medicine and advanced biopharmaceuticals screening committee, Article 13 of the Act) A committee jointly affiliated with the MOHW and the MFDS Joint Committee is composed of up to 20 related experts, for consultations on the evaluation of advanced biopharmaceuticals and the review on advanced regenerative medicine research plans ○ The scope of human cells used as materials for advanced regenerative medicine and advanced biopharmaceuticals is regulated as containing cells and tissues originated from humans and animals, organs originated from animals and those processed using these as ingredients [2] Establishment of basic plans, etc. (Article 5 to 9 of the Proposal) ○ Specifies related governmental departments*, annual implementation plan establishment procedures according to pan-governmental support measures for the advanced regeneration field on a 5-year cycle * (MOHW, MFDS) + Ministry of Economy and Finance, Ministry of Science and ICT, Ministry of Trade, Industry and Energy, Ministry of SMEs and Startups, Ministry of Agriculture, Food and Rural Affairs, Ministry of Education, Ministry of Oceans and Fisheries [3] Implementing advanced regenerative medicine clinical research (Article 10 to 29 of the Proposal) ○ (Regenerative medical institutions*) Preparing research plan and submission methods, implementation standards that must be followed when carrying out clinical research following approval of the research plan * Institutions wishing to perform clinical research designated by the Minister of Health and Welfare when requirements are satisfied ○ (Composition and operation of review committee) Specifies additional matters to review* other than compatibility of research plans, review and approval procedures, etc. * Deciding suspension, halt, and restart of research when research result reporting and research plan change, abnormality occurs, etc. - Provides procedures for quick and transparent review, such as having regular meetings 6 times a year, presenting research plans to the review committee within 60 days from official application*, public release of review commit tee meeting minutes*, etc. * (Specified in Enforcement Rule) Presentation within 60 days, release of minutes within 30 days after the conclusion of review - To increase professionalism of the reviews, 4 expert committees are established and assigned to each treatment type, and the responsibility is given to write review reports for verifying effectiveness and safety of the review plan - A MOHW-MFDS joint executive office is established to provide various types of clerical support, such as dealing with administrative process including the reception of research plans, working on writing review reports, operating the review and expert committees, and responding to inquiries from regenerative medicine institutions, etc. ○ (Cell processing facilities*) Specifying facility, equipment, personnel standards for the approval as well as procedures to comply with and consent requirement for cell collection * A newly-established type of business that supplies human cells, etc. to regenerative medical institutions (Article 15 of law) which can professionally handle only the processing of cells ○ (Safety management) Specifying procedures for establishing plans for long-term follow-up of clinical research subjects*, necessary public health measures for emergencies such as epidemiology studies and construction/operation of a clinical research information system * Implemented at advanced regenerative medical safety management institution (Korea Centers for Disease Control and Prevention) [4] Standards of advanced biopharmaceutical manufacturer and importer facilities, etc. (Article 30 to 31 of the Proposal) ○ Establishing facility standards (production location, storage location, laboratory, etc.) for advanced biopharmaceutical manufacturer approvals and importer declarations, and specifying the scope of entrusted parties and matters to be observed [5] Facility, equipment, personnel standards for management businesses of human cell, etc. (Article 32 to 33 of Proposal) ○ Providing facility, equipment, personnel standards necessary for human cell, etc. management business* approval, and specifying matters to include in a quality management system * Newly established business type that supplies human cells to be used as ingredients in advanced biopharmaceuticals (Article 28 of act) [6] Targets of long-term monitoring and designation of regulatory science center, etc. (Article 34 to 37 of the Proposal) ○ Providing procedures for the registration of medication history and reporting of adverse events, and designating a target of advanced biopharmaceuticals* for which long-term follow-ups are necessary * Stem cell treatments, medications containing animal tissue and cells, and gene therapy products can be designated ○ Korea Institute of Drug Safety & Risk Management designated as a regulatory science center, specifying the tasks of the regulatory science center* * Advanced biopharmaceutical long-term follow-up plan examination and execution confirmation, committee establishment and operation, side effect causality investigations, safety information management system construction and operation, etc. ≪ A Proposal for Regulations (ordinance of the Prime Minister) on the Safety of and Support for Advanced Biopharmaceuticals≫ [1] Define approval-related procedures for cell processing facilities and human cell management businesses ○ Specify procedures for approval/prior-approval for changes/license renewal of cell processing facilities and human cell management businesses ○ Define compliance requirements for human cell management businesses for the sake of securing quality and safety in the process of collecting/importing/processing/storing/transporting/supplying human cells. [2] Stipulate matters related to the approval, product approval, license renewal of advanced biopharmaceutical manufacturers ○ Specify approval/reporting/license renewal procedures and methods for advanced biopharmaceutical manufacturers, consigned manufacturing and sales businesses, and importers ○ Require safety/efficacy documentation, standards and testing methods, risk management plans to be included in the approval application when applying for the product approval in manufacturing and sales/importing. [3] Define matters related to the establishment of long-term follow-up plans and compliance requirements ○ Specify necessary items when establishing long-term follow-up plan, such as follow-up purpose, duration, and items, and identify compliance requirements for long-term follow-ups [4] Product categorization of advanced bio-pharmaceuticals ○ Prescribe application procedures and required dossiers (product information, explanatory documentation, documentation comparing products with similar domestic/foreign products, etc.) when applying for product categorization of advanced biopharmaceuticals [5] Introduction of tailored examinations/conditional approvals and designation of products eligible for quick approval ○ Stipulate procedures necessary to designate advanced biopharmaceuticals eligible for expedited processing* and specify procedures for tailored review and conditional approvals applied to eligible advanced biopharmaceuticals * Eligible drugs include those intended for the treatment of serious and life-threatening diseases, provided that there are no alternatives available, rare diseases, prevention of a pandemic, and treatment of infectious diseases ≪ Draft Enforcement Rule (ordinance of the Ministry of Health and Welfare) on the Safety of and Support for Advanced Regenerative Medicine ≫ [1] Designation/management of regenerative medicine institutions ○ Prescribe obligations to report through a clinical research information system and requirements for facility/equipment/personnel* necessary to conduct clinical research * Deliberation committee will re-evaluate whether additional facility/equipment/personnel needed for the execution of research plans have been secured. ※ Information on the application for regenerative medicine institution designation, will be provided during the enactment period of subordinate statute. [2] Definition of compliance requirements of the heads of cell processing facilities, etc. ○ Define required facilities, equipment, among others, for cell processing tasks by regenerative medicine institutions, and compliance requirements of cell processing facilities and regenerative medicine institutions regarding cell processing tasks, such as storing human cells and designating human cell storage/task record managers. * Obligatory storage/management of records for 20 years, obligation deemed to have been fulfilled if registered in clinical research information system [3] Stipulate safety management institution designation and task fulfillment procedures ○ Designate Korea Centers for Disease Control and Prevention as advanced regenerative medicine safety management institution, and define safety management institution’s task execution procedures * Investigation of actual conditions at regenerative medicine institutions, reporting system for adverse patient reactions, etc. □ The MFDS and the MOHW, while collecting wide-ranging opinions from the public during the pre-announcement of legislation period to push forward the enactment of this legislation of subordinate statute, also plans to prepare concrete operational plans such as the enactment of notices. ○ Any organization or individuals with a comment regarding these legal proposals can submit their opinion to the Ministry of Food and Drug Safety’s Biopharmaceutical Policy Division, the Ministry of Health and Welfare’s Healthcare Technology Development Division, or the Citizen Participation Legislation Center by June 1st (Mon) 2020. ○ Detailed information regarding the proposals is available on the MFDS website (www.mfds.go.kr), MOHW website (www.mohw.go.kr), or the Citizen Participation Legislation Center (opinion.lawmaking.go.kr). Please find the attached file for more information