Module 9 2024

03/09/2024

Other Major Reg Authority Worldwide

Swissmedic ● Regulated under Swiss Federal Act on Medicinal Products and Medical Devices of 15 December 2000 (Therapeutic Products Act, TPA) ● Federal Human Research Act of 30 September 2011 ● Ordinance on Clinical Trials in Human Research (ClinO/KlinV) of 20 September 2013 ● Federal Law of 21 March 2003 on Non-Human Gene Technology (Gene Technology Law, GTG / LGG) ● Details “Requirements relating to the authorisation documentation for transplant products (TP), gene therapy medicinal products (GT) and medicinal products consisting of or containing genetically modified organisms (GMO)” – May 2016 ● If the product is a GMO, it needs to be identified as such ● All GT need an accompanying Environmental Risk Assessment (https://www.swissmedic.ch/bewilligungen) Canada ● Regulated under Part C of the Food and Drug Regulations ● Cell & Gene regulations are presently being drafted. In the meanwhile the existing biologic regulations are applied. ● BGTD is very active participant in international fora (ASGCT and ESCGT, ISCT, Regulators Forum etc...) and often takes positions close to the CAT. ● https://www.canada.ca/en/health-canada/services/drugs-health-products/biologics-radiopharmaceuticals-genetic-therapies.html Japan ● Two types of product: Cell/Tissue based products and gene therapy products ● Regulated under the Regenerative Medicines Act, Nov 2013 and are defined separately from Drugs and Devices ● Conditional/Time-limited Approval System ● Expedited approval system based on the estimation of efficacy and confirmation of safety ● Cartagena protocol: mandatory approval prior clinical trials with GTs

The Organisation for Professionals in Regulatory Affairs

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International Council for Harmonisation of Technical Requirements for Pharmaceutical (ICH) Regulators and Industry Associations ● 20 members (EMA, FDA, PMDA, but also EFPIA, JPMA, PhRMA, etc.), 36 observer members (incl. USP, EDQM, WHO) Scope of guidelines excludes cell and gene therapy by default ● ICH Q5(E) guidelines can be applied to some extent (viral safety, comparability). ● ICHQ2R1 (Analytical method validation) Gene therapy discussion group was disbanded, M6 guideline on vector shedding never finalized ICH S12 - Nonclinical Biodistribution Considerations for Gene Therapy Products (Draft) Lack of common experience in approval between regions and of regulatory

alignment due to wide diversity of scientific opinions Lengthy process to get guidelines approved there

The Organisation for Professionals in Regulatory Affairs

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