Guidance of marketing authorizations for Advanced Therapy Medicinal Products (ATMPs) in The USA The EU and JAPAN for a pre-requisite in Thailand

สำนักงานคณะกรรมการอาหารและยา. แนวทางการขึ้นทะเบียนตำรับยาที่เป็นผลิตภัณฑ์การแพทย์ขั้นสูง ชนิดผลิตภัณฑ์เซลล์บำบัด [อินเทอร์เน็ต]. 2561 [เข้าถึง เมื่อ 25 ม.ค. 2562]; เข้าถึงได้จาก: http://www.fda.moph.go.th/sites/drug/Shared%20Documents/Law04-Notification-ThFDA/FDA-20180510.pdf

Hanna E, Rémuzat C, Auquier P, Toumi M. Advanced therapy medicinal products: current and future perspectives. Journal of market access health policy 2016;4(1):310-36.

Alliance for Regenerative Medicine. State of the Industry [Internet]. 2019 [cited 2019 May 29]. Available from: https://alliancerm.org/publication/q3-2019-data-report/

Prabhasawat P, Ekpo P, Uiprasertkul M, Chotikavanich S, Tesavibul N. Efficacy of cultivated corneal epithelial stem cells for ocular surface reconstruction. Clin Ophthalmol 2012;6:1483-492.

Prasongtanakij S, Anurathapan U, Vanichapol T, et al. Production and characterization of haploidentical CD19 CAR T cells: validated to induce a continuous complete remission in a patient with relapsed refractory B-cell ALL [published online ahead of print, 2020 Sep 24]. Asia Pac J Clin Oncol 2020;18(1):44-51.

European Medicines Agency. GUIDELINES on Good Clinical Practice specific to Advanced Therapy Medicinal Products [Internet]. 2019 [cited 2019 May 19]. Available from: https://ec.europa.eu/health/sites/health/files/files/eudralex/vol-10/atmp_guidelines_en.pdf

European Medicines Agency. Draft Guideline on quality, non-clinical and clinical requirements for investigational advanced therapy medicinal products in clinical trials [Internet]. 2019 [cited 2019 May 10]. Available from: https://www.ema.europa.eu/en/documents/scientific-guideline/draft-guideline-quality-non-clinical-clinical-requirements-investigational-advanced-therapy_en.pdf

United State Food and Drug Administration. Considerations for the Design of Early-Phase Clinical Trials of Cellular and Gene Therapy Products [Internet]. 2015 [cited 2019 May 9]. Available from: https://www.fda.gov/media/106369/download

European Medicines Agency. Regulation (EC) No 1394/2007 Of The European Parliament And Of The Council [Internet]. Official Journal of the European Union. 2007 [cited 2019 May 9]. Available from: https://eur-lex.europa.eu/LexUriServ/LexUriServ.do?uri=OJ:L:2007:324:0121:0137:en:PDF

United State food and drug administration. Cellular & Gene Therapy Products [Internet]. 2019 [cited 2019 Jun 10 ]. Available from: https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products

Azuma K. Regulatory Landscape of Regenerative Medicine in Japan. Current Stem Cell Reports 2015;1(2):118 - 28.

Okada K, Koike K, Sawa Y. Consideration of and expectations for the Pharmaceuticals, Medical Devices and Other Therapeutic Products Act in Japan. Regen Ther 2015;1:80-3.

Nagai S. Flexible and Expedited Regulatory Review Processes for Innovative Medicines and Regenerative Medical Products in the US, the EU, and Japan. Int J Mol Sci. 2019 3;20(15):3801.

United state Department of Health and Human Services, Food and Drug Administration Center for Biologics Evaluation and Research. Guidance for Industry Expedited Programs for Serious Conditions – Drugs and Biologics [Internet]. 2014 [cited 2019 Jan 24] Available from: https://www.fda.gov/media/86377/download

The European Medicines Agency. Guideline on the scientific application and the practical arrangements necessary to implement Commission Regulation (EC) No 507/2006 on the conditional marketing authorisation for medicinal products for human use falling within the scope of Regulation (EC) No 726/2004. [Internet]. 2016. [cited 2019 Jan 24 ] Available from: https://www.ema.europa.eu/en/documents/scientific-guideline/guideline-scientific application-practical-arrangements-necessary-implement-commission-regulation-ec/2006-conditional-marketing-authorisation-medicinal-products-human-use-falling_en.pdf

The European Medicines Agency. Accelerated Assessment [Internet]. [cited 2019 Jan 25 ] Available from: https://www.ema.europa.eu/en/human-regulatory/marketing-authorisation/accelerated-assessment

The European Medicines Agency. PRIME: priority medicines. [Internet]. [cited 2019 Jan 25] Available from: https://www.ema.europa.eu/en/human-regulatory/research-development/prime-priority-medicines

The European Medicines Agency. Pilot project on adaptive licensing. [Internet]. 2014. [cited 2019 Jan 25] Available from: http://www.ema.europa.eu/docs/en_GB/document_ library/Other/2014/03/WC500163409.pdf

The European Medicines Agency. Guidance for companies considering the adaptive pathways approach [Internet]. 2016 [cited 2019 Jan 25] Available from: https://www.ema.europa.eu/en/documents/regulatory-procedural-guideline/guidance-companies-considering-adaptive-pathways-approach_en.pdf

Ministry of Health, Labour, and Welfare. Strategy of SAKIGAKE. [Internet]. 2014. [cited 2019 Jan 24] Available from: https://www.mhlw.go.jp/english/policy/health-medical/pharmaceuticals/140729-01.html

Schothorst M V, Krijn J W, Oortwijn W S, Hoekman J, Coppens D. Study on the regulation of advanced therapies in selected jurisdictions. [Internet]. 2016 [cited 2019 May 9] Available from: https://ec.europa.eu/health//sites/health/files/human-use/docs/20147306_rfs_chafea_2014_health_24_060516.pdf

United State food and drug administration. Interactions with Office of Tissues and Advanced Therapies [Internet]. [updated 2022 Jun 23; cited 2023 May 20] Available from: https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/interactions-office-tissues-and-advanced-therapies

United state Department of Health and Human Services, Food and Drug Administration Center for Biologics Evaluation and Research. Guidance for Industry Expedited Programs for Serious Conditions – Drugs and Biologics [Internet]. 2014 [cited 2019 Jan 24] Available from: https://www.fda.gov/media/86377/download

Japan Pharmaceutical Manufacturers Association. INFORMATION ON JAPANESE REGULATORY AFFAIRS Regulatory Information Task Force Japan [Internet]. 2018 [cited 2019 Jan 25] Available from: http://www.jpma.or.jp/english/parj/whole.html

Okura H, Matsuyama AJJoSCR. History of development and regulations for regenerative medicines in Japan. J Stem Cell Res Ther 2017;7(1):1-7.

The European Medicines Agency. Guideline on procedures for the granting of a marketing authorisation under exceptional circumstances, pursuant to article 14 (8) of regulation (EC) no 726/2004. [Internet]. 2005. [cited 2019 Jan 24] Available from: https://www.ema.europa.eu/en/documents/regulatory-procedural-guideline/guideline-procedures-granting-marketing-authorisation-under-exceptional-circumstances-pursuant/2004_en.pdf

United State food and drug administration. Approved Cellular and Gene Therapy Products [Internet]. 2023 [cited 2023 May 20] [Available from: https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products

Pharmaceutical and Medical Devices Agency. Review Reports: Regenerative Medical Products [Internet]. 2023 [cited 2023 May 20]. Available from: http://www.pmda.go.jp/english/review-services/reviews/approved-information/0004.html.

The European Medicines Agency. CAT quarterly highlights and approved ATMPs January 2023 [Internet]. 2023 [cited 2023 May 26]. Available from: https://www.ema.europa.eu/en/documents/report/cat-quarterly-highlights-approved-atmps-january-2023_en.pdf