Appendix B. EU, Japan, and FDA Preclinical Guidance Available for Cell Therapy Products.
| Title | Year | |
|---|---|---|
| EU | Guideline on the minimum quality and non-clinical data for certification of advanced therapy medicinal products | 2010 |
| Procedural advice on the certification of quality and non-clinical data for small and medium-sized enterprises developing advanced therapy medicinal products | 2010 | |
| Quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells | 2012 | |
| IPRP [A] expectations for biodistribution (BD) assessments for gene therapy (GT) products | 2018 | |
| Japan | PFSB [B] / ELD [C] notification no. 0210001, IDRAC [D] number: 49230 | 2005 |
| Assurance of quality and safety of medical devices and pharmaceutical products utilizing cells and tissues, amendments, IDRAC number: 118427 | 2010 | |
| Points to Consider when completing the application form for confirmation of quality and safety of cell/tissue processed medicinal products and medical devices, IDRAC number: 107478 | 2010 | |
| Ordinance on conducting non-clinical study on safety (good laboratory practice guideline) for regenerative medicine, IDRAC number: 200574 [Japanese] | 2014 | |
| Notice: PSEHB [E] / ELD: Technical guidance for conducting quality, nonclinical and clinical studies of the regenerative medicinal products (human cell-based product), IDRAC number: 229830 [English and Japanese] | 2016 | |
| MHLW [F] document for public comment: Recommendation for Undifferentiated pluripotent stem cell / transformed cell detection test, tumorigenicity study and genetic stability evaluation of human cell-based therapeutic products, draft version, IDRAC number: 257728 [Japanese] | 2017 | |
| US | Preclinical assessment of investigational cellular and gene therapy products | 2013 |
| Guidance for industry: Source Animal, product, preclinical, and clinical issues concerning the use of xenotransplantation products in humans | 2016 |
SOURCE: Reprinted with permission from Snehal Naik, Brian Dow, and Melody Eble.
NOTES: [A] IPRP = International Pharmaceutical Regulators Forum [B] PFSB = Pharmaceutical and Food Safety Bureau [C] ELD = Evaluation and Licensing Division [D] IDRAC = International Drug Regulatory Affairs Compendium [E] PSEHB = Pharmaceutical Safety and Environmental Health Bureau [F] MHLW = Ministry of Health, Labor, and Welfare