Regulatory approval for autologous human cells and tissue products in the United States, the European Union, and Japan

Regenerative Therapy

Volumn 1, Issue , 2015, Pages 45-56

  Yano, Kazuo ^a,b   Watanabe, Natsumi ^a   Tsuyuki, Kenichiro ^a   Ikawa, Taisuke ^a   Kasanuki, Hiroshi ^a,b   Yamato, Masayuki ^a  

^a TOKYO WOMEN'S MEDICAL UNIVERSITY   (Japan)

^b WASEDA UNIVERSITY   (Japan)

Author keywords

Autologous cell; Chondrocyte; Epithelial cell; Fibroblast; Macrophage; Regenerative medicine; Regulatory science; Tissue engineering

Indexed keywords

ALIPOGENE TIPARVOVEC; AUTOLOGOUS CELL PRODUCT; AZFICEL T; BIOLOGICAL PRODUCT; SIPULEUCEL T; TISSUE PRODUCT; UNCLASSIFIED DRUG;

ARTICLE; BIOLOGICAL THERAPY; CHONDROCYTE IMPLANT; CONTROLLED STUDY; DRUG APPROVAL; DRUG LEGISLATION; EUROPEAN UNION; GRAFT REJECTION; HUMAN; HUMAN CELL; HUMAN TISSUE; IMMUNOSUPPRESSIVE TREATMENT; JAPAN; PERIPHERAL BLOOD MONONUCLEAR CELL; POSTMARKETING SURVEILLANCE; QUALITY CONTROL; RANDOMIZED CONTROLLED TRIAL (TOPIC); TISSUE ENGINEERING; UNITED STATES;

EID: 84941315998     PISSN: None     EISSN: 23523204     Source Type: Journal    
DOI: 10.1016/j.reth.2014.10.001     Document Type: Article

References (66)

1. US Food and Drug Administration Strategic Plan for regulatory science and research 2012-2016 2012, Available from: URL:. http://www.fda.gov/downloads/BiologicsBloodVaccines/ScienceResearch/UCM303542.pdf.
2. US Food and Drug Administration Human cells, tissues, and cellular and tissue-based products, 21CFR1271 2012, Available from: URL:. http://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/CFRSearch.cfm?CFRPart=1271.
3. European Parliament and the Council of the European Union Regulation (EC) No 1394/2007 of the European Parliament and of the Council of 13 November 2007 on advanced therapy medicinal products and amending Directive 2001/83/EC and Regulation (EC) No 726/2004. Off J Eur Union 2007, L324:121-137.
4. Pharmaceutical and Food Safety Bureau Quality and safety assurance for drug products or medicinal devices with processed (homogeneous) human-derived cells or tissue 2008, Yakushokuhatsu. 0912006.
5. Pharmaceutical and Food Safety Bureau Quality and safety assurance for drug products or medicinal devices with processed (autologus) human-derived cells or tissue 2008, Yakushokuhatsu. 0208003.
6. US Food and Drug Administration Definition of primary mode of action of a combination product. Fed Regist 2005, 70:49848-49862.
7. ® cultured epidermal autograft (CEA) - H990002 2007, Available from: URL:. http://www.accessdata.fda.gov/cdrh_docs/pdf//h990002a.pdf.
8. Pharmaceuticals and Medical Devices Agency Approval letter: other surgical/orthpedic materials, autologous cultured epidermis 2007, JACE, Available from: URL:, [in Japanese]. http://www.pmda.go.jp/operations/shonin/info/new/h19medicaldevice:beppyo.html.
9. Malarkey M.A., Witten C.M. Approval letter - Laviv. 2011 June 21, 2011, Available from: URL:. http://www.fda.gov/BiologicsBloodVaccines/CellularGeneTherapyProducts/ApprovedProducts/ucm260486.htm.
10. European Medicines Agency Authorisation: ChondroCelect 2009, Available from: URL:. http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/human/medicines/000878/human_med_000698.jsp%26mid=WC0b01ac058001d124.
11. Siegel J.P., Donlon J.A. Approval letter - Carticel. 1997 August 22, 1997, Available from: URL:. http://www.fda.gov/BiologicsBloodVaccines/CellularGeneTherapyProducts/ApprovedProducts/ucm171702.htm.
12. European Medicines Agency Authorisation: MACI 2013, Available from: URL:. http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/human/medicines/002522/human_med_001660.jsp%26mid=WC0b01ac058001d124.
13. Pharmaceutical and Food Safety Bureau Approval letter: human autologous cells and tissues. JACC 2012, Available from: URL:, [in Japanese]. http://www.pmda.go.jp/operations/shonin/info/new/h24medicaldevice:beppyo.html.
14. Malarkey M.A., Witten C.M. Approval letter April 29, 2010, Provenge, Available from: URL:. http://www.fda.gov/BiologicsBloodVaccines/CellularGeneTherapyProducts/ApprovedProducts/ucm210215.htm.
15. European Medicines Agency Authorisation 2013, Provenge, Available from: URL:. http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/human/medicines/002513/human_med_001680.jsp%26mid=WC0b01ac058001d124.
16. Yano K., Tsuyuki K., Watanabe N., Kasanuki H., Yamato M. The regulation of allogeneic human cells and tissue products as biomaterials. Biomaterials 2013, 34:3165-3173.
17. US Food and Drug Administration Vaccines, blood & biologics, marketed products, approved products 2012 2012, Available from: URL:. http://www.fda.gov/BiologicsBloodVaccines/CellularGeneTherapyProducts/ApprovedProducts/default.htm.
18. US Food and Drug Administration Listing of CDRH humanitarian device exemptions 2012, Available from: URL:. http://www.fda.gov/MedicalDevices/ProductsandMedicalProcedures/DeviceApprovalsandClearances/HDEApprovals/ucm161827.htm.
19. US Food and Drug Administration Summary for basis of approval: autologous cultured chondrocytes 1997, Carticel™, Available from: URL:. http://www.fda.gov/downloads/BiologicsBloodVaccines/CellularGeneTherapyProducts/ApprovedProducts/UCM109341.pdf.
20. US Food and Drug Administration Summary of safety and probable benefit: cultured epidermal autograft (CEA) 2007, Epicel®, Available from: URL:. http://www.accessdata.fda.gov/cdrh_docs/pdf/H990002b.pdf.
21. US Food and Drug Administration Clinical review: Sipulecel-T 2010, Provenge®, Available from: URL:. http://www.fda.gov/downloads/BiologicsBloodVaccines/CellularGeneTherapyProducts/ApprovedProducts/UCM214540.pdf.
22. US Food and Drug Administration Clinical review: autologous human fibroblast cells 2009, Available from: URL:. http://www.fda.gov/downloads/BiologicsBloodVaccines/CellularGeneTherapyProducts/ApprovedProducts/UCM263421.pdf.
23. European Medicines Agency Public assessment report: ChondroCelect 2009, Available from: URL:. http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Public_assessment_report/human/000878/WC500026035.pdf.
24. European Medicines Agency Public assessment report 2013, MACI, Available from URL:. http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Public_assessment_report/human/002522/WC500145888.pdf.
25. European Medicines Agency Public assessment report 2012, Provenge, Available from: URL:. http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Public_assessment_report/human/002513/WC500151101.pdf.
26. European Medicines Agency Public assessment report 2012, Glybera, Available from: URL:. http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Public_assessment_report/human/002145/WC500135476.pdf.
27. Pharmaceuticals and Medical Devices Agency Review report: other surgical/orthopedic material (autologous cultured epidermis). JACE 2007, Available from: URL:. http://www.pmda.go.jp/english/service/pdf/medical_devices/jace:oct2007_e.pdf.
28. Pharmaceutical and Food Safety Bureau Review report 2012, JACC, (in Japanese). Available from: URL:. http://www.info.pmda.go.jp/nmdevices/M201200024/340938000_22400BZX00266000_R100_2.pdf.
29. US Food and Drug Administration Guidance on applications for products comprised of living autologous cells manipulated ex vivi and intendeded for structual repair or reconstruction; availability. Fedral Regist 1996, 61:26523-26524.
30. US Food and Drug Administration Code of Federal Regulations Title 21, subpart E - accelated approval of biological products for serious or life-threating illnesses, Sec. 601.40 Scope 2012, Available from: URL:. http://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/CFRSearch.cfm?CFRPart=601%26showFR=1%26subpartNode=21:7.0.1.1.2.5.
31. Zaslav K., Cole B., Brewster R., DeBerardino T., Farr J., Fowler P., et al. Aprospective study of autologous chondrocyte implantation in patients with failed prior treatment for articular cartilage defect of the knee: results of the Study of the Treatment of Articular Repair (STAR) clinical trial. Am J Sports Med 2009, 37:42-55.
32. US Food and Drug Administration Code of Federal Regulation Title 21, subpart H - humanitarian use device 814.100-126 2012, Available from: URL:. http://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/CFRSearch.cfm?CFRPart=814%26showFR=1%26subpartNode=21:8.0.1.1.11.7.
33. Kantoff P.W., Higano C.S., Shore N.D., Berger E.R., Small E.J., Penson D.F., et al. Sipuleucel-T immunotherapy for castration-resistant prostate cancer. NEng J Med 2010, 363:411-422.
34. Smith S.R., Munavalli G., Weiss R., Maslowski J.M., Hennegan K.P., Novak J.M. Amulticenter, double-blind, placebo-controlled trial of autologous fibroblast therapy (Azficel-T) for the treatment of nasolabial fold wrinkles. Dermatol Surg 2012.
35. Levine W.D. Carticel (autologous cultured chondrocytes) dear healthcare professional letter Mar 2000, Available from: URL:. http://www.fda.gov/Safety/MedWatch/SafetyInformation/SafetyAlertsforHumanMedicalProducts/ucm176061.htm.
36. US Food and Drug Administration Appoval letter of dermagraft temporary covering 1997, Dermagraft-TC™, Available from: URL:. http://www.accessdata.fda.gov/cdrh_docs/pdf/p960007.pdf.
37. US Food and Drug Administration Apligraf™ (Graftskin) - P950032 1998, Available from: URL:. http://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfTopic/pma/pma.cfm?num=p950032.
38. US Food and Drug Administration OrCel™ bilayered cellular matrix - P010016 2001, Available from: URL:. http://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfTopic/pma/pma.cfm?num=p010016.
39. ® - P000036 2001, Available from: URL:. http://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfTopic/pma/pma.cfm?num=P000036.
40. US Food and Drug Administration GINTUIT (allogeneic cultured keratinocytes and fibroblasts in bovine collagen) 2012, Available from: URL:. http://www.fda.gov/BiologicsBloodVaccines/CellularGeneTherapyProducts/ApprovedProducts/ucm295465.htm.
41. European Parliament and the Council of the European Union Directive 2001/83/EC of the European Parliament and of the Council of 6 November 2001 on the community code relating to medicinal products for human use. Off J Eur Commun 2001, L311:67-128.
42. European Parliament and the Council of the European Union Regulation (EC) No 726/2004 0f the European Parliament and of the Council of 31 March 2004 laying down community procedure for the authorisation and supervision of medicinal products for human and veterinary use and establishing a European Medicines Agency. Off J Eur Union 2004, R0726:1-51.
43. Yakuji Nippo Ltd Outline of regulation for medical devices under the pharmaceutical affairs law. Guide to medical device registration in Japan 2004, 1-19. Yakuji Nippo, Ltd., Tokyo. 7th ed.
44. Arai H., Arai S., Takehiko A., Eiichi C., Jiro H., Yoko I., et al. Applications for biologics. Fundamentals of Japanese Regulatory Affairs 2010, 245-260. DC Regulatory Affairs Professionals Society, Washington. N. Susumu, A. Hideyuki (Eds.).
45. Shinzo Abe Prime Minister Law of Partial Revision of Pharmaceutical Affairs Law (Law of Efficacy and Safety Assurance for Medicinal Products, Medical Devices, etc.). Off Gazette 2013, Extra Edition 225, November 27.
46. US Food and Drug Administration Summary of basis of regulatory basis 2011, Available from: URL:. http://www.fda.gov/downloads/BiologicsBloodVaccines/CellularGeneTherapyProducts/ApprovedProducts/UCM262780.pdf.
47. National Institute of Biochemical Innovation Services to promote development of medicinal products for rare diseases 2012, Available from: URL:. http://www.nibio.go.jp/shinko/orphan/english/CCP011.html.
48. European Medicines Agency COMP (The Committee for Orphan Medicinal Products): overview 2012, Available from: URL:. http://www.ema.europa.eu/ema/index.jsp?curl=pages/about_us/general/general_content_000123.jsp%26mid=WC0b01ac0580028e32.
49. US Food and Drug Administration Guidance for industry fast track drug development programs - designation, development, and application review 2012, Available from: URL:. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM079736.pdf.
50. Wood J.J., Malek M.A., Frassica F.J., Polder J.A., Mohan A.K., Bloom E.T., et al. Autologous cultured chondrocytes: adverse events reported to the United States Food and Drug Administration. JBone Jt Surg Am 2006, 88:503-507.
51. Eichler H.G., Oye K., Baird L.G., Abadie E., Brown J., Drum C.L., et al. Adaptive licensing: taking the next step in the evolution of drug approval. Clin Pharmacol Ther 2012, 91:426-437.
52. US Food and Drug Administration Proposed approach to relation of cellular and tissue-based products 1997, Available from: URL:. http://www.fda.gov/downloads/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/Tissue/UCM062601.pdf.
53. US Food and Drug Administration Guidance for industry eligibility determination for donor of human cells, tissues, and cellular and tissue-based products (HCT/Ps) 2007, Available from: URL:. http://www.fda.gov/downloads/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/Tissue/ucm091345.pdf.
54. US Food and Drug Administration Guidance for industry current good tissue practice (CGTP) and additional requirements for manufactures of human cells, tissues, and cellular and tissue-based products (HCT/Ps) 2011, Available from: URL:. http://www.fda.gov/downloads/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/Tissue/UCM285223.pdf.
55. ® (azficel-T) package insert and patient information sheet 2011, Available from: URL:. http://www.fda.gov/downloads/BiologicsBloodVaccines/CellularGeneTherapyProducts/ApprovedProducts/UCM260489.pdf.
56. Japan Tissue Engineering Cop.Ltd. Pacakage insert: other surgical/orthopedic materials (autologus cultured epidermis). JACE 2010, Available from: URL. http://www.info.pmda.go.jp/downfiles/md/PDF/340938_21900BZZ00039000_A_01_04.pdf.
57. Japan Tissue Enginerring Cop.Ltd. Pacakage Insert: human autologous cells and tissue. JACC 2013, Available from: URL:. http://www.info.pmda.go.jp/downfiles/md/PDF/340938_22400BZX00266000_A_01_01.pdf.
58. ®) 2007, Available from: URL:. http://www.accessdata.fda.gov/cdrh_docs/pdf/H990002d.pdf.
59. European Medicines Agency Product information, annex III labeling and package leaflet 2013, MACI, Available from: URL:. http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Product_Information/human/002522/WC500145886.pdf.
60. ®: characterised viable autologous cartilage cells expanded exvivo expressing specific marker proteins) 2009, Available from: URL:. http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Product_Information/human/000878/WC500026031.pdf.
61. Dendreon Co. Package insert and patient information 2010, Provenge, Available from: URL:. http://www.fda.gov/downloads/BiologicsBloodVaccines/CellularGeneTherapyProducts/ApprovedProducts/UCM210031.pdf.
62. European Medicines Agency Product information, annex III labelling and pakage leaflet 2013, Provenge, Available from: URL:. http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Product_Information/human/002513/WC500151099.pdf.
63. Genzyme Tissue Repair Package insert (Carticel: autologous cultured chondrocytes) 2007, Available from: URL:. http://www.fda.gov/downloads/BiologicsBloodVaccines/CellularGeneTherapyProducts/ApprovedProducts/UCM109339.pdf.
64. US Food and Drug Administration Human cell & tissue products (HCT/P) adverse reaction reporting 2007, Available from: URL:. http://www.fda.gov/BiologicsBloodVaccines/SafetyAvailability/ReportaProblem/ucm152576.htm.
65. EudraVigilance Mandatory e-reporting essentials 2012, Available from: URL:. http://eudravigilance.ema.europa.eu/human/index.asp.
66. Pharmaceuticals and Medical Devices Agency Post-marketing safety 2012, Available from: URL:. http://www.pmda.go.jp/english/service/outline_p.html.