Reference
1. 식품의약품안전처 홈페이지 Available from: https://www.mfds.go.kr/brd/m_99/view.do?seq=45541&srchFr=&srchTo=&srchWord=&srchTp=&itm_seq_1=0&itm_seq_2=0&multi_itm_seq=0&company_cd=&company_nm=&page=2
2. 식품의약품안전처 홈페이지 Available from:
https://www.mfds.go.kr/brd/m_1060/view.do?seq=14860&srchFr=&srchTo=&srchWord=&srchTp=&itm_seq_1=0&itm_seq_2=0&multi_itm_seq=0&company_cd=&company_nm=&page=2
3. 식품의약품안전처 홈페이지 Available from: https://www.mfds.go.kr/brd/m_1060/view.do?seq=14849&srchFr=&srchTo=&srchWord=&srchTp=&itm_seq_1=0&itm_seq_2=0&multi_itm_seq=0&company_cd=&company_nm=&page=1
4. 식품의약품안전처 홈페이지 Available from: https://www.mfds.go.kr/brd/m_99/view.do?seq=45451&srchFr=&srchTo=&srchWord=&srchTp=&itm_seq_1=0&itm_seq_2=0&multi_itm_seq=0&company_cd=&company_nm=&page=3
5. 식품의약품안전처 홈페이지 Available from: https://www.mfds.go.kr/brd/m_99/view.do?seq=45434&srchFr=&srchTo=&srchWord=&srchTp=&itm_seq_1=0&itm_seq_2=0&multi_itm_seq=0&company_cd=&company_nm=&page=2
6. “MIDD Immunogenicity Workshop” US FDA Accessed May 28, 2021 Available from: https://www.fda.gov/media/147871/download
7. “Important Patient and Consumer Information About Regenerative Medicine Therapies” US FDA Accessed June 3, 2021
Available from: https://www.fda.gov/vaccines-blood-biologics/consumers-biologics/important-patient-and-consumer-information-about-regenerative-medicine-therapies
8. “Regulatory Education for Industry (REdI) Annual Conference 2021” US FDA Accessed June 3, 2021
Available from: https://www.fda.gov/drugs/regulatory-education-industry-redi-annual-conference-2021-07192021-07232021
9. “SUPPLEMENT APPROVAL ” US FDA Accessed June 11, 2021 Available from: https://www.fda.gov/media/150087/download
10. “FDA Approves StrataGraft for the Treatment of Adults with Thermal Burns“ Accessed June 15, 2021
Available from: https://www.fda.gov/news-events/press-announcements/fda-approves-stratagraft-treatment-adults-thermal-burns
11. “BLA APPROVAL” US FDA Accessed June 15, 2021 Available from: https://www.fda.gov/media/150131/download
12. “Chemistry, Manufacturing, and Controls Changes to an Approved Application: Certain Biological Products; Guidance for Industry” US FDA Accessed June 21, 2021
Available from: https://www.fda.gov/media/109615/download
13. “SUPPLEMENT APPROVAL” US FDA Accessed June 30, 2021 Available from: https://www.fda.gov/media/150463/download
14. “FDA approves first autologous cellularized scaffold for the repair of cartilage defects of the knee” US FDA Accessed Dec 13, 2016
Available from: https://www.fda.gov/news-events/press-announcements/fda-approves-first-autologous-cellularized-scaffold-repair-cartilage-defects-knee
15. “Transcripts for the Patient Engagement & Regenerative Medicine An FDA Workshop for Patient Advocate” US FDA Accessed June 30, 2021
Available from: https://www.fda.gov/media/150446/download
16. “Slides for the Patient Engagement & Regenerative Medicine: An FDA CBER Workshop for Patient Advocates” US FDA Accessed June 30, 2021
Available from: https://www.fda.gov/media/150445/download
17. “ Committee for Advanced Therapies (CAT): work-plan-2021-Jun” EMA. Accessed Jun. 16, 2021 Available from:
https://www.ema.europa.eu/en/documents/agenda/agenda-cat-agenda-16-18-june-2021-meeting_en.pdf
18. “Abecma” EMA. Accessed Jun. 25, 2021 Available from:
https://www.ema.europa.eu/en/news/first-cell-based-gene-therapy-treat-adult-patients-multiple-myeloma
19. “Orphan designations-eu304194” EMA. Accessed May. 31, 2021
Available from: https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu304194
20. “Orphan designations-eu3161740” EMA. Accessed Jun. 11, 2021
Available from: https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3161740
21. “Orphan designations-eu3202297” EMA. Accessed Jun. 24, 2021
Available from: https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3202297
22. “Orphan designations-eu3151434” EMA. Accessed Jun. 25, 2021
Available from: https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3151434
23. “Orphan designations-eu3151578” EMA. Accessed Jun. 24, 2021
Available from: https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3151578
24. “Orphan designations-eu3202312” EMA. Accessed Jun. 24, 2021
Available from: https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3202312
25. “Orphan designations-eu3202297” EMA. Accessed Jun. 23, 2021
Available from: https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3202297
26. “Dacogen” EMA. Accessed Jun. 04, 2021 Available from: https://www.ema.europa.eu/en/medicines/human/EPAR/dacogen
27. “Takara Bio grants BioNTech a commercial patents license of CAR T-cell Therapy drug” Biospectrum. Accessed June. 25, 2021
https://www.biospectrumasia.com/news/50/18447/takara-bio-grants-biontech-a-commercial-patents-license-of-car-t-cell-therapy-drug.html
28. “Japan's Fujifilm invests ¥90 B to increase capacity for biologics” Biospectrum. Accessed July. 6, 2021
https://www.biospectrumasia.com/news/50/18513/japans-fujifilm-invests-90-b-to-increase-capacity-for-biologics.html
29. “RepliCel Applies for Manufacturing Approval for Its Collagen and Tissue Regeneration Cell Therapies” Biospace. Accessed July. 7, 2021
https://www.biospace.com/article/releases/replicel-applies-for-manufacturing-approval-for-its-collagen-and-tissue-regeneration-cell-therapies/
30. 보건복지부 보도자료. Available from: http://www.mohw.go.kr/react/al/sal0301vw.jsp?PAR_MENU_ID=04&MENU_ID=0403&CONT_SEQ=350418&page=1
31. Iglesias-Lopez C., et al. Comparison of regulatory pathways for the approval of advanced therapies in the European Union and the United States. Cytotherapy. 2021 Mar;23(3):261-274.
32. ARM (Alliance for Regenerative Medicine), 2020: Growth & Resilience in Regenerative Medicine Annual Report, 2021. 3.
Available from: https://alliancerm.org/sector-report/2020-annual-report/
33. 한국바이오협회, 글로벌 첨단바이오의약품(재생의료) 산업 동향 보고서
Available from: https://www.koreabio.org/board/board.php?bo_table=report&key_type=b_subject&key_word=%EA%B8%80%EB%A1%9C%EB%B2%8C&idx=37
34. 생명공학정책연구센터, BioINwatch: 유전자, 세포 및 RNA 치료제 개발 활기
Available from: https://www.bioin.or.kr/board.do?num=307290&cmd=view&bid=issue&cPage=1&cate1=all&cate2=all2&s_key=title&s_str=%20RN&sdate=&edate=
35. 생명공학정책연구센터, BioINregulation: 미국 21세기 치유법 (21st Century Cures)의 바이오 규제혁신
Available from: https://www.bioin.or.kr/regulationView.do?num=307084&cmd=view&bid=regulation&cPage=1
36."Regenerative Medicine Advanced Therapy Designation" US FDA. Last modifed July 6, 2021 Available from:
https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/regenerative-medicine-advanced-therapy-designation
37. “PRIME: priority medicines” EU EMA. Accessed Jul 12, 2021
Available from: https://www.ema.europa.eu/en/human-regulatory/research-development/prime-priority-medicines
38. “Strategy of SAKIGAKE by MHLW” Japan PMDA. Accessed Jul 12, 2021
Available from: https://www.pmda.go.jp/english/review-services/reviews/advanced-efforts/0001.html
39. “Cumulative CBER Regenerative Medicine Advanced Therapy (RMAT) Designation Requests Received by Fiscal Year” US FDA. Last modifed June 30, 2021
Available from: https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/cumulative-cber-regenerative-medicine-advanced-therapy-rmat-designation-requests-received-fiscal
40. “Annual report 2020 published” EU EMA. Updated June 14, 2021
Available from: https://www.ema.europa.eu/en/documents/annual-report/2020-annual-report-european-medicines-agency_en.pdf
41. 미국약물정보학회 “Sakigake System: From Pilot to Permanent Summary and Impact of Pharmaceutical & Medical Device Act Amendments in Japan” Akiko Ikeda Janssen Pharmaceutical K.K.
Available from: https://globalforum.diaglobal.org/issue/december-2020/sakigake-system-from-pilot-to-permanent-summary-and-impact-of-pharmaceutical-medical-device-act-amendments-in-japan/
42. Qui T., Hanna E., and Toumi M. PB149 The expedited regulatory programs for regenerative medicines in the US, EU and Japan. Value in Health 2019 Vol. 22, Supplement S426.
43. 채수미, 박은자, 주민희, 구현민, 유원곤, 박실비아* 희귀의약품 제도의 국가별 비교 연구. 보건사회연구 2013. 33(2), 525-548
44. 한국보건산업진흥원, 국내외 희귀의약품 (Orphan Drug) 시장 및 연구개발 현황 분석
Available from: https://www.khidi.or.kr/board/view?&linkId=48774874&menuId=MENU01783&link=%26linkId%3D48774874%26menuId%3DMENU01783
45. 생명공학정책연구센터, BioINwatch: 희귀의약품 글로벌 시장 현황 및 전망
Available from: https://www.bioin.or.kr/board.do?num=259072&cmd=view&bid=issue&cPage=1&cate1=all&cate2=all2
46. 식품의약품안전평가원, [Apec 규제조화센터] 유럽의약품청 의약품허가제도
Available from: http://www.nifds.go.kr/brd/m_95/view.do?seq=30960&srchFr=&srchTo=&srchWord=&srchTp=&itm_seq_1=0&itm_seq_2=0&multi_itm_seq=0&company_cd=&company_nm=&page=1
47. “Incentives and Regulatory consideration in orphan drug,medical device development” Japan PMDA. Accessed Jul 12, 2021
Available from: https://www.pmda.go.jp/files/000205953.pdf
48. “2017 Summary Basis for Regulatory Action – YESCARTA” US FDA. Last updated Oct 18, 2017
Available from: https://www.fda.gov/media/108788/download
49. “2017 Summary Basis for Regulatory Action – KYMRIAH” US FDA. Last updated Oct 30, 2017
Available from: https://www.fda.gov/media/107962/download
50. “Kymriah : EPAR - Public assessment report” EU EMA. Last updated Sep 19 2018
Available from: https://www.ema.europa.eu/en/documents/assessment-report/kymriah-epar-public-assessment-report_en.pdf
51. “Yescarta : EPAR - Public assessment report” EU EMA. Last updated Sep 10 2018
Available from: https://www.ema.europa.eu/en/documents/assessment-report/yescarta-epar-public-assessment-report_en.pdf
52. “Health technology assessment bodies” EU EMA. Accessed July 12, 2021
Available from: https://www.ema.europa.eu/en/partners-networks/health-technology-assessment-bodies
53. Seimetz D., Heller K., Richter J. Approval of First CAR-Ts: Have we Solved all Hurdles for ATMPs? Cell Med. 2019 Jan 22;11:2155179018822781.
54. “Draft toolbox guidance on scientific elements and regulatory tools to support quality data packages for PRIME marketing authorisation applications” EU EMA. Updated Feb 2, 2021
Available from:
https://www.ema.europa.eu/en/documents/scientific-guideline/draft-toolbox-guidance-scientific-elements-regulatory-tools-support-quality-data-packages-prime_en.pdf
55. “Recommendations on medication errors” EU EMA. Accessed July 12, 2021
Available from:
https://www.ema.europa.eu/en/human-regulatory/post-authorisation/pharmacovigilance/medication-errors/recommendations-medication-errors
56. “2018 Assessment report – KYMRIAH” EU EMA. Updated June 28 2018
Available from: https://www.ema.europa.eu/en/documents/assessment-report/kymriah-epar-public-assessment-report_en.pdf
57. 식품의약품안전평가원, “2018년도 ”의약품위해성관리계획 (RMP) 교육 워크숍 발표자료“
Available from: https://www.nifds.go.kr/brd/m_13/view.do?seq=9640&srchFr=&srchTo=&srchWord=&srchTp=&itm_seq_1=0&itm_seq_2=0&multi_itm_seq=0&company_cd=&company_nm=&page=3
58. Seoane-Vazquez E., Shukla V., Rodriguez-Monguio R Innovation and competition in advanced therapy medicinal products. EMBO Mol Med. 2019 Mar;11(3):e9992.
59. 생명공학정책연구센터, BioINdustry: 글로벌 희귀의약품 시장 및 전망
Available from: https://www.bioin.or.kr/board.do?num=301797&cmd=view&bid=watch
60. ”PMDA, Regenerative Medical Product, List of Approved Product“ Japan PMDA. Updated March, 2021
Available from: https://www.pmda.go.jp/english/review-services/reviews/approved-information/0002.html
61. Tanaka M., et al. Achievements and challenges of the Sakigake designation system in Japan. Br J Clin Pharmacol. 2021. Mar10; doi: 10.1111/bcp.14807.