White paper on how to go forward with cell-based advanced therapies in Europe

Tissue Engineering - Part A

Volumn 20, Issue 19-20, 2014, Pages 2549-2554

  Erben, Reinhold G ^a   Silva Lima, Beatriz ^b   Reischl, Ilona ^c   Steinhoff, Gustav ^d   Tiedemann, Gudrun ^d   Dalemans, Wilfried ^e   Vos, Alexander ^f   Janssen, Rob T A ^g   Le Blanc, Katarina ^h   Van Osch, Gerjo J V M ⁱ   Luyten, Frank P ^j  

^a UNIVERSITY OF VETERINARY MEDICINE   (Austria)

^b RESEARCH INSTITUTE FOR MEDICINES IMED ULISBOA   (Portugal)

^c AUSTRIAN AGENCY FOR HEALTH AND FOOD SAFETY   (Austria)

^d UNIVERSITY OF ROSTOCK   (Germany)

^e TiGenix   (Belgium)

^f Pharmacell   (Netherlands)

^g Scalable Life Sciences   (Netherlands)

^h KAROLINSKA UNIVERSITY HOSPITAL   (Sweden)

ⁱ ERASMUS MEDICAL CENTER   (Netherlands)

^j SKELETAL BIOLOGY AND ENGINEERING RESEARCH CENTER   (Belgium)

more..

Author keywords

[No Author keywords available]

Indexed keywords

PRODUCT DESIGN;

APPROPRIATE DESIGNS; INNOVATIVE SYSTEMS; MEDICAL RESEARCH COUNCILS; MODE OF ACTION; PRE-CLINICAL TESTING; PRODUCT CHARACTERIZATIONS; REGENERATIVE MEDICINE; VIENNA , AUSTRIA;

SAFETY TESTING;

ALGORITHM; ARTICLE; AUSTRIA; CELL BASED ADVANCED THERAPY; CELL THERAPY; CLINICAL PRACTICE; EUROPEAN UNION; HOSPITAL FINANCE; HUMAN; MEDICAL RESEARCH; OUTCOME ASSESSMENT; PATIENT SAFETY; PREDICTION; REIMBURSEMENT; RISK ASSESSMENT; STUDY DESIGN; THERAPY EFFECT; BIOLOGICAL THERAPY; CLINICAL TRIAL (TOPIC); ORGANIZATION; REGENERATIVE MEDICINE;

AUSTRIA; CELL- AND TISSUE-BASED THERAPY; CLINICAL TRIALS AS TOPIC; CONGRESSES AS TOPIC; EUROPEAN UNION; HUMANS; REGENERATIVE MEDICINE;

EID: 84907187544     PISSN: 19373341     EISSN: 1937335X     Source Type: Journal    
DOI: 10.1089/ten.tea.2013.0589     Document Type: Article

References (19)

1. European Commission. Medicinal Products for Human Use. Advanced Therapies. Available at http://ec.europa.eu/ health/human-use/advanced-therapies/index-en.htm (Last accessed on April 16, 2014).
2. European Medicines Agency. Advanced-therapy medicinal products. Available at www.ema.europa.eu/ema/index.jsp? curl = pages/regulation/general/general-content-000294 .jsp&mid = WC0b01ac05800241e0 (Last accessed on April 16, 2014).
3. U.S.Food and Drug Administration. Cellular & Gene Therapy Products. Available at www.fda.gov/Biologics-BloodVaccines/CellularGeneTherapyProducts/default.htm (Last accessed on April 16, 2014).
4. Tiedemann, G., and Sethe, S. Regulatory Frameworks for Cell and Tissue Based Therapies in Europe and the USA. In: Steinhoff, G., ed. Regenerative Medicine - from Protocol to Patient. Springer, Berlin, 2011.
5. European Parliament and Council. Regulation (EC) No 1394/2007 of the European Parliament and of the Council on advanced therapy medicinal products and amending Directive 2001/83/EC and Regulation (EC) No 726/2004. Available at http://eur-lex.europa.eu/LexUriServ/LexUriServ.do?uri =CELEX:32007R1394:en:NOT (Last accessed on April 16, 2014).
6. European Parliament and Council. Directive 2001/83/EC of the European Parliament and of the Council on the Community code relating to medicinal products for human use. Available at http://eur-lex.europa.eu/LexUriServ/LexUriServ .do?uri =CELEX:32001L0083:en:NOT (Last accessed on April 16, 2014).
7. European Commission. Regulation (EC) No. 1394/2007 on advanced therapy medicinal products: summary of the responses to the public consultation. Available at http://ec .europa.eu/health/files/advtherapies/2013-05-pc-atmp/2013-04-03-pc-summary.pdf (Last accessed on April 16, 2014).
8. Bianco, P., Cao, X., Frenette, P.S., Mao, J.J., Robey, P.G., Simmons, P.J., and Wang, C.Y. The meaning, the sense and the significance: translating the science of mesenchymal stem cells into medicine. Nat Med 19, 35, 2013.
9. European Medicines Agency. Guideline on Human Cellbased Medicinal Product, Doc. Ref. EMEA/CHMP/ 410869/2006. Available at www.ema.europa.eu/docs/en-GB/document-library/Scientific-guideline/2009/09/ WC500003898.pdf (Last accessed on April 16, 2014).
10. Rosenzweig, M., Connole, M., Glickman, R., Yue, S.P., Noren, B., DeMaria, M., and Johnson, R.P. Induction of cytotoxic T lymphocyte and antibody responses to enhanced green fluorescent protein following transplantation of transduced CD34+ hematopoietic cells. Blood 97, 1951, 2001.
11. Andersson, G., Illigens, B.M., Johnson, K.W., Calderhead, D., LeGuern, C., Benichou, G., White-Scharf, M.E., and Down, J.D. Nonmyeloablative conditioning is sufficient to allow engraftment of EGFP-expressing bone marrow and subsequent acceptance of EGFP-transgenic skin grafts in mice. Blood 101, 4305, 2003.
12. Odörfer, K.I., Unger, N.J., Weber, K., Sandgren, E.P., and Erben, R.G. Marker tolerant, immunocompetent animals as a new tool for regenerative medicine and long-term cell tracking. BMC Biotechnol 7, 30, 2007.
13. European Medicines Agency. Guideline on the risk-based approach according to annex I, part IV of Directive 2001/ 83/EC applied to advanced therapy medicinal products, EMA/CAT/CPWP/686637/2011. Available at www.ema .europa.eu/docs/en-GB/document-library/Scientific-guideline/ 2013/03/WC500139748.pdf (Last accessed on April 16, 2014).
14. European Medicines Agency. EMA EPAR - Public assessment report, ChondroCelect. Available at www.ema.europa.eu/docs/en-GB/document-library/EPAR-Summary- for-the-public/human/000878/WC500026033.pdf (Last accessed on April 16, 2014).
15. Rosenblatt, B. Meeting regulatory challenges for cell-based therapies. BioProcess Int 10, 8, 2012.
16. Baum, E., Littman, N., Ruffin, M., Ward, S., and Aschheim, K. Key tools and technology hurdles in advancing stem-cell therapies. a white paper sponsored by: california institute for regenerative medicinealliance for regenerative medicine cell therapy catapult. Available at www.cirm.ca .gov/sites/default/files/files/funding-page/Key-Tools-Tech-Hurdles-in-Advancing-Stem-Cell-Therapies.pdf (Last accessed on April 16, 2014).
17. Scott, C. A Decade of Processing. BioProcess Int 10, 24, 2012.
18. Abou-El-Enein, M., Romhild, A., Kaiser, D., Beier, C., Bauer, G., Volk, H.D., and Reinke, P. Good Manufacturing Practices (GMP) manufacturing of advanced therapy medicinal products: a novel tailored model for optimizing performance and estimating costs. Cytotherapy 15, 362, 2013.
19. Maciulaitis, R., D'Apote, L., Buchanan, A., Pioppo, L., and Schneider, C.K. Clinical development of advanced therapy medicinal products in Europe: evidence that regulators must be proactive. Mol Ther 20, 479, 2012.