메뉴 건너뛰기




Volumn 18, Issue 8, 2016, Pages 1056-1061

Putting a price tag on novel autologous cellular therapies

Author keywords

adoptive T cell therapy; commercialization; cost of goods; good manufacturing practice; immunotherapy; market adoption; price; regulation; reimbursement; scale up

Indexed keywords

ADOPTIVE IMMUNOTHERAPY; ADOPTIVE TRANSFER; ARTICLE; AUTOTRANSPLANTATION; CELL THERAPY; GENE THERAPY; GOOD MANUFACTURING PRACTICE; HEALTH CARE COST; HUMAN; HUMAN CYTOMEGALOVIRUS; IMMUNOCOMPROMISED PATIENT; INVESTMENT; NONHUMAN; PRIORITY JOURNAL; REIMBURSEMENT; SCALE UP; T LYMPHOCYTE; BIOLOGICAL THERAPY; CELL ENGINEERING; COMMERCIAL PHENOMENA; ECONOMICS; EXPERIMENTAL THERAPY; PROCEDURES; TRANSPLANTATION;

EID: 84974815570     PISSN: 14653249     EISSN: 14772566     Source Type: Journal    
DOI: 10.1016/j.jcyt.2016.05.005     Document Type: Article
Times cited : (33)

References (30)
  • 1
    • 84884178203 scopus 로고    scopus 로고
    • Multicenter study of banked third-party virus-specific T cells to treat severe viral infections after hematopoietic stem cell transplantation
    • [1] Leen, A.M., Bollard, C.M., Mendizabal, A.M., Shpall, E.J., Szabolcs, P., Antin, J.H., et al. Multicenter study of banked third-party virus-specific T cells to treat severe viral infections after hematopoietic stem cell transplantation. Blood 121 (2013), 5113–5123.
    • (2013) Blood , vol.121 , pp. 5113-5123
    • Leen, A.M.1    Bollard, C.M.2    Mendizabal, A.M.3    Shpall, E.J.4    Szabolcs, P.5    Antin, J.H.6
  • 2
    • 84887501471 scopus 로고    scopus 로고
    • Safety and clinical efficacy of rapidly-generated trivirus-directed T cells as treatment for adenovirus, EBV, and CMV infections after allogeneic hematopoietic stem cell transplant
    • [2] Gerdemann, U., Katari, U.L., Papadopoulou, A., Keirnan, J.M., Craddock, J.A., Liu, H., et al. Safety and clinical efficacy of rapidly-generated trivirus-directed T cells as treatment for adenovirus, EBV, and CMV infections after allogeneic hematopoietic stem cell transplant. Mol Ther 21 (2013), 2113–2121.
    • (2013) Mol Ther , vol.21 , pp. 2113-2121
    • Gerdemann, U.1    Katari, U.L.2    Papadopoulou, A.3    Keirnan, J.M.4    Craddock, J.A.5    Liu, H.6
  • 4
    • 67649635053 scopus 로고    scopus 로고
    • Adoptive T-cell therapy of a lung transplanted patient with severe CMV disease and resistance to antiviral therapy
    • [4] Brestrich, G., Zwinger, S., Fischer, A., Schmück, M., Röhmhild, A., Hammer, M.H., et al. Adoptive T-cell therapy of a lung transplanted patient with severe CMV disease and resistance to antiviral therapy. Am J Transplant 9 (2009), 1679–1684.
    • (2009) Am J Transplant , vol.9 , pp. 1679-1684
    • Brestrich, G.1    Zwinger, S.2    Fischer, A.3    Schmück, M.4    Röhmhild, A.5    Hammer, M.H.6
  • 5
    • 37349073878 scopus 로고    scopus 로고
    • Infection in solid-organ transplant recipients
    • [5] Fishman, J.A., Infection in solid-organ transplant recipients. N Engl J Med 357 (2007), 2601–2614.
    • (2007) N Engl J Med , vol.357 , pp. 2601-2614
    • Fishman, J.A.1
  • 6
    • 64449085766 scopus 로고    scopus 로고
    • Delayed onset CMV disease in solid organ transplant recipients
    • [6] Husain, S., Pietrangeli, C.E., Zeevi, A., Delayed onset CMV disease in solid organ transplant recipients. Transpl Immunol 21 (2009), 1–9.
    • (2009) Transpl Immunol , vol.21 , pp. 1-9
    • Husain, S.1    Pietrangeli, C.E.2    Zeevi, A.3
  • 7
    • 84874828991 scopus 로고    scopus 로고
    • Cytomegalovirus in solid organ transplantation
    • [7] Razonable, R.R., Humar, A., Cytomegalovirus in solid organ transplantation. Am J Transplant 13:Suppl. 4 (2013), 93–106.
    • (2013) Am J Transplant , vol.13 , pp. 93-106
    • Razonable, R.R.1    Humar, A.2
  • 8
    • 80053490673 scopus 로고    scopus 로고
    • Antiviral drugs for viruses other than human immunodeficiency virus
    • [8] Razonable, R.R., Antiviral drugs for viruses other than human immunodeficiency virus. Mayo Clin Proc 86 (2011), 1009–1026.
    • (2011) Mayo Clin Proc , vol.86 , pp. 1009-1026
    • Razonable, R.R.1
  • 9
    • 84901985882 scopus 로고    scopus 로고
    • The clinical and financial burden of pre-emptive management of cytomegalovirus disease after allogeneic stem cell transplantation—implications for preventative treatment approaches
    • [9] Jain, N.A., Lu, K., Ito, S., Muranski, P., Hourigan, C.S., Haggerty, J., et al. The clinical and financial burden of pre-emptive management of cytomegalovirus disease after allogeneic stem cell transplantation—implications for preventative treatment approaches. Cytotherapy 16 (2014), 927–933.
    • (2014) Cytotherapy , vol.16 , pp. 927-933
    • Jain, N.A.1    Lu, K.2    Ito, S.3    Muranski, P.4    Hourigan, C.S.5    Haggerty, J.6
  • 10
    • 84892471446 scopus 로고    scopus 로고
    • The time is now: moving toward virus-specific T cells after allogeneic hematopoietic stem cell transplantation as the standard of care
    • [10] Saglio, F., Hanley, P.J., Bollard, C.M., The time is now: moving toward virus-specific T cells after allogeneic hematopoietic stem cell transplantation as the standard of care. Cytotherapy 16 (2014), 149–159.
    • (2014) Cytotherapy , vol.16 , pp. 149-159
    • Saglio, F.1    Hanley, P.J.2    Bollard, C.M.3
  • 11
    • 33845800093 scopus 로고    scopus 로고
    • Commission directive 2003/94/EC laying down the principles and guidelines of good manufacturing practice in respect of medicinal products for human use and investigational medicinal products for human use
    • [11] The European Commission, Commission directive 2003/94/EC laying down the principles and guidelines of good manufacturing practice in respect of medicinal products for human use and investigational medicinal products for human use. Off J Eur Union 13 (2003), 22–26 http://ec.europa.eu/health/files/eudralex/vol-1/dir_2003_94/dir_2003_94_en.pdf.
    • (2003) Off J Eur Union , vol.13 , pp. 22-26
  • 12
    • 84991822523 scopus 로고    scopus 로고
    • EudraLex—Volume 4 Good manufacturing practice (GMP) Guidelines
    • [12] EudraLex, EudraLex—Volume 4 Good manufacturing practice (GMP) Guidelines. http://ec.europa.eu/health/documents/eudralex/vol-4/index_en.htm, 2009.
    • (2009)
    • EudraLex1
  • 13
    • 84875476872 scopus 로고    scopus 로고
    • Good Manufacturing Practices (GMP)manufacturing of advanced therapy medicinal products: a novel tailored model for optimizing performance and estimating costs
    • [13] Abou-El-Enein, M., Römhild, A., Kaiser, D., Beier, C., Bauer, G., Volk, H.-D., et al. Good Manufacturing Practices (GMP)manufacturing of advanced therapy medicinal products: a novel tailored model for optimizing performance and estimating costs. Cytotherapy 15 (2013), 362–383.
    • (2013) Cytotherapy , vol.15 , pp. 362-383
    • Abou-El-Enein, M.1    Römhild, A.2    Kaiser, D.3    Beier, C.4    Bauer, G.5    Volk, H.-D.6
  • 14
    • 84903820011 scopus 로고    scopus 로고
    • Manufacturing models permitting roll out/scale out of clinically led autologous cell therapies: regulatory and scientific challenges for comparability
    • [14] Hourd, P., Ginty, P., Chandra, A., Williams, D.J., Manufacturing models permitting roll out/scale out of clinically led autologous cell therapies: regulatory and scientific challenges for comparability. Cytotherapy 16 (2014), 1033–1047.
    • (2014) Cytotherapy , vol.16 , pp. 1033-1047
    • Hourd, P.1    Ginty, P.2    Chandra, A.3    Williams, D.J.4
  • 15
    • 84922188493 scopus 로고    scopus 로고
    • The business case for cell and gene therapies
    • [15] Abou-El-Enein, M., Bauer, G., Reinke, P., The business case for cell and gene therapies. Nat Biotechnol 32 (2014), 1192–1193.
    • (2014) Nat Biotechnol , vol.32 , pp. 1192-1193
    • Abou-El-Enein, M.1    Bauer, G.2    Reinke, P.3
  • 16
    • 84991824487 scopus 로고    scopus 로고
    • “See How” Octane Technology can impact your goals
    • [16] OCTANE, “See How” Octane Technology can impact your goals. http://octaneco.com/octane-download.pdf.
    • OCTANE1
  • 18
    • 84962367238 scopus 로고    scopus 로고
    • Hurdles in clinical implementation of academic Advanced Therapy Medicinal Products: a national evaluation
    • [18] de Wilde, S., Veltrop-Duits, L., Hoozemans-Strik, M., Ras, T., Blom-Veenman, J., Guchelaar, H.-J., et al. Hurdles in clinical implementation of academic Advanced Therapy Medicinal Products: a national evaluation. Cytotherapy 18:6 (2016), 797–805, 10.1016/j.jcyt.2016.02.010.
    • (2016) Cytotherapy , vol.18 , Issue.6 , pp. 797-805
    • de Wilde, S.1    Veltrop-Duits, L.2    Hoozemans-Strik, M.3    Ras, T.4    Blom-Veenman, J.5    Guchelaar, H.-J.6
  • 19
    • 84911997803 scopus 로고    scopus 로고
    • The puzzling situation of hospital exemption for advanced therapy medicinal products in Europe and stakeholders’ concerns
    • [19] Cuende, N., Boniface, C., Bravery, C., Forte, M., Giordano, R., Hildebrandt, M., et al. The puzzling situation of hospital exemption for advanced therapy medicinal products in Europe and stakeholders’ concerns. Cytotherapy 16 (2014), 1597–1600.
    • (2014) Cytotherapy , vol.16 , pp. 1597-1600
    • Cuende, N.1    Boniface, C.2    Bravery, C.3    Forte, M.4    Giordano, R.5    Hildebrandt, M.6
  • 20
    • 84945551120 scopus 로고    scopus 로고
    • The current status of orphan drug development in Europe and the US
    • [20] Hall, A.K., Carlson, M.R., The current status of orphan drug development in Europe and the US. Intractable Rare Dis Res 3 (2014), 1–7.
    • (2014) Intractable Rare Dis Res , vol.3 , pp. 1-7
    • Hall, A.K.1    Carlson, M.R.2
  • 21
    • 84909644152 scopus 로고    scopus 로고
    • A roadmap toward clinical translation of genetically-modified stem cells for treatment of HIV
    • [21] Abou-El-Enein, M., Bauer, G., Reinke, P., Renner, M., Schneider, C.K., A roadmap toward clinical translation of genetically-modified stem cells for treatment of HIV. Trends Mol Med 20 (2014), 632–642.
    • (2014) Trends Mol Med , vol.20 , pp. 632-642
    • Abou-El-Enein, M.1    Bauer, G.2    Reinke, P.3    Renner, M.4    Schneider, C.K.5
  • 22
    • 84890948712 scopus 로고    scopus 로고
    • Regulation of cell-based therapies in europe: current challenges and emerging issues
    • [22] Blasimme, A., Rial-Sebbag, E., Regulation of cell-based therapies in europe: current challenges and emerging issues. Stem Cells Dev 22 (2013), 14–19.
    • (2013) Stem Cells Dev , vol.22 , pp. 14-19
    • Blasimme, A.1    Rial-Sebbag, E.2
  • 23
    • 84857756807 scopus 로고    scopus 로고
    • Clinical development of advanced therapy medicinal products in Europe: evidence that regulators must be proactive
    • [23] Maciulaitis, R., D'Apote, L., Buchanan, A., Pioppo, L., Schneider, C.K., Clinical development of advanced therapy medicinal products in Europe: evidence that regulators must be proactive. Mol Ther 20 (2012), 479–482.
    • (2012) Mol Ther , vol.20 , pp. 479-482
    • Maciulaitis, R.1    D'Apote, L.2    Buchanan, A.3    Pioppo, L.4    Schneider, C.K.5
  • 24
    • 84960929762 scopus 로고    scopus 로고
    • Deciphering the EU clinical trials regulation
    • [24] Abou-El-Enein, M., Schneider, C.K., Deciphering the EU clinical trials regulation. Nat Biotechnol 34 (2016), 231–233.
    • (2016) Nat Biotechnol , vol.34 , pp. 231-233
    • Abou-El-Enein, M.1    Schneider, C.K.2
  • 25
    • 84991815883 scopus 로고    scopus 로고
    • Summary of the responses to the targeted stakeholder consultation on the development of Guidelines on Good Manufacturing Practice for Advanced Therapy Medicinal Products pursuant to Article 5 of Regulation 1394/2007
    • [25] The European Commission, Summary of the responses to the targeted stakeholder consultation on the development of Guidelines on Good Manufacturing Practice for Advanced Therapy Medicinal Products pursuant to Article 5 of Regulation 1394/2007. http://ec.europa.eu/health/files/advtherapies/2015_11_pc_gmp_atmp/2015_11_pc_gmp_atmp_summary.pdf, 2015.
    • (2015)
  • 27
    • 84930892969 scopus 로고    scopus 로고
    • Gene therapy: a possible future standard for HIV care
    • [27] Abou-El-Enein, M., Bauer, G., Reinke, P., Gene therapy: a possible future standard for HIV care. Trends Biotechnol 33 (2015), 374–376.
    • (2015) Trends Biotechnol , vol.33 , pp. 374-376
    • Abou-El-Enein, M.1    Bauer, G.2    Reinke, P.3
  • 28
    • 77957305203 scopus 로고    scopus 로고
    • Adverse events following infusion of T cells for adoptive immunotherapy: a 10-year experience
    • [28] Cruz, C.R., Hanley, P.J., Liu, H., Torrano, V., Lin, Y.-F., Arce, J.A., et al. Adverse events following infusion of T cells for adoptive immunotherapy: a 10-year experience. Cytotherapy 12 (2010), 743–749.
    • (2010) Cytotherapy , vol.12 , pp. 743-749
    • Cruz, C.R.1    Hanley, P.J.2    Liu, H.3    Torrano, V.4    Lin, Y.-F.5    Arce, J.A.6
  • 29
    • 22544478762 scopus 로고    scopus 로고
    • HLA type-independent generation of antigen-specific T cells for adoptive immunotherapy
    • [29] Hammer, M.H., Meyer, S., Brestrich, G., Moosmann, A., Kern, F., Tesfa, L., et al. HLA type-independent generation of antigen-specific T cells for adoptive immunotherapy. Eur J Immunol 35 (2005), 2250–2258.
    • (2005) Eur J Immunol , vol.35 , pp. 2250-2258
    • Hammer, M.H.1    Meyer, S.2    Brestrich, G.3    Moosmann, A.4    Kern, F.5    Tesfa, L.6
  • 30
    • 84879076574 scopus 로고    scopus 로고
    • Impact of viral reactivations in the era of pre-emptive antiviral drug therapy following allogeneic haematopoietic SCT in paediatric recipients
    • [30] Hiwarkar, P., Gaspar, H.B., Gilmour, K., Jagani, M., Chiesa, R., Bennett-Rees, N., et al. Impact of viral reactivations in the era of pre-emptive antiviral drug therapy following allogeneic haematopoietic SCT in paediatric recipients. Bone Marrow Transplant 48 (2013), 803–808.
    • (2013) Bone Marrow Transplant , vol.48 , pp. 803-808
    • Hiwarkar, P.1    Gaspar, H.B.2    Gilmour, K.3    Jagani, M.4    Chiesa, R.5    Bennett-Rees, N.6


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.