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Editorial

Gene therapies, uncertainty, and decision-making: thinking about the last mile at the first step

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Pages 853-856 | Received 03 Jul 2023, Accepted 02 Aug 2023, Published online: 09 Aug 2023

References

  • Hanna E, Toumi M, Dussart C, et al. Funding breakthrough therapies: A systematic review and recommendation. Health Policy. 2018;122:217–229. doi: 10.1016/j.healthpol.2017.11.012
  • Rosenberg SA, Aebersold P, Cornetta K, et al. Gene transfer into humans — immunotherapy of patients with advanced melanoma, using tumor-infiltrating lymphocytes modified by retroviral gene transduction. N Engl J Med. 1990;323(9):570–578. doi: 10.1056/NEJM199008303230904
  • “U.S. National Library of Medicine“ ClinicalTrials.Gov.
  • Gottlieb S Statement from FDA Commissioner Scott Gottlieb, M.D., Marks P. Director of the center for biologics evaluation and research on new policies to advance development of safe and effective cell and gene therapies. Administration, USFD. Montgomery County: (MD), 2019.
  • Eder C, Wild C. Technology forecast: advanced therapies in late clinical research, EMA approval or clinical application via hospital exemption. J Mark Access Health Policy. 2019;7(1):1600939. doi: 10.1080/20016689.2019.1600939
  • Hollier-Hann G, Cork D, Ralston S, et al. Health technology assessment of gene therapies for inherited genetic disorders in the US and Europe. Value Health. 2018;21:S210. doi: 10.1016/j.jval.2018.04.1421
  • Amanda Whittal EI, Hutchings A. Exploring the economics of gene therapy innovation and price. London, UK: Dolon Ltd; 2022. p. 18. https://bit.ly/3AhEoG1
  • Goncalves E. Value-based pricing for advanced therapy medicinal products: emerging affordability solutions. Eur J Health Econ. 2021;23(2):155–163. doi: 10.1007/s10198-021-01276-2
  • Carr DR, Bradshaw SE. Gene therapies: The challenge of super-high-cost treatments and how to pay for them. Regen Med. 2016;11(4):381–393. doi: 10.2217/rme-2016-0010
  • Salzman R, Cook F, Hunt T, et al. Addressing the value of gene therapy and enhancing patient access to transformative treatments. Mol Ther. 2018;26(12):2717–2726. doi: 10.1016/j.ymthe.2018.10.017
  • Barlow J, Bens C, Brent T, et al. AMCP partnership forum: designing benefits and payment models for innovative high-investment medications. J Manag Care Spec Pharm. 2019;25:156–162.
  • Drummond MF, Neumann PJ, Sullivan SD, et al. Analytic considerations in applying a general economic evaluation reference case to gene therapy. Value Health. 2019;22(6):661–668. doi: 10.1016/j.jval.2019.03.012
  • Jørgensen J, Kefalas P. Annuity payments can increase patient access to innovative cell and gene therapies under England’s net budget impact test. J Mark Access Health Policy. 2017;5(1):1355203. doi: 10.1080/20016689.2017.1355203
  • Cook K, Forbes SP, Adamski K, et al. Assessing the potential cost-effectiveness of a gene therapy for the treatment of hemophilia a. J Med Econ. 2020;23(5):501–512. doi: 10.1080/13696998.2020.1721508
  • Hettle R, Corbett M, Hinde S, et al. The assessment and appraisal of regenerative medicines and cell therapy products: An exploration of methods for review, economic evaluation and appraisal. Health Technol Assess. 2017;21(7):1–204. doi: 10.3310/hta21070
  • Angelis A, Naci H, Hackshaw A. Recalibrating health technology assessment methods for cell and gene therapies. PharmacoEconomics. 2020;38(12):1297–1308. doi: 10.1007/s40273-020-00956-w
  • Project” MNF. Designing precision financing for cures. In Financing and reimbursement of cures in the US. Initiatives. MNDDP Cambridge, MA: Massachusetts Institute of Technology; 2018. p. 1–3. https://newdigs.tuftsmedicalcenter.org/designing-precision-financing-for-cures/
  • Claxton K, Palmer S, Longworth L, et al. A comprehensive algorithm for approval of health technologies with, without, or only in research: the key principles for informing coverage decisions. Value Health. 2016;19(6):885–891. doi: 10.1016/j.jval.2016.03.2003
  • ten Ham RMT, Hövels AM, Klungel OH, et al. Development and regulation of gene and cell-based therapies in europe: a quantification and reflection. Trends Pharmacol Sci. 2020;41(2):67–71. doi: 10.1016/j.tips.2019.11.007
  • Pasi KJ, Rangarajan S, Mitchell N, et al. Multiyear follow-up of AAV5-Hfviii-SQ gene therapy for hemophilia a. N Engl J Med. 2020;382(1):29–40. doi: 10.1056/NEJMoa1908490
  • Ozelo MC, Mahlangu J, Pasi KJ, et al. Valoctocogene roxaparvovec gene therapy for hemophilia a. N Engl J Med. 2022;386(11):1013–1025. doi: 10.1056/NEJMoa2113708
  • Rothe M, Modlich U, Schambach A. Biosafety challenges for use of lentiviral vectors in gene therapy. Curr Gene Ther. 2013;13(6):453–468. doi: 10.2174/15665232113136660006
  • CfMPfHUC. Guideline on safety and efficacy follow-up and risk management of advanced therapy medicinal products. EMA London: (UK); 2018. https://www.ema.europa.eu/documents/scientific-guideline/guideline-safety-efficacy-follow-risk-management-advanced-therapy-medicinal-products_en.pdf
  • Garrison LP, Kamal-Bahl S, Towse A. Toward a broader concept of value: identifying and defining elements for an expanded cost-effectiveness analysis. Value Health. 2017;20(2):213–216. doi: 10.1016/j.jval.2016.12.005
  • Gardner J, Webster A, Barry J. Anticipating the clinical adoption of regenerative medicine: Building institutional readiness in the UK. Regen Med. 2018;13(1):29–39. doi: 10.2217/rme-2017-0121
  • Gardner J, Faulkner A, Mahalatchimy A, et al. Are there specific translational challenges in regenerative medicine? Lessons from other fields. Regen Med. 2015;10(7):885–895. doi: 10.2217/rme.15.50
  • Hopkins MM, Martin PA, Nightingale P, et al. The myth of the biotech revolution: An assessment of technological, clinical and organisational change. Res Policy. 2007;36(4):566–589. doi: 10.1016/j.respol.2007.02.013
  • Shire SJ, Shahrokh Z, Liu J. Challenges in the development of high protein concentration formulations. J Pharmaceut sci. 2004;93(6):1390–1402. doi: 10.1002/jps.20079
  • Shire SJ. Formulation and manufacturability of biologics. Curr Opin Biotechnol. 2009;20(6):708–714. doi: 10.1016/j.copbio.2009.10.006
  • Edlin R, Hall P, Wallner K, et al. Sharing risk between payer and provider by leasing health technologies: an affordable and effective reimbursement strategy for innovative technologies? Value Health. 2014;17(4):438–444. doi: 10.1016/j.jval.2014.01.010
  • Persson KSC U, Jonsson B. Alternative payment models in haemophilia treatment. Lund:IHE; 2016. https://ihe.se/wp-content/uploads/2016/12/IHE-Report-2016_10_.pdf
  • Quinn C, Ciarametaro M, Sils B, et al. Value-based performance arrangements for chronic conditions: an economic simulation of medicaid drug rebate program reforms. Expert Rev Pharmacoecon Outcomes Res. 2023;23(5):535–546. doi: 10.1080/14737167.2023.2193331
  • Project” MNF. How can self-insured employers prepare for the portfolio impact of highcost gene therapies coming to market? In Financing and reimbursement of cures in the US. Cambridge, MA:Massachusetts Institute of Technology; 2022. https://newdigs.tuftsmedicalcenter.org/how-can-self-insured-employers-prepare-for-the-portfolio-impact-of-highcost-gene-therapies-coming-to-market/
  • Trusheim MR, Cassidy WM, Bach PB. Alternative state-level financing for hepatitis C treatment-the “netflix model”. JAMA. 2018;320(19):1977–1978. doi: 10.1001/jama.2018.15782
  • van Nimwegen KJM, Lilford RJ, van der Wilt GJ, et al. Headroom beyond the quality- adjusted life-year: the case of complex pediatric neurology. Int J Technol Assess Health Care. 2017;33(1):5–10. doi: 10.1017/S0266462317000046

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