References
- Gammie T, Lu CY, Babar ZU. Access to orphan drugs: a comprehensive review of legislations, regulations and policies in 35 countries. PLoS One. 2015;10(10):e0140002.
- Khosla N, Valdez R. A compilation of national plans, policies and government actions for rare diseases in 23 countries. Intractable Rare Dis Res. 2018;7(4):213–222.
- Benjamin K, Vernon MK, Patrick DL, et al. Patient-reported outcome and observer-reported outcome assessment in rare disease clinical trials: an ISPOR COA emerging good practices task force report. Value Health. 2017;20(7):838–855.
- Jayasundara K, Hollis A, Krahn M, et al. Estimating the clinical cost of drug development for orphan versus non-orphan drugs. Orphanet J Rare Dis. 2019;14(1):12.
- Lacaze P, Millis N, Fookes M, et al. Rare disease registries: a call to action. Intern Med J. 2017;47(9):1075–1079.
- Sun W, Zheng W, Simeonov A. Drug discovery and development for rare genetic disorders. Am J Med Genet A. 2017;173(9):2307–2322.
- Hanna E, Remuzat C, Auquier P, et al. Advanced therapy medicinal products: current and future perspectives. J Mark Access Health Policy.. 2016;4(1):31036.
- Food and Drug Administration. Regenerative medicine advanced therapy designation. 2019. cited 2020 Apr 28. Available from:https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/regenerative-medicine-advanced-therapy-designation.
- Qiu T, Dabbous M, Chachoua L, et al. The diversity in regenerative medicines regulations in Europe, USA and Japan. Cell Gene Ther Insights. 2019;5(8):1031–1042.
- Coherent Market Insights. Global cell and gene therapy market to surpass US$ 35.4 Billion by 2026. Globe newswire. 2019. cited 2020 Mar 14. Available from: http://www.globenewswire.com/newsrelease/2019/02/05/1710761/0/en/Global-Cell-and-Gene-Therapy-Market-to-Surpass-US-35-4-Billion-by-2026.html
- Korchagina D, Millier A, Vataire A-L, et al. Determinants of orphan drugs prices in France: A regression analysis. Orphanet J Rare Dis. 2017;12(1):75
- Jayasundara K, Hollis A, Krahn M, et al. Estimating the clinical cost of drug development for orphan versus non-orphan drugs. Orphanet J Rare Dis. 2019;14(1):12.
- Grace Hampson AT, Pearson SD, Dreitlein WB, et al. Gene therapy: evidence, value and affordability in the US health care system. J Comp Eff Res. 7(1): 15–28. 2018.
- Ronfard V, Vertes AA, May MH, et al. Evaluating the past, present, and future of regenerative medicine: a global view. Tissue Eng Part B Rev. 2017;23(2):199–210.
- Wurster CD, Ludolph AC. Nusinersen for spinal muscular atrophy. Ther Adv Neurol Disord.. 2018;11: 1756285618754459.
- Pipes SC How this gene therapy drug earned its $2.1 million price tag. 2020. cited 2020 Apr 28. Available from: https://fortune.com/2020/02/07/zolgensma-high-drug-prices/
- European Medicines Agency. Committee for orphan medicinal products (COMP). 2020. cited 2020 Apr 28. Available from: https://www.ema.europa.eu/en/committees/committee-orphan-medicinal-products-comp
- Medic G, Korchagina D, Young KE, et al. Do payers value rarity? An analysis of the relationship between disease rarity and orphan drug prices in Europe. J Mark Access Health Policy. 2017;5(1):1299665.
- Farkas AM, Mariz S, Stoyanova-Beninska V, et al. Advanced therapy medicinal products for rare diseases: state of play of incentives supporting development in Europe. Front Med (Lausanne). 2017;4:53.
- Alliance for Regenerative Medicine. Leading innovation-the UK’s ATMP landscape. 2019.
- BIOTECH S. Why France is leading the gene and cell therapy field in Europe. 2019. cited 2019 Dec 14. Available from: https://www.sirion-biotech.com/newsroom_post/why-france-is-leading-the-gene-and-cell-therapy-field-in-europe/.
- Fraldi A, Serafini M, Sorrentino NC, et al. Gene therapy for mucopolysaccharidoses: in vivo and ex vivo approaches. Ital J Pediatr. 2018 Nov 16;44(Suppl 2):130.
- Mark A Kay SLW, Woo SLC. Gene therapy for metabolic disorders. Trends Genet. 1994;10(7):253–257.
- Batty P, Lillicrap D. Advances and challenges for hemophilia gene therapy. Hum Mol Genet. 2019;28(R1):R95–R101.
- Perrin GQ, Herzog RW, Markusic DM. Update on clinical gene therapy for hemophilia. Blood. 2019;133(5):407–414.
- Today HN BioMarin seeks marketing approval in Europe for its investigational gene therapy, valoctocogene roxaparvovec. 2020. cited 2020 Jul 29. Available from: https://hemophilianewstoday.com/2019/11/25/biomarin-submits-maa-to-ema-for-valoctocogene-roxaparvovec/
- Ziccardi L, Cordeddu V, Gaddini L, et al. Gene therapy in retinal dystrophies. Int J Mol Sci. 2019;20(22):5722.
- Augustine EF, Adams HR, Mink JW. Clinical trials in rare disease: challenges and opportunities. J Child Neurol. 2013;28(9):1142–1150.
- Gagne JJ, Thompson L, O’Keefe K, et al. Innovative research methods for studying treatments for rare diseases: methodological review. BMJ. 2014;349(nov24 15): g6802.
- Abou-El-Enein M, Elsanhoury A, Reinke P. Overcoming challenges facing advanced therapies in the EU market. Cell Stem Cell. 2016;19(3):293–297.
- Kilix S. Trends in cell and gene therapy clinical development for rare and ultra-rare diseases. Cell Gene Ther Insights. 2020;6(3):543–547.
- De Castro Lopez MJ. Advanced therapy clinical trials for rare pediatric disorders: key challenges and lessons learned. Cell Gene Ther Insights. 2020;6(3):511–515.
- Potter BK, Khangura SD, Tingley K, et al. Translating rare-disease therapies into improved care for patients and families: what are the right outcomes, designs, and engagement approaches in health-systems research? Genet Med. 2016;18(2):117–123.
- Abrahamyan L, Feldman BM, Tomlinson G, et al. Alternative designs for clinical trials in rare diseases. Am J Med Genet C Semin Med Genet. 2016;172(4):313–331.
- Food and Drug Administration. Adaptive designs for clinical trials of drugs and biologics 2019. 2019. cited 2020 Feb 10. Available from: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/adaptive-design-clinical-trials-drugs-and-biologics-guidance-industry
- Zheng PP, Kros JM, Approved LJ. CAR T cell therapies: ice bucket challenges on glaring safety risks and long-term impacts. Drug Discov Today. 2018;23(6):1175–1182.
- Kim HT, Armand P. Clinical endpoints in allogeneic hematopoietic stem cell transplantation studies: the cost of freedom. Biol Blood Marrow Transplant. 2013;19(6):860–866.
- Nestler-Parr S, Korchagina D, Toumi M, et al. Challenges in research and health technology assessment of rare disease technologies: report of the ISPOR rare disease special interest group. Value Health. 21(5): 493–500. 2018.
- Mercieca-Bebber R, King MT, Calvert MJ, et al. The importance of patient-reported outcomes in clinical trials and strategies for future optimization. Patient Relat Outcome Meas.. 2018;9:353–367.
- Czech M, Baran-Kooiker A, Atikeler K, et al. A review of rare disease policies and orphan drug reimbursement systems in 12 Eurasian countries. Front Public Health. 2019;7:416.
- Young KE, Soussi I, Hemels M, et al. A comparative study of orphan drug prices in Europe. J Mark Access Health Policy. 2017;5(1):1297886.
- David Tordrup VT, Kanavos P. Orphan drug considerations in health technology assessment in eight european countries. Rare dis orphan drugs. 2014;1(3):86–97.
- Lakdawalla DN, Doshi JA, Garrison LP, et al. Defining elements of value in health care-a health economics approach: an ISPOR special task force report. Value Health. 2018;21(2):131–139.
- 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.
- Bubela T, Bonter K, Lachance S, et al. More haste, less speed: could public-private partnerships advance cellular immunotherapies? Front Med (Lausanne). 2017;4:134.
- Jaroslawski S, Toumi M. Non-profit drug research and development: the case study of Genethon. J Mark Access Health Policy. 2019;7(1):1545514.
- Investor’s Business Daily. Biotech stocks burn a hole in big pharma’s pocket; look for more mergers in 2020. 2019. cited 2020 Apr 15. Available from: https://www.investors.com/news/technology/biotech-stocks-catch-fire-big-pharma-opens-wallet/
- Chang D. Innovating CAR T cell therapy for today and tomorrow. Cell Gene Ther Insights. 2020;6(6):783–788.
- Liu C, Wu J. The rise of human induced pluripotent stem cell technology in drug development. Cell Gene Ther Insights. 2020;6(1):127–135.
- Bailey SR, Maus MV. Gene editing for immune cell therapies. Nat Biotechnol. 2019 Dec 01;37(12):1425–1434.
- Ashmore-Harris C, Fruhwirth GO. The clinical potential of gene editing as a tool to engineer cell-based therapeutics. Clin Transl Med. 2020 Feb 07;9(1):15.
- Food and Drug Administration. Rare disease therapy development and access remain top FDA priorities during COVID-19 2020. 2020. cited 2020 Aug 20. Available from: https://www.fda.gov/news-events/fda-voices/rare-disease-therapy-development-and-access-remain-top-fda-priorities-during-covid-19
- McKinsey. COVID-19 and cell and gene therapy: how to keep innovation on track 2020. 2020. cited 2020 Aug 20. Available from: https://www.mckinsey.com/industries/pharmaceuticals-and-medical-products/our-insights/covid-19-and-cell-and-gene-therapy-how-to-keep-innovation-on-track.
- Tarnowski J, Krishna D, Jespers L, et al. Delivering advanced therapies: the big pharma approach. Gene Ther. 2017 Sep;24(9):593–598.
- Hampson G, Towse A, Dreitlein WB, et al. Real-world evidence for coverage decisions: opportunities and challenges. J Comp Eff Res.. 2018 Dec;7(12):1133–1143.
- White W. A rare disease patient/caregiver perspective on fair pricing and access to gene-based therapies. Gene Ther. 2019 Nov 13.Epub ahead of print. PMID:31723211.DOI:10.1038/s41434-019-0110-7.
- Malik NN, Durdy MB. CAR T-cell therapies: formulation-product-proposition framework for commercialisation. Drug Discov Today. 2016 Nov;21(11):1731–1734.
- Firestone G. Immuno-oncology cell therapies: commercial considerations and strategies for the new decade. Cell Gene Ther Insights. 2020;6(6): 798.
- Keesara S, Jonas A, Schulman K. Covid-19 and health care’s digital revolution. N Engl J Med. 2020 Jun 04;382(23):e82.
- Whitelaw S, Mamas MA, Topol E, et al. Applications of digital technology in COVID-19 pandemic planning and response. Lancet Digital Health. 2020;2(8):e435–e440.