Open Access
Med Sci (Paris)
Volume 36, Number 4, Avril 2020
Page(s) 389 - 393
Section Repères
Published online 01 May 2020
  1. High KA, Roncarolo MG. Gene Therapy. N Engl J Med 2019 ; 381 : 455–464. [Google Scholar]
  2. Fischer A, Hacein-Bey-Abina S. Gene therapy for severe combined immunodeficiencies and beyond. J Exp Med 2020; 217 : e20190607. [CrossRef] [PubMed] [Google Scholar]
  3. Editorial. Gene therapy’s next installment. Nat Biotechnol 2019; 37 : 697. [CrossRef] [PubMed] [Google Scholar]
  4. Bach PB. National coverage analysis of CAR-T therapies - Policy, evidence, and payment. N Engl J Med 2018 ; 379 : 1396–1398. [Google Scholar]
  5. Mazzucato M.. High cost of new drugs. BMJ 2016 ; 354 : i4136. [Google Scholar]
  6. Fischer A, Dewatripont M, Goldman M. Benefit corporation: a path to affordable gene therapies?. Nat Med 2019 ; 25 : 1813–1814. [CrossRef] [PubMed] [Google Scholar]
  7. Middleton PG, Mall MA, Drevinek P, et al. Elexacaftor-tezacaftor-ivacaftor for cystic fibrosis with a single Phe508del allele. N Engl J Med 2019 ; 381 : 1809–1819. [Google Scholar]
  8. Collins FS. Realizing the dream of molecularly targeted therapies for cystic fibrosis. N Engl J Med 2019 ; 381 : 1863–1865. [Google Scholar]
  9. Bach PB. Anchoring’ was at work in setting the price of Novartis’ new gene therapy. June 4, 2019. [Google Scholar]
  10. Evaluate Pharma. World Preview 2019, Outlook to 2024 (2019). [Google Scholar]
  11. Stevens AJ, Jensen JJ, Wyller K, et al. The role of public-sector research in the discovery of drugs and vaccines. N Engl J Med 2011 ; 364 : 535–541. [Google Scholar]
  12. Nayak RK, Avorn J, Kesselheim AS. Public sector financial support for late stage discovery of new drugs in the United States: cohort study. BMJ 2019 ; 367 : l5766. [Google Scholar]
  13. Sarpatwari A, LaPidus AK, Kesselheim AS. Revisiting the National institutes of health fair pricing condition: promoting the affordability of drugs developed with government support. Ann Intern Med 2020. [Google Scholar]
  14. World Health Organization. Improving the transparency of markets for medicines, vaccines, and other health products. SEVENTY-SECOND WORLD HEALTH ASSEMBLY72/A/CONF./2 Rev. 1 Agenda item 11.7; 28 May 2019: [Google Scholar]
  15. Luzzatto L, Hyry HI, Schieppati A, et al. Outrageous prices of orphan drugs: a call for collaboration. Lancet 2018 ; 392 : 791–794. [CrossRef] [PubMed] [Google Scholar]
  16. Green A. Biotech companies defend prices of one-off gene therapy. Financial Times December 9, 2019. [Google Scholar]
  17. New York University Stern school of business. httpi// [Google Scholar]
  18. Thakor RT, Anaya N, Zhang Y, et al. Just how good an investment is the biopharmaceutical sector?. Nat Biotechnol 2017 ; 35 : 1149–1157. [CrossRef] [PubMed] [Google Scholar]
  19. Tay-Teo K, Ilbawi A, Hill SR. Comparison of sales income and research and development costs for FDA-approved cancer drugs sold by originator drug companies. JAMA Netw Open 2019 ; 2 : e186875. [PubMed] [Google Scholar]
  20. Bolton P, Dewatripont M. Contract theory. Cambridge : The MIT Press, 2004. [Google Scholar]
  21. Hart O. Firms, contracts, and financial structure. Clarendon Press-Oxford University Press, 1995. [CrossRef] [Google Scholar]
  22. Wineinger NE, Zhang, Topol EJ. Trends in prices of popular brand-name prescription drugs in the United States. JAMA Netw Open 2019; 2 : e194791. [PubMed] [Google Scholar]
  23. La Cour des Comptes. Le rapport public annuel 2017. 8 février 2017. [Google Scholar]
  24. Marsden G, Towse A, Pearson SD, et al. Gene therapy: understanding the science, assessing the evidence, and paying for value. ICER - Institute for clinical and economic review, a report from the 2016 ICER membership policy. March 2017. [Google Scholar]
  25. Business rountable. Our commitment. ourcommitment. Released: 2019; signatures updated: September 2019, December 2019, February 2020. [Google Scholar]
  26. Cummings B.. Benefit corporations : how to enforce a mandate to promote the public interest. Columbia Law Rev 2012 ; 112 : 578–627. [Google Scholar]
  27. DNDi - Drugs for Neglected Disease Initiative. [Google Scholar]
  28. Nielsen TB, Brass EP, Gilbert DN, et al. Sustainable discovery and development of antibiotics - Is a nnonprofit approach the future?. N Engl J Med 2019 ; 381 : 503–505. [Google Scholar]
  29. Barkats M. SMA: de la découverte du gène à la thérapie génique. Med Sci (Paris) 2020; 36 : 137–40. [CrossRef] [EDP Sciences] [PubMed] [Google Scholar]
  30. Braun S. Thérapies génétiques de l’amyotrophie spinale infantile : un morceau d’histoire de la médecine. Med Sci (Paris) 2020; 36 : 141–6. [CrossRef] [EDP Sciences] [PubMed] [Google Scholar]
  31. Pécoul B.. Un modèle alternatif et innovant de recherche et développement pour garantir l’accès aux médicaments. Maladies négligées, populations négligées et système d’innovation biomédicale. Med Sci (Paris) 2016 ; 32 : 1049–1050. [CrossRef] [EDP Sciences] [PubMed] [Google Scholar]

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