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Original article

Vol. 150 No. 5153 (2020)

Towards novel reimbursement models for expensive advanced therapy medicinal products (ATMPs)

Cite this as:
Swiss Med Wkly. 2020;150:w20355


Currently, a major focus of biomedical research and clinical application are the so-called advanced therapy medicinal products (ATMPs), which are highly complex medicines that enable the targeted and personalised treatment of patients. The potential of ATMPs in future cancer treatment is invaluable. However, this novel class of treatments is often extremely expensive. Consequently, these therapies push established reimbursement models to their limits. Because of the high costs, as well as the lack of appropriate reimbursement models, access to these potentially lifesaving therapies is currently not guaranteed to all patients. This paper analyses the current legal framework in Switzerland and critically evaluates existing reimbursement models, particularly with respect to their adaptation for ATMPs. As a promising reimbursement arrangement, this paper proposes a model combining outcome-based instalment payments with aspects of the pay for performance and the annuity payment model. According to this performance-based shared risk model, instalment payments are due when defined treatment goals are achieved.


  1. Ferlay J, Colombet M, Soerjomataram I, Dyba T, Randi G, Bettio M, et al. Cancer incidence and mortality patterns in Europe: Estimates for 40 countries and 25 major cancers in 2018. Eur J Cancer. 2018;103:356–87. doi:.
  2. Henn V. Kymriah: CAR-T-Zellen bekämpfen Leukämien und Lymphome. In: [cited 2020 August 4]. Available from:
  3. Darvin P, Toor SM, Sasidharan Nair V, Elkord E. Immune checkpoint inhibitors: recent progress and potential biomarkers. Exp Mol Med. 2018;50(12):1–11. doi:.
  4. Buchbinder EI, Desai A. CTLA-4 and PD-1 Pathways: Similarities, Differences, and Implications of Their Inhibition. Am J Clin Oncol. 2016;39(1):98–106. doi:.
  5. Prasad V, Kaestner V. Nivolumab and pembrolizumab: Monoclonal antibodies against programmed cell death-1 (PD-1) that are interchangeable. Semin Oncol. 2017;44(2):132–5. doi:.
  6. Syn NL, Teng MWL, Mok TSK, Soo RA. De-novo and acquired resistance to immune checkpoint targeting. Lancet Oncol. 2017;18(12):e731–41. doi:.
  7. European Medicines Agency. Darvadstrocel-Alofisel [cited 2020 August 4]. Available from:
  8. Scott LJ. Darvadstrocel: A Review in Treatment-Refractory Complex Perianal Fistulas in Crohn’s Disease. BioDrugs. 2018;32(6):627–34. doi:.
  9. Verstockt B, Ferrante M, Vermeire S, Van Assche G. New treatment options for inflammatory bowel diseases. J Gastroenterol. 2018;53(5):585–90. doi:.
  10. European Medicines Agency. Advanced Therapy Medicinal Products: Overview [cited 2020 August 4]. Available from:
  11. European Medicines Agency. Sperox-spheroids of human autologous matrix-associated chondrocytes [cited 2020 August 4]. Available from:
  12. Armoiry X, Cummins E, Connock M, Metcalfe A, Royle P, Johnston R, et al. Autologous Chondrocyte Implantation with Chondrosphere for Treating Articular Cartilage Defects in the Knee: An Evidence Review Group Perspective of a NICE Single Technology Appraisal. Pharmacoeconomics. 2019;37(7):879–86. doi:.
  13. Zhao Z, Chen Y, Francisco NM, Zhang Y, Wu M. The application of CAR-T cell therapy in hematological malignancies: advantages and challenges. Acta Pharm Sin B. 2018;8(4):539–51. doi:.
  14. Hucks G, Rheingold SR. The journey to CAR T cell therapy: the pediatric and young adult experience with relapsed or refractory B-ALL. Blood Cancer J. 2019;9(2):10. doi:.
  15. Dotti G, Gottschalk S, Savoldo B, Brenner MK. Design and development of therapies using chimeric antigen receptor-expressing T cells. Immunol Rev. 2014;257(1):107–26. doi:.
  16. Jackson HJ, Rafiq S, Brentjens RJ. Driving CAR T-cells forward. Nat Rev Clin Oncol. 2016;13(6):370–83. doi:.
  17. Neelapu SS, Tummala S, Kebriaei P, Wierda W, Gutierrez C, Locke FL, et al. Chimeric antigen receptor T-cell therapy - assessment and management of toxicities. Nat Rev Clin Oncol. 2018;15(1):47–62. doi:.
  18. Maude SL, Frey N, Shaw PA, Aplenc R, Barrett DM, Bunin NJ, et al. Chimeric antigen receptor T cells for sustained remissions in leukemia. N Engl J Med. 2014;371(16):1507–17. doi:.
  19. Maude SL, Laetsch TW, Buechner J, Rives S, Boyer M, Bittencourt H, et al. Tisagenlecleucel in Children and Young Adults with B-Cell Lymphoblastic Leukemia. N Engl J Med. 2018;378(5):439–48. doi:.
  20. Neelapu SS, Locke FL, Bartlett NL, Lekakis LJ, Miklos DB, Jacobson CA, et al. Axicabtagene Ciloleucel CAR T-Cell Therapy in Refractory Large B-Cell Lymphoma. N Engl J Med. 2017;377(26):2531–44. doi:.
  21. European Medicines Agency. Kymriah (tisagenlecleucel) [cited 2020 August 4]. Available from:
  22. Food and Drug Administration. Kymriah (tisagenlecleucel) [cited 2020 August 4]. Available from:
  23. European Medicines Agency. Yescarta (axicabtagene ciloleucel) [cited 2020 August 4]. Available from:
  24. Food and Drug Administration. Yescarta (axicabtagene ciloleucel) [cited 2020 August 4]. Available from:
  25. Swissmedic. Yescarta, 0,4–2 x 108 Zellen Infusionsdispersion (axicabtagene ciloleucel) [cited 2020 August 4]. Available from:
  26. Swissmedic. Kymriah, Zellsuspension zur Infusion (Tisagenlecleucelum) [cited 2020 August 4]. Available from:
  27. Supreme Court Decision 129 V 167 c. 3.1 p. 169 et seq.
  28. Federal Administrative Court Decisions C-6243/2014 of 2 May 2017 c. 4.1; C-6460/2011 of 24 June 2014 c. 4.2.1.
  29. Supreme Court Decisions 133 V 115 c. 3.1 p. 117; 128 V 159 c. 5c/aa p. 165.
  30. Supreme Court Decisions 130 V 299 c. 6.1 p. 304; 127 V 138 c. 5 p. 146; 9C_1011/2012 of 18 April 2013 c. 2.2.
  31. Federal Council, Statement on the revision of the health insurance of 6 November 1991, BBl 1992 I 93 et seq. p. 159.
  32. Supreme Court Decisions 142 V 26 c. 5.2.1 p. 35; 136 V 395 c. 7.4 p. 407 et seq.
  33. Federal Administrative Court Decision 2015/51 c. 8.2.2 p. 768.
  34. Supreme Court Decision 136 V 395 c. 5.1 p. 399.
  35. Rüefli C, Bolliger C. Off-Label-Use in der obligatorischen Krankenpflegeversicherung. Evaluation der Umsetzung von Artikel 71a und 71b KVV. Schlussbericht im Auftrag des Bundesamts für Gesundheit. Berne; 2014. p. 47 et seq.
  36. Cerny T, Lenz F. Innovative Medikamente: faire Preise und fairer Zugang für alle – ein Widerspruch? im dialog CSS 2019;3:10–11. p. 10; Wanner C. Die Krankenkasse zahlt nicht immer – der Bund will wissen wieso. Off-Label-Use von Medikamenten. In: SRF HeuteMorgen of 5 June 2019 [cited 2020 August 4]. Available from:
  37. Bauer K. Umstrittener Geheimvertrag für teure Krebstherapie. Preispoker in Pharmabranche. In: SRF 10vor10 of 17 June 2019 [cited 2020 August 4]. Available from:
  38. Handelszeitung. Abrechnung neuer Gentherapien: Santésuisse skizziert Lösung. In: Handelszeitung of 30 March 2019 [cited 2020 August 4]. Available from:
  39. Walter N. Die unglaubliche Heilung des Peter Rohr. In: Sonntagszeitung of 28 July 2019 [cited 2020 August 4]. Available from:
  40. Swiss DRGAG. Basisinformationen für Gesundheitsfachleute. Berne; 2015. p. 2.
  41. Inter alia, Supreme Court Decision 141 V 206 c. 2.1.2 p. 210.
  42. Oggier W. Zusatz- und Innovationsentgelte unter SwissDRG. Warum und wie gehandelt werden sollte. Berne; 2012. p. 6 et seq.
  43. Kipfer B, Witzmann C. Die Vergütung von Arzneimitteln im Einzelfall nach Art. 71a–d KVV. LSR 2019;2:89–109. p. 91.
  44. Haas V. Bundesrat genehmigt Tarifvertrag für innovative Gentherapien. HSK Einkaufsgemeinschaft Press Release of 6 December 2019 [cited 2020 August 4]. Available from:
  45. Burkhardt P. Novartis erhält für neues Krebsmittel bis zu 370’000 Franken. In: Tagesanzeiger of 16 June 2019 [cited 2020 August 4]. Available from:
  46. Kipfer B, Witzmann C. Die Vergütung von Arzneimitteln im Einzelfall nach Art. 71a–d KVV. LSR 2019;2:89–109. p. 108.
  47. FOPH. Der Bundesrat verabschiedet Tarifvereinbarung zur Vergütung einer innovativen Krebstherapie [cited 2020 August 30]. Available from,ist%20g%C3%BCltig%20bis%20zum%2031.
  48. Cohen J. At Over $2 Million Zolgensma Is The World’s Most Expensive Therapy, Yet Relatively Cost-Effective. In: Forbes of 5 June 2019 [cited 2020 August 4]. Available from:
  49. Danzon PM. Differential Pricing of Pharmaceuticals: Theory, Evidence and Emerging Issues. Pharmacoeconomics. 2018;36(12):1395–405. doi:.
  50. Harris E. Potential Solutions To Current Pricing Models For Cell And Gene Therapies. In: Life Science Leader of 1 October 2019 [cited 2020 August 4]. Available from:
  51. Alliance for Regenerative Medicine. Annual Regenerative Medicine Data Report 2018 [cited 2020 August 4]. Available from:
  52. Towse A, Garrison LP, Jr. Can’t get no satisfaction? Will pay for performance help?: toward an economic framework for understanding performance-based risk-sharing agreements for innovative medical products. Pharmacoeconomics. 2010;28(2):93–102. doi:.
  53. Antoñanzas F, Rodríguez-Ibeas R, Juárez-Castelló CA. Personalized Medicine and Pay for Performance: Should Pharmaceutical Firms be Fully Penalized when Treatment Fails? Pharmacoeconomics. 2018;36(7):733–43. doi:.
  54. European Medicines Agency. Luxturna (voretigene neparvovec) [cited 2020 August 4]. Available from:
  55. Food and Drugs Administration. Luxturna. (voretigene neparvovec-rzyl) [cited 2020 August 4]. Available from:
  56. Swissmedic. Luxturna (Wirkstoff: Voretigen Neparvovec). Public Summary SwissPAR vom 11.06.2020 [cited 2020August 4]. Available from:
  57. Spark Therapeutics. Spark Therapeutics Announces First-of-their-kind Programs to Improve Patient Access to LUXTURNA™ (voretigene neparvovec-rzyl), a One-time Gene Therapy Treatment. Press Release of 3 January 2018 [cited 2020 August 4]. Available from:
  58. NICE. NICE recommends novel gene therapy treatment for rare inherited eye disorder. Press Release of 4 September 2019 [cited 2020 August 4]. Available from:
  59. Jack Z. Novartis’s gene therapy Luxturna receives ‘considerable added benefit’ in Germany. Global Pricing Innovations Press Release of 18 October 2019 [cited 2020 August 4]. Available from:
  60. Novartis. AveXis Announces Innovative Zolgensma® Gene Therapy Access Programs for US Payers and Families. Press Release of 24 May 2019 [cited 2020 August 4]. Available from:
  61. Express Scripts Canada. Express Scripts Canada Introduces New Business Model. Press Release of 15 November 2011 [cited 2020 August 4]. Available from:
  62. Malik NN. Pay-for-performance pricing for a breakthrough heart drug: learnings for cell and gene therapies. Regen Med. 2016;11(3):225–7. doi:.
  63. Swissmedic. Requirements rel. to the authorisation documentation for TP/GT/GMO. AW-Information sheet, version 5.0. Berne; 2019. p. 4.
  64. Supreme Court Decision 136 V 84 c. 2.1 p. 86.
  65. Blaser N. Roche setzt Bundesamt unter Druck, Poker um Medikamentenpreise. In: SRF Rundschau of 31 January 2019 [cited 2020 August 4]. Available from:
  66. Fossgreen A. Ein Medikament nur für Mila. In: Tagesanzeiger of 4 November 2019 [cited 2020 August 4]. Available from:
  67. Viciano A, Catanzaro M. Krankenhäuser stellen eigene Krebsmedikamente her. In: Süddeutsche Zeitung of 27 June 2020 [cited 2020 August 4]. Available from:

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