Skip to main content
Log in

Highly Priced Gene Therapies: A Wake-Up Call for Early Price Regulation

  • Commentary
  • Published:
PharmacoEconomics Aims and scope Submit manuscript

This is a preview of subscription content, log in via an institution to check access.

Access this article

Price excludes VAT (USA)
Tax calculation will be finalised during checkout.

Instant access to the full article PDF.

Institutional subscriptions

Fig. 1
Fig. 2

References

  1. US Food and Drug Administration. FDA approval brings first gene therapy to the United States: CAR T-cell therapy approved to treat certain children and young adults with B-cell acute lymphoblastic leukemia. Silver Spring: US FDA; 2017.

    Google Scholar 

  2. Novartis. Novartis receives first ever FDA approval for a CAR-T cell therapy, Kymriah™ (tisagenlecleucel, CTL019), for children and young adults with B-cell ALL that is refractory or has relapsed at least twice. 30 Aug 2017. Available at: https://www.pharma.us.novartis.com/news/media-releases/novartis-receives-first-ever-fda-approval-car-t-cell-therapy-kymriahtm. Accessed 5 Jan 2018.

  3. Bach PB, Giralt SA, Saltz LB. FDA approval of tisagenlecleucel: promise and complexities of a $475000 cancer drug. JAMA. 2017;318:1861–2.

    Article  Google Scholar 

  4. Institute for Clinical and Economic Review. Chimeric Antigen Receptor T-Cell Therapy for B Cell Cancers: Effectiveness and Value. Available at: https://icer-review.org/wp-content/uploads/2017/07/ICER_CAR_T_Draft_Evidence_Report_121917.pdf. Accessed 10 Mar 2018.

  5. DiMasi JA, Grabowski HG, Hansen RW. Innovation in the pharmaceutical industry: new estimates of R&D costs. J Health Econ. 2016;47:20–33.

    Article  Google Scholar 

  6. Morgan S, Grootendorst P, Lexchin J, Cunningham C, Greyson D. The cost of drug development: a systematic review. Health Policy. 2011;100:4–17.

    Article  Google Scholar 

  7. Chit A, Parker J, Halperin SA, Papadimitropoulos M, Krahn M, Grootendorst P. Toward more specific and transparent research and development costs: the case of seasonal influenza vaccines. Vaccine. 2014;32:3336–40.

    Article  Google Scholar 

  8. Prasad V, De JK, Mailankody S. The high price of anticancer drugs: origins, implications, barriers, solutions. Nat Rev Clin Oncol. 2017;14:381–90.

    Article  Google Scholar 

  9. Kesselheim AS, Avorn J, Sarpatwari A. The high cost of prescription drugs in the United States: origins and prospects for reform. JAMA. 2016;316:858–71.

    Article  Google Scholar 

  10. Bach PB. Indication-specific pricing for cancer drugs. JAMA. 2014;312:1629–30.

    Article  CAS  Google Scholar 

  11. Schuster SJ, Bishop MR, Tam CS, et al. Primary analysis of juliet: a global, pivotal, phase 2 trial of CTL019 in adult patients with relapsed or refractory diffuse large B-cell lymphoma. Blood. 2017;130:577.

    Google Scholar 

  12. Prasad V, Mailankody S. Research and development spending to bring a single cancer drug to market and revenues after approval. JAMA Intern Med. 2017;177:1569–75.

    Article  Google Scholar 

  13. Munos B. Can open-source R&D reinvigorate drug research? Nat Rev Drug Discov. 2006;5:723–9.

    Article  CAS  Google Scholar 

  14. Schuhmacher A, Gassmann O, Hinder M. Changing R&D models in research-based pharmaceutical companies. J Transl Med. 2016;14:105.

    Article  Google Scholar 

  15. Ardal C, Rottingen JA. Open source drug discovery in practice: a case study. PLoS Negl Trop Dis. 2012;6:e1827.

    Article  Google Scholar 

  16. Ardal C, Rottingen JA. An open source business model for malaria. PLoS One. 2015;10:e0117150.

    Article  Google Scholar 

  17. Van Voorhis WC, Adams JH, Adelfio R, et al. Open source drug discovery with the malaria box compound collection for neglected diseases and beyond. PLoS Pathog. 2016;12:e1005763.

    Article  Google Scholar 

  18. Danzon PM, Towse A. Differential pricing for pharmaceuticals: reconciling access, R&D and patents. Int J Health Care Finance Econ. 2003;3:183–205.

    Article  Google Scholar 

  19. Papanicolas I, Woskie LR, Jha AK. Health care spending in the United States and other high-income countries. JAMA. 2018;319:1024–39.

    Article  Google Scholar 

  20. Anderson GF, Reinhardt UE, Hussey PS, Petrosyan V. It’s the prices, stupid: why the United States is so different from other countries. Health Aff (Millwood). 2003;22:89–105.

    Article  Google Scholar 

  21. Dieleman JL, Squires E, Bui AL, et al. Factors associated with increases in US health care spending, 1996–2013. JAMA. 2017;318:1668–78.

    Article  Google Scholar 

Download references

Author information

Authors and Affiliations

Authors

Corresponding author

Correspondence to Feng Xie.

Ethics declarations

Funding

No funding was received for this study.

Conflict of interest

Dr. Feng Xie has no conflicts of interest to declare.

Rights and permissions

Reprints and permissions

About this article

Check for updates. Verify currency and authenticity via CrossMark

Cite this article

Xie, F. Highly Priced Gene Therapies: A Wake-Up Call for Early Price Regulation. PharmacoEconomics 36, 883–888 (2018). https://doi.org/10.1007/s40273-018-0664-z

Download citation

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1007/s40273-018-0664-z

Navigation