Skip to main content

Thank you for visiting nature.com. You are using a browser version with limited support for CSS. To obtain the best experience, we recommend you use a more up to date browser (or turn off compatibility mode in Internet Explorer). In the meantime, to ensure continued support, we are displaying the site without styles and JavaScript.

  • Perspective
  • Published:

Access to affordable medicines: obligations of universities and academic medical centers

Gene Therapy volume 30, pages 753–755 (2023)Cite this article

Subjects

Over the last decade, the development and approval of remarkably effective therapeutic interventions, many based on technologies directed at the human genome, have altered the treatment landscape for patients facing a range of serious and life-threatening conditions. These novel therapies include chimeric antigen receptor T cells (CAR-T) for lymphoma, childhood leukemia, and multiple myeloma; antisense oligonucleotide (ASO) therapy for spinal muscular atrophy (SMA); and gene therapy for SMA, Leber’s congenital amaurosis Type 2 (LCA, a rare form of inherited blindness), beta thalassemia major, and hemophilia B. Advances not directed at the genome include drugs targeting the cystic fibrosis transmembrane conductance protein, which have transformed the lives of most patients with that disease.

These therapies are effective but also expensive. For example, the U.S. list price of tisagenlecleucel CAR-T cell therapy for childhood leukemia is $475,000; the first year of ASO therapy for SMA is $750,000 ($375,000 for each subsequent year); and gene therapy ranges from $850,000 for LCA (both eyes) to $3.5 million for hemophilia B. High prices are not unique to cell and gene therapies. Combination therapy for cystic fibrosis runs approximately $300,000/year, and median inflation-adjusted launch prices for novel self-administered cancer drugs increased more than 20% from $188,842 to $232,788 per year of therapy between 2017 and 2021 [1, 2]. Prices for many of these therapies exceed accepted cost-effectiveness thresholds. [3]. Furthermore, even when therapies are cost-effective, high prices prevent many patients from accessing necessary drugs, whether through payers’ decisions not to cover them or through unaffordable copayments, resulting in worsened health outcomes [4]. Patients who receive these therapies can experience financial toxicity due to the substantial out-of-pocket costs that typically accompany receipt of costly drugs [5]. Finally, high prices impose burdens on all members of society through rising health insurance premiums, taxes, and the opportunity costs of forgone investment in other societal priorities.

This is a preview of subscription content, access via your institution

Access options

Buy this article

  • Purchase on Springer Link
  • Instant access to full article PDF
Buy now

Prices may be subject to local taxes which are calculated during checkout

References

  1. Institute for Clinical and Economic Review. Modulator treatments for cystic fibrosis: effectiveness and value. 2020. https://icer.org/wp-content/uploads/2020/08/ICER_CF_Final_Report_092320.pdf. Accessed 7 Jan 2023.

  2. Office of Representative Katie Porter. Skyrocketing: how big pharma exploits launch pricest to cash in on cancer. 2020. https://porter.house.gov/uploadedfiles/skyrocketing_-_how_big_pharma_exploits_launch_prices_to_cash_in_on_cancer.pdf. Accessed 7 Jan 2023.

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

    Article  PubMed  Google Scholar 

  4. Dusetzina SB, Huskamp HA, Rothman RL, Pinheiro LC, Roberts AW, Shah ND, et al. Many Medicare beneficiaries do not fill high-price specialty drug prescriptions. Health Aff. 2022;41:487–96.

    Article  Google Scholar 

  5. Yabroff KR, Dowling EC, Guy GP Jr., Banegas MP, Davidoff A, Han X, et al. Financial hardship associated with cancer in the united states: findings from a population-based sample of adult cancer survivors. J Clin Oncol. 2016;34:259–67.

    Article  PubMed  Google Scholar 

  6. Nayak RK, Lee CC, Avorn J, Kesselheim AS. Public-sector contributions to novel biologic drugs. JAMA Intern Med. 2021;181:1522–5.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  7. Galkina Cleary E, Beierlein JM, Khanuja NS, McNamee LM, Ledley FD. Contribution of NIH funding to new drug approvals 2010–2016. PNAS. 2018;115:2329–34.

    Article  PubMed  PubMed Central  Google Scholar 

  8. Edwards M, Murray F, Yu R. Gold in the ivory tower: equity rewards of outlicensing. Nat Biotechnol. 2006;24:509–15.

    Article  CAS  PubMed  Google Scholar 

  9. AUTM. AUTM 2020 Licensing Activity Survey. 2021. https://register.autm.net/detail.aspx?id=2020_APPENDIX. Accessed 7 Jan 2023.

  10. Mission Statement of the Perelman School of Medicine. https://www.med.upenn.edu/psom/overview.html. Accessed 7 Jan 2023.

  11. Chokshi DA. Improving access to medicines in poor countries: the role of universities. PLoS Med. 2006;3:e136.

    Article  PubMed  PubMed Central  Google Scholar 

  12. Nguyen T-Y, Veras J, Shahzad MA. Recent experiences in policy implementation of socially responsible licensing in select universities across Europe and North America: identifying key provisions to promote global access to health technologies. Les Nouvelles. 2018;53. https://papers.ssrn.com/sol3/papers.cfm?abstract_id=3218516. Accessed 7 Jan 2023.

  13. AUTM. Nine Points to Consider in Licensing University Technology. 2007. https://autm.net/about-tech-transfer/principles-and-guidelines/nine-points-to-consider-when-licensing-university. Accessed 7 Jan 2023.

  14. Contreras JL “In the public interest” – University technology transfer and the Nine Points Document – an empirical assessment. U.C. Irvine L Rev. 2022;12: forthcoming. https://papers.ssrn.com/sol3/papers.cfm?abstract_id=3990450. Accessed 7 Jan 2023.

  15. National Research Council. Managing university intellectual property in the public interest. Washington, DC: The National Academies Press: 2011. https://nap.nationalacademies.org/catalog/13001/managing-university-intellectual-property-in-the-public-interest. Accessed 7 Jan 2023.

  16. Fernandez Lynch H, Bateman-House A, Rivera SM. Academic advocacy: opportunities to influence health and science policy under U.S. lobbying law. Acad Med. 2020;95:44–51.

    Article  PubMed  Google Scholar 

  17. Gagnon D, Ming E. Higher education is investing in ESG. https://info.kpmg.us/news-perspectives/advancing-the-profession/higher-ed-investing-in-esg.html. Accessed 7 Jan 2023.

  18. 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  PubMed  Google Scholar 

  19. Patent and Trademark Law Amendments Act of 1980, Pub. L. 96-517, 94 Stat. 3015 (December 12, 1980), codified at 35 U.S.C. §§ 200–212.

Download references

Acknowledgements

The authors thank Katharine Gleason, MPH and Mariah Johnson, MPH for research assistance and administrative support. The authors also thank Merith Basey, MSc, Rebecca Eisenberg, JD, Sarah Emond, MPP, Heather Pierce, JD, MPH, Reshma Ramachandran, MD, MPP, MHS, and Jon Soderstrom, PhD for helpful discussions that informed the arguments presented herein (acknowledgment does not necessarily imply agreement).

Funding

The research presented here was funded by a grant to the University of Pennsylvania from the National Center for Advancing Translational Research (NCATS) of the National Institutes of Health (5UL1TR001878). The sponsor had no input into this research or publication.

Author information

Authors and Affiliations

  1. Department of Medical Ethics and Health Policy, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA

    Steven Joffe, Emily A. Largent, Holly Fernandez Lynch & Matthew S. McCoy

  2. Leonard Davis Institute for Health Economics, University of Pennsylvania, Philadelphia, PA, USA

    Steven Joffe, Emily A. Largent, Holly Fernandez Lynch & Matthew S. McCoy

  3. Department of Markets, Public Policy and Law, Questrom School of Business, Boston University, Boston, MA, USA

    Rena M. Conti

  4. S.J. Quinney College of Law, University of Utah, Salt Lake City, UT, USA

    Jorge L. Contreras

  5. Patients for Affordable Drugs, Washington, DC, USA

    David Mitchell

  6. Washington University School of Law, St. Louis, MO, USA

    Rachel E. Sachs

  7. Georgia State University College of Law, Atlanta, GA, USA

    Allison M. Whelan

Authors
  1. Steven Joffe
    View author publications

    You can also search for this author in PubMed Google Scholar

  2. Rena M. Conti
    View author publications

    You can also search for this author in PubMed Google Scholar

  3. Jorge L. Contreras
    View author publications

    You can also search for this author in PubMed Google Scholar

  4. Emily A. Largent
    View author publications

    You can also search for this author in PubMed Google Scholar

  5. Holly Fernandez Lynch
    View author publications

    You can also search for this author in PubMed Google Scholar

  6. David Mitchell
    View author publications

    You can also search for this author in PubMed Google Scholar

  7. Rachel E. Sachs
    View author publications

    You can also search for this author in PubMed Google Scholar

  8. Allison M. Whelan
    View author publications

    You can also search for this author in PubMed Google Scholar

  9. Matthew S. McCoy
    View author publications

    You can also search for this author in PubMed Google Scholar

Contributions

All authors contributed to the research and arguments presented in this article. SJ wrote the first draft of the manuscript; all authors revised it critically for important intellectual content. All authors approved the final draft of the manuscript.

Corresponding author

Correspondence to Steven Joffe.

Ethics declarations

Competing interests

SJ reports receiving research funding from Pfizer through the University of Pennsylvania until May 2020 and is a member of a Data and Safety Monitoring Board for CSL Behring. DM is a patient with incurable blood cancer. He is Founder of Patients for Affordable Drugs (P4AD), a not-for-profit organization, for which he serves as an unpaid volunteer. P4AD does not accept funding from any organizations that profit from development or distribution of prescription drugs. RES reports receiving grant funding and personal fees from the Institute for Clinical and Economic Review, the National Academy for State Health Policy, and West Health. HFL reports research funding from Arnold Ventures and consulting with the Robert Wood Johnson Foundation outside the present work. RMC, JLC, EAL, MSM, and AMW have no financial relationships to report.

Ethical approval

No ethical approval was required as this research did not involve human or animal subjects.

Additional information

Publisher’s note Springer Nature remains neutral with regard to jurisdictional claims in published maps and institutional affiliations.

Rights and permissions

Springer Nature or its licensor (e.g. a society or other partner) holds exclusive rights to this article under a publishing agreement with the author(s) or other rightsholder(s); author self-archiving of the accepted manuscript version of this article is solely governed by the terms of such publishing agreement and applicable law.

Reprints and permissions

About this article

Check for updates. Verify currency and authenticity via CrossMark

Cite this article

Joffe, S., Conti, R.M., Contreras, J.L. et al. Access to affordable medicines: obligations of universities and academic medical centers. Gene Ther 30, 753–755 (2023). https://doi.org/10.1038/s41434-023-00393-2

Download citation

  • Received:

  • Revised:

  • Accepted:

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1038/s41434-023-00393-2

This article is cited by

Search

Advanced search

Quick links