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Non-profit Drug Research and Development at a Crossroads

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Abstract

In wealthy nations, non-profit drug R&D has been proposed to reduce the prices of medicines. We sought to review the ethical and economic issues concerning non-profit drug R&D companies, and the possible impact that their pricing strategy may have on the innovation efforts from for-profit companies targeting the same segment of the pharmaceutical market. There are two possible approaches to pricing drugs developed by non-profit R&D programs: pricing that maximises profits and “affordable” pricing that reflects the cost of manufacturing and distribution, plus a margin that ensures sustainability of the drug supply. Overall, the non-profits face ethical challenges - due to the lack of resources, they are unable to independently commercialize their products on a large scale; however, the antitrust law does not permit them to impose prices on potential licensees. Also, reduced prices for the innovative products may result in drying the for-profit R&D in the area.

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Abbreviations

AFM-Telethon:

Association Française contre les Myopathies

CFF:

US Cystic Fibrosis Foundation

DNDi:

Drugs for Neglected Diseases initiative

MA:

Marketing authorization

PDP:

Product development partnerships

R&D:

Research and development

References

  1. Maxmen A. Busting the billion-dollar myth: how to slash the cost of drug development. Nature. 2016;536(7617):388–90.

    Article  CAS  PubMed  Google Scholar 

  2. Hale VG, Woo K, Lipton HL. Oxymoron No More: The Potential Of Nonprofit Drug Companies To Deliver On The Promise Of Medicines For The Developing World. Health Aff. 2005;24(4):1057–63.

    Article  Google Scholar 

  3. Butler D. Neglected diseases fund touted. Nature. 2010;465(7296):277.

    Article  CAS  PubMed  Google Scholar 

  4. Dougherty G, Harjivan C. Drug Development for Government, Nonprofit, and Developing-World Markets. Pharmaceutical Technology Sourcing and Management. 2011;7(3). http://www.pharmtech.com/drug-development-governmentnonprofit-and-developing-world-markets. Accessed 29 Jan 2018.

  5. Conti RM, Meltzer DO, Ratain MJ. Nonprofit biomedical companies. Clin Pharmacol Ther. 2008;84(2):194–7.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  6. Murteira S, El Hammi E, Toumi M. Fixing the price of the orphan drug Siklos®: The Council of State takes over the decision. European Journal of Health Law. 2014;21(5):505–15.

    Article  PubMed  Google Scholar 

  7. Meekings KN, Williams CS, Arrowsmith JE. Orphan drug development: an economically viable strategy for biopharma R&D. Drug Discov Today. 2012;17(13–14):660–4.

    Article  PubMed  Google Scholar 

  8. Morel T, Arickx F, Befrits G, Siviero P, van der Meijden C, Xoxi E, et al. Reconciling uncertainty of costs and outcomes with the need for access to orphan medicinal products: a comparative study of managed entry agreements across seven European countries. Orphanet J Rare Dis. 2013;8:198.

    Article  PubMed  PubMed Central  Google Scholar 

  9. Pollack A. Deal by Cystic Fibrosis Foundation Raises Cash and Some Concern. The New York Times 2014. https://www.nytimes.com/2014/11/19/business/for-cystic-fibrosis-foundation-ventureyields-windfall-in-hope-and-cash.html. Accessed 29 Jan 2018.

  10. Young B, Surrusco M. Rx R&D Myths: The Case Against The Drug Industry’s R&D “Scare Card”. Public Citizen Congress Watch; 2001. Available from: https://www.citizen.org/sites/default/files/rdmyths.pdf.

  11. NIH Contributions to Pharmaceutical Development, Administrative Document,. National Institutes of Health; 2000. Available from: https://www.citizen.org/sites/default/files/appc.pdf.

  12. Pills and profts. Stop AIDS. Global Justice Now.; 2017. Available from: https://www.globaljustice.org.uk/sites/default/files/files/news_article/pills-and-profits-report-web.pdf.

  13. NIH Director's Blog. What a Year It Was! A Look Back at Research Progress in 2017. National Institutes of Health 2018. Available from: https://directorsblog.nih.gov/2018/01/02/what-a-year-it-was-a-look-back-at-research-progress-in-2017.

  14. Silverman D. At $475,000, new cancer drug raises thorny questions about drug pricing — and value.; 2017. Available from: https://www.statnews.com/2017/09/04/cancer-drug-pricing-novartis/.

  15. Regalado A. Gene-Therapy Cure Has Money-Back Guarantee. MIT Technology Review 2016.

  16. Medic G, Korchagina D, Young KE, Toumi M, Postma MJ, Wille M, et al. Do payers value rarity? An analysis of the relationship between disease rarity and orphan drug prices in Europe. Journal of Market Access & Health Policy. 2017;5(1):1299665.

    Article  Google Scholar 

  17. Messori A, Cicchetti A, Patregani L. Orphan drugs. Relating price determination to disease prevalence BMJ. 2010;341:c4615.

    PubMed  Google Scholar 

  18. Jarosławski S, Auquier P, Toumi M. No correlation between the prices of oncology orphan drugs in the US and their patient population sizes. J Cancer Policy. 2017;14:1–4.

    Article  Google Scholar 

  19. McPherson JD, Marra M, Hillier L, Waterston RH, Chinwalla A, Wallis J, et al. A physical map of the human genome. Nature. 2001;409(6822):934–41.

    Article  CAS  PubMed  Google Scholar 

  20. Chumakov I, Rigault P, Guillou S, Ougen P, Billaut A, Guasconi G, et al. Continuum of overlapping clones spanning the entire human chromosome 21q. Nature. 1992;359(6394):380–7.

    Article  CAS  PubMed  Google Scholar 

  21. Cohen D, Chumakov I, Weissenbach J. A first-generation physical map of the human genome. Nature. 1993;366(6456):698–701.

    Article  CAS  PubMed  Google Scholar 

  22. Bellanne-Chantelot C, Lacroix B, Ougen P, Billault A, Beaufils S, Bertrand S, et al. Mapping the whole human genome by fingerprinting yeast artificial chromosomes. Cell. 1992;70(6):1059–68.

    Article  CAS  PubMed  Google Scholar 

  23. Butler D. Genethon builds up 'genetic valley'. Nature. 1994;370(6484):4.

    Google Scholar 

  24. Chakravarthy R, Cotter K, DiMasi J, Milne CP, Wendel N. Public and Private Sector Contributions to the Research & Development of the Most Transformational Drugs of the Last 25 Years. Boston, MA: Tufts Center for the Study of Drug Development. Tufts University School of Medicine; 2015. Available from: http://csdd.tufts.edu/files/uploads/PubPrivPaper2015.pdf.

  25. Pioneering ways of working through innovative partnerships 2002–2015. The successful development of a fixed dose combination of artesunate plus amodiaquine antimalarial. Drugs for Neglected Diseases initiative; 2015. Available from: https://www.dndi.org/wp-content/uploads/2014/11/DNDi_ASAQ-story_2002-2015.pdf.

  26. Janssen Global Public Health: HIV Medicines Access & Partnerships Program. 2014. Available from: http://partnerships.ifpma.org/partnership/janssen-generic-arv-licensing.

  27. Goldman DP, Jena AB, Philipson T, Sun E. Drug licenses: a new model for pharmaceutical pricing. Health Aff (Millwood). 2008;27(1):122–9.

    Article  Google Scholar 

  28. Simoens S. Pricing and reimbursement of orphan drugs: the need for more transparency. Orphanet J Rare Dis. 2011;6:42.

    Article  PubMed  PubMed Central  Google Scholar 

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Authors declare no conflicts of interest or sources of funding related to the topics presented in the article.

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Correspondence to Mondher Toumi.

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Jarosławski, S., Toumi, M., Auquier, P. et al. Non-profit Drug Research and Development at a Crossroads. Pharm Res 35, 52 (2018). https://doi.org/10.1007/s11095-018-2351-3

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