Affordable Orphan Drugs: a Role for Not-for-profit Organizations

Overview

Journal Br J Clin Pharmacol

Specialty Pharmacology

Date 2017 Jan 22

PMID 28109021

Citations 21

Authors

Elin H Davies

Emma Fulton

Daniel Brook

Dyfrig A Hughes

Affiliations

Soon will be listed here.

Abstract

Aims: The success of the Regulation on Orphan Medicinal Products in the European Union is evidenced by the 127 orphan drugs that have had market authorization since 2000. However, the incentives aimed at stimulating research and development have had the unintended consequence of increasing drug cost, resulting in many orphan drugs not being cost-effective. Orphan drugs command an increasing share of the pharmaceutical market and account for a disproportionate amount of healthcare expenditure. Orphan drug ownership by socially motivated, not-for-profit organizations may facilitate access to more affordable orphan drugs, for the benefit of patients and healthcare systems alike. This study aims to describe opportunities for such organizations to become orphan drug Market Authorization Holders.

Methods: We reviewed data on the ownership of EMA designated and approved orphan drugs, identified funding opportunities and business models for not-for-profit organizations, and summarised relevant legal and policy documents concerning intellectual property rights and drug regulation.

Results: Using repurposed drugs as a paradigm, this narrative review navigates the regulatory hurdles, describes the legal context and identifies funding opportunities, in a bid to facilitate and encourage not-for-profit organizations to lead on the development of affordable orphan drugs.

Conclusions: Although the regulatory steps required to obtain an MA for an orphan drug are numerous and challenging, they are not insurmountable and can be achieved by not-for-profit organizations that are socially motivated to reduce the costs of orphan drugs to the payers of healthcare. Opportunities for orphan drug development resulting in affordable products lie mainly with repurposed drugs.

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References

Ashburn T, Thor K . Drug repositioning: identifying and developing new uses for existing drugs. Nat Rev Drug Discov. 2004; 3(8):673-83. DOI: 10.1038/nrd1468. View

Cohen D, Raftery J . Paying twice: questions over high cost of cystic fibrosis drug developed with charitable funding. BMJ. 2014; 348:g1445. DOI: 10.1136/bmj.g1445. View

Kesselheim A, Tan Y, Avorn J . The roles of academia, rare diseases, and repurposing in the development of the most transformative drugs. Health Aff (Millwood). 2015; 34(2):286-93. DOI: 10.1377/hlthaff.2014.1038. View

Houston A, Beall R, Attaran A . Upstream solutions for price-gouging on critical generic medicines. J Pharm Policy Pract. 2016; 9:15. PMC: 4852412. DOI: 10.1186/s40545-016-0064-8. View

Hughes D, Poletti-Hughes J . Profitability and Market Value of Orphan Drug Companies: A Retrospective, Propensity-Matched Case-Control Study. PLoS One. 2016; 11(10):e0164681. PMC: 5074462. DOI: 10.1371/journal.pone.0164681. View

Stakisaitis D, Spokiene I, Juskevicius J, Valuckas K, Baiardi P . Access to information supporting availability of medicines for patients suffering from rare diseases looking for possible treatments: the EuOrphan Service. Medicina (Kaunas). 2007; 43(6):441-6. View

Collier J . Getting new drugs to market: how individuals could do this without leaving their desks. BMJ. 2006; 333(7582):1315-7. PMC: 1761173. DOI: 10.1136/bmj.39055.610764.47. View

Lincker H, Ziogas C, Carr M, Porta N, Eichler H . Regulatory watch: Where do new medicines originate from in the EU?. Nat Rev Drug Discov. 2014; 13(2):92-3. DOI: 10.1038/nrd4232. View

Hughes D, Tunnage B, Yeo S . Drugs for exceptionally rare diseases: do they deserve special status for funding?. QJM. 2005; 98(11):829-36. DOI: 10.1093/qjmed/hci128. View

10.

DiMasi J, Grabowski H, Hansen R . Innovation in the pharmaceutical industry: New estimates of R&D costs. J Health Econ. 2016; 47:20-33. DOI: 10.1016/j.jhealeco.2016.01.012. View

11.

Maxmen A . Busting the billion-dollar myth: how to slash the cost of drug development. Nature. 2016; 536(7617):388-90. DOI: 10.1038/536388a. View

12.

Morgan S, Grootendorst P, Lexchin J, Cunningham C, Greyson D . The cost of drug development: a systematic review. Health Policy. 2011; 100(1):4-17. DOI: 10.1016/j.healthpol.2010.12.002. View

13.

Hay M, Thomas D, Craighead J, Economides C, Rosenthal J . Clinical development success rates for investigational drugs. Nat Biotechnol. 2014; 32(1):40-51. DOI: 10.1038/nbt.2786. View

14.

Onakpoya I, Spencer E, Thompson M, Heneghan C . Effectiveness, safety and costs of orphan drugs: an evidence-based review. BMJ Open. 2015; 5(6):e007199. PMC: 4480037. DOI: 10.1136/bmjopen-2014-007199. View

15.

Fagnan D, Gromatzky A, Stein R, Fernandez J, Lo A . Financing drug discovery for orphan diseases. Drug Discov Today. 2013; 19(5):533-8. DOI: 10.1016/j.drudis.2013.11.009. View

16.

Meekings K, Williams C, Arrowsmith J . Orphan drug development: an economically viable strategy for biopharma R&D. Drug Discov Today. 2012; 17(13-14):660-4. DOI: 10.1016/j.drudis.2012.02.005. View

17.

Hale V, Woo K, Lipton H . Oxymoron no more: the potential of nonprofit drug companies to deliver on the promise of medicines for the developing world. Health Aff (Millwood). 2005; 24(4):1057-63. DOI: 10.1377/hlthaff.24.4.1057. View

18.

Schuller Y, Hollak C, Biegstraaten M . The quality of economic evaluations of ultra-orphan drugs in Europe - a systematic review. Orphanet J Rare Dis. 2015; 10:92. PMC: 4520069. DOI: 10.1186/s13023-015-0305-y. View

19.

Dragojlovic N, Lynd L . What will the crowd fund? Preferences of prospective donors for drug development fundraising campaigns. Drug Discov Today. 2016; 21(12):1863-1868. DOI: 10.1016/j.drudis.2016.07.002. View

20.

Davies E, Fulton E, Brook D, Hughes D . Affordable orphan drugs: a role for not-for-profit organizations. Br J Clin Pharmacol. 2017; 83(7):1595-1601. PMC: 5465340. DOI: 10.1111/bcp.13240. View