Pay to play: Participant funded clinical research for rare diseases

Angela J. Ballantyne1, Mike R. King2

1 Department of Primary Health Care and General Practice, University of Otago, Wellington New Zealand, angela.ballantyne@otago.ac.nz
2 Bioethics Centre, University of Otago, Dunedin, New Zealand, mike.king@otago.ac.nz

Medical care relies on research to test and refine existing treatment options and to bring better treatments to the market. This is estimated to cost as much as US$2 billion per new drug.[1]  Many promising products do not make it to the point of clinical trials, often because there is insufficient prospective market for the product, for example because a disease is rare. [2]

In response to this problem, author and patient advocate Alexander Masters made an innovative and unconventional proposal: wealthy donors with a personal interest in an underfunded potential drug could privately fund clinical trials in exchange for special rights, such as access to the experimental drug during the trial. Masters’ proposal was used to raise over US$2M to fund an Uppsala University study into a viral therapy for neuroendocrine cancer.[3]

Charging for the right to access clinical studies is an issue rarely addressed in the research ethics literature. However, the potential benefits that could result from this are a reason to consider it seriously. In this paper we consider three funding options: charging each participant[4], crowdfunding clinical trials[5], and private donors.  For each of these options we explore the following potential criticisms: psychological exploitation of desperate patients and families; commodification of research; undermining the social value of research by skewing the research agenda; undermining scientific validity of the study by allowing the funders to dictate recruitment and allocation; and undermining fair subject selection.

We provide responses to these criticisms, concluding that participant funded clinical research for rare diseases represents a promising option for supporting drug development, and an example of the creativity and value of advocates offering innovative solutions. This method of funding warrants further investigation in research ethics.

[1] Avorn, Jerry. 2015. “The $2.6 Billion Pill — Methodologic and Policy Considerations.” New England Journal of Medicine 372 (20): 1877–79.
[2] Aronson, JK. 2006. “Rare Diseases and Orphan Drugs.” British Journal of Clinical Pharmacology 61 (3): 243–45.
[3] Masters, Alexander. 2014. “A Plutocratic Proposal.” Mosaic. October 27.  Http://mosaicscience.com/story/plutocratic-proposal.
[4] Emanuel EJ, Joffe S, Grady C, Wendler D, Persad G. Clinical research: Should patients pay to play?
Sci Transl Med. 2015 Jul 29;7(298):298ps16.
[5] Dragojlovic, Nick, and Larry D. Lynd. 2014. “Crowdfunding Drug Development: The State of Play in Oncology and Rare Diseases.” Drug Discovery Today 19 (11): 1775–80.


Biography

Dr. Angela Ballantyne is a Senior Lecturer in the Bioethics Department at the University of Otago and is President of the International Association of Bioethics. She received her PhD from from Monash University and since then has worked around the world most notably as a visiting scholar at Yale University and for the World Health Organisation.

Mike King has published in both bioscience and bioethics, and his research focuses on the intersection between these disciplines.

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