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Patient-Driven Drug Development

Jessica Flanigan

Autonomy is a foundational principle of medical ethics. Patients are entitled to make their own treatment decisions, even if they don’t make the medically advisable choice. Patients are also entitled to know their options. Health workers must inform patients of all available treatments that a reasonable person would consider. Patients also have the right to seek out approved alternative therapies as long as they can find a willing provider. Even when all existing treatments are disclosed and made available, though, some patients have very few options for fighting their diseases. These patients encounter the limits of medical autonomy—one can have the freedom to choose and refuse treatment but that freedom is of little value when effective treatments do not exist or are unknown.

For example, patients with rare diseases may not have effective treatment options because manufacturers do not typically invest in therapies that treat relatively small patient populations. Even when existing therapies can be used for rare-disease treatment, physicians may not be equipped to inform patients about off-label treatment options because they are not familiar with the latest research about their patient’s rare condition. Patients who suffer from emerging diseases face similar hurdles. When patients’ medical needs outpace the scientific community’s medical knowledge, medical autonomy really amounts to the right to wait for the scientific community to develop an effective treatment.

In this essay, I discuss an emerging approach to drug development that is forged in rare-disease communities and patient advocacy networks. Drug development has historically advanced within the context of research institutions, such as universities and pharmaceutical companies. Within and outside of this framework, some patients are now taking greater control of their treatment options even when few options exist—not only as clinical trial participants but also as researchers. These citizen- scientists refuse to wait, even when there are few available treatments.

J. Flanigan (*)

The Jepson School of Leadership Studies, The University of Richmond, Richmond, VA, USA e-mail: This email address is being protected from spam bots, you need Javascript enabled to view it

© Springer Science+Business Media B.V. 2017

D. Ho (ed.), Philosophical Issues in Pharmaceutics, Philosophy and Medicine 122, DOI 10.1007/978-94-024-0979-6_2

Yet the patient-driven approach to drug development is controversial. Patients are not medical experts, so when citizen-scientists with rare diseases publicize information about untested and experimental treatments to rare-disease communities, their example could cause some people to harm themselves. Patient-driven drug development could also compromise the clinical trial system, thereby making it harder for other patients to know if a treatment is working. Another concern is that citizen-scientists will threaten existing pathways of drug development if patients’ adverse reactions to experimental treatments ultimately discourage manufacturers from developing the therapy. On the other hand, patient-driven drug development could also divert resources from more promising therapies if resources for medical resources are very limited. Or patient-driven development could slow progress toward effective treatment insofar as including laypeople’s priorities or judgments makes medical research less efficient. Citizen-scientists are not impartial. They mix research with advocacy in ways that undermine their credibility with academics. Outside rare-disease communities, some people wonder whether wealthy patients should set our priorities in medicine, when research funding would otherwise help a greater number of patients (Salmon 2014).

Despite these concerns, I argue that patient-driven development is best understood as a legitimate method of drug discovery and medical treatment that should be valued as a complement to the conventional development pipeline. The main reason in favor of including patients as partners in the development process is that patients are entitled to pursue treatment options. Including patients in the development process can also promote drug discovery and help manufacturers and physicians better understand emerging therapies.

I first explain some of the policies and practices that currently limit patients’ treatment options. The current development pathway is often harmful and should be reformed to give patients greater access to experimental drugs, but I will not argue for that claim in this essay. Rather, I am concerned with the ethics of some patients’ response to the current system, when they become partners in research and development. Next, I describe some forms of patient-driven development. I then argue that patient-driven development is morally permissible, even if it causes some patients to make risky treatment decisions. I also respond to concerns that patient-driven development would threaten drug development on balance and I make the case for patient-driven drug development, despite concerns about political bias and inequality.

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