Under the designation “patient-led research” (PLR) or “citizen-driven biomedical research,” citizens, patients, and families have increasingly become the leading force in the initiation or conduct of health research projects, pursuing a range of activities from analyses of genomic data for diagnosing rare diseases, identification of potential therapeutic drugs, organization and crowdfunding of clinical trials’ cohorts, and even self-surveillance or selfexperimentation. Many of the participants in citizen-driven biomedical research are patients and families confronted with a condition that is the subject of their research, therefore facing new epistemic and governance challenges, and often testing the ethical and regulatory limits within which health research has traditionally operated.
This new form of research where citizens and patients are the primary producers and mobilizers or instigators of knowledge promises to break new ground in underserved health domains, but also suffers from a lack of legitimacy when it comes to assessing the quality of patients’ experiential data. Moreover, this endeavor gradually transfers the responsibility to preserve safety and ethics to lay experts, probing new ethical matters of concerns – from blurring boundaries between treatments and self-experimentation, peer-pressure to participate in trial, exploitation of vulnerable individuals or third parties (children), to a lack of regulation concerning quality control and risk of harm. Very little research currently focuses on adequate ways to adapt or design responsible governance and ethical standards tailored to citizen-driven biomedical research.
The present report addresses important unresolved issues that could compromise the
development and recognition of citizen and patient-driven biomedical research: by (1) analyzing the practices and methods enacted by citizens and patients to produce and mobilize experiential biomedical knowledge as evidence; by (2) identifying the specific ethical and governance challenges patients and citizens encountered when conducting research; by (3) evaluating and adapting oversight mechanisms tailored to prevent these controversial epistemic and governance issues. In the process, the report also unveils matters of concern that are of interest to both citizens and regulators, and which are zones of undone science and unregulated science, such as underserved condition-areas like rare genetic diseases. Another important merit of this report is to provide regulatory institutions with an analysis of the different practices enacted by citizens and patients to articulate “experiential knowledge” with “credentialed knowledge,” therefore contributing to the current development of real-world evidence-based medicine.
Notwithstanding ongoing challenges, we should not simply disregard medical research conducted outside of traditional institutions as de facto less safe, less reproducible, or unethical. Patients often have in-depth experiential knowledge of their conditions along with a vested interest to make sure that a treatment or device will be effective, safe, and beneficial. Yet, facing regulatory uncertainty, they might not overcome the “chill factor” – a phenomenon described by citizen scientists and DIY inventors as the fear to confront regulators by sharing the recipe for a new invention. The press has recently covered cases of biohackers who self-experimented with unregulated gene-therapies. But the stories encountered in community biolabs, such as Biocurious and Denver Biolabs, are different: mentors, amateurs, and students want their proof of concept to be safe and reproducible, achieving specific standards in the research processes and evidences they rely on
The next step is to foster legitimacy for citizen-driven biomedical innovation by supporting citizens and patients to document and share their data, evidence, and ethical concerns in ongoing conversations with regulators and society. Because they respect biological safety
levels and function as a peer-review culture, community biolabs constitute an ideal ecosystem for mentorship in the most current bioengineering techniques and their related risk-benefit trade-offs. These labs might be the perfect place to start a continuing dialogue about how to adapt our regulatory standards to a more democratized form of biomedical innovation. What we need is empowerment, but also more collective intelligence. If risks are properly managed without dampening the now more-democratized reality, then we might all gain in the process.
Table of Contents
* Case- Studies
* Health Pioneers
The Gray Family
Sonia Vallabh and Eric Minikel
* Inside Community Biolabs
Counter Culture Labs (Open Insulin)
Four Thieves Vinegar (EpiPencil)
Vardhaan Ambati & Elodie Rebesque
* Inside the Health Makerspace
* Topography of Innovations & Regulatory Barriers
* Regulatory Considerations: A Research & Regulatory Toolkit
Perspective of Regulators
Inspiration for Taxonomy as a Legitimacy-Building Tool
* Analysis: Barriers & Opportunities in Democratizing Health Research