Patient participation and innovation

Technological developments and changing social norms can outpace existing regulations and institutions. Paul Wicks and James Heywood, two pioneers in the area of patient activism and citizen science, have recently argued in the American Journal of Bioethics (1) that this may be happening in the organization of clinical trials and health care systems. They have been experiencing for years how patients are eager to produce and share data concerning their diseases to hasten clinical researches that might address their own and other people’s condition, and how patient-powered research networks have greatly improved our capabilities in gathering and analysing such data. They have for instance co-authored a paper, published in Nature Genetics, where they aggregate self-reported data on the use of lithium in patients with amyotrophic lateral sclerosis – a rapidly fatal degenerative disease for which no cure is known – through their online platform PatientsLikeMe (2). Their results, unfortunately negative, were confirmed by subsequent clinical trials, the ‘gold standard’ for medical evidence. Remarkably, recruitment through PatientsLikeMe was very efficient, and they were able to disclose their results fastly. Wicks and Heywood add that network-based studies could tackle some entrenched issues of the regulatory pipeline for the approval of drugs based on randomized controlled trials, such as ‘multiple comorbidities, a lack of patient-centered outcome measures, long-term safety, or drug interactions that occur in the real world, while also allowing every patient to contribute to the fight against disease’. On this basis, they propose to extrapolate to the whole health system their strategy: ‘every time an individual receives treatment but does not have his or her experience of efficacy, tolerability, and outcomes shared systematically is a loss to us all’ – a loss that arguably is unnecessary, given patients’ willingness to contribute to biomedical researches. Wicks and Heywood acknowledge some potential risks, such as the risks involved in creating undue incentives for the experimentation of potentially dangerous drugs, and some scientific limitations, especially with regards to the quality of data, arguably the major obstacle that has so far slowed the uptake of data generated by ‘citizen scientists’ within the scientific community. Relying on patients’ aspiration to participate — be it self-centered, altruistic, or both — in order to overcome some restrictions to the use of patients’ data is not a new proposal. Almost ten years ago, Kohane and Altmann, now at Harvard and Stanford Medical School respectively, proposed to relax regulations regarding data privacy and consent in order to harvest citizens’ willingness to ‘annotate’ their own genome with phenotypic information (3). Back then, the scientific and industrial context was the perceived gap between investment (and hype), and medical returns of the human genome project. According to Wicks and Heywood, health care systems constructed on the ‘blueprint’ of patient-powered research networks might close another innovation gap, the gap between our rising computational capabilities — and the associated decline of costs in data production and sharing — and stagnating innovation, especially in the biomedical domain (as measured by declining drug-related patent per dollar spent in R&D). The search for a ‘personalized medicine’ — explicitly mentioned in both articles — underlies much thought about biomedical innovation in the last decade (4-5). The concept that technological and scientific breakthroughs will be achieved in the future by ‘going diffuse’ and collecting the reservoir of data and creative capacities of the multitude was common currency in debates over the construction of a ‘knowledge economy’. The promises of a ‘sharing’ (=donation-based) economy in terms of creation of value is an equally important thread beyond these socio-technical developments. The biomedical domain represents an important case study here: health care systems, and biomedical innovation, are central for human well-being, and human motivations to participate to biomedical research are several, and sometimes strongly felt. There are important questions to be asked as well. Shall we protect citizens and patients when they participate in biomedical research as citizen scientists? If so, how (6)? And in case some of the promises of these approaches do materialize, how shall we deliver benefits and dividends? To whom? Facing a stagnation in biomedical innovation that could become recrudescent — if not secular — by recruiting citizen scientists is an inspirational concept, yet not one devoid of risks and unknowns that we shall want to assess properly. References  

  1. P. Wicks & J.A. Heywood. 2014. Data Donation Could Power the Learning Health Care System, Including Special Access Programs. The American Journal of Bioethics. doi: 10.1080/15265161.2014.957627
  1. P. Wicks, T.E. Vaughan, M.P. Massagli & J. Heywood. 2011. Accelerated clinical discovery using self-reported patient data collected online and a patient-matching algorithm. Nat Biotechnol. doi: 10.1038/nbt.1837.
  1. I.S. Kohane & R.B. Altman 2005. Health-information Altruists – a Potentially Critical Resource. N Engl J Med. doi: 10.1056/NEJMsb051220
  2. European Science Foundation (ESF). 2012. Personalised Medicine for the European Citizen – towards more precise medicine for the diagnosis, treatment and prevention of disease. Strasbourg: ESF.
  3. B. Prainsack. 2014. Personhood and solidarity: What kind of personalized medicine do we want? Personalized Medicine 11/7: 651-657.
  4. E. Vayena & J. Tasioulas. 2013. Adapting Standards: Ethical Oversight of Participant-Led Health Research. PLOS Medicine. doi: 10.1371/journal.pmed.1001402

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