Data Donation Could Power the Learning Health Care System, Including Special Access Programs

摘要

In this issue, Walker and colleagues (Walker, Rogers, and Entwistle 2014) review the ethical dimensions of special access programs (SAPs) for unapproved medical interventions in advance of regulatory approval. Our own experience as advocates and researchers is with amyotrophic lateral sclerosis (ALS), a rapidly progressive and terminal condition with no effective treatment (Wijesekera and Leigh 2009) Over the past 15 years we built online forums for thousands of engaged ALS patients at BUILD-UK.net and ALS.net, and more recently a patient-powered research network (PPRN) at PatientsLikeMe.com (Brownstein et al. 2009). The PatientsLikeMe communities have nurtured many patient leaders across many diseases, but it is ALS patients who have most consistently advocated for earlier access to experimental treatments.