Is crowdfunding an effective way to raise money for clinical trials? And, if so, are such trials in the best interest of patients, researchers and science? Yes, and not yet, found two recent papers that looked at these questions.

To assess the success rate of crowdfunding clinical research, investigators scoured 14 popular crowdfunding websites, including 4 dedicated solely to medical research, to identify clinical trial fundraising campaigns (Lancet 386, 338; 2015). They found 20 in total, including 7 that were ongoing. Of the 13 completed campaigns, 8 (62%) had met their fundraising objectives. These campaigns raised from US$3,600, for a pilot trial looking at the effect of cannabis on attention deficit disorder, to $3 million, for a Phase I trial of an oncolytic virus. The average amount raised was $540,000, and the median was $167,000.

Given decreasing funding from national public agencies, “this strategy might be especially useful for pilot or Phase I studies,” the authors write.

But an ethical analysis of “patient-funded trials”, which include crowdfunded trials and pay-for-access trials, raised several red flags about this approach (Cell Stem Cell 17, 135–137; 2015). These trials side step scientific peer review, co-opting the 'therapeutic hope' of desperate patients to proceed despite potentially “little or no scientific foundation”. A short-term incentive structure for sponsors of crowdfunded trials can lead to poor trial design and low-quality clinical data, the authors also note, and these trials can recruit patients away from studies grounded in stronger scientific rationale.

While crowdfunded trials can “empower patients, expand available research resources and accelerate the pace of translation”, the authors of the ethical analysis argue that as yet they harbour “important liabilities for both patients and the broader clinical research enterprise”. Funding bodies and research institutions need to establish novel mechanisms to review the merit and design of patient-funded trials if such trials are going to meet the interests of patients, science and society, they write.