Many health startups, like Theranos, lack peer-reviewed data to back claims
- Startups generate hype for their disruptive potential to advance healthcare goals, but few of the most highly valued startups publish evidence to support their claims, an analysis by Stanford University researchers finds.
- Researchers reviewed published research output of 47 "unicorns," or startups valued at more than $1 billion. Of 18 current unicorns, 10 had no highly cited published medical papers. The rate for exited unicorns was 12 of 29.
- Investors, patients and providers should be wary of "stealth research" — data generated internally rather than through peer-reviewed research, researchers say.
The most infamous unicorn miscalculation in recent history is likely Theranos, the much-hyped startup that promised to upend conventional diagnostics with low-cost finger prick tests, but then imploded when massive fraud was uncovered.
While fraud is a concern, stealth research points to a larger problem, the researchers say. They note, for example, that three digital health unicorns — Outcome Health, GuaHao and Oscar Health — had no published papers, while Clover Health and Zocdoc each had just one.
Of the exited unicorns, five had no published papers and two had only two. Nearly half of all the 425 published papers that were reviewed were from 23andMe (107) and Adaptive Biotechnologies (89).
Moreover, of the 34 highly cited papers, 16 were genetic association studies of which only one — a retrospective study — detailed trial results.
The researchers raise broader transparency concerns as well. For example, Intarcia Therapeutics, the top-valued startup at $5.5 billion, published six papers on its ITCA 650 glucagon-like peptide 1 receptor, though none with more than 50 citations. When the FDA denied the device's manufacturing plan for the device, the company was mum on why or how it would remedy the agency's concerns.
The lack of publications wasn't limited to unicorns. If anything, it may be worse with lesser-funded startups, the researchers say.
"We are not arguing that startups should divert excessive resources to having peer-reviewed papers. However, when their products are destined to affect patient health, they should neither be solely doing marketing," they write. "Confidential data sharing with potential investors or regulators cannot replace more open scrutiny by the scientific community. Transparently reported research does not threaten proprietary rights to innovation under patent protection."
The study jibes with a recent Health Affairs report showing few digital health companies study their impact on disease burden and overall health costs. Of the 20 top-funded, private U.S.-based digital health companies, only a few had published studies and those mostly involved healthy volunteers. Few tried to determine outcomes in high-burden populations or impact on patient access and costs.
- Wiley Online Library Stealth research: lack of peer‐reviewed evidence from healthcare unicorns