Former FDA chief Scott Gottlieb’s favorite real-world evidence platform is getting a new stream of cash, as it nears what will likely be the biggest test of the young company so far.
Aetion, the New York health tech startup, has for the second time raised an extension to their Series B, adding $19 million from J&J’s VC arm, Greenspring Associates and EDBI to bring their round to $82 million. Gottlieb, who championed real-world evidence as commissioner and later joined the company’s board, said in a statement that the new cash would help fuel a global expansion.
The funding comes as a pandemic has rapidly transformed questions around the usefulness of real-world data from a subject for biopharma wonks debating the near-future of drug development, to an issue that has — often dubiously — rapidly informed and changed treatment decisions around the globe. And it comes as Aetion, in partnership with the FDA and a long list of other organizations, looks to prove their platform can give earlier and broader look at treatments for a novel virus, separating them from a field that experts say has yet to turn in much useful info and is filled with flawed approach.
“You could argue that never have our insights been more important,” CEO Carolyn Magill told Endpoints News.
Researchers turned to real-world data early in the pandemic for the first signs of what was working and what wasn’t, but the results often left something to be desired. Most notably, two influential studies that looked at hydroxychloroquine use around the world were pulled from top journals after it emerged that some of the data were flawed. Later, Gilead used a real-world comparator in one trial to try to prove remdesivir improved survival — a method promptly pilloried by outside experts.
And just this past week, controversy erupted after the FDA authorized convalescent plasma based on observational data from tens of thousands of patients who received it under an expanded protocol (which differs slightly from real-world evidence platforms). The data and analysis were regarded as sound, but researchers differed on whether it met the bar for an authorization.
Asked about real-world data during the pandemic, Yale cardiologist and health outcomes researcher Harlan Krumholz said such analyses had “awesome potential” but that scientists still needed to agree on standards that differentiate good analyses from shoddy ones.
“To date, there are few examples [where] these types of studies have tipped the balance,” Krumholz, who directs Yale’s center for outcomes research and evaluation, said via email
For Magill, the hydroxychloroquine debacle underscored the need for platforms like Aetion’s, where there are guard-rails in place to ensure data is extracted and analyzed in rigorous ways.
“There are key aspects of principled database epidemiology that we think should be incorporated into every study,” she said. “And I think what we saw with hydroxychloroquine, for example, reinforces that notion.”
In April, Aetion announced a partnership with HealthVerity to build a real-time treatment tracker, allowing doctors and researchers to look at how Covid-19 practice is changing day-by-day. Recently, Magill said, the tracker spotlighted how quickly physicians adopted dexamethasone after a UK team showed it improved survival in severe patients.
In May, they teamed with the FDA to use real-world evidence to learn more about the Covid-19 disease progression and how treatments and diagnostics were being used. The effort emerged out of the Covid-19 Evidence Accelerator, a broader initiative from the Reagan-Udall Foundation and Friends of Cancer Research. Magill said they are particularly looking at differences in how the disease progresses and how patients from different populations are treated.
So far, Aetion has published little on Covid-19: most notably a study with Brigham and Women’s Hospital on ace inhibitors and hospitalization risk. They have also looked at differences between hydroxychloroquine users and non-users, Magill said. The direct usefulness of that analysis is likely limited — hydroxychloroquine has been shown to have little effect in multiple trials and is no longer widely used for Covid-19— but Magill said it informed how they do future analyses and set up comparisons.
Now, the company is preparing to aid vaccine makers on the post-approval monitoring that the FDA and HHS have said will be essential for ensuring the safety of a vaccine. Magill declined to disclose which vaccine producers it worked with, but the company has previously announced a partnership with Sanofi. And J&J was a contributor to their latest round.
The monitoring will present new challenges in safety monitoring, Magill said, ones they are preparing for now.
”You can imagine that these vaccines are new, and anytime something is new there is no precedent and data could be collected in different ways, depending on the health system, depending on who’s administering the vaccine, as an example, where you are geographically,” she said. “And all of that has implications on the real world data.”