Clinical Rockstar Pamela Tenaerts Advancing Patient-Centric Trials at Medable
Photo courtesy of Medable.
Patient advocacy groups, trial sponsors, and scientists alike have been clamoring for innovations like decentralized clinical trials (DCT), novel endpoints, and quality by design for years. Leading the charge has been Medable Inc.’s new chief scientific officer, Dr. Pamela Tenaerts.
With more than 30 years of clinical experience, including most recently at the helm of Duke University’s Clinical Trials Transformation Initiative (CTTI), Tenaerts is an obvious choice to take Medable, a leading cloud platform for patient-centric drug development, to the next level.
According to Tenaerts, whose resume also includes stints as a clinical trials investigator, hospital research administrator, and academic in both the U.S. and Europe, the COVID-19 pandemic has opened a lot of eyes to the value of decentralized research.
“Before, it was perceived that it was risky to take on a new methodology of getting evidence like a decentralized clinical trial. With Covid, it became risky not to do it, because if you didn’t adopt decentralized methods, all of a sudden you wouldn’t have a trial anymore. So Medable technology really went from an edge case, sort of a risky situation, to mainstay,” Tenaerts told BioSpace.
You could take Tenaerts’ well-earned word for it, but the ultimate proof is in Medable’s year-over-year revenue increase and ever-increasing funding. The company’s revenue grew by 400% in the first half of 2020, and on April 15, Medable added another $78 million in financing to the $91 million Series C round it completed in November, for a total of $217 million overall.
As Tenaerts takes the scientific helm at Medable, she will leverage the company’s proprietary platform to advance decentralized research methodologies with evidence-based best practices for patient-centric clinical trials.
“We found that 72% of participants preferred a digital health trial,” said Tenaerts. “There is a lot of burden that is decreased and it certainly provides a way to do more research about more diseases and find more medical products that can help patients. Digital health gives you an opportunity to measure something that you’ve been measuring, in a better way.”
Decentralized research also has the potential to address the persistent lack of diversity in traditionally-run trials.
“That’s been a big topic in Covid as well – the minority populations were affected disproportionately. Typically, in clinical trials over the years, we have not done a really good job at making sure that minorities are well represented. We think that with decentralization of clinical trials, there might be an opportunity to represent better because it’s easier for people to participate,” Tenaerts said.
Access to clinical trials is just as much a geographical challenge, particularly if one happens to be stricken with a rare disease.
“The other thing that happens in regular clinical trials is that there’s a geography issue. More than 70% of potential participants live more than two hours away from a research site. So if you’re somebody with a rare disease, you kind of have to be lucky to live around an area where there’s research done for your disease,” said Tenaerts.
The accessibility resolution is a relatively easy argument to make. More difficult will be convincing the scientific community-at-large that the datasets collected from decentralized trials are on par with the traditional brick-and-mortar methodology.
“I’m totally convinced that we can work with that, and the quality of the data can be as good as in a typical clinical trial. We just have to show the evidence that that’s the case,” Tenaerts said.
Tenaerts is also working to transform the quality of the data collected in clinical trials with technology-enabled novel endpoints.
“The burden of clinical trials has been long-contemplated as an issue. At least 25% of typical brick-and-mortar clinical trials collect tests and procedures, endpoint data points, that are never used for eventual submission. So one of the things patients are also asking for is that clinical trials are simplified and measure what matters,” she said.
To this end, Medable works closely with a patient advocacy council and patient champions network. In fact, the company does not release any new platform or product before it goes through a patient review process.
“It’s really an opportunity to turn research on its head and measure what’s important to patients living with a disease rather than what we’ve always measured anyway. How can we include their perspectives in how this new clinical trial methodology works?” Tenaerts said.
For example, Michael Henson, co-founder of the "No More Excuses" ALS Advocacy Group, would like to see less reliance on the ALS Functional Rating Scale, a 48-point scale made up of 12 questions.
“Our trials in ALS are literally graded completely on the ALS FRS score. One of the questions is: As compared to how your handwriting was before you had ALS, is your handwriting today legible, not legible, etc. That varies greatly day-to-day in ALS. Another question is: How well can you climb stairs? Well, no ALS patient is ever going to regrow motor neurons, so that question never gets better,” stated Henson, who is not involved in any trials with Medable.
Referencing a 2019 review of U.S. and EU cardiology guidelines, Tenaerts explained that the concept of quality by design in clinical trials can also yield more comprehensive and evidence-based data.
“Cardiology is one of the most researched diseases out there and less than 10% of their guidelines are evidence-based. We need to think about focusing on what matters, so that we can get to more products that help patients and decrease disease burden around the world,” she said.
Also on Tenaerts’ list of priorities at Medable is looking at design options to scale clinical trials for faster real-time answers and a wider range of treatments.
“Medable is looking at how we can keep our participant community engaged and leverage that for future research, sort of creating a cohort where instead of doing one-off trials where one product is tested at a time against a control, we create the opportunity for multiple medical products to be tested at the same time. I think the Medable system can definitely be the platform for those things to happen,” she said.
This type of model is currently playing out in the RECOVERY trial in the U.K., a randomized study of multiple therapies that could benefit patients hospitalized with suspected or confirmed COVID-19.
Tenaerts, who has devoted her entire career to the advancement of innovation in clinical trials, believes that patient-centric research will lead to greater outcomes.
“I think decentralized clinical trials is one of the ways that we can make research more accessible to everyone, and where patients drive their own research, I think we’ll get to a lot more answers,” she said.