A ‘Holy Grail Moment’ for Clinical Research

It was a familiar scenario: A rare disease area of oncology. A new drug candidate. A desire to assess efficacy as safely and quickly as possible, so that if successful, a new therapy could get approved and change lives. But this desire could have been tempered by reality: the trial was going to be challenging and slow. And with a typical controlled study, patients would be faced with a difficult choice: participation meant the possibility of being randomly assigned to receive an inactive or possible less-effective substance.

In looking for ways to innovate and improve efficiency, Parexel saw an opportunity to take a different approach and worked with FDA and a sponsor to harness real-world evidence  (RWE). “We were able to match, patient for patient, the trial’s active treatment arm (drug + standard of care), with an external cohort of patients using RWE,” explained Peyton Howell, Executive Vice President and Chief Commercial & Strategy Officer at Parexel. “This is where clinical research is becoming a care option.”

Using data in new ways

This trial went beyond what is typically thought of as RWE – claims and billing activities, mobile device data and more. Pivotal data were generated and the sponsor saw their BLA approved – believed to be a first approval with the use of synthetic control arm to demonstrate contribution of effect. And the approval came in months. “From a patient perspective, that time savings is exciting,” said Howell. “But it also changes clinical research. If it was your loved one looking at the trial, this is a ‘no regrets’ trial.”

Close collaboration was crucial to this work. Parexel’s regulatory and biostatistics experts worked with FDA to ensure that the approach would generate data that would be acceptable to the agency. Howell sees this greater level of detail producing more confidence than would have otherwise been seen. “Because it’s real-world evidence, the bar is even higher. You actually know exactly what happened to the patient before, during and after their treatment. It’s a wider lens from which to draw data.”

The resulting approval showed that FDA believed this study and approach was valid, and this medicine became the only second line therapy in this area of cancer. If patients fail frontline treatment, there is another approved therapy now available to them because of this study.

To Howell, a synthetic control arm for approval feels like a kind of ‘holy grail moment,’ where distinct elements aligned to truly advance the conduct of clinical research. While synthetic control arms may not be right for every study, this collaborative effort revealed the kinds of positive impacts that are possible. Augmenting data sets with RWE can be a powerful addition to a study, even when there is not wholesale adoption of synthetic control arms.

Tokenizing data to increase RWE use

“Now that we can connect data across EMRs and other datasets, more information is available.” Safeguarding sensitive information through tokenizing data can help increase the use of RWE by enabling connections to other research efforts. “If a patient allows their data to be tokenized, it’s a HIPAA-compliant way to be able to connect their information with future data sets, so you can then ask the questions that you don’t even know are questions yet. You can’t go backwards [in time] and ask patients. [Tokenization] should reduce the cost of clinical research and improve outcomes.” Howell wants to see this become routine. “Patients, when given an option, do opt in. They want to help…The more that it can become the normal, and we can get encouragement from FDA, the better.”

Clinical research and RWE as patient centricity

Applying RWE across the continuum of clinical research is seen as a way to strengthen evidence and accelerate timelines. At an industry roundtable in September, former FDA Commissioner Scott Gottlieb explained that “the pandemic has reinforced the importance of practical implementation of RWE and accelerated trends that were underway, which will drive greater adoption. We are not going back.”

“There’s not an oncology trial on the planet that’s not behind in enrolling patients right now,” said Howell, owing to the introduction of some inconvenience or even deviation from the standard of care. She sees increased RWE and synthetic control arms as an opportunity to increase true patient centricity. “When clinical research is just a component of your own overall care, and it doesn’t feel out of the ordinary, and it’s integrated, that’s when we’re really moving patients through to more treatments more quickly.”

Peyton Howell is Executive Vice President and Chief Commercial & Strategy Officer at Parexel

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