How we can innovate in decentralized clinical trials: “People do not change when they see the light, they change when they feel the heat.”

“People do not change when they see the light, they change when they feel the heat,” Dr Amir Kalali, a physician-scientist, drug developer, founder of the Collaborating for Novel Solutions (CNS) Summit, and co-founder of the Decentralized Clinical Trials and Research Alliance, DTRA, tells IDEA Pharma. So now that the imminent crisis of coronavirus has retrenched, what changes and adaptations must the industry continue to make to keep that heat smoldering?

For one thing, the ability to select decentralized methodologies is the way forward for more patient-centered clinical research. “Experiments need to stop being done to patients and start being done with them,” says Kalali. This is also reflective of the IDEA Pharma philosophy, a core tenet of which is that meaningful medicines are created as the result of the right design decisions. Patient input must be considered when designing hybrid trials to maximize the opportunities for a molecule in development

To level-set: a decentralized clinical trial is one that uses direct-to-patient and other technologies that allow patients to participate in research in a location of their choosing. It reduces the burden on the patient directly by eliminating the need to go in person to the research site and indirectly through less time away from work, not needing to organize child-care, and saved time and money on transportation.

Historically, clinical trials are labor intensive and disruptive to a patient’s life. This has not bode well for pharma companies because lack of patient enrollment in trials is one of the leading causes of their failure—and not necessarily because the drug did not work. Kalali agrees. “If you can open up that throttle and be able to recruit patients more efficiently, that means more drugs can be developed,” he says. And the more drugs that are developed means more patients can be treated.

For the drug development industry, hybrid trials are the way forward for more equitable access to research. These trials require some in-person components and some virtual components, and this is the time to ask patients individually what works for them and their disease. As Kalali puts it, “optionality is key.” For instance, a patient with children may elect to participate in a more remote fashion so they don’t have to find child-care. A patient who is frightened of needles may elect to go into the research site, so they won’t have to learn how to self-administer injections. These considerations matter because it enables patients to participate in research on their own terms so that they will be more engaged, and the trials will be able to retain a more diverse set of patients with divergent needs.

“The impact is here to stay in terms of how we have modern, fit-for-purpose trials that give access to a much wider population of patients and allow us to assess those patients on an ongoing basis,” Kalali explains.

Not only is finding patients in general a challenge—finding patients that are truly representative of the population that will receive the drug is an even greater challenge. Because clinical trials are typically conducted on white men, pharma has a pressing need for patient diversity and retention in clinical trials, and decentralized trial methodologies can be a tool that, if deployed thoughtfully, reduce the cost and time it takes to recruit patients in the pursuit of providing more equitable access to research.

Although the potential of decentralized clinical methodologies is undeniable, the field is still more nascent than the headlines make it out to be. To address some of its growing pains, the Decentralized Trial and Research Alliance, DTRA has taken on the mission of enabling stakeholder collaboration to make research participation accessible and widely adopted via decentralized methodologies. The organization has four priorities it plans to tackle through 12 initiatives, the two with initial output are 1) a Decentralized Research Glossary to standardize the language used surrounding decentralized trials; and 2) Crowdsourcing the Evidence of Impact to aide with aggregation of data to demonstrate the need for promotion of the education of decentralized clinical trials.

So What?

Decentralized clinical trial methodologies are still in relatively early stages. They have been put on the fast track the past few years, exposing long-standing problems and barriers within the biomedical research ecosystem. Currently, federal officials report only 6% of clinical trials yield actionable results. Of the molecules in the 94% of clinical trials that fail, too many are not for scientific reasons—meaning the studies failed to recruit enough patients or have been inadequately designed (1).

This record of failure needs to be addressed with innovative and modern approaches such as expanded decentralized clinical trial use. Given that these trials are at the heart of drug development, the industry must undertake constant improvement to create a mean and lean clinical trial infrastructure—and that can be achieved by thoughtfully adopting innovative approaches such as decentralized trials which maximally benefit patients with unmet needs. The life sciences industry has both seen the light and felt the heat. Now is the time to make sure the innovative fire remains ablaze, and the advances keep coming.

References:

(1) Sue Sutter, “COVID-19 Should Force ‘Soul Searching’ over Fragmented US Clinical Trials System, Woodcock Says,” Pink Sheet, July 22, 2022,https://pink.pharmaintelligence.informa.com/PS143029/ COVID19-Should-Force-Soul-Searching-Over-Fragmented-US-Clinical-Trials-System-Woodcock-Says.