Participating in a Clinical Trial – Does the Heart Rule the Head?

Since 1995, I’ve been working with the pharmaceutical industry and patient groups as a “bridge” to create multi-collaborative research teams with the objective of bringing new medicines to people in need as soon as possible. In the industry, we know this as “R&D.” and it sits within “Clin Ops.” An investigational medicine advances through development via a clinical trial program, and ideally at the end, there is sufficient data to show it is effective and safe, regulators approve it, and, after considerable financial negotiation with different national and international approval bodies and reimbursement systems, the new medicine becomes available for clinicians to prescribe to patients. The sooner this process advances, the sooner people get the medicines they need.

Hope – the Bridging Emotion in R&D

Introducing patient perspectives and preferences and patient-reported outcomes into the drug development process has become a critical part of R&D. In the pharma industry, this falls under the auspices of groups such as Patient-Focused Medicines Development (PFMD). I can’t think of a pharmaceutical company that doesn’t have some level of patient-centricity/engagement infrastructure.

We’ve come a long, long way. I can still recall the thrill in 1996 of sharing data faxed from the lab at MSD with patient groups about the efficacy of its protease inhibitor, indinavir, against HIV. There was shared exhilaration and a sense of hope. As we scrambled to explain to regulators what surrogate markers would ratify efficacy for approval, we were hopeful the clinical responses would speak for themselves. People with HIV who were taking indinavir were not dying.

In drug development, researchers are hopeful an investigational medicine will deliver the data that regulators require. At the same time, patients and their families are hopeful they will have better outcomes and improved quality of life. Hope is the common ground.

Hypocrisy – Do We Ignore the Emotions that Drive Decision-Making?

Given the decades I’ve worked to broker hope between the pharma industry and patients, why then, when a letter arrived from two prestigious organizations inviting me to enroll my son in a trial to evaluate an investigational COVID-19 vaccine did I barely read it? Why haven’t I enrolled him? The world needs these data and insights to keep coming up with solutions and continue to help people struggling with the effects of COVID-19 and long-COVID. But I hesitated. Does that make me a hypocrite who has spent two decades persuading people to participate in clinical trials? Or does it mean I’m a human who has forgotten that all decisions have an emotional component to them.

Action or inaction is promulgated on the four basic emotions of happiness, sadness, anger and fear – which, broadly speaking, lead to confidence, longing, frustration and worry respectively? Examining my conscience, I think (I hope!) I’ve just lost sight of the personal in being the professional. This is a wake-up call, reminding me and my colleagues in R&D that, if we don’t prioritize getting the human part right for participation in clinical trials, is it any surprise that people don’t enroll in clinical trials or that they struggle to remain in them?

To learn more about this, I conducted a survey of my colleagues who are co-invested in R&D to determine their perceptions and interest in participating in a clinical trial. I found the responses to be both honest and remarkable:

• 57% were curious about participating in a clinical trial.

• 24% said they would be concerned about participating.

• The majority (55%) said they only had a basic awareness about what would be expected of them in a clinical trial, and 18% said they felt ”clueless” about the phases of a clinical trial program.

• 85% said a family member or friend with a medical condition would motivate them to follow trials more closely, with 55% expressing that this would encourage them to try to participate themselves.

• Altruism was the top reason to learn more about the clinical trial process, with 42% saying they would like to be able to pass knowledge on to their community.

• The majority (51%) do not seek information about clinical trials; of those who do, 27% prefer to trust specialist medical sites.

What do these findings indicate about asking people to personally contribute to a clinical trial program? Again, the four basic emotions are at play, but fear predominates. People don’t seek information about trials until they have to or until a family member or friend becomes ill, at which point they step right up to participate in a trial themselves to help, even if they are “clueless” about how the trial actually works. This reinforces the human commonality of hope – that something can be done and, even if we don’t fully understand the science part, we can and will enroll in a trial if appropriate.

Harnessing Hope Not Hubris

Given this hope and trust that a person is prepared to “give” to a research organization, what responsibility does the pharmaceutical industry have to accept so it is harnessing this and not just creating hubris where patient inclusion is stated as a ‘mission’ but not part of the standard infrastructure or implemented in routine, everyday ways that matter to patients?

A few years ago, I heard Anna Cederber, former director at Lilly CoLabs and co-founder of Melo, a grassroots, patient-designed experiments platform state: “Justin Bieber can sell out Madison Square Garden in five minutes, but we can’t recruit and retain people in clinical trials.” That was a wake-up call to the industry to remind us that, when people care about something, they will take action. With a platform like Melo, patients are invited to “be a guest in an existing product trial or create your own.” This is a start-up mentality where “done is better than perfect.” This would likely induce fear in any person in Clin Ops or clinical trial design, but we are going to have to come to terms with greater agility in clinical trial design.

There is still considerable inertia (and hubris) around implementing patient-focused trial design, collaboration, engagement and experiences, because the pharma industry is concerned about potentially losing control. This is where support and reassurance from patients and patient groups are necessary. There is a co-responsibility to look for innovative and scalable ways to run clinical trials. The pharma industry must recognize that current clinical trials, which are designed to generate regulatory data, don’t account for human emotions that drive action. Patients, on the other hand, must accept the reciprocal emotions that drive business decision-making within pharma, such as the disappointment that occurs when a clinical trial doesn’t deliver the necessary safety and efficacy data to pursue a regulatory pathway.

With Clinical Trials Day approaching on Friday, May 20, I’m optimistic that we can start to have an ongoing and enduring conversation about the approaches we can take to re-humanize clinical trials. It starts with a simple alignment of ideology – humans make decisions based on underlying emotions and beliefs, and we must recognize that both the decision to run a clinical trial and the decision to participate in a clinical trial are unified by one core emotion – hope for a good outcome.

This is part one of a three-part series of articles focusing on “Re-Humanizing Clinical Trials.” In part two, Emma will outline what patients specifically want when participating in a clinical trial. Part three will provide recommendations on what industry can do to improve the patient experience at scale.