Patient Engagement, Pharma

Why designing patient-centered clinical trials is like conducting an orchestra

At the sixth annual BIO Patient and Health Advocacy Summit in Washington, D.C. this week, a discussion on clinical trial design explored some of the challenges of ensuring patient's needs are balanced with those of drug developers and regulatory needs.

What tends to become lost in the course of developing clinical trials is the voice of the patient — something Sharon Tan knows all too well.

As global project head of Sanofi Genzyme, Tan works with the Massachusetts-based company’s in-house unit on rare diseases, and specifically on Niemann-Pick syndrome, an inherited disease that affects the way the body metabolizes lipids. People with Type A and B Niemann-Pick suffer from fat buildup in their cells, which eventually causes cells to die. Currently, there are no treatments available for Niemann-Pick, and because the disease is so rare, it’s not only hard to find enough patients for clinical trials, but also to keep those patients’ lives stable during the course of them.

“Our trials are very long, sometimes five years, just because of the rarity of disease,” said Tan. “Participating in a clinical trial takes a lot of commitment that impedes family life, work life — some patients have said they’ve lost jobs because of this.”

So designing clinical trials with patients in mind from the start is key, and was one of the salient insights from a panel discussion Tan participated in Monday at the sixth annual BIO Patient and Health Advocacy Summit in Washington, D.C., this week.

But doing that takes some skilled maneuvering, as there are multiple stakeholders all interested in obtaining something different from a clinical trial. It’s like conducting an orchestra, to borrow a metaphor used by Amy Kant, Reinvention Director at the National Niemann-Pick Disease Foundation. “You need to align the different interests of groups,” she said, referring to sponsoring organizations, research groups, clinicians, and patients.

Still, the needs of the patients ought to come first, a common theme throughout the entire discussion. When T.J. Sharpe was diagnosed five years ago with Stage IV melanoma, clinical trials became his saving grace. The second trial he was a part of was successful, and over the summer, after four years, doctors found no metastatic disease in his body.

“I never saw myself as a subject; I saw myself as part of something that was hopefully going to create a cure or a treatment,” Sharpe said. “Being the best option for healthcare should be what clinical trials should be.”

In other words, they’re not just chances for research. In Sharpe’s case, it was the best — and only — option he had to make sure he could stay alive “to see my kids grow up.”

How to shift the focus of clinical trials more toward patients’ needs was the point Zachary Hallinan drove home. Hallinan is a project manager at the Clinical Trials Transformation Initiative (CTTI), a public-private partnership established by the U.S. Food and Drug Administration and Duke University in 2007. Since inception, CTTI’s focus has been on trying to understand the relationship between patient groups and sponsors in clinical trials, and how each side thinks about the value of engaging patients in clinical trials.

After the discussion, in an interview, Hallinan offered up two general observations on how clinical trials can be designed with patients in mind from the get-go. “Make sure you’re asking questions that are meaningful to patients,” he said. “And make sure the clinical trial is as minimally burdensome as possible.”

It’s something Tan and Sanofi Genzyme have been thinking about more and more. After relaying the story about how some patients in their clinical trials have been so burdened by their participation that they’ve lost jobs, Tan told the audience that a new system has since been implemented: a service of nurses who administer drugs by going to the homes of patients participating in clinical trials.

“That’s thinking about what the patients’ needs are,” she said.

Photo: XinXinXing, Getty Images

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