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The process of designing and implementing clinical trials hasn’t changed much over the decades — which may be why 1 in 5 clinical trials fail due to insufficient patient enrollment, and fewer than 5 percent of adult patients take part in clinical trials at all.

“The Federal Food, Drug, and Cosmetic Act of 1938 really continues to guide the whole industry,” notes Scott Schliebner, senior vice president of the Center for Rare Diseases, at PRA Health Sciences. “And our model hasn’t evolved much since. Clinical trials are taking longer to develop new medicines. That creates a snowball effect that affects overall healthcare costs.”

Not patient-focused

One of the main problems with the current state of affairs is the burden that traditional clinical trials place on patients. “The pharma-biotech space is very risk-averse,” Schliebner says, “and hasn’t adopted a customer-focused or patient-focused approach. We cannot move forward without patients — we’re 100 percent dependent on them, their data, and their participation — if we want to develop new life-changing therapies. Yet, they are kind of an afterthought in how we design these trials.”

This is especially true for rare diseases and disorders — something Luke Rosen knows firsthand. After his daughter Susannah was diagnosed with KIF1A Associated Neurological Disorder (KAND), the actor and activist founded KIF1A.ORG with his wife, Sally, in order to accelerate research into treatments for the disease.

“We have been involved in clinical research,” Rosen notes, “and it has been so difficult. Susannah sometimes has upwards of 100 seizures a day. She has a service dog and a wheelchair — when we travel, we travel big. It takes an hour to get out of the house, and fatigue is a trigger for a lot of the difficulty she has medically.”

Rosen thinks that a patient-focused attitude should extend to the goals of a trial as well. “Understanding and identifying what a meaningful endpoint would be is crucial,” he says. “Getting a therapeutic that would allow her to run a marathon would be awesome, but just getting her five more steps would be life-changing.”

New tools

The technology that would solve some of these problems already exists and is finally making inroads into the clinical trial space, thanks in part to the COVID-19 pandemic. “People are saying ‛That technology that we were hesitant to really jump on board with? Well, we kind of need that right now,’” Schliebner notes.

One example is PRA Health Sciences’ mobile health platform. Patients can download an app, connect with a clinical trial, review study information, and even sign a consent form electronically. A secure telemedicine component then allows patients to interact with the research investigator or physician.

“That can really eliminate travel,” notes Schliebner. “Beyond that, it gets really interesting. With Bluetooth, we’re now able to leverage all kinds of connected devices to measure lung capacity, or activity, or glucose and blood sugar levels.” That means improved data for the trial, as well as higher recruitment rates and fewer dropouts due to reduced disruption and costs facing patients.

Rosen endorses tools like this. “I think that certainly if there is a visit that could be remote, that would be wonderful,” he says. “Because it’s not one individual participating in a clinical trial — it’s a family participating in a clinical trial. It’s not a clinical trial for just Susannah. It’s a clinical trial that my wife, myself, and my eight-year-old son have to participate in.”

Schliebner stresses that technology is just a tool. “Tech can be a great part of the solution. But it’s more important to change our mindset to focus on patients, saying, ‛How can we make this a little bit easier so that it fits into patients’ lives?’”

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