Clinical trials for neurological conditions can be some of the hardest to fill and conduct. But there are dedicated groups working with patient and caregiver communities, and with neurological disease researchers to make progress. Clinical Informatics News spoke with three such initiatives to explore how participant recruitment and other aspects of clinical trials are changing. –The Editor  

By Maxine Bookbinder

February 27, 2018 | Neurological disorders are difficult to diagnose and challenging to treat; recruiting for these clinical trials requires novelty, patience, and a willingness to meet patients at their needs level. Limited mobility or cognition due to degenerative diseases, financial struggles, long travel distances, and lack of awareness about trial purpose and procedures are all potential obstacles for both patients and investigative teams. However, researchers are working to changing that.

“I don’t like to see failed trials because we can’t recruit,” says Clinton Wright, Director of the Division of Clinical Research at the National Institute of Neurological Disorders and Strokes (NINDS), part of the National Institutes of Health (NIH).  “We work hard with investigators to set milestones and stay on top of these to get data and complete studies.”

Part of the challenge, particularly in neurological trials, is finding the right patients.

“It’s important to recruit representative populations,” says Wright. “We must engage the community.  We must make sure studies are available to those groups.” This includes holding informational sessions at community and cultural centers and other venues where preferred patients normally gather.

He stresses the importance of asking the right questions and taking action on the answers. “We need to ask them, ‘What do you think are barriers to you participating in a trial?’ We need to design the recruitment process addressing those needs. We want reviewers to look at these things,” says Wright.

Recruiting control participants is also challenging, says Wright. In a placebo-controlled trial, patients might not want the risk of being randomized into a placebo group, though if the treatment is only available in a trial, they may be willing to participate if they know a placebo is combined with standard therapy. Patients may drop out, though, if experimental, cutting-edge treatments can be accessed off-label outside of the trial.

To address these challenges, NINDS is attempting to make recruitment easier, faster, and cheaper through creative infrastructure that bypasses slower, traditional start-up procedures within four clinical trial networks:

• SIREN, Strategies to Innovate EmeRgENcy Care, facilitates clinical trials in neurologic, heart, lung, blood, and traumatic emergencies;
• NeuroNEXT, Neurology Network of Excellence in Clinical Trials, facilitates early-phase clinical trials and biomarker studies preparatory to phase III clinical trials in neurological disorders;
• StrokeNet covers phase III clinical trials as well as early phase trials and biomarker studies preparatory to phase III clinical trials in stroke prevention, treatment and recovery; and
• The Neurological Emergencies Treatment Trials (NETT) Network comprises phase III trials of acute injuries and illnesses affecting the brain, spinal cord and peripheral nervous system.

NINDS has created master trial agreements to which network member institutions agree to abide, thus eliminating frustrating and time-consuming negotiations; it also has a central IRB. Thanks to these time-saving startup steps, “We can focus on building teams and running trials,” says Wright. “It’s helpful to have sites that have the infrastructure in place. It provides stability and quality.” He also emphasizes the importance of having knowledgeable and experience staff at sites. “We must have high quality coordinators at the sites who engage with the community, identify potential participants, and handle regulatory aspects,” he said.

Wright defines success as, “finding a meaningful answer,” but he’s realistic about what that answer may be. The real goal, he says, is forward progress. “Maybe [we] find a treatment, or a biomarker that’s helpful in a particular disease, but that doesn’t always happen. A meaningful result might reveal treatment is ineffective. That’s an answer to move forward. It’s not as great as finding a treatment or cure but it still helps to move forward.”