Patients as partners, communication key to designing rare disease clinical trials
The American Society of Pediatric Nephrology has established a Therapeutics Development Committee to establish public-private partnerships and outline strategies to design and conduct pediatric nephrology clinical trials in a more efficient manner.
The committee includes academic pediatric nephrologists, patient advocates, private pharmaceutical company representatives, and employees from the US Food and Drug Administration (FDA) and the National Institutes of Health (NIH).
Read more: The challenges of pediatric clinical trials
“The committee hopes to help by creating a roadmap that can be used to design clinical trials and, with hope, facilitate the interaction between the key stakeholders to smooth the process,” Marva Moxey-Mims, M.D., chief of nephrology at the Children’s National Health System and founding committee member told Outsourcing-Pharma.com.
A paper summarizing the efforts was recently published online by Pediatric Research.
Rare disease, common challenge
The biggest challenge in conducting nephrology trials is the small numbers of patients diagnosed with any individual type of kidney disease, Moxey-Mims explained.
“This is exacerbated when it comes to pediatric kidney disease trials, where each type of disease essentially meets the definition of a rare disease,” she said. “This then means that a larger number of sites are needed to conduct adequately powered trials, which can increase the expense and complicate logistics.”
Yet, as Moxey-Mims noted, these challenges are not unique to pediatric kidney disease research, but apply all rare diseases – of which there are approximately 7,000 and only 5% with an FDA-approved treatment.
“As these varied groups share their own best practices, all of our patients will benefit,” added Moxey-Mims.
According to the report, the committee members have learned that by working together they can achieve better results – and that early communication can help avoid flaws in designing clinical trials.
The committee is meeting next month at the American Society of Nephrology conference to discuss next steps, which includes partnering with other groups.
Designing clinical trials
In the report, committee members note the role patients and their families play in early research study design, a role that has evolved as researchers recognize the benefits of their contributions.
Specifically, Moxey-Mims said patients and families can be helpful by telling study investigators what trial outcomes are personally most important.
“They also can help investigators see the clinical trial from their unique point of view, in terms of aspects that they might consider particularly burdensome or unacceptable, as well as things families would find particularly helpful to keep them engaged for the duration of the trial,” she explained.
“We are now more likely to think of participants as partners in the research endeavor, rather than ‘subjects’ of the research.”
Source: Pediatric Research
Title: Enhancing clinical trial development for pediatric kidney diseases
Authors: H William Schnaper, Joseph T Flynn, Coleman Gross, Anne B Cropp, Bastian Dehmel, Leah B Patel, Larry A Greenbaum, Elisabeth Houtsmuller, Frederick Kaskel, Marva Moxey-Mims, Karen Nowak, Douglas Silverstein, Aliza Thompson, Lynne Yao, Edress Darsey, and William E Smoyer