Rare Disease Report

Making Patients the Focus of Pediatric Clinical Trials

SEPTEMBER 29, 2017
Mark Sorrentino, MD

At the Global Genes Patient and Advocacy Summit, Rare Disease Report spoke with Mark Sorrentino, M.D. of PRA Health Sciences about the push to change clinical trial designs to make them more convenient for patients to participate in clinical trials.

Rare Disease Report: What is PRA Health Sciences?
Sorrentino: PRA Health Sciences is a global contract research organization we help operationalize and develop the clinical research for a number of entities including the pharmaceutical and biotech industries.

Within PRA we have the center for pediatric clinical development and I'm the vice president of that group. We just launched in April of this year and we are a global group of professionals that help bring innovative medicines to the pediatric space.

RDR: What can PRA Health Sciences offer to the clinical trial community?
Sorrentino: We bring some objectivity to an industry that has really been driven by subjectivity. What I mean by that is that the metrics to date have been rather poor in that companies would call a doctor or send him a Survey Monkey to see if the doctor had certain patients or whether they could join a global trial. We don't believe that that's the most effective way to find patients, families, and physicians that are in these areas.

We use a whole bunch of objective measures. For example, we do social listening campaigns and find out what the patients are talking about that have a given disease. And for that disease, we find out if there is a key opinion leader, etc. And we find out if there is a tertiary specialty center and if not, how do we connect those patients with the proper centers. 

Then we go out and look again at data-driven metrics about where the physicians, patients, and caregivers are and then match them all up to be able to join the trials.

RDR: The Duchenne muscular dystrophy families have argued that the 6 minute walks test is not an appropriate measure for measuring DMD drug efficacy. Are you finding that other patients with other conditions can educate pharmaceutical companies and regulators about what outcomes should be measured in clinical trials?
Sorrentino: That is a perfect example of where our patient experience team that does social listening is good at. It’s not just for DMD and other muscular dystrophies but for any disease. It could be for atopic dermatitis that the patients and the families might be having a completely different conversation than the regulators and maybe even the physicians as to what's important to them.

We find that being able to bring those patients in early to the conversation is really the hallmark of how you make change.

Change is slow, especially with the regulator's although I was just at the FDA conference on pediatric trial modeling and they were very forward-thinking in possibly including adolescents in adult trials in our industry. We've been saying that forever. Now you have the regulators saying that's something that we should be doing and that we haven't done in in the past. You still have a little bit of pushback from industry but again that's going to change now that they're hearing from the regulators.

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