Nick Kenny, PhD, Chief Scientific Officer at Syneos Health, understands the important role patients play in developing orphan drugs and designing clinical trials. Recently, we talked to Dr. Kenny at the BIO International Convention held in Philadelphia, PA.

Dr. Kenny noted that most parties, including investors, clinicians, and regulators, understand that patient input is essential for the development new medicines.

“I was listening to a panel discussion at JP Morgan earlier this year and investment groups are now insisting in many instances that for people that are bringing them interesting science in the rare disease space, if they have not already engaged with patients or patient advocates to truly understand the disease from the patient perspective, not just the clinical perspective, and create that wraparound value and assessment of was important to patients, then they’re probably not going to entertain funding for that drug,” noted Dr. Kenny.

And it is not just investors that are demanding patient input – so are regulatory agencies like the Food and Drug Administration (FDA). “We’ve all seen the guidances that the agency is beginning to put out. I think they have 4 new guidances planned and 1 has been issued that focused, and this sounds a bit odd, but I think that focus on final patient centricity.”

Historically, the words ‘patient centricity’ were thought of as collecting more data from the patient but today, it is taken on a new meaning, according to Dr. Kenny. Today, the ’patient centricity’ theme really does indicate that the industry and the regulators want to develop a drug that takes into account the patient’s perspective.

“I’m tremendously encouraged when we talk to patient advocacy groups about them finally getting a true voice and I think that’s being led not just by regulatory bodies and advocacy’s but by not-for-profits like the Leukemia Lymphoma Society that are truly trying to make their trials patient centric. And I think you see a lot more of that in other rare diseases too,” concluded Dr. Kenny.

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