FDA Commissioner Speaks on Agency’s Efforts for Patient-Focused Drug DevelopmentBy
The US Food and Drug Administration (FDA) has issued one of four planned draft guidances to describe how patient-experience data and other information from patients and caregivers can be collected and used for medical-product development and regulatory decision-making. The guidances are part of the FDA’s recently initiated patient-focused drug development (PFDD) efforts.
“We learn through scientific advances, but also by listening to patients,” FDA Commissioner Scott Gottlieb said in a June 12, 2018 statement. “Our work demands that we must continue to reflect on how we can make the science of drug development and review more modern and more patient-centered, so that approved products impact the metrics that real-world patients and families value most…Done well, the result is more and more of our review of new medical products benefits from a better understanding of the patient’s experience, providing our reviewers with the critically important context of a disease, and helping them to understand what’s most important to patients related to treatment benefits, risks and disease burden.”
In-line with these patient-centered goals, the FDA issued a draft guidance this month (June 2018) to inform patients and product developers on these principles. The guidance is the first of four guidances the FDA will issue, according to information from the FDA. The first draft guidance Patient-Focused Drug Development: Collecting Comprehensive and Representative Input, addresses sampling methods for collecting representative information on the patient experience to inform the development and evaluation of medical products throughout the medical product lifecycle. This draft guidance also discusses methods on how to operationalize and standardize the collection, analysis, and dissemination of patient experience data. It includes a glossary of terms that the FDA says it intends to use in one or more of the four draft guidance documents.
The four guidances are part of the FDA’s Patient Focused Drug Development (PFDD) efforts in accordance with the 21st Century Cures Act and the FDA Reauthorization Act of 2017. Through the PFDD initiative, started under the Prescription Drug User Fee Act V, the FDA has been addressing the need to better enable patients to provide input into drug and biologic drug development.
As part of that effort, Gottlieb noted that the FDA has held PFDD meetings in more than 20 disease areas to hear directly from those impacted by diseases, including opioid-use disorder, autism, HIV, Parkinson’s disease, and various conditions involving pain. “These meetings have given the FDA’s professional staff a deeper understanding of patient and caregiver experiences,” Gottlieb said in his statement. “We have gained from this experience. Also important to this effort is educating companies about rigorous approaches to obtaining and incorporating this important and unique input into product development. We must also explain how the FDA will incorporate this information into our regulatory decision-making.”