America’s biopharmaceutical research companies have long been dedicated to researching and developing new medicines that enable patients to live longer, healthier lives. Patients’ voices are becoming increasingly important in a patient-centric approach to drug development.
Patients, their families, caregivers and others are integral to the delivery of care and can provide unique and valuable perspectives on a specific disease or condition, as well as the treatment options available to the patient. These perspectives can then inform the U.S. Food and Drug Administration’s (FDA) evaluation of a medicine’s benefits and risks and provide the context for FDA’s regulatory decision making.
In recent years, biopharmaceutical researchers and the FDA have been increasingly integrating the patient perspective into the drug development and regulatory review process. As part of the 2012 reauthorization of the Prescription Drug User Fee Act (PDUFA V), the FDA established the Patient-Focused Drug Development (PFDD) initiative, which seeks to gather patient input on specific illnesses systematically and incorporate these data into the drug evaluation process. The FDA has committed to holding 24 disease area-specific PFDD meetings with both individual patients and patient organizations to hear their perspectives on their disease and treatments.
In addition to the PFDD meetings, patient data on improvements to their condition have also been used by the FDA to help inform its decision making. For example, melanoma patients provided information about the cosmetic and psychological advantages of seeing their lesions decrease in size. This information was instrumental in the FDA’s advisory committee’s overall favorable benefit-risk determination and the Agency’s decision to approve a new melanoma treatment.
Advancing science-based approaches to collect robust and meaningful patient and caregiver input can more consistently inform drug development and regulatory decision making. For example, since 2014, PhRMA has collaborated with the Genetic Alliance to explore the use of a technology-enabled, crowd-sourcing approach to gather insights into patients’ experiences with a disease or condition.
Continuing to develop a science-based and systematic approach to gathering data from patients robust enough to support FDA’s regulatory decision making will be necessary in order to realize the goal of a patient-centered approach to drug development and regulatory review. Additionally, it will be important to continue building upon the FDA’s PFDD efforts to encourage the development and use of patient experience data in the drug development and review process.
Future efforts that clarify how the FDA will collect and translate patient perspective data into tangible outcomes will help advance the science of patient input, enhancing drug discovery and development to better focus on patient needs.
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