As important stakeholders in the drug development and approval process, patients provide a unique and valuable perspective when considering the benefits and risks of potential new and innovative medicines. Patients can identify areas of unmet medical need, provide critical perspective on the impact of a disease and its manifestations, and uniquely inform the development of outcome measures that are meaningful to patients. A scientific approach to gathering patient input is necessary to realize the shared goal of a patient-centered approach to drug development and regulatory review. PhRMA
The most recent reauthorization of the Prescription Drug User Fee Act (PDUFA) included new efforts by the Food and Drug Administration (FDA) to enable patients and patient groups to become actively involved in FDA evaluations of new medicines. Two programs in particular--the Patient-Focused Drug Development (PFDD) Initiative and Benefit/Risk Assessment--work in tandem to inform the FDA's decisions on whether or not to approve a medicine for patient use.
We at NORD believe that patients need to play a central and much more active role in the drug development process, from start to approval. Until very recently, the process has largely ignored the patient as an active participant.
An astute marketer will tell you that success of any product is dependent on the wants and needs of the end user. In health care, we need a similar mindset. By collaborating with patients early in drug development, there is an opportunity to improve the success rates in the preclinical stage of the development, which could ultimately accelerate the delivery of new treatments to patients and enhance patient adherence to their treatment plans. The end result will be a win for all stakeholders.
Over the past several years, Direct Relief has been privileged to work with and support several deeply dedicated partners in Liberia and Sierra Leone working hard and thoughtfully to expand and improve health services for people and build back the health systems that were decimated by civil wars that ended a decade ago. Of the many severe, chronic problems their efforts were seeking to address, the Ebola virus was not among them. When Ebola arrived, Direct Relief’s partner organizations were on the front lines of what has since become the worst, deadliest outbreak in history.