Patient-reported outcomes in vaccines research: relevance for decision-making

The development and demand for effective vaccines have witnessed an exponential growth over the last century. In the meantime, the vaccine market involves more knowledgeable stakeholders, with a shift in emphasis by regulatory agencies on understanding the patient perception and experience. The Food and Drug Administration's publication of the patient-reported outcomes (PRO) guidance has elevated the discipline of PROs and has resulted in a transition from clinician reports of patient outcomes to PROs. This review reports various research methods, which utilize PROs, including qualitative and quantitative research, clinical trials, and patient preference studies. With the advancement of electronic PRO data capture, additional advantages of PROs are being observed and utilized (e.g. as a trigger for clinical endpoints). We discuss uses and advantages of including PROs into the clinical trial program to improve efficiencies, clinical relevance and overall validity of the program in the vaccine field. (See Plain Language Summary) What is the context? Potential vaccine recipients want to understand the benefits and risks of a vaccine directly from the patient perspective. Well-defined Patient-reported outcomes (PROs) provide this perspective. The Food and Drug Administration (FDA) bases their approvals for interventions on how the patients feel, function and survive, with PROs providing important quantitative estimates of how patients feel and function. What is new? The FDA and European Medicines Agency (EMA) have developed frameworks that ensure that patients' experiences, perspectives, needs, and priorities are captured and meaningfully incorporated into drug/vaccine development and evaluation. This has led to an increased interest in PRO evaluations by other stakeholders including regulatory authorities, ministry of health, health technology assessment bodies, national immunization technical advisory groups (NITAGs), payer groups, key opinion leaders, healthcare providers and potential vaccine recipients. The availability of new technologies (e.g. smartphones) has increased the role of virtual observational and clinical studies, using mobile clinical trial platforms assessing PROs with no in-person site visits required. What is the impact? PROs may be incorporated into the research and development program of a vaccine using virtual technology, resulting in a more representative sample that is easier to recruit and retain. This introduces efficiencies and im