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

Abstract

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)

Impact of COVID-19 and effects of BNT162b2 on patient-reported outcomes: quality of life, symptoms, and work productivity among US adult outpatients

“…However, there is limited research on the potential benefits of vaccination on physical, mental, social, emotional functioning and economic well-being. It has been increasingly recognized that such aspects of health and well-being are best described by the patients themselves through Patient-Reported Outcomes (PRO), without amendment or interpretation of the patient's response by a clinician or anyone else [ 10 – 12 ]. Both generic and disease-specific PROs have been widely used in vaccine research to measure the patient experience of disease-related symptoms, disease impact and HRQoL [ 12 ].…”

“…It has been increasingly recognized that such aspects of health and well-being are best described by the patients themselves through Patient-Reported Outcomes (PRO), without amendment or interpretation of the patient's response by a clinician or anyone else [ 10 – 12 ]. Both generic and disease-specific PROs have been widely used in vaccine research to measure the patient experience of disease-related symptoms, disease impact and HRQoL [ 12 ]. Leveraging a US national retail pharmacy SARS-CoV-2 test database and using validated PRO measures (PROMs), the objective of this study was to address such evidence gaps by assessing COVID-19 symptoms, HRQoL and WPAI prior to through one month following SARS-CoV-2 infection in outpatients, and compared results between unvaccinated individuals and those vaccinated with BNT162b2.…”

Vaccines – beliefs and concerns: the voice of patients with inflammatory immunomediated diseases

“…This study has several strengths including the large sample size, the patient-friendly questionnaire design, and the focus on patients' knowledge and perceptions. This particular focus is not common in studies and research in the field of vaccination; however, medical and political authorities may take valuable lessons from analyzing patients' perspectives on relevant issues for public health [9,[18][19][20][21].…”

Ensuring equity with pre-clinical planning for chlamydia vaccines

“…The Food and Drug Administration has begun considering patient experiences as well through the use of patient-reported outcomes (PROs), with an argument that patients are uniquely qualified to report this context of vaccine development 90 . PROs often measure experiences of disease-related symptoms, disease impact, health-related quality of life related to the disease, and the impact of an intervention (like a vaccine) on a patient 90 . This may be through questionnaires, symptom diaries, and validated instruments to assess quality of life 90 .…”

“…PROs often measure experiences of disease-related symptoms, disease impact, health-related quality of life related to the disease, and the impact of an intervention (like a vaccine) on a patient 90 . This may be through questionnaires, symptom diaries, and validated instruments to assess quality of life 90 . However, PROs are typically implemented during a clinical trial.…”