PMU115 A QUALITATIVE ANALYSIS OF THE MOST COMMON THEMES FROM THE VOICE OF THE PATIENT REPORTS

Abstract

Under the fifth Prescription Drug User Fee Act (PDUFA V), the Food and Drug Administration (FDA) held a series of Voice-of-the-Patient (VoP) meetings intended to improve medical-product development by gathering patients’ perspectives on their condition and treatments. This analysis explored most common themes identified across the 27 FDA-led and 23 externally-led VoP meetings with available reports. Publicly available VoP meeting reports were retrieved from the FDA or, for externally-led meetings, corresponding patient-advocacy organization websites. Data were extracted by three researchers and thematically analyzed. Four categories were created for common symptom themes: physical symptoms, mental impacts, psychosocial burden, and work/finance/social burden. Eight categories were created for common treatment themes: condition characteristics, benefits and risks, barriers, prescription treatments, alternatives to drugs, future treatment needs, clinical-trial design, and placebo. Common symptom themes: Mental impact subthemes included psychological impact (n=48); work/finance/social burden subthemes included participating in physical or social activities (n=40), unable to work/workplace concerns (n=39), performing ADLs/chores (n=35); Physical symptom subthemes included sleep disturbance (n=35), pain (n=35), and fatigue (n=34); and psychosocial burden subthemes included quality of life (n=35), worry about and living with uncertainty (n=33). Common treatment themes: benefits and risks subthemes included side effects (n=42), risk and benefit tradeoff (n=26); barriers subthemes included financial burden/unaffordability (n=32), fair access to medications (n=29); alternative treatments to prescriptions subthemes included diet modifications (n=34), dietary supplements (n=31); condition characteristics subthemes included substantial symptom heterogeneity (n=30); and future treatment needs subthemes included need for additional therapies (n=36), treatment with less side effects (n=29), and need for disease modifying treatment (n=25). Examination across diverse VoP reports provides insights across diverse patient populations and illnesses on patient-experiences. Common themes emerged that can help stakeholders understand the symptoms, impacts, and challenges patients face. These findings can be useful in medical-product development and value assessment.