Abstract
Background
Defining and measuring unmet medical needs (UMN) from all health care stakeholders is essential for optimizing health care resource allocation decisions and guiding research and development priorities to address them.Objective
To understand UMN experience and prioritize UMN elements from the perspective of patients and caregivers.Methods
The mixed-method study consisted of a qualitative listening workshop and a quantitative ranking exercise. Adult patients with self-reported chronic diseases or disabilities and unpaid family caregivers were recruited through patient advocacy organizations, physician specialty organizations, academic centers, and social media. We conducted a virtual listening workshop to solicit patients' and unpaid family caregivers' perceptions of UMN and what has impacted them the most. Recruited patients were those living with a chronic disease or disability, including cancer, autoimmune, mental health, and rare diseases. A presurvey was designed to capture demographic and other qualitative information. Coding was performed for transcript analysis and reviewed by the patient moderator for accuracy. Following the qualitative workshop, we conducted a quantitative prioritization exercise to identify the most important domains of UMN for patients and caregivers.Results
We recruited 22 patients and caregivers to participate in the workshop via patient advocacy organizations. The majority (90.5%) of participants were patients, with a few having dual roles as unpaid family caregivers (23.8%). Patients and caregivers defined UMN as gaps in access to appropriate and quality care, specialists, providers knowledgeable about the specifics of their needs, patient access to trusted information about the condition, mental health, and financial support. Perceptions of UMN that patients experienced and considered important included financial hardship, mental health, barriers to care, caregiving burden, employment impacts, and health care system challenges. The ranking exercise identified health care system functioning, economic burden, and impacts on quality of life as the top-priority UMN domains, followed by more traditional measures anchored in clinical effectiveness.Conclusions
Our exploratory findings support both the depth and breadth of UMN faced by patients and unpaid caregivers, highlighting that UMN experienced by and important to them is not limited to traditional clinical effectiveness but also includes additional patient-centered components. Given the exploratory nature of this study owing to its small sample size, these findings are limited in generalizability and should be interpreted as hypothesis generating rather than definitive. Decision-makers should expand existing UMN frameworks to capture the full spectrum of patient-centered unmet medical needs.