Patients and caregivers widen definition of unmet medical need

Two new National Pharmaceutical Council studies found that patients, caregivers and a broader stakeholder group define unmet medical need more broadly than the federal clinical standard used in drug policy. The findings could influence how CMS, FDA and payers weigh access, cost and quality-of-life in future decisions.

Why it matters: - The studies suggest federal policy may be using too narrow a lens when judging unmet medical need. - A broader definition could affect drug pricing, FDA expedited pathways and coverage decisions. - The findings add evidence that access, cost and quality of life matter alongside clinical effectiveness.

What happened: - The National Pharmaceutical Council released two studies on how different groups define unmet medical need. - One workshop included 22 patients and family caregivers. - The other included 17 stakeholders from research, industry, payer, policymaker and patient-group backgrounds. - Both groups ranked unmet-need dimensions identified through a literature review.

The details: - The literature review pulled in patient, caregiver and societal perspectives, including views already reflected in federal policy. - The review also identified broader domains: quality-of-life impacts, economic burden, treatment administration attributes and health system functioning. - Patients and family caregivers ranked health system functioning first. - That group then ranked economic burden, quality of life and availability of treatment alternatives ahead of traditional clinical effectiveness, which placed fifth. - The multi-stakeholder group ranked traditional clinical effectiveness first. - That group placed availability of other treatments second, followed by quality of life and economic burden. - Both groups ranked economic burden and quality of life as important dimensions missing from the government’s current definition. - Jonathan D. Campbell, NPC chief science officer and co-author of both studies, said unmet medical need extends beyond whether a treatment works to include access, cost and life impact. - NPC managed both workshops with the University of Colorado Anschutz Medical Campus. - The National Health Council co-managed the patient workshop.

Between the lines: - The two groups did not fully agree on what should come first, but they did converge on a broader concept than the clinical standard. - The patient and caregiver ranking put system performance and affordability ahead of efficacy, which suggests lived experience can diverge from policy frameworks. - The authors describe the research as exploratory, so the work is more signal than final answer. - Their broader point is that unmet need should be measured in a way that captures where patient pain is greatest, not just where treatment options are limited.

What's next: - The authors call for future definitions of unmet need to draw on both perspectives. - They also call for additional research to better define and measure unmet need. - Decisionmakers at CMS, FDA and in coverage policy may use this kind of evidence as pressure builds for a wider framework.

The bottom line: - Patients and experts are pushing unmet medical need beyond clinical effectiveness alone, toward a definition that includes access, cost and quality of life. More information: NPC