Expanding the definition of unmet medical need: Findings from a multistakeholder workshop.

چکیده اصلی

BACKGROUND: Unmet medical need (UMN) is frequently referenced in health policy and health care decision-making, including the Centers for Medicare & Medicaid Services' (CMS) Drug Price Negotiation Program (DPNP). However, the concept of UMN is often defined inconsistently and has a narrow focus, primarily centered on clinical effectiveness and the availability of treatment. This approach may overlook the wider needs of patients, caregivers, and society as a whole. A broader conceptualization of UMN has been proposed, yet there remains a lack of an actionable implementation framework. OBJECTIVE: To examine how stakeholders interpret and distinguish among UMN elements, prioritize these elements based on perceived importance, and elicit perspectives on the applicability of a prioritized UMN framework in a decision-making context. METHODS: A multistakeholder workshop was conducted in June 2025 involving 17 participants representing researchers, industry, payers, policymakers, and a patient organization. Participants evaluated 38 UMN elements grouped into 7 domains derived from a prior structured literature review. Using an interactive polling platform, participants assessed the conceptual distinctness of elements within domains, ranked domains by importance, and completed a pairwise weighting exercise grounded in the Simple Multi-Attribute Rating Technique. Quantitative voting results were aggregated, and qualitative feedback from plenary discussions was recorded and thematically synthesized. RESULTS: Participants confirmed the conceptual distinctness of the proposed domains and elements. In the weighting exercise, traditional clinical effectiveness received the highest importance (24%), followed by availability of other treatments (19%) and impacts on patient, caregiver, or family quality of life (16%), although differences across domains were modest and should be interpreted with caution, given the exploratory sample. Economic burden on patients and families (13%), societal perspective elements (11%), and economic burden on society (10%) received moderate weights, whereas treatment administration attributes received the lowest weight (7%). Participants agreed that the broader element set captures dimensions of UMN not addressed by the current framework under CMS's DPNP. Key qualitative themes included perceived overlap within elements of the quality-of-life domain, the need for clearer conceptual boundaries between UMN and comparative effectiveness, and feasibility concerns around implementation. CONCLUSIONS: UMN is inherently multidimensional and extends beyond traditional clinical measures. These exploratory findings suggest that structured prioritization of UMN domains may support more transparent and policy-relevant frameworks for evaluating unmet need in the United States, including within CMS's DPNP.

ترجمه فارسی چکیده

نیاز پزشکی نام fulfillment (UMN) اغلب در سیاست‌های سلامت و تصمیم‌گیری‌های درمانی ارجاع داده می‌شود، اما تعریف آن اغلب ناهمگون است و تمرکز محدودی بر اثربخشی بالینی و در دسترس بودن درمان دارد. این رویکرد ممکن است نیازهای وسیع‌تر بیماران، مراقبان و جامعه را نادیده بگیرد. هدف این مطالعه بررسی نحوه تفسیر و تفکیک عناصر UMN توسط ذینفعان، اولویت‌بندی این عناصر بر اساس اهمیت percepsted و درک دیدگاه‌ها در مورد کاربرد یک چارچوب اولویت‌بندی شده UMN در زمینه تصمیم‌گیری است. در این کارگاه چندجانبه که در ژوئن 2025 برگزار شد، ۱۷ شرکت‌کننده (شامل پژوهشگران، صنعت، تأمین‌کنندگان، سیاست‌گذاران و یک سازمان بیمار) ۳۸ عنصر UMN را در ۷ حوزه ارزیابی کردند. نتایج نشان داد که اثربخشی بالینی سنتی بیشترین اهمیت را داشت (۲۴٪)، و اثرات بر کیفیت زندگی بیمار یا خانواده ۱۶٪ بود. عناصر مربوط به بار اقتصادی و دیدگاه اجتماعی وزن‌های متوسطی داشتند. ذینفعان موافقت کردند که مجموعه عناصر وسیع‌تر ابعادی از UMN را پوشش می‌دهد که در چارچوب فعلی تحت برنامه مذاکره قیمت داروهای مراقبت سلامت (DPNP) پوشش داده نمی‌شود.

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