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.
Plain language summary
We brought together patients and unpaid family caregivers to understand what unmet medical needs (UMN) matter most to them. Through a qualitative listening workshop and a follow-up ranking exercise, participants described how unmet needs affect their lives and prioritized what is most important. They reported substantial unmet needs and ranked health care system barriers, financial burden, and impact on quality of life as the top concerns.
Implications for managed care pharmacy
Our study demonstrates both the depth and breadth of UMN faced by patients and unpaid caregivers. By integrating qualitative insights with quantitative ranking, we provide foundational evidence to inform development of patient-centered UMN framework, assisting decision-makers to comprehensively identify and measure unmet need, thereby steering health care innovation toward areas of greatest need and value. These findings have limited generalizability and should be interpreted as hypothesis generating. Future research should validate and refine identified domains.
Defining and measuring unmet medical needs (UMN) across all health care stakeholders is essential for optimizing health care resource allocation and guiding research and development priorities. There is growing recognition of the importance of incorporating patient-centered experiences and perspectives to inform UMN assessment.1–4 Incorporating patient-centered perspectives can help ensure that UMN assessments better reflect the real-world experiences and priorities of those affected by diseases, which ultimately guides health care innovation, regulatory, and access decisions toward areas of greatest need and value.
Concerns have been raised that the existing UMN scope may be too narrow to fully reflect patient-centered dimensions and broader societal priorities.5 Existing UMN frameworks have largely been developed for regulatory decision-making and primary emphasize clinical effectiveness and treatment availability.1,6–8 For instance, the US Food and Drug Administration (FDA) guidance has defined UMN as a “condition whose treatment or diagnosis is not addressed adequately by available therapy.”8 Such a definition not only underlies regulatory pathways and incentive programs but is also critical in informing important health care decision-making beyond the regulatory domain. For instance, the Centers for Medicare & Medicaid Services (CMS) considers the degree of UMN and the extent to which a selected drug addresses that need when implementing policies under the Medicare Drug Price Negotiation Program.9 Payers also consider incorporating UMN considerations into formulary design and reimbursement decision-making.10
Additionally, UMN is rarely defined in a way that is actionable and meaningful for decision-making, particularly from a patient-centered perspective.1,2,7,11 Stakeholders’ viewpoints differ regarding which domains should be included to define UMN and how it should be measured.1,2,7,11 Although a growing body of literature has proposed expanding UMN beyond traditional clinical effectiveness to include quality of life (QoL) of patients, caregivers, and families, economic burden at the household and societal levels, and other societal impacts, limited evidence exists in assessing the relative importance of UMN components, which limits decision-makers’ ability to incorporate patient-centered UMN measures into existing UMN assessment structures.2
Therefore, it is pivotal to assess how patients and caregivers perceive and prioritize the UMN they face. We aimed to understand UMN experience and prioritize UMN elements based on the literature and input from patients and caregivers, with the goal of informing the development of an actionable framework that meaningfully integrates patient-centered perspectives.
Methods
The mixed-method study consisted of a qualitative listening workshop and a quantitative ranking exercise. We conducted a listening workshop to solicit patients’ and unpaid family caregivers’ perceptions of UMN and what has impacted them the most. The listening session was conducted virtually and recorded over Zoom. Textual analysis and thematic coding were carried out by an independent researcher and confirmed through the review of a supervisory researcher. To ensure a patient-centered approach to the study, textual analysis was additionally reviewed by the patient moderator for accuracy. However, we did not preidentify the domains of unmet needs before engaging with the participants in the qualitative portion of the workshop; instead, they were extracted through analysis using Braun and Clarke’s framework, incorporating both deductive and inductive coding.12 Deductive coding was due to some understanding of top unmet needs ascertained through a pre-event survey as well as via the listening session question structure. Given the data derived from 1 single listening session, intercoder reliability statistics were not calculated. Rather, coding rigor was established through methodological triangulation of pre-event survey data.
RECRUITMENT
Patients were recruited if they met the inclusion criteria of being an adult (aged 18 years and older) and having a self-reported chronic disease or disability across 4 main disease areas, including cancer, autoimmune, mental health, and rare disease. No participants were excluded based on criteria, but after saturation was reached (n = 22 participants), some patients and caregivers were not able to be accepted as the workshop was at full capacity. Patients and unpaid family caregivers who participated in this virtual listening workshop were recruited through a variety of methods, including National Health Council social media channels, advertisements on individual patient advocacy organizations, physician specialty organizations, and academic centers. Recruitment began in January 2025, and the workshop was conducted via Zoom on March 24, 2025, for 90 minutes. An institutional review board exemption was obtained from the University of Colorado (COMIRB No. 25-0574). Participants were given an honorarium ($400.00 per participant) for their time. A patient familiar with patient engagement and value assessment/health technology assessment was enlisted to moderate the listening session to provide a flat power dynamic. As patients’ and caregivers’ roles overlap and are often dual circumstances within a family (eg, a patient may have a chronic condition and also be a caregiver for a family member with a chronic condition), we recruited both patients and unpaid family caregivers for a single session.
PRESURVEY AND LISTENING WORKSHOP
Patient-facing materials were developed, including an informed consent document, a discussion guide, an agenda, a presentation, and a pre-event survey (Supplementary Materials (90.1KB, pdf) , available in online article). The pre-event survey was designed to capture demographic and other qualitative information, including participants’ understanding of UMN and identification of their unmet priorities, in open-ended formats. All recruited patients and unpaid family caregivers were given the opportunity to respond to the optional presurvey before the event. Open-ended questions in the presurvey were designed to understand patients’ top challenges and unmet needs across the disease journey, as well as the broader impacts of disease on family members, caregivers, employers, and others in their social circle. Methodological triangulation was employed by integrating data from the presurvey event with data from listening session, enabling convergence of findings and themes. Additionally, to understand how the UMN has impacted patients’ lives in general, participants were asked to rate the impact of UMN during their worst disease period on a scale of 1 (least severe) to 10 (most severe) and then rate the overall significance of these unmet needs today.
The qualitative listening workshop discussion guide was developed based on associated patient-led qualitative work and on important topics noted by the listening session participants during the presurvey.13 Patients and caregivers during the listening session were asked the following: (1) How do you define an unmet medical need? (2) What are the top 3 to 5 challenges related to unmet needs with your condition that you have faced? (3) How do these unmet medical needs impact your day-to-day activities? (4) What challenges impact others around you, such as your family, caregivers, employers, and others in your social circle?
CODING ANALYSIS
The listening session was recorded via a secured Zoom session, and a raw transcript was extracted for coding analysis. Given the listening session was structurally guided across various topics in relation to unmet needs, deductive codes were identified. Followed by transcript review, inductive codes were identified, providing a deductive/inductive mixed coding approach to this study.
RANKING EXERCISE
Following the qualitative workshop, we conducted a quantitative prioritization exercise to identify the most important domains and elements of UMN for patients and caregivers. The domains shown to patients and caregivers were extracted and previously categorized from the UMN concepts synthesized from a targeted literature review.2 The elements were synthesized and categorized into 6 patient-centered domains derived from the original 40 in the previously published review. The 6 patient-centered domains include traditional clinical effectiveness; impacts on QoL; economic burden on patients, caregivers, and families; attributes of treatment administration; availability of other treatments; and health system functioning.
Exercises were based on a modified version of Simple Multi-Attribute Rating Technique (SMART) to generate a prioritized list of domains.14,15 SMART ranking and weighting simplifies the ranking process by asking participants to rank only 3 domains at a time followed by an exercise that compares the “space” between the ranks. First, participants were asked to rank the 3 most important domains. The top-3-ranked domains were then summed at the group level where the highest-ranked domains had more “points,” that is, ranked highest among more individual participants during the ranking exercise as compared with the lower-ranked domains. The top 3 were removed and then participants were asked to rank the final 3 domains, which were then summed at the group level again until the full list was exhausted. Themes from the presurvey were used as examples of each domain to give context for participants. To generate space between ranks, we used weighting exercises that asked participants to compare adjacent domains in the rankings. The relative importance between pairs was based on a zero-percentage difference (equally important) up to a 50-percentage difference (maximum difference). The mean percentages were then used to generate the final weighted domain list, normalized to 100%. We report both the relative weights and the frequency of responses above the median importance when comparing (ie, greater than or equal to 25% difference based on the scale from 0% difference to 50% difference). The ranking and weighting exercises, as well as the scoring rules, have been used in prior patient engagement exercises.16 Mentimeter, a voting software tool, was used to generate responses from participants anonymously (ie, participant demographics were not linked to individual responses).
Results
PARTICIPANTS
We recruited 22 patients and caregivers to participate in the workshop, and a total of 21 (95.5%) responded to the presurvey regarding their demographic and clinical characteristics (Table 1). The majority (n = 19, 90.5%) of the participants were patients, with a few also serving simultaneously as caregivers (unpaid family members, n = 5, 23.8%). Participants tended to be living with autoimmune (n = 11, 52.4%) or rare conditions (n = 7, 33.3%). Participants were not fully representative of the US population, particularly with respect to gender, regional location, condition type, and insurance type. They were predominantly female (n = 18, 85.7%), Black/African American (n = 10, 47.6%) or White (n = 9, 42.9%), older than 35 years (n = 19, 90.5%), living in urban or suburban communities in the South (n = 17, 81.0%), earning between $25,000 and $150,000 per year per household (n = 16, 76.2%), and covered by private insurance (n = 15, 71.4%). Caregivers all represented patients older than 18 years, often spouses or parents, and were primarily connected to the adult neurological rare disease community.
TABLE 1.
Demographic Characteristics From the Presurvey
| Question | n | % |
|---|---|---|
| Which community do you represent?a | ||
| Patient | 19 | 90.5 |
| Caregiver (unpaid family member) | 5 | 23.8 |
| If you are a caregiver, is the patient you are representing over or under the age of 18?a | ||
| Patient is aged 18 years or older (adult) | 8 | 42.1 |
| Patient is younger than 18 years (a child or juvenile) | 0 | 0 |
| Not applicable (not a caregiver) | 11 | 57.9 |
| Which disease community are you most closely aligned with?a | ||
| Autoimmune | 11 | 52.4 |
| Cancer | 4 | 19.1 |
| Mental health | 2 | 9.5 |
| Rare | 7 | 33.3 |
| What is your sex? | ||
| Male | 3 | 14.3 |
| Female | 18 | 85.7 |
| What is your age range? | ||
| 18-24 | 1 | 4.8 |
| 25-34 | 1 | 4.8 |
| 35-44 | 4 | 19.1 |
| 45-54 | 5 | 23.8 |
| 55-64 | 4 | 19.1 |
| 65+ | 6 | 28.6 |
| What is your racial or ethnic identity?a | ||
| Asian | 2 | 9.5 |
| Black or African American | 10 | 47.6 |
| Hispanic/Latino | 0 | 0 |
| American Indian/Alaskan Native | 0 | 0 |
| Pacific Islander or Hawaiian Native | 0 | 0 |
| Middle Eastern | 0 | 0 |
| White | 9 | 42.9 |
| Other | 0 | 0 |
| What is your census region? | ||
| Midwest | 4 | 19.1 |
| Northeast | 7 | 33.3 |
| South | 9 | 42.9 |
| West | 1 | 4.8 |
| What is your community setting? | ||
| Rural | 4 | 19.1 |
| Suburban | 8 | 38.1 |
| Urban | 9 | 42.9 |
| What is your yearly household income before taxes? | ||
| Less than $25,000 | 1 | 4.8 |
| Between $25,000 and $49,999 | 6 | 28.6 |
| Between $50,000 and $99,999 | 4 | 19.1 |
| Between $100,000 and $149,999 | 6 | 28.6 |
| More than $150,000 | 4 | 19.1 |
| What type of health insurance covers most of your medical expenses?a | ||
| Private (through employer, spouse’s insurance, or purchased individually) | 15 | 71.4 |
| Public (Medicare, Medicaid, CHIP, Tricare) | 8 | 38.1 |
| No insurance (self-pay) | 0 | 0 |
Represents questions for which participants could choose multiple options,
so the percentages/numerical values may be more than 100%.
UNMET NEEDS FROM THE PATIENT AND CAREGIVER PERSPECTIVE
In the presurvey and listening workshop, patients and unpaid family caregivers defined UMN through the following emergent themes: (1) lack of access to care that is timely, (2) lack of education among providers of treatment options and services, (3) financial burden, (4) treatment burden, and (5) life factors or social determinants of health. For example, participants mentioned other social determinants of health that affected their experience such as long travel times to receive care, short office hours for providers, lack of consistent transportation, lack of quality food, housing, location, and so on, and severe side effects from medications (eg, chemo, mental health medications) being part of unmet needs (Table 2).
TABLE 2.
Patient and Caregiver Inputs Related to Unmet Medical Needs From Qualitative Workshop
| Topic | Example quote |
|---|---|
| Defining unmet medical needs | “I define it as needs that go beyond physical. These unmet needs are clear medical communication and information, transportation, access to healthy affordable food, clean safe housing and mental therapy” Presurvey participant answer “Unmet need refers to situations where patients are unable to receive quality care when needed. It also refers to the often not measured or recognized side impacts of an illness possibly related to availability, affordability, accessibility, and acceptability of services.” Presurvey participant answer “And unmet need to me is a gap between what people actually need and what they are actually receiving or experiencing, whether that’s from a health care perspective education, access to different types of resources or even daily support they need…” Participant comment from workshop |
| Highest unmet medical needs | “Late diagnosis of my chronic disease. Lack of compassionate communication when explaining my chronic disease. No referral to mental health counselor. I had depression/sadness on how I found out about my disease state and no one gave me useful coping tips on how to manage my chronic illness. Lack of health literacy or preventative measures prior to my diagnosis. Too many micro- and macro-aggressions.” Presurvey participant answer “Parkinson’s medication have notorious side effects, yet we are provided almost no information on them or avenue for timely medication management.” Participant comment from workshop “There was a time when I had to purchase private insurance and only qualified to get high-risk insurance at $1,000 monthly premium! My meds cost more than $700 monthly. It broke me after 2 years!” Participant comment from workshop “I know what proper coordination of care looks like, what, that could be, and we just simply don’t have it. And we’re almost made to feel like shamed to even have that expectation, because we should be able to be our own advocates…” Participant comment from workshop |
| Impact on others | “I am not able to spend nearly enough time with my family and friends as I used to because of my caregiving responsibilities. I often find myself torn and struggling to balance all aspects of my life while caring for someone with a terminal disease. I’m not sure what others would say about the challenges they are living with as my life has become much more narrowed since my husband’s diagnosis.” Presurvey participant answer “I was a mom of young children 5 and 7, and I had to travel across the country for brain surgeries for over a month. And I look at how that impacted them. You know that suddenly Mom wasn’t available for a month, and I came home, and I couldn’t read, and I couldn’t talk for a while.” Participant comment from workshop “There are a lot of OTHER things that add onto. Not just the actual disease itself, but all the other. We travel 2 hours to Cleveland Clinic—so we use PTO or sick time, then there is the drive; if I have a test my husband may also have to take the day off as well. Then we also need to make sure someone is able to take care of our kids while we are gone.” Participant comment from workshop “My husband’s diagnosis has been devastating for my life. My world has closed in such that I spend most of my time that isn’t spent working my job, with my husband either directly taking care of him or managing all things he used to handle. His family has largely avoided getting involved and his friends have disappeared. If I were not in his life, he would be in full-time care in a nursing home. At this point, this is too devastating for me to consider, but at some point I will have to place him.” Participant comment from workshop |
When asked in the presurvey on a scale of 1 to 10, participants, on average, rated the impact of their UMN during their worst period as 9 (1 being less severe and 10 being most severe). The average response was 7.3 (SD=2.2) when asked how UMN impact their life today. The top challenges patients experienced when their disease was at its worst included inaccessibility to a specialist/referral wait times, information/education for the patient and family from the provider, side effects/knowledge about side effects, financial hardship (because of medication costs, insurance, or other), mental health support, lack of quality care/compassion in the medical system, and lack of a community or support system. Regarding challenges extending beyond patients themselves, the most frequent responses were inability to work/impact on job performance, low family finances because of medical costs, isolation/inability to leave home often, time burden/general burden of navigating the system while sick, and parents not being fully present with their children. Some participants were both caregivers and patients and thus experienced challenges due to their dual roles.
Other answers during the presurvey or qualitative listening workshop are documented in Table 2.
RANKING EXERCISE
In the quantitative exercise (relative weight; frequency of responses above median importance), patients and caregivers ranked health system functioning (25.6%; 76%) as the most important domain when considering UMN, followed by economic burden (21.7%; 53%), impacts on QoL (18.4%; 47%), availability of other treatments (14.3%; 41%), traditional clinical effectiveness (11.2%; 24%), and attributes of treatment administration (8.7%; 76%) (Table 3). Traditional clinical effectiveness was ranked fifth in relative importance.
TABLE 3.
Patient/Caregiver Ranking of Unmet Medical Need Domains
| Rank | Value domain | Elements | Normalized weight |
|---|---|---|---|
| 1 | Health system functioning | Proximity/geographic access to care; appropriateness of care; care process factors; care transitions; health system performance; compassionate care; explanation of treatment benefits and risks | 25.6% (59% of responses above the median relative difference) |
| 2 | Economic burden on the patient, caregiver, and family | Impact on income; affordability to patient; financial burden of disease (medical and nonmedical costs) | 21.7% (53% of responses above the median relative difference) |
| 3 | Impacts on quality of life | Physical health; psychological/emotional health; cognitive functioning; social relationships; activities of daily living; aesthetic/cosmetic; independence; reproduction; career; education; caregiver quality of life; family quality of life | 18.4% (47% of responses above the median relative difference) |
| 4 | Availability of other treatments | No treatment available; lack of ideal (eg, curative) treatment; number of treatments available | 14.3% (41% of responses above the median relative difference) |
| 5 | Traditional clinical effectiveness | Safety/adverse events; time to event; impact on key clinical/biological parameters; symptoms; durability of treatment effect | 11.2% (24% of responses above the median relative difference) |
| 6 | Attributes of treatment administration | Overall complexity of treatment; convenience of administration; length of treatment regimen; medication frequency; route of administration; needle/tablet size; polypharmacy need; medicine storage requirements; drug-drug interactions; uncertainty of treatment effectiveness and safety; interaction with religion/culture; patient subjective experience of treatment | 8.7% (76% of responses above the median relative difference) |
Discussion
Our findings support both the depth and breadth of UMN faced by patients and unpaid family caregivers. The UMN experienced by and important to patients are not limited to traditional clinical effectiveness measures but also include additional patient-centered components. Patients and caregivers reported difficulty in accessing appropriate and quality care and/or treatments, challenges in accessing specialists and providers knowledgeable about their condition/needs, limited access to mental health support, financial hardship, and inability to work and socialize. Additionally, our ranking exercise revealed the relative importance of key UMN domains perceived by patients and caregivers, with health care system functioning, economic burden, and impacts on QoL as the top priorities, followed by more traditional measures.
The finding that patients and caregivers ranked health care system functioning as the most important revealed barriers in navigating the health care system and challenges in accessing appropriate and high-quality care. These structural barriers can substantially shape day-to-day experience, affect QoL, and even impact health outcomes. For this particular sample, it can be hypothesized that current therapies were perceived as generally meeting the core clinical needs, thereby shifting attention to deficiencies in system-level support. In such contexts, some of these UMN may not be able to be addressed by therapeutic innovation alone but instead require system intervention to resolve. At the same time, continued pharmaceutical innovation remains critical to help mitigate barriers related to health care system functioning and others. For example, the transition from intravenous to subcutaneous administration can reduce or eliminate the need to travel to infusion centers.
Defining UMN and rigorously measuring all its aspects is pivotal to steering health care innovation toward the area of greatest need and value.5,17–19 When assessing UMN, understanding patients’ experiences is fundamental to informing industry investment, regulatory approval, and reimbursement decisions.1 However, existing UMN frameworks used by regulators to inform incentive programs and accelerated pathways are often operationalized narrowly around clinical effectiveness and traditional measures.1,6–8 For example, the FDA’s guidance defines the UMN as a “condition whose treatment or diagnosis is not addressed adequately by available therapy.”8 Additionally, under Medicare’s Drug Price Negotiation Program, CMS refers to the FDA’s guidance to define and assess UMN, without incorporating broader elements reflecting patient, caregiver, and societal perspectives. Similar limitations are evident in formulary design and reimbursement decision-making, where a limited focus on conventional clinical endpoints may inadequately capture the true UMN experienced by patients and consequently introduce additional barriers to treatment access.10
Our findings are consistent with previous work emphasizing the importance of incorporating patient and caregiver perspectives. Previous studies have proposed multidimensional definitions of UMN.1,2,7,11,20 For example, DiStefano et al conducted a targeted literature review and identified 40 distinct elements, grouped into 7 UMN domains from perspectives of patients and society.2 Zhang et al proposed a principle-based framework in understanding UMN, including concerns for need, inequity in access and outcomes, and aggregate well-being. Additionally, recent work has also highlighted the need for standardized patient-centered approaches to assessing UMN.11 Marsh et al proposed using patient preference data to generate a quantitative, disease-agnostic measure of UMN (ie, length-of-life equivalent).1 However, additional work is needed to clarify the relative importance of different UMN domains to support real-world policy applications. Our study complements these efforts by advancing actionable prioritization of patient-centered UMN domains.
Thus, we encourage health care decision-makers to broaden existing approaches for identifying and assessing UMN. Our work provides preliminary evidence to inform future UMN framework development, while recognizing the practical challenges of incorporating a large number of UMN components. Future research should build on this foundation by advancing more specific and measurable approaches to assessing patient-centered UMN. The UMN priorities may differ across patients by disease severity, socioeconomic status, insurance type, and other clinical and demographic characteristics. Thus, future research is needed to better capture these differences and their implications. More effort is needed to better understand how UMN priorities differ across other stakeholders, which is critical and might differ from our findings. Having a holistic perspective is crucial to inform more transparent investment decisions, health policy conversations, and formulary decision-making, with the goal of improving patient access to innovations that address these unmet needs.
LIMITATIONS
This study has several limitations. First, the generalizability of our findings may be limited. Although we captured perspectives across multiple disease areas, the sample was overrepresented by individuals with autoimmune and rare diseases. Our sample was also geographically concentrated in the Northeastern and Southern United States. Given the limited distribution across therapeutic areas and the small sample size, we acknowledge that the findings may vary across different conditions not captured in our study. Owing to the small sample size, we did not conduct a subgroup analysis by caregivers and patients. In addition, participants were recruited primarily through patient advocacy organizations; thus, our findings may not fully reflect the experiences of patients who did not have the opportunity to participate, including those from lower socioeconomic backgrounds or with limited access to research engagement. Disease characteristics were self-reported and were not independently verified through medical records, introducing the potential for misclassification or reporting bias. Furthermore, we acknowledge the exploratory nature of the ranking exercise, which was intended to generate preliminary insights into the relative importance of UMN domains. The ranking exercise relied on a set of predefined UMN domains informed by published literature. Although this approach ensured feasibility and comparability, it may not have captured additional UMN concepts important to patients and caregivers that are underrepresented in the existing literature, resulting in potential anchoring bias and influencing the prioritization of UMN domains. Although the ranking exercise captured the relative importance of UMN domains, we did not estimate quantitative weights reflecting the magnitude of trade-offs between them. Consequently, the findings alone cannot be used to construct a weighted UMN framework or directly inform quantitative decision-making.
Conclusions
Our study suggests that patients and unpaid caregivers experience substantial UMN that extend beyond clinical aspects. Although clinical outcomes remain important, challenges related to health care system functioning, economic burden, and impacts on QoL are emphasized. The ranking exercise identified health care system functioning, economic burden, and impacts on QoL as the top-priority UMN domains, followed by more traditional measures anchored in clinical effectiveness. Although our findings are exploratory, they provide valuable evidence to inform the continued refinement of a UMN framework that integrates a patient-centered perspective. Decision-makers should broaden existing UMN frameworks to capture the full spectrum of patient-centered needs, supporting transparent, shared decision-making across investment, which may help stakeholders more explicitly and consistently consider patient-centered UMN domains in health care decision-making related to research investment, regulatory evaluation, reimbursement, and policy. Future research is needed to further refine and validate UMN measurement approaches, as well as to integrate perspectives from key stakeholders.
Disclosures
Hanke Zheng and Jonathan D. Campbell are employees of the National Pharmaceutical Council. Silke C. Schoch and Omar A. Escontrías are employees of the National Health Council. R. Brett McQueen, Michael J. DiStefano, and Antal Zemplenyi are employees of the University of Colorado. This study was funded by the National Pharmaceutical Council. Researchers from the National Pharmaceutical Council, in collaboration with other investigators, participated in the study design and execution and the decision to publish.
Acknowledgments
The authors of this article acknowledge the National Health Council’s membership and other stakeholders for their contributions and promoting this study among their patient and caregiver constituents.
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