Drug-dispensing limits within Medicaid during the COVID-19 pandemic.

SUMMARY The 1-month drug-dispensing limit is a common drug utilization tool used by state Medicaid agencies to control spending. Since the beginning of the COVID-19 pandemic, many states relaxed the 1-month dispensing limit restriction in order to align with social distancing recommendations. Yet, some states have not relaxed this limit and have differed substantially regarding the policies that have been implemented. Among states that relaxed the 1-month supply limit, determining which chronic disease drugs qualified for this extension can be challenging for patients and clinicians. As more commercial and Medicare insurance beneficiaries are offered 90-day drug supplies, the 30-day drug supply limit with Medicaid has become a health equity issue, since many individuals insured by Medicaid have already experienced a disproportionate impact from and remain at high risk for severe COVID-19 disease. Thus, we propose policy solutions to ensure that Medicaid beneficiaries have safe and uninterrupted access to chronic disease medications during and beyond the COVID-19 pandemic. DISCLOSURES: No funding was received for this work. Alpern has received funding from Arnold Ventures for research related to the use and spending of off-patent drugs, unrelated to this work, and is a member of the Pharmacy and Therapeutics Committee at Regions Hospital, St. Paul, MN. DeSilva has received CDC support for work on Vaccine Safety Datalink, VISION network, and Center of Excellence for Newcomer Health, unrelated to this work. Chomilo is Medicaid Medical Director for the State of Minnesota's Department of Human Services.

Improving clinical documentation of evaluation and management care and patient acuity improves reimbursement as well as quality metrics.

OBJECTIVE: Accurate documentation of patient care and acuity is essential to determine appropriate reimbursement as well as accuracy of key publicly reported quality metrics. We sought to investigate the impact of standardized note templates by inpatient advanced practice providers (APPs) on evaluation and management (E/M) charge capture, including outside of the global surgical package (GSP), and quality metrics including case mix index (CMI) and mortality index (MI). We hypothesized this clinical documentation initiative as well as improved coding of E/M services would result in increased reimbursement and quality metrics. METHODS: A documentation and coding initiative on the heart and vascular service line was initiated in 2016 with focus on improving inpatient E/M capture by APPs outside the GSP. Comprehensive training sessions and standardized documentation templates were created and implemented in the electronic medical record. Subsequent hospital care E/M (current procedural terminology codes 99231, 99232, 99233) from the years 2015 to 2017 were audited and analyzed for charge capture rates, collections, work relative value units (wRVUs), and billing complexity. Data were compared over time by standardizing CMS values and reimbursement rates. In addition, overall CMI and MI were calculated each year. RESULTS: One year following the documentation initiative, E/M charges on the vascular surgery service line increased by 78.5% with a corresponding increase in APP charges from 0.4% of billable E/M services to 70.4% when compared with pre-initiative data. The charge capture of E/M services among all inpatients rose from 21.4% to 37.9%. Additionally, reimbursement from CMS increased by 65% as total work relative value units generated from E/M services rose by 78.4% (797 to 1422). The MI decreased over the study period by 25.4%. Additionally, there was a corresponding 5.6% increase in the cohort CMI. Distribution of E/M encounter charges did not vary significantly. Meanwhile, the prevalence of 14 clinical comorbidities in our cohort as well as length of stay (P = .88) remained non-statistically different throughout the study period. CONCLUSIONS: Accurate clinical documentation of E/M care and ultimately inpatient acuity is critical in determining quality metrics that serve as important measures of overall hospital quality for CMS value-based payments and rankings. A system-based documentation initiative and expanded role of inpatient APPs on vascular surgery teams significantly improved charge capture and reimbursement outside the GSP as well as CMI and MI in a consistently complex patient population.

Association of Supporting Trial Evidence and Reimbursement for Off-Label Use of Cancer Drugs.

Importance: In many health systems, access to off-label drug use is controlled through reimbursement restrictions by health insurers, especially for expensive cancer drugs. Objective: To determine whether evidence from randomized clinical trials is associated with reimbursement decisions for requested off-label use of anticancer drugs in the Swiss health system. Design, Setting, and Participants: This cross-sectional study used reimbursement requests from routinely collected health records of 5809 patients with drug treatment for cancer between January 2015 and July 2018 in 3 major cancer centers, covering cancer care of approximately 5% of the Swiss population, to identify off-label drug use. For each off-label use indication with 3 or more requests, randomized clinical trial evidence on treatment benefits was systematically identified for overall survival (OS) or progression-free survival (PFS). Data were analyzed from August 2018 to December 2020. Exposures: Available randomized clinical trial evidence on benefits for OS or PFS for requested off-label use indications. Main Outcomes and Measures: The main outcome was the association between evidence for treatment benefit (expressed as improved OS or PFS) and reimbursement in multivariable regression models. Results: Among 3046 patients with cancer, 695 off-label use reimbursement requests in 303 different indications were made for 598 patients (median [interquartile range] age, 64 [53-73] years; 420 [60%] men). Off-label use was intended as first-line treatment in 311 requests (45%). Reimbursement was accepted in 446 requests (64%). For 71 indications, including 431 requests for 376 patients, there were 3 or more requests. Of these, 246 requests (57%) had no supporting evidence for OS or PFS benefit. Reimbursement was granted in 162 of 246 requests without supporting evidence (66%). Of 117 requests supported by OS benefit, 79 (67%) were reimbursed, and of 68 requests supported by PFS benefit alone, 54 (79%) were reimbursed. Evidence of OS benefit from randomized clinical trials was not associated with a higher chance of reimbursement (odds ratio, 0.76, 95% CI, 0.45-1.27). Conclusions and Relevance: These findings suggest that in a health care system enabling access to off-label use, it was frequently intended as a first-line treatment in cancer care. Availability of randomized clinical trial evidence showing survival benefit was not associated with reimbursement decisions for off-label anticancer drug treatment in Switzerland. A transparent process with criteria considering clinical evidence is needed for evidence-based reimbursement decisions to ensure fair access to cancer treatments.

Improving Payment for Collaborative Mental Health Care in Primary Care.

BACKGROUND: There is strong evidence supporting implementation of the Collaborative Care Model within primary care. Fee-for-service payment codes, published by Current Procedural Terminology in 2018, have made collaborative care separately reimbursable for the first time. These codes (ie, 99492-99494) reimburse for time spent per month by any member of the care team engaged in Collaborative Care, including behavioral care managers, primary care providers, and consulting psychiatrists. Time-based billing for these codes presents challenges for providers delivering Collaborative Care services. OBJECTIVES: Based on experience from multiple health care organizations, we reflect on these challenges and provide suggestions for implementation and future refinement of the codes. CONCLUSIONS: Further refinements to the codes are encouraged, including moving from a calendar month to a 30-day reimbursement cycle. In addition, we recommend payers adopt the new code proposed by the Centers for Medicare and Medicaid Services to account for smaller increments of time.

What Is the Quality of Surgical Care for Patients with Hip Fractures at Critical Access Hospitals?

BACKGROUND: Critical access hospitals (CAHs) play an important role in providing access to care for many patients in rural communities. Prior studies have shown that these facilities are able to provide timely and quality care for patients who undergo various elective and emergency general surgical procedures. However, little is known about the quality and reimbursement of surgical care for patients undergoing surgery for hip fractures at CAHs compared with non-CAH facilities. QUESTIONS/PURPOSES: Are there any differences in 90-day complications, readmissions, mortality, and Medicare payments between patients undergoing surgery for hip fractures at CAHs and those undergoing surgery at non-CAHs? METHODS: The 2005 to 2014 Medicare 100% Standard Analytical Files were queried using ICD-9 procedure codes to identify Medicare-eligible beneficiaries undergoing open reduction and internal fixation (79.15, 79.35, and 78.55), hemiarthroplasty (81.52), and THA (81.51) for isolated closed hip fractures. This database was selected because the claims capture inpatient diagnoses, procedures, charged amounts and paid claims, as well as hospital-level information of the care, of Medicare patients across the nation. Patients with concurrent fixation of an upper extremity, lower extremity, and/or polytrauma were excluded from the study to ensure an isolated cohort of hip fractures was captured. The study cohort was divided into two groups based on where the surgery took place: CAHs and non-CAHs. A 1:1 propensity score match, adjusting for baseline demographics (age, gender, Census Bureau-designated region, and Elixhauser comorbidity index), clinical characteristics (fixation type and time to surgery), and hospital characteristics (whether the hospital was located in a rural ZIP code, the average annual procedure volume of the operating facility, hospital bed size, hospital ownership and teaching status), was used to control for the presence of baseline differences in patients presenting at CAHs and those presenting at non-CAHs. A total of 1,467,482 patients with hip fractures were included, 29,058 of whom underwent surgery in a CAH. After propensity score matching, each cohort (CAH and non-CAH) contained 29,058 patients. Multivariate logistic regression analyses were used to assess for differences in 90-day complications, readmissions, and mortality between the two matched cohorts. As funding policies of CAHs are regulated by Medicare, an evaluation of costs-of-care (by using Medicare payments as a proxy) was conducted. Generalized linear regression modeling was used to assess the 90-day Medicare payments among patients undergoing surgery in a CAH, while controlling for differences in baseline demographics and clinical characteristics. RESULTS: Patients undergoing surgery for hip fractures were less likely to experience many serious complications at a critical access hospital (CAH) than at a non-CAH. In particular, after controlling for patient demographics, hospital-level factors and procedural characteristics, patients treated at a CAH were less likely to experience: myocardial infarction (3% (916 of 29,058) versus 4% (1126 of 29,058); OR 0.80 [95% CI 0.74 to 0.88]; p < 0.001), sepsis (3% (765 of 29,058) versus 4% (1084 of 29,058); OR 0.69 [95% CI 0.63 to 0.78]; p < 0.001), acute renal failure (6% (1605 of 29,058) versus 8% (2353 of 29,058); OR 0.65 [95% CI 0.61 to 0.69]; p < 0.001), and Clostridium difficile infections (1% (367 of 29,058) versus 2% (473 of 29,058); OR 0.77 [95% CI 0.67 to 0.88]; p < 0.001) than undergoing surgery in a non-CAH. CAHs also had lower rates of all-cause 90-day readmissions (18% (5133 of 29,058) versus 20% (5931 of 29,058); OR 0.83 [95% CI 0.79 to 0.86]; p < 0.001) and 90-day mortality (4% (1273 of 29,058) versus 5% (1437 of 29,058); OR 0.88 [95% CI 0.82 to 0.95]; p = 0.001) than non-CAHs. Further, CAHs also had risk-adjusted lower 90-day Medicare payments than non-CAHs (USD 800, standard error 89; p < 0.001). CONCLUSION: Patients who received hip fracture surgical care at CAHs had a lower risk of major medical and surgical complications than those who had surgery at non-CAHs, even though Medicare reimbursements were lower as well. Although there may be some degree of patient selection at CAHs, these facilities appear to provide high-value care to rural communities. These findings provide evidence for policymakers evaluating the impact of the CAH program and allocating funding resources, as well as for community members seeking emergent care at local CAH facilities. LEVEL OF EVIDENCE: Level III, therapeutic study.

Telemedicine platforms and their use in the coronavirus disease-19 era to deliver comprehensive vascular care.

Implementation of telemedicine for patient encounters optimizes personal safety and allows for continuity of patient care. Embracing telehealth reduces the use of personal protective equipment and other resources consumed during in-person visits. The use of telehealth has increased to historic levels in response to the coronavirus disease 2019 (COVID-19) pandemic. Telehealth may be a key modality to fight against COVID-19, allowing us to take care of patients, conserve personal protective equipment, and protect health care workers all while minimizing the risk of viral spread. We must not neglect vascular health issues while the coronavirus pandemic continues to flood many hospitals and keep people confined to their homes. Patients are not immune to diseases and illnesses such as stroke, critical limb ischemia, and deep vein thrombosis while being confined to their homes and afraid to visit hospitals. Emerging from the COVID-19 crisis, incorporating telemedicine into routine medical care is transformative. By leveraging digital technology, the authors discuss their experience with the implementation, workflow, coding, and reimbursement issues of telehealth during the COVID-19 era.

Telehealth Beyond COVID-19.

Because of the COVID-19 pandemic, many mental health care services have been shifted from face-to-face to virtual interactions. Several health policy changes have influenced telehealth uptake during this time, including changes in technology, Internet connectivity, prescriptions, and reimbursement for services. These changes have been implemented for the duration of the pandemic, and it is unclear if all, some, or none of these new or amended policies will be retained after the pandemic has ended. Accordingly, in the wake of changing policies, mental health care providers will need to make decisions about the future of their telehealth programs. This article briefly reviews telehealth policy changes due to the COVID-19 pandemic and highlights what providers should consider for future delivery and implementation of their telehealth programs.

Value assessment of antimicrobials and the implications for development, access, and funding of effective treatments: Australian stakeholder perspective.

BACKGROUND: The frameworks used by Health Technology Assessment (HTA) agencies for value assessment of medicines aim to optimize healthcare resource allocation. However, they may not be effective at capturing the value of antimicrobial drugs. OBJECTIVES: To analyze stakeholder perceptions regarding how antimicrobials are assessed for value for reimbursement purposes and how the Australian HTA framework accommodates the unique attributes of antimicrobials in cost-effectiveness evaluation. METHODS: Eighteen individuals representing the pharmaceutical industry or policy-makers were interviewed. Interviews were transcribed verbatim, coded, and thematically analyzed. RESULTS: Key emergent themes were that reimbursement decision-making should consider the antibiotic spectrum when assessing value, risk of shortages, the impact of procurement processes on low-priced comparators, and the need for methodological transparency when antimicrobials are incorporated into the economic evaluation of other treatments. CONCLUSIONS: Participants agreed that the current HTA framework for antimicrobial value assessment is inadequate to properly inform funding decisions, as the contemporary definition of cost-effectiveness fails to explicitly incorporate the risk of future resistance. Policy-makers were uncertain about how to incorporate future resistance into economic evaluations without a systematic method to capture costs avoided due to good stewardship. Lacking financial reward for the benefits of narrower-spectrum antimicrobials, companies will likely focus on developing broad-spectrum agents with wider potential use. The perceived risks of shortages have influenced the funding of generic antimicrobials in Australia, with policy-makers suggesting a willingness to pay more for assured supply. Although antibiotics often underpin the effectiveness of other medicines, it is unclear how this is incorporated into economic models.

Leveraging Health Department Capacities, Partnerships, and Health Insurance for Infectious Disease Response in Massachusetts, 2014-2018.

Policies facilitating integration of public health programs can improve the public health response, but the literature on approaches to integration across multiple system levels is limited. We describe the efforts of the Massachusetts Department of Public Health to integrate its HIV, viral hepatitis, sexually transmitted infection (STI), and tuberculosis response through policies that mandated contracted organizations to submit specimens for testing to the Massachusetts State Public Health Laboratory; co-test blood specimens for HIV, hepatitis C virus (HCV), and syphilis; integrate HIV, viral hepatitis, and STI disease surveillance and case management in a single data system; and implement an integrated infectious disease drug assistance program. From 2014 through 2018, the number of tests performed by the Massachusetts State Public Health Laboratory increased from 16 321 to 33 674 for HIV, from 11 054 to 33 670 for HCV, and from 19 169 to 30 830 for syphilis. Service contracts enabled rapid response to outbreaks of HIV, hepatitis A, and hepatitis B. Key challenges included lack of a billing infrastructure at the Massachusetts State Public Health Laboratory; the need to complete negotiations with insurers and to establish a retained revenue account to receive health insurance reimbursements for testing services; and time to train testing providers in phlebotomy for required testing. Investing in laboratory infrastructure; creating billing mechanisms to maximize health insurance reimbursement; proactively engaging providers, community members, and other stakeholders; and building capacity to transform practices are needed. Using multilevel policy approaches to integrate the public health response to HIV, STI, viral hepatitis, and tuberculosis is feasible and adaptable to other public health programs.

Beyond the COVID Pandemic, Telemedicine, and Health Care.

This article reviews the current experience and the flaws encountered in the rush to deploy telemedicine as a substitute for in-person care in response to the raging coronavirus (COVID-19) pandemic; the preceding fault lines in the U.S. health care system that exacerbated the problem; and the importance of emerging from this calamity with a clear vision for necessary health care reforms. It starts with the premise that the precursors of catastrophes of this magnitude provide a valid basis for planning corrective measures, improved preparedness, and ultimately serious health reform. Such reform should include standardized protocols for proper deployment of telemedicine to triage patients to the appropriate level and source of care at the point of need, proper use of relevant technological innovations to deliver precision medicine, and the development of regional networks to coordinate and improve access to care while streamlining the care process. The other essential element is a universal payment system that puts the United States at par with the rest of the industrialized countries, regardless of variation among them. The ultimate goal is creating an efficient, effective, accessible, and equitable system of care. Although timing is uncertain, the pandemic will be brought under control. The path to a better future after the pandemic offers some consolation for the massive loss of life and treasure during this pandemic.

Barriers in accessing medical cannabis for children with drug-resistant epilepsy in Canada: A qualitative study.

INTRODUCTION: The use of medical cannabis to treat drug-resistant epilepsy in children is increasing; however, there has been limited study of the experiences of parents with the current system of accessing medical cannabis for their children. METHODS: In this qualitative study, we used a patient-centered access to care framework to explore the barriers faced by parents of children with drug-resistant epilepsy when trying to access medical cannabis in Canada. We conducted semistructured interviews with 19 parents to elicit their experiences with medical cannabis. We analyzed the data according to five dimensions of access, namely approachability, acceptability, availability, affordability, and appropriateness. RESULTS: Parents sought medical cannabis as a treatment because of a perceived unmet need stemming from the failure of antiepileptic drugs to control their children's seizures. Medical cannabis was viewed as an acceptable treatment, especially compared with adding additional antiepileptic drugs. After learning about medical cannabis from the media, friends and family, or other parents, participants sought authorization for medical use. However, most encountered resistance from their child's neurologist to discuss and/or authorize medical cannabis, and many parents experienced difficulty in obtaining authorization from a member of the child's existing care team, leading them to seek authorization from a cannabis clinic. Participants described spending up to $2000 per month on medical cannabis, and most were frustrated that it was not eligible for reimbursement through public or private insurance programs. CONCLUSIONS: Parents pursue medical cannabis as a treatment for their children's drug-resistant epilepsy because of a perceived unmet need. However, parents encounter barriers in accessing medical cannabis in Canada, and strategies are needed to ensure that children using medical cannabis receive proper care from healthcare professionals with training in epilepsy care, antiepileptic drugs, and medical cannabis.

Implications of the Revisions and Revaluation of Office/Outpatient Evaluation and Management Codes for Neuroradiology Reimbursement.

In the 2020 Final Rule, the Center for Medicare & Medicaid Services adopted a new coding structure and accepted the substantial increase in valuation for office/outpatient Evaluation and Management codes set to begin in 2021. Given budget neutrality requirements, the projected increase in reimbursement will require a reduction in the conversion factor to offset such increases. The aim is to inform neuroradiologists the impact of these proposed changes on reimbursement and the profession.

The BAPRAS screening tool for reimbursement in a postbariatric population.

INTRODUCTION: Reimbursement of body-contouring surgery (BCS) is a worldwide problem: there is no objective instrument to decide which postbariatric patients should qualify for reimbursement. The British Association of Plastic, Reconstructive and Aesthetic Surgeons (BAPRAS) has developed a screening tool for this purpose. In this study, we used a modified version of this screening tool in a postbariatric population and describe which patients would qualify for reimbursement using this tool. METHODS: In this cross-sectional study postbariatric patients were asked to fill in an online questionnaire based on the BAPRAS screening tool with questions regarding complaints of overhanging skin and medical history. Weight loss data were extracted from a prospective database. The BODY-Q was added to assess patient-reported outcomes. RESULTS: Patients who wanted to undergo BCS (n = 90) had higher screening tool scores and lower BODY-Q scores compared to patients who did not want BCS (n = 24). In total, 25 patients (26%) qualified for reimbursement, these patients had higher weight loss (33.5% versus 29.2%, p = 0.008), lower BMI (27.3 kg/m2 versus 30.4 kg/m2, p = 0.014) and more medical (4.0 versus 2.0, p = 0.004) and psychological complaints (88% versus 61%, p = 0.009). There was a significant, negative correlation between the screening tool scores and almost all BODY-Q scales. CONCLUSIONS: Patients with a desire for BCS have more complaints of excess skin, which negatively impacts their well-being. With the modified BAPRAS screening tool, patients with the best weight (loss) and most medical and psychological complaints of excess skin qualified for referral and reimbursement of BCS.

Ambulatory care pharmacy practice in China: status and future efforts.

In recent years, as the era of deepening healthcare reform in China progresses, there is a gradual development of ambulatory care pharmacy practice. This commentary reviews the current state of ambulatory care pharmacy practice in China and discusses future efforts to advance the practice. Areas of focus include practice standardization, transitions of care, and reimbursements of ambulatory care pharmacy practice.

Ethical Issues in Physician Billing Under Fee-For-Service Plans.

Medical ethics has become an important and recognized component of physician training. There is one area, however, in which medical students receive little guidance. There is practically no discussion of the financial aspects of medical practice. My objective in this paper is to initiate a discussion about the moral dimension of physician billing practices. I argue that physicians should expand their conception of professional responsibility in order to recognize that their moral obligations toward patients include a commitment to honest and forthright billing practices. I argue that physicians should aspire to a standard of clinical accuracy-not legal adequacy-in describing their activities. More generally, physicians should strive to promote an integrity-based professional culture, first and foremost by stigmatizing rather than celebrating creative billing practices, as well as condemning the misguided sense of solidarity that currently makes it taboo for physicians to criticize each other on this score.

New evidence of state variation in Medicaid payment policies for dual Medicare-Medicaid enrollees.

OBJECTIVE: To develop the first longitudinal database of state Medicaid policies for paying the cost sharing in Medicare Part B for services provided to dual Medicare-Medicaid enrollees ("duals") and an index summarizing the impact of these policies on payments for physician office services. DATA SOURCES: Medicaid policy data collected from electronic sources and inquiries with states. STUDY DESIGN: We constructed a national database of Medicaid payment policies for the period 2004-2018, consolidating information from online Medicaid policy documents, state laws, and policy data reported to us by state Medicaid programs. Using this database and state Medicaid fee schedules, we constructed a Medicaid payment index for duals. This index represented the proportion of the Medicare allowed amount that physicians would expect to be paid from Medicare and Medicaid for a subset of physician office services (evaluation and management services) based on annual state payment policies and Medicaid fee schedules. PRINCIPAL FINDINGS: In 2018, 42 states had policies to limit Medicaid payments of Medicare cost sharing when Medicaid's fee schedule was lower than Medicare's-an increase from 36 such states in 2004. In the preponderance of states with these policies, combined Medicare and Medicaid payments for evaluation and management services provided to duals averaged 78 percent of the Medicare allowed amount for these services, reflecting relatively low Medicaid fee schedules in these states. In 2013 and 2014, physicians who qualified for the Affordable Care Act's Medicaid "fee bump" were paid 100 percent of the Medicare allowed amount for these services. CONCLUSIONS: Medicaid programs vary across states and over time in their payments of cost sharing for physician office services provided to duals. Our database and index can facilitate monitoring of these policies and research on the consequences of policy changes for duals.

Principles of Financing the Medical Home for Children.

A well-implemented and adequately funded medical home not only is the best approach to optimize the health of the individual patient but also can function as an effective instrument for improving population health. Key financing elements to providing quality, effective, comprehensive care in the pediatric medical home include the following: (1) first dollar coverage without deductibles, copays, or other cost-sharing for necessary preventive care services as recommended by Bright Futures: Guidelines for Health Supervision of Infants, Children, and Adolescents; (2) adoption of a uniform definition of medical necessity across payers that embraces services that promote optimal growth and development and prevent, diagnose, and treat the full range of pediatric physical, mental, behavioral, and developmental conditions, in accord with evidence-based science or evidence-informed expert opinion; (3) payment models that promote appropriate use of pediatric primary care and pediatric specialty services and discourage inappropriate, inefficient, or excessive use of medical services; and (4) payment models that strengthen the patient- and family-physician relationship and do not impose additional administrative burdens that will only erode the effectiveness of the medical home. These goals can be met by designing payment models that provide adequate funding of the cost of medical encounters, care coordination, population health services, and quality improvement activities; provide incentives for quality and effectiveness of care; and ease administrative burdens.

Unmet Medical Need: An Introduction to Definitions and Stakeholder Perceptions.

BACKGROUND: Despite increasing informal and formal use of unmet medical need (UMN) in drug development, regulation, and assessment, there is no insight into its definitions in use. This study aims to provide insight into the current definitions in use and to provide a starting point for a multi-stakeholder discussion on alignment. METHODS: A scoping and a gray literature review were performed to locate definitions of UMN in literature and on stakeholder websites. These definitions were categorized and then discussed among the multi-stakeholder author group via semistructured group discussions and open session workshops with a broader stakeholder audience. Issues with the formation of a common definition and mechanisms for use were discussed. RESULTS: The reviews yielded 16 definitions. Differences were evident, but all included 1 or more of the following elements: (adequacy of) available treatments (16 of 16: 100%), disease severity or burden (6 of 16: 38%), and patient population size (1 of 16: 6%). The stakeholder discussions led to a suggestion for a definition including the first 2 items and, depending on context, population size. The discussions also showed that quantification of UMN is highly dependent on the scope and the value framework in which it is used based on different stakeholder preferences and responsibilities. CONCLUSION: We encourage stakeholders that want to promote alignment on the concept of UMN to prospectively discuss the scope in which they want to apply the concept, what elements they find important for consideration in each case, and how they would measure UMN within the broader regulatory or value framework applicable.