Considerations for US coverage and reimbursement: Which digital health products to evaluate? Findings from a modified Delphi study.

BACKGROUND: The rapid growth of digital health tools, including digital applications, wearables, sensors, diagnostics, digital therapeutics (DTx), and prescription DTx, offers new ways to treat patients and close gaps in care. Payers need transparent, credible, and efficient processes to differentiate products for potential reimbursement from the larger universe of digital health products. OBJECTIVE: To identify areas of agreement, disagreement, and rationale for payers to determine which digital health products should be evaluated for formulary consideration and to develop generalizable criteria for health care decision-makers developing policies and approaches for digital health products. METHODS: Experts from the Academy of Managed Care Pharmacy DTx Advisory Group Payer Evaluation subcommittee rated whether a pharmacy and therapeutics committee, health technology assessment group, or an innovation center within a health plan or pharmacy benefit manager should consider 14 hypothetical products for potential formulary coverage. Using a 4-step modified Delphi approach, experts rated whether it was appropriate for a payer to evaluate each product on a scale of 1 (strongly disagree) to 9 (strongly agree). Quantitative agreement was assessed using terciles of responses, medians, and the distribution of appropriateness scores. The corresponding discussions are summarized to identify generalizable criteria for payers to consider as they develop approaches to determine which digital health products to evaluate. RESULTS: Among the 14 hypothetical products, 4 achieved quantitative agreement that payers should evaluate the product. 5 products had quantitative disagreement, and the remaining were indeterminant. Payers were most likely to review a product if it (1) was reviewed by the US Food and Drug Administration, (2) required a prescription, (3) was intended to be paid for using premium dollars, (4) treated rather than diagnosed or monitored a clinical condition, (5) had a low clinical opportunity cost, and (6) could address population health metrics. CONCLUSIONS: The rapid availability of digital health and DTx options can be daunting for health care decision-makers when determining which products to evaluate. These generalizable criteria can help payers develop a more efficient process.

What Should Clinicians in Organizations Without Established MLP Programs Do When Their Patients Need Lawyers to Meet Their Health Needs?

Undocumented people in the United States face innumerable legal and structural barriers to health and health care services, including for kidney failure. Their experiences vary across states and regions due to wide variation in insurance coverage and unreliable access to health-promoting resources, including medical-legal partnerships. This commentary on a case canvasses key policy about structural and legal determinants of health for undocumented persons.

The Role of Individual Coverage Health Reimbursement Arrangements-The Liger of Health Insurance?

This JAMA Forum discusses aspects of individual coverage health reimbursement arrangements and their expanded use over the last few years.

Rural-urban disparities and socioeconomic determinants of caesarean delivery rates in Zimbabwe: Evidence from the 2019 National Multiple Indicator Cluster Survey.

Caesarean sections (CSs) have increased globally, with concerns being raised involving overutilisation and inequalities in access. In Zimbabwe, where healthcare access varies greatly, we aimed to analyse factors associated with ever having a CS using the 2019 National Multiple Indicator Cluster Survey. The weighted national CS rate was 10.3%, and CS happened more commonly among women in urban than rural areas (15.7% v. 7.4%; odds ratio (OR) 2.34; (95% confidence interval (CI)) 1.71 - 3.20; p=0.001). Percentages of those having a CS significantly increased with education: overall χ2 for a trend of p=0.001 and wealth quintile, and overall χ2 for a trend of p=0.001. Women with insurance coverage were more likely to have had a CS than those without: 26.7% v. 8.7%; OR 3.82; 95% CI 2.51 - 5.83; p=0.001. The same was the case for women with access to the internet: 15.4% v. 7.0%, OR 2.42; 95%CI 1.71 - 3.41; p=0.001). These findings show an association that could indicate this being overutilised by insured women in urban settings, rather than being accessible based on clinical needs. Further research should explore reasons for these disparities and inform interventions to ensure equitable access to optimum childbirth in Zimbabwe.

Factors affecting decisions of an HMO Drug Exemptions Committee on individual patient requests for coverage of non-formulary drugs.

BACKGROUND: In Israel, coverage of health needs is delivered by four health maintenance organizations (HMOs), which are budgeted by the government according to the recommendations of the National Drug Formulary (NDF) Committee. For medications not listed in the NDF, individuals may request to cover the costs by the HMO Exemptions Committee (DEC). The objectives of the current study, a first of its kind, are to document the DEC decision process, to identify its components and to determine the decisions' clinical outcome. METHODS: This retrospective cohort study included all members (≥ age 18) of the Maccabi Healthcare Service (MHS) who submitted a request to the DEC between June 2017 and December 2018. Collected data include patient demographics, clinical information and components of the decision process. Decision success (i.e., clinical outcome correlated with DEC decision) was determined by clinical outcome over at least one-year follow-up. RESULTS: A total of 335 requests were included. Strong evidence and rare disease were positively associated with approvals, while the availability of alternative treatments and costs were negatively associated. The majority of decisions (75%) met predicted clinical outcomes. Only estimated costs were found to be associated with decision success. CONCLUSIONS: Factors that reduce the potential costs of a requested drug are significantly associated with higher odds for drug approval, but only when the evidence supports potential benefit.

Does Insurance Provide Adequate Coverage for Physical Therapy Visits for Common Orthopedic Diagnoses? A Survey Study.

OBJECTIVES: This study aimed to analyze the association between physical therapists' recommended number of visits for a full recovery from common orthopedic injuries/surgeries and the extent of insurance coverage for these visits. METHODS: A prospective observational study was conducted with board-certified physical therapists. A qualitative questionnaire was used to gather physical therapists' demographics and the recommended number of physical therapy visits to achieve a full recovery after 11 common orthopedic diagnoses. Physical therapists also were asked to report whether they believe that insurance provides an adequate number of visits overall. In addition to the qualitative survey, insurance coverage details of major Alabama companies were obtained for comparison. Descriptive statistics of the participating therapists were analyzed for sex, age, degree/training, and years of experience. Kruskal-Wallis statistics were used to analyze variance between the aforementioned groupings when compared with the reported average number of sessions. RESULTS: The survey (N = 251) collected data on the average number of physical therapy sessions that are necessary for a complete recovery as recommended by physical therapists for 11 common orthopedic diagnoses. From this survey, the average number of necessary visits ranged from 11.3 visits (ankle sprains) to 37.3 visits (anterior cruciate ligament reconstruction), with the overall average number of visits being 23.8. Only 24% of physical therapists believed that insurance companies provided enough coverage. Insurance coverage varied but often required additional procedures to allocate the adequate number of visits for the studied orthopedic pathologies. CONCLUSIONS: The majority of practicing physical therapists in Alabama perceive insufficient insurance coverage for physical therapy visits for most orthopedic diagnoses. This study has implications for healthcare decision making and patient-centered rehabilitation goals. Physicians and physical therapists can use this information to optimize treatment decisions and rehabilitation goals. Patients will benefit from improved physical and economic well-being. This study has the potential to drive further research and influence national insurance policies to better serve patients' needs.

Most States Allow Medicaid Managed Care Plans Discretion To Restrict Substance Use Disorder Treatment Benefits.

Managed care plans, which contract with states to cover three-quarters of Medicaid enrollees, play a crucial role in addressing the drug epidemic in the United States. However, substance use disorder benefits vary across Medicaid managed care plans, and it is unclear what role states play in regulating their activities. To address this question, we surveyed thirty-three states and Washington, D.C., regarding their substance use disorder treatment coverage and utilization management requirements for Medicaid managed care plans in 2021. Most states mandated coverage of common forms of substance use disorder treatment and prohibited annual maximums and enrollee cost sharing in managed care. Fewer than one-third of states forbade managed care plans from imposing prior authorization for each treatment service. For most treatment medications, fewer than two-thirds of states prohibited prior authorization, drug testing, "fail first," or psychosocial therapy requirements in managed care. Our findings suggest that many states give managed care plans broad discretion to impose requirements on covered substance use disorder treatments, which may affect access to lifesaving care.

Overlap In Carriers And Networks In Medicaid And The Individual Marketplaces.

As people lose Medicaid because of the end of the COVID-19 public health emergency, many states will route former Medicaid managed care enrollees into Affordable Care Act Marketplace coverage with the same carrier. In 2021, 52.1 percent of Medicaid managed care enrollees were enrolled by a carrier that also had a plan on the Marketplace in the same county.

Medicare Eligibility and Changes in Coverage, Access to Care, and Health by Sexual Orientation and Gender Identity.

Importance: Medicare provides nearly universal insurance coverage at age 65 years. However, how Medicare eligibility affects disparities in health insurance coverage, access to care, and health status among individuals by sexual orientation and gender identity is poorly understood. Objective: To assess the association of Medicare eligibility with disparities in health insurance coverage, access to care, and self-reported health status among individuals by sexual orientation and by gender identity. Design, Setting, and Participants: This cross-sectional study used the age discontinuity for Medicare eligibility at age 65 years to isolate the association of Medicare with health insurance coverage, access to care, and self-reported health status, by their sexual orientation and by their gender identity. Data were collected from the Behavioral Risk Factor Surveillance System for respondents from 51 to 79 years old from 2014 to 2021. Data analysis was performed from September 2022 to April 2023. Exposures: Medicare eligibility at age 65 years. Main Outcomes and Measures: Proportions of respondents with health insurance coverage, usual source of care, cost barriers to care, influenza vaccination, and self-reported health status. Results: The study population included 927 952 individuals (mean [SD] age, 64.4 [7.7] years; 524 972 [56.6%] females and 402 670 [43.4%] males), of whom 28 077 (3.03%) identified as a sexual minority-lesbian, gay, bisexual, or another sexual minority identity (LGB+) and 3286 (0.35%) as transgender or gender diverse. Respondents who identified as heterosexual had greater improvements at age 65 years in insurance coverage (4.2 percentage points [pp]; 95% CI, 4.0-4.4 pp) than those who identified as LGB+ (3.6 pp; 95% CI, 2.3-4.8 pp), except when the analysis was limited to a subsample of married respondents. For access to care, improvements in usual source of care, cost barriers to care, and influenza vaccination were larger at age 65 years for heterosexual respondents compared with LGB+ respondents, although confidence intervals were overlapping and less precise for LGB+ individuals. For self-reported health status, the analyses found larger improvements at age 65 years for LGB+ respondents compared with heterosexual respondents. There was considerable heterogeneity by state in disparities by sexual orientation among individuals who were nearly eligible for Medicare (close to 65 years old), with the US South and Central states demonstrating the highest disparities. Among the top-10 highest-disparities states, Medicare eligibility was associated with greater increases in coverage (6.7 pp vs 5.0 pp) and access to a usual source of care (1.4 pp vs 0.6 pp) for LGB+ respondents compared with heterosexual respondents. Conclusions and Relevance: The findings of this cross-sectional study indicate that Medicare eligibility was not associated with consistently greater improvements in health insurance coverage and access to care among LGBTQI+ individuals compared with heterosexual and/or cisgender individuals. However, among sexual minority individuals, Medicare may be associated with closing gaps in self-reported health status, and among states with the highest disparities, it may improve health insurance coverage, access to care, and self-reported health status.

Effect of Medicaid expansion on cancer treatment and survival among Medicaid beneficiaries and the uninsured.

BACKGROUND: The Affordable Care Act expanded Medicaid coverage for people with low income in the United States. Expanded insurance coverage could promote more timely access to cancer treatment, which could improve overall survival (OS), yet the long-term effects of Medicaid expansion (ME) remain unknown. We evaluated whether ME was associated with improved timely treatment initiation (TTI) and 3-year OS among patients with breast, cervical, colon, and lung cancers who were affected by the policy. METHODS: Medicaid-insured or uninsured patients aged 40-64 with stage I-III breast, cervical, colon, or non-small cell lung cancer within the National Cancer Database (NCDB). A difference-in-differences (DID) approach was used to compare changes in TTI (within 60 days) and 3-year OS between patients in ME states versus nonexpansion (NE) states before (2010-2013) and after (2015-2018) ME. Adjusted DID estimates for TTI and 3-year OS were calculated using multivariable linear regression and Cox proportional hazards regression models, respectively. RESULTS: ME was associated with a relative increase in TTI within 60 days for breast (DID = 4.6; p < 0.001), cervical (DID = 5.0 p = 0.013), and colon (DID = 4.0, p = 0.008), but not lung cancer (p = 0.505). In Cox regression analysis, ME was associated with improved 3-year OS for breast (DID hazard ratio [HR] = 0.82, p = 0.009), cervical (DID-HR = 0.81, p = 0.048), and lung (DID-HR = 0.87, p = 0.003). Changes in 3-year OS for colon cancer were not statistically different between ME and NE states (DID-HR, 0.77; p = 0.075). CONCLUSIONS: Findings suggest that expanded insurance coverage can improve treatment and survival outcomes among low income and uninsured patients with cancer. As the debate surrounding ME continues nationwide, our findings serve as valuable insights to inform the development of policies aimed at fostering accessible and affordable healthcare for all.

Medicaid Coverage in Early Childhood for Children With Sickle Cell Disease.

This cohort study examines patterns of Medicaid coverage in the first 3 years of life among children with sickle cell disease across 5 states.

Disparities in satisfaction among insured and uninsured adult outpatient department service users in Southern Ethiopia, 2022: a comparative cross-sectional study.

BACKGROUND: Patient satisfaction is a critical measure of the quality of healthcare services provided by healthcare facilities. However, very few studies, particularly in Ethiopia, which includes the study area, have specifically examined these discrepancies among people who use outpatient care. In this study, satisfaction levels and associated factors were compared between insured and uninsured patients receiving outpatient services at public health institutions in Hadiya Zone, southern Ethiopia. METHODS: A facility-based comparative cross-sectional study design was employed on 630 patients using multistage and systematic random sampling. Data were collected using a pretested and structured interviewer-administered questionnaire. Results of the analysis were presented in text, tables, and graphs as appropriate. Multivariable logistic regression was used to predict associations between predictors and the outcome variable. Statistical significance was declared at p-value < 0.05. RESULTS: Overall, 344(55.48%) patients were satisfied with the service they received, of which 206(65.8%) out of 313 with a 95% CI [60.7-71.2%] were insured and 138(44.95%) out of 307 with a 95% CI [39.4-5.1%] were uninsured. Among insured patients, factors associated with higher satisfaction included having a family size less than five members [AOR = 3.3, 95% CI; 1.5, 7.4], perceived fair waiting time to be seen[AOR = 2.35, 95% CI; 1.02, 5.5], perceived short waiting time to be seen[AOR = 8.12, 95% CI; 1.6, 41.3], having all ordered laboratory tests available within the facility[AOR = 7.89, 95% CI; 3.5, 17.5], having some ordered laboratory tests within the facility[AOR = 2.97, 95% CI; 1.25, 7.01] having all prescribed medications available within the facility[AOR = 16.11, 95% CI; 6.25, 41.5], having some prescribed medications available within the facility[AOR = 13.11, 95% CI; 4.7, 36.4]. Among non-insured patients, factors associated with higher satisfaction included urban residency, a fair and short perceived time to be seen, having ordered laboratory tests within the facility, and having prescribed drugs within the facility. CONCLUSION: This study identified lower overall satisfaction, particularly among uninsured patients. Enrollment in the CBHI program significantly impacted satisfaction, with both groups reporting lower levels compared to enrollment periods. Access to essential services, wait times, and socio-demographic factors identified as associated factors with patient satisfaction regardless of insurance status.

Quantifying the insurance value for rare diseases: Duchenne muscular dystrophy.

OBJECTIVES: To quantify the magnitude of an ISPOR novel value element, insurance value, as applied to new treatments for a rare, severe disease with pediatric onset: Duchenne muscular dystrophy (DMD). STUDY DESIGN: Prospective survey of individuals planning to have children in the future. METHODS: A survey was administered to US adults (aged ≥ 21 years) planning to have a child in the future to elicit willingness to pay (WTP) for insurance coverage for a new hypothetical DMD treatment that improved mortality and morbidity relative to the current standard of care. To identify an indifference point between status quo insurance and insurance with additional cost that would cover the treatment if respondents had a child with DMD, a multiple random staircase design was used. Insurance value-the value individuals receive from a reduction in future health risks-was calculated as the difference between respondent's WTP and what a risk-neutral individual would pay. The risk-neutral value was the product of the (1) probability of having a child with DMD (decision weighted), (2) quality-adjusted life-years (QALYs) gained from the new treatment, and (3) WTP per QALY. RESULTS: Among 207 respondents, 80.2% (n = 166) were aged 25 to 44 years, and 59.9% (n = 124) were women. WTP for insurance coverage of the hypothetical treatment was $973 annually, whereas the decision-weighted risk-neutral value was $452 per year. Thus, insurance value constituted 53.5% ($520) of value for new DMD treatments. CONCLUSIONS: Individuals planning to have children in the future are willing to pay more for insurance coverage of novel DMD treatments than is assumed under risk-neutral, QALY-based frameworks.

Predeductible Coverage and Receipt of Telemental Health Visits.

This cohort study examines the utilization changes associated with the reintroduction of cost sharing for patients receiving telemental health services.

Insurance Type and Withdrawal of Life-Sustaining Therapy in Critically Injured Trauma Patients.

Importance: Withdrawal of life-sustaining therapy (WLST) decisions for critically injured trauma patients are complicated and multifactorial, with potential for patients' insurance status to affect decision-making. Objectives: To determine if patient insurance type (private insurance, Medicaid, and uninsured) is associated with time to WLST in critically injured adults cared for at US trauma centers. Design, Setting, and Participants: This retrospective registry-based cohort study included reported data from level I and level II trauma centers in the US that participated in the American College of Surgeons Trauma Quality Improvement Program (TQIP) registry. Participants included adult trauma patients who were injured between January 1, 2017, and December 31, 2020, and required an intensive care unit stay. Patients were excluded if they died on arrival or in the emergency department or had a preexisting do not resuscitate directive. Analyses were performed on December 12, 2023. Exposures: Insurance type (private insurance, Medicaid, uninsured). Main Outcomes and Measures: An adjusted time-to-event analysis for association between insurance status and time to WLST was performed, with analyses accounting for clustering by hospital. Results: This study included 307 731 patients, of whom 160 809 (52.3%) had private insurance, 88 233 (28.6%) had Medicaid, and 58 689 (19.1%) were uninsured. The mean (SD) age was 40.2 (14.1) years, 232 994 (75.7%) were male, 59 551 (19.4%) were African American or Black patients, and 201 012 (65.3%) were White patients. In total, 12 962 patients (4.2%) underwent WLST during their admission. Patients who are uninsured were significantly more likely to undergo earlier WLST compared with those with private insurance (HR, 1.54; 95% CI, 1.46-1.62) and Medicaid (HR, 1.47; 95% CI, 1.39-1.55). This finding was robust to sensitivity analysis excluding patients who died within 48 hours of presentation and after accounting for nonwithdrawal death as a competing risk. Conclusions and Relevance: In this cohort study of US adult trauma patients who were critically injured, patients who were uninsured underwent earlier WLST compared with those with private or Medicaid insurance. Based on our findings, patient's ability to pay was may be associated with a shift in decision-making for WLST, suggesting the influence of socioeconomics on patient outcomes.

Orthopaedic Nurse Navigators and Total Joint Arthroplasty Preoperative Optimization: Payer Status and Medication Management-Part Six of the Movement Is Life Special ONJ Series.

This article is the sixth in the Movement is Life series and focuses on insurance coverage and medication management in the perioperative period, 2 tangentially related variables that affect patient outcomes. Our aim is to use current practices and literature to develop recommendations for nurse navigators' execution of preoperative optimization protocols related to payer status and medication management. Discussions with nurse navigators and a literature search were used to gather information and develop recommendations specific to optimizing payer status and medication management. Nurse navigators connected patients to resources and provided education regarding financial concerns and medications, and findings from the literature discussed insurance status among TJA patients. Nurse navigators can contribute to payer status and medication management optimization by providing patient education and resource referrals. In addition, we recommend conducting repeated medication reconciliation and developing awareness of financial resources and perioperative medication management guidelines.

Influence of health insurance on withdrawal of life sustaining treatment for patients with isolated traumatic brain injury: a retrospective multi-center observational cohort study.

BACKGROUND: Healthcare inequities for patients with traumatic brain injury (TBI) represent a major priority area for trauma quality improvement. We hypothesized a relationship between health insurance status and timing of withdrawal of life sustaining treatment (WLST) for adults with severe TBI. METHODS: This multicenter retrospective observational cohort study utilized data collected between 2017 and 2020. We identified adult (age ≥ 16) patients with isolated severe TBI admitted participating Trauma Quality Improvement Program centers. We determined the relationship between insurance status (public, private, and uninsured) and the timing of WLST using a competing risk survival analysis framework adjusting for baseline, clinical, injury and trauma center characteristics. Multivariable cause-specific Cox regressions were used to compute adjusted hazard ratios (HR) reflecting timing of WLST, accounting for mortality events. We also quantified the between-center residual variability in WLST using the median odds ratio (MOR) and measured insurance status association with access to rehabilitation at discharge. RESULTS: We identified 42,111 adults with isolated severe TBI treated across 509 trauma centers across North America. There were 10,771 (25.6%) WLST events in the cohort and a higher unadjusted incidence of WLST events was evident in public insurance patients compared to private or uninsured groups. After adjustment, WLST occurred earlier for publicly insured (HR 1.07, 95% CI 1.02-1.12) and uninsured patients (HR 1.29, 95% CI 1.18-1.41) compared to privately insured patients. Access to rehabilitation was lower for both publicly insured and uninsured patients compared to patients with private insurance. Accounting for case-mix, the MOR was 1.49 (95% CI 1.43-1.55), reflecting significant residual between-center variation in WLST decision-making. CONCLUSIONS: Our findings highlight the presence of disparate WLST practices independently associated with health insurance status. Additionally, these results emphasize between-center variability in WLST, persisting despite adjustments for measurable patient and trauma center characteristics.

Insurance Instability for Patients With Opioid Use Disorder in the Year After Diagnosis.

Importance: Transitions in insurance coverage may be associated with worse health care outcomes. Little is known about insurance stability for individuals with opioid use disorder (OUD). Objective: To examine insurance transitions among adults with newly diagnosed OUD in the 12 months after diagnosis. Design, Setting, and Participants: Longitudinal cohort study using data from the Massachusetts Public Health Data Warehouse. The cohort includes adults aged 18 to 63 years diagnosed with incident OUD between July 1, 2014, and December 31, 2014, who were enrolled in commercial insurance or Medicaid at diagnosis; individuals diagnosed after 2014 were excluded from the main analyses due to changes in the reporting of insurance claims. Data were analyzed from November 10, 2022, to May 6, 2024. Exposure: Insurance type at time of diagnosis (commercial and Medicaid). Main Outcomes and Measures: The primary outcome was the cumulative incidence of insurance transitions in the 12 months after diagnosis. Logistic regression models were used to generate estimated probabilities of insurance transitions by insurance type and diagnosis for several characteristics including age, race and ethnicity, and whether an individual started medication for OUD (MOUD) within 30 days after diagnosis. Results: There were 20 768 individuals with newly diagnosed OUD between July 1, 2014, and December 31, 2014. Most individuals with newly diagnosed OUD were covered by Medicaid (75.4%). Those with newly diagnosed OUD were primarily male (67% in commercial insurance, 61.8% in Medicaid). In the 12 months following OUD diagnosis, 30.4% of individuals experienced an insurance transition, with adjusted models demonstrating higher transition rates among those starting with Medicaid (31.3%; 95% CI, 30.5%-32.0%) compared with commercial insurance (27.9%; 95% CI, 26.6%-29.1%). The probability of insurance transitions was generally higher for younger individuals than older individuals irrespective of insurance type, although there were notable differences by race and ethnicity. Conclusions and Relevance: This study found that nearly 1 in 3 individuals experience insurance transitions in the 12 months after OUD diagnosis. Insurance transitions may represent an important yet underrecognized factor in OUD treatment outcomes.

Impact of mental health in persons living with rare disease: Findings from the AMCP Market Insights Program.

Within the framework of its Market Insights Program, AMCP convened a panel of experts representing diverse stakeholders to identify alterations to plan design and/or coverage options geared toward improving the diagnosis and treatment of mental health conditions among persons living with rare diseases (PLWRD). PLWRD face unique mental health challenges because of the misunderstood nature of their conditions, potential misdiagnosis, and limited treatment options. Economic burdens arise from increased medical needs, reliance on caregivers, and work disruptions. The interplay of these factors, along with health insurance coverage, creates a distinctive mental health landscape for PLWRD and a need to prioritize mental health support for this patient population. This article aims to (1) summarize expert perspectives on health care system challenges and areas of agreement concerning the management of mental health conditions and (2) advance payers' understanding of their role in supporting mental health care for patients with rare diseases. Addressing mental health needs of PLWRD presents multifaceted challenges. Managed care organizations play a pivotal role in supporting mental health care for PLWRD through their quality improvement initiatives and policies for coverage and reimbursement, which can impact both the rare disease treatment and mental health services PLWRD receive.

Utilization and affordability of health insurance coverage for rare disease drugs in a first-tier city in Northeast China from 2018 to 2021: a study based on the health insurance claims database.

OBJECTIVE: The accessibility issue of orphan drugs in China is prominent. Based on real-world data from a tier-one city in Northeast China, this study aims to analyze the current usage and affordability of orphan drugs for rare diseases. METHODS: The data was sourced from the health insurance claims data of a certain city from 2018 to 2021, including a total of 16 orphan drugs. The utilization of orphan drugs is assessed using four indicators: frequency of medical insurance claims, medication cost, defined daily doses (DDDs), and defined daily drug cost (DDDc). Affordability is measured using the concept of catastrophic health expenditure (CHE). RESULTS: Between January 2018 and December 2021, there were a total of 2,851 medical insurance claims in the city, with a total medication costs of $3.08 million. Overall, during the study, there was a year-on-year increase in the utilization frequency of individual rare disease drugs in the city, with DDDs rising from 140.22 in 2018 to 3983.63 in 2021. Additionally, the annual medication costs of individual drugs showed a consistent upward trend, increasing from $10,953.53 in 2018 to $120,491.36 in 2021. However, the DDDc of individual drugs decreased from $398.12 in 2018 to $96.65 in 2021.The number of sales and the amount of sales for orphan drugs in community pharmacies have significantly increased. Prior to medical insurance coverage, out of the 16 orphan drugs, 9 drugs had annual treatment costs exceeding CHE for urban residents, and 15 drugs had annual treatment costs exceeding CHE for rural residents. After medical insurance coverage, there were no drugs with out-of-pocket costs exceeding CHE for urban residents, while 8 drugs had out-of-pocket costs exceeding CHE for rural residents. Furthermore, both before and after medical insurance coverage, the four treatment drugs for idiopathic pulmonary arterial hypertension were more affordable compared to the four treatment drugs for multiple sclerosis. CONCLUSION: The usage frequency of orphan drugs in a certain city increased gradually, but the disease burden remained heavy. More policy support should be provided to the priority rare disease populations, and the rare disease medical security and diagnosis and treatment systems should be improved.