Prevalence and clinical characteristics of people with HIV with limited treatment options in Japan: A Japanese claims data analysis.

INTRODUCTION: Long-term medication leads some people with HIV (PWH) to limited treatment options (LTO) due to multiple factors. The present study investigated the prevalence of PWH with LTO in Japan and their clinical characteristics, persistence, and adherence. METHODS: PWH who received antiretroviral therapy (ART) between 2017 and 2022 were identified in the Medical Data Vision (MDV) Japanese claims database. PWH with LTO were defined as: 1) receiving regimens indicative for LTO or 2) having a complex treatment history (≥4 different core agents, ≥11 ART agents). Prevalence by calendar year, clinical characteristics, persistence, and adherence measured by the proportion of days covered (PDC) of ART were investigated. RESULTS: A total of 5740 PWH were included, and 207 (3.6 %) were identified as LTO. Mean (SD) age was 50.3 (11.8) years, 148 (71.5 %) had evidence of AIDS-defining condition, and 25 (12.1 %) had hemophilia. The prevalence of PWH with LTO increased from 2.58 % in 2017 to 3.55 % in 2022. Persistence at 1 year was estimated as 70.3 % and mean PDC through 1 year was 96.7 %. CONCLUSION: Between the years 2017-2022, 3.6 % (approximately 200) Japanese PWH were identified as having LTO. The results of this analysis found clinical characteristics of PWH with LTO as older age and higher percentages with an AIDS-defining condition and hemophilia than the general HIV population. Low persistence indicates that treatment optimization is required in this population. These results will help health care providers to understand the clinical characteristics of PWH with LTO and may contribute to the establishment of appropriate treatment strategies.

Indirect Treatment Comparisons of Lenacapavir Plus Optimized Background Regimen Versus Other Treatments for Multidrug-Resistant Human Immunodeficiency Virus.

BACKGROUND/AIMS: Heavily treatment-experienced (HTE) people with human immunodeficiency virus (HIV) (PWH) may not achieve virologic suppression (VS) with combination antiretroviral therapy due to multidrug resistance (MDR), intolerance, and safety concerns. These PWH often receive highly individualized treatment regimens, but these regimens may not enable PWH to achieve VS, thereby halting disease progression. Novel medications are required for treating individuals with MDR HIV. Lenacapavir (LEN), a first-in-class HIV capsid inhibitor, is under investigation for the treatment of HTE individuals with MDR HIV in the phase 2/3 CAPELLA study. This study aimed to compare LEN plus optimized background regimen (OBR) with fostemsavir (FTR) + OBR, ibalizumab (IBA) + OBR, and OBR alone in terms of VS, CD4 cell count change from baseline, immunologic recovery, and discontinuation due to adverse events, using indirect treatment comparisons. METHODS: A systematic review identified clinical evidence on HIV-1 treatments in HTE PWH. A feasibility assessment evaluated the identified studies for indirect treatment comparison analyses based on population characteristics, interventions, comparators, and outcomes of interest. Unanchored simulated treatment comparisons of LEN + OBR versus comparators were conducted. RESULTS: LEN + OBR had 6.57 times higher odds of VS at weeks 24 to 28 than FTR + OBR (95% confidence interval [CI] 1.34-32.28), 8.93 times higher odds of VS than IBA + OBR (95% CI 2.07-38.46), and 12.74 times higher odds of VS than OBR alone (95% CI 1.70-95.37). Change from baseline in CD4 cell count was similar across LEN + OBR, FTR + OBR, and IBA + OBR. CONCLUSION: LEN + OBR has statistically significantly greater odds of VS at weeks 24 to 28 than its comparators and represents a novel treatment for people with MDR HIV.

Cost-effectiveness analysis of HIV pre-exposure prophylaxis in Japan.

BACKGROUND: While global efforts have been made to prevent transmission of HIV, the epidemic persists. Men who have sex with men (MSM) are at high risk of infection. Despite evidence of its cost-effectiveness in other jurisdictions, pre-exposure prophylaxis (PrEP) for MSM is neither approved nor reimbursed in Japan. METHOD: The cost-effectiveness analysis compared the use of once daily PrEP versus no PrEP among MSM over a 30-year time horizon from a national healthcare perspective. Epidemiological estimates for each of the 47 prefectures informed the model. Costs included HIV/AIDS treatment, HIV and testing for sexually transmitted infections, monitoring tests and consults, and hospitalization costs. Analyses included health and cost outcomes, as well as the incremental cost-effectiveness ratio (ICER) reported as the cost per quality-adjusted life year (QALY) for all of Japan and each prefecture. Sensitivity analyses were performed. FINDINGS: The estimated proportion of HIV infections prevented with the use of PrEP ranged from 48% to 69% across Japan, over the time horizon. Cost savings due to lower monitoring costs and general medical costs were observed. Assuming 100% coverage, for Japan overall, daily use of PrEP costs less and was more effective; daily use of PrEP was cost-effective at a willingness to pay threshold of ¥5,000,000 per QALY in 32 of the 47 prefectures. Sensitivity analyses found that the ICER was most sensitive to the cost of PrEP. INTERPRETATION: Compared to no PrEP use, once daily PrEP is a cost-effective strategy in Japanese MSM, reducing the clinical and economic burden associated with HIV.

HIV remains an epidemic, and men who have sex with men (MSM) are at higher risk of infection. Pre-exposure prophylaxis (PrEP) is a preventive treatment that can reduce someone's risk of getting infected with HIV and has been shown to provide good value for money. PrEP, however, is neither approved nor reimbursed in Japan. In order to determine the value for money in Japan, an economic model was developed to estimate the number of HIV infections and AIDS cases that could be avoided, along with whether daily use of PrEP among MSM in Japan is cost effective. Findings showed that with use of daily PrEP, the proportion of HIV infections and AIDS cases prevented was 63% and 59%, respectively, across Japan. Over a 30-year time horizon, daily use of PrEP would cost the health system less and be more effective than no use of PrEP. Daily PrEP should therefore be considered for reimbursement in MSM in Japan, given its value for money.

Primary Prophylaxis With Biosimilar Filgrastim for Patients at Intermediate Risk for Febrile Neutropenia: A Cost-Effectiveness Analysis.

PURPOSE: Temporary COVID-19 guideline recommendations have recently been issued to expand the use of colony-stimulating factors in patients with cancer with intermediate to high risk for febrile neutropenia (FN). We evaluated the cost-effectiveness of primary prophylaxis (PP) with biosimilar filgrastim-sndz in patients with intermediate risk of FN compared with secondary prophylaxis (SP) over three different cancer types. METHODS: A Markov decision analytic model was constructed from the US payer perspective over a lifetime horizon to evaluate PP versus SP in patients with breast cancer, non-small-cell lung cancer (NSCLC), and non-Hodgkin lymphoma (NHL). Cost-effectiveness was evaluated over a range of willingness-to-pay thresholds for incremental cost per FN avoided, life year gained, and quality-adjusted life year (QALY) gained. Sensitivity analyses evaluated uncertainty. RESULTS: Compared with SP, PP provided an additional 0.102-0.144 LYs and 0.065-0.130 QALYs. The incremental cost-effectiveness ranged from $5,660 in US dollars (USD) to $20,806 USD per FN event avoided, $5,123 to $31,077 USD per life year gained, and $7,213 to $35,563 USD per QALY gained. Over 1,000 iterations, there were 73.6%, 99.4%, and 91.8% probabilities that PP was cost-effective at a willingness to pay of $50,000 USD per QALY gained for breast cancer, NSCLC, and NHL, respectively. CONCLUSION: PP with a biosimilar filgrastim (specifically filgrastim-sndz) is cost-effective in patients with intermediate risk for FN receiving curative chemotherapy regimens for breast cancer, NSCLC, and NHL. Expanding the use of colony-stimulating factors for patients may be valuable in reducing unnecessary health care visits for patients with cancer at risk of complications because of COVID-19 and should be considered for the indefinite future.

Evaluation of the cost-utility of a prescription digital therapeutic for the treatment of opioid use disorder.

Background: The opioid epidemic continues to generate a significant mental and physical health burden on patients, and claims the life of almost 150 Americans daily. Making matters worse, an increase in relapses and/or opioid-related deaths has been reported in more than 40 U.S. states since the start of the COVID-19 pandemic. Opioid use disorder (OUD) is one of the single most expensive disorders in the United States, generating average medical costs of $60B from just 2 million Americans diagnosed with the disorder. In commercial use since 2019, reSET-O is a non-drug, prescription digital therapeutic (PDT) that delivers evidence-based neurobehavioral treatment for OUD and helps overcome the barriers associated with access to care, stigma, and social distancing. Although shown to be cost effective and efficacious in clinical trials and real-world evidence studies, respectively, information on its value for money from a health utilities and cost per quality-adjusted life-year is needed to inform policy discussions.Objectives: To evaluate the impact of reSET-O on health utilities and assess its overall cost per quality-adjusted life year (QALY) gained vs. treatment-as-usual (TAU).Methods: Decision analytic model comparing reSET-O plus TAU to TAU alone (i.e. buprenorphine, face-to-face counseling, and contingency management) over 12 weeks. Clinical effectiveness data (abstinence and health utility) were obtained from a clinical trial, and resource utilization and cost data were adapted from a recent claims data analysis to reflect less frequent face-to-face counseling with the therapeutic.Results: The addition of reSET-O to TAU decreases total health care costs by -$131 and resulted in post-treatment utility values within population norms, with a corresponding gain of 0.003 QALYs. reSET-O when used adjunctively to TAU was economically dominant (less costly, more effective) vs. TAU alone.Conclusion: reSET-O is an economically-dominant adjunctive treatment for OUD and is associated with an overall reduction in total incremental cost vs TAU.

Evaluating the Presence of Cognitive Biases in Health Care Decision Making: A Survey of U.S. Formulary Decision Makers.

BACKGROUND: Behavioral economics is a field of economics that draws on insights from psychology to understand and identify patterns of decision making. Cognitive biases are psychological tendencies to process information in predictable patterns that result in deviations from rational decision making. Previous research has not evaluated the influence of cognitive biases on decision making in a managed care setting. OBJECTIVE: To assess the presence of cognitive biases in formulary decision making. METHODS: An online survey was conducted with a panel of U.S. pharmacy and medical directors who worked at managed care organizations and served on pharmacy and therapeutics committees. Survey questions assessed 4 cognitive biases: relative versus absolute framing effect, risk aversion, zero-risk bias, and delay discounting. Simulated data were presented in various scenarios related to adverse event profiles, drug safety and efficacy, and drug pricing for new hypothetical oncology products. Survey questions prompted participants to select a preferred drug based on the information provided. Survey answers were analyzed to identify decision patterns that could be explained by the cognitive biases. Likelihood of bias was analyzed via chi-square tests for framing effect, risk aversion, and zero-risk bias. The delay discounting section used a published algorithm to characterize discounting patterns. RESULTS: A total of 35 pharmacy directors and 19 medical directors completed the survey. In the framing effect section, 80% of participants selected the suboptimal choice in the relative risk frame, compared with 38.9% in the absolute risk frame (P < 0.0001). When assessing risk aversion, 42.6% and 61.1% of participants displayed risk aversion in the cost- and efficacy-based scenarios, respectively, but these were not statistically significant (P = 0.27 and P = 0.10, respectively). In the zero-risk bias section, results from each scenario diverged. In the first zero-risk bias scenario, 90.7% of participants selected the drug with zero risk (P < 0.001), but in the second scenario, only 32.1% chose the zero-risk option (P < 0.01). In the section assessing delay discounting, 54% of survey participants favored a larger delayed rebate over a smaller immediate discount. A shallow delay discounting curve was produced, which indicated participants discounted delayed rewards to a minimal degree. CONCLUSIONS: Pharmacy and medical directors, like other decision makers, appear to be susceptible to some cognitive biases. Directors demonstrated a tendency to underestimate risks when they were presented in relative risk terms but made more accurate appraisals when information was presented in absolute risk terms. Delay discounting also may be applicable to directors when choosing immediate discounts over delayed rebates. However, directors neither displayed a statistically significant bias for risk aversion when assessing scenarios related to drug pricing or clinical efficacy nor were there significant conclusions for zero-risk biases. Further research with larger samples using real-world health care decisions is necessary to validate these findings. DISCLOSURES: This research was funded by Xcenda. Mezzio, Nguyen, and O'Day are employees of Xcenda. Kiselica was employed by Xcenda at the time the study was conducted. The authors have nothing to disclose. A portion of the preliminary data was presented as posters at the 2017 AMCP Managed Care & Specialty Pharmacy Annual Meeting; March 27-30, 2017; in Denver, CO, and the 2017 International Society for Pharmacoeconomics and Outcomes Research 22nd Annual International Meeting; May 20-24, 2017; in Boston, MA.

Association of ABC (HbA1c, blood pressure, LDL-cholesterol) goal attainment with depression and health-related quality of life among adults with type 2 diabetes.

AIMS: To determine the relationship between ABC goal attainment, depression, and health-related quality of life (HRQoL) among a national sample of patients with type 2 diabetes (T2DM). METHODS: A retrospective, cross-sectional analysis was performed examining 808 non-pregnant patients ≥20 years old with T2DM from the National Health and Nutrition Examination Survey (NHANES) 2007-2012. ABC goals were defined as HbA1c<7%, BP<130/80 mm Hg, and LDL-C<100 mg/dL. Patient characteristics associated with ABC goal attainment were examined. RESULTS: Overall, 23.7% of participants achieved simultaneous ABC goals. Severe depression was significantly associated with lower rates of ABC goal attainment compared to those with no depression (5.0% vs. 25.4%, p=0.048). ABC goal attainment rates were lower among females, Hispanic and non-Hispanic black minority groups, and patients with a duration of diabetes over five years, while increased visits with health care professionals were significantly associated with meeting all three ABC goals for patients with T2DM. CONCLUSIONS: The relationship between simultaneous ABC goal attainment, depression and HRQoL is complex. Patients with T2DM unable to meet ABC goals may benefit from increased contact with health care professionals.