Journal of Medical Economics最新文献

Background: Ulcerative colitis (UC) imposes persistent clinical and economic burden on patient and healthcare management in Japan.

Purpose: To evaluate impact of prolonged oral corticosteroid (OC) use on healthcare resource utilization (HCRU) and treatment-related costs, and to assess discontinuation patterns of 5-amino salicylic acid (5-ASA), immunomodulators (IMs), and OCs in Japanese UC patients after biologic initiation.

Methods: Data were extracted from the Japan Medical Data Centre for patients diagnosed with UC with ≥1 prescription of OC with 5-ASA and/or IM, prior or at the index date (first biologic initiation) between 2016 and 2022, grouped by

Results: For all identified patients (N = 1,494; mean ± SD age: 38.6 ± 13.7 years; male: 65.3%), HCRU (inpatient and outpatient visits, length of stay, and procedures) per patient-year (PPY) declined after biologics initiation. Direct inpatient medical costs decreased throughout the study; outpatient costs increased from pre-index to the 1-year post-index period, followed by slight decrease in the 2-year and 3-year post-index. PPY costs of non-biologic UC-related drugs (OCs, 5-ASA, IMs) increased slightly during the post-index period. Overall, HCRU and costs dynamics were similar in patients with <180 days and those with ≥180 days of OC use. Patients with <180 days of OC use had shorter median time to OC discontinuation after biologic initiation compared with ≥180 days group (3.1 months vs 9.5 months).

Conclusions: Biologic initiation was associated with reduced HCRU and inpatient costs, with similar trends observed regardless of prolonged or shorter OC use duration.

Background: Traditional cost effectiveness analyses frequently use quality-adjusted life years (QALYs) to quantify health benefits. The Medicare Drug Price Negotiation program, however, cannot use QALYs, but may consider alternative, non-discriminatory metrics.

Objective: To examine the impact of using alternative quantitative health benefit metrics on the economic value of new medications. The framework was applied to assess the cost-effectiveness of rifaximin for preventing overt hepatic encephalopathy (OHE) recurrence in adults.

Methods: A cost-effectiveness analysis evaluated the economic value of rifaximin ± lactulose versus standard of care ± lactulose in preventing recurrent OHE in adults over a lifetime horizon from US payer and societal perspectives. Clinical outcomes included time in remission and overt health states, number of liver transplants, and life years (LYs). Health benefit was quantified using QALYs, health years in total (HYT), equal-value of life years gained (evLYG), and generalized risk-adjusted cost-effectiveness (GRACE). Treatment value was measured using incremental cost effectiveness ratio (ICER). A societal perspective scenario added productivity and caregiving impacts to the model.

Results: Rifaximin patients spent >3 times as long in remission (54.4 vs. 17.3 months), comparable time in the overt health state (1.44 vs. 1.44 months), and had twice as many liver transplants (20 vs. 9), driven by longer survival (8.80 vs. 4.17 LYs), resulting in incremental gains of 3.12 QALYs, 3.26 HYT, 2.78 evLYG, and 3.16 GRA-QALYs. Total costs were higher with rifaximin ($182,369 vs. $38,313, Δ = $144,056), mainly due to drug costs (Δ = $133,330). Including caregiving and productivity reduced the incremental cost to $136,866. From a payer perspective, rifaximin ICERs were $46,215/QALY, $44,198/HYT, $51,847/evLYG, and $45,609/GRA-QALY. After incorporating societal costs, ICERs improved to $43,908/QALY, $41,992/HYT, $49,259/evLYG, and $43,332/GRA-QALY.

Conclusion: Rifaximin is a cost-effective treatment for preventing OHE recurrence in adults using QALY and non-QALY health benefit measures.

Aim: Nirmatrelvir-ritonavir (NMV/r; Paxlovid) treatment reduces COVID-19-related morbidity, mortality, and direct healthcare burdens. To investigate the potential impact of NMV/r on indirect disease burdens, we compared workplace productivity between employees diagnosed with COVID-19 who were and were not treated with NMV/r.

Methods: Adult employees with COVID-19 at high-risk of severe disease were identified in the Merative MarketScan Health and Productivity Management Database. Index date was the first COVID-19 diagnosis on or after 12/16/2021; employees were followed over a six-month pre-period and ≥30-day post-period. Individuals treated with NMV/r (claim within 5 days of index) were exactly matched 1:1 to untreated employees based on age, sex, index quarter, Charlson Comorbidity Index, and pre-period acute care visits. Lost workdays per-patient-per-month (PPPM) and associated estimated indirect costs due to absence, short-term disability (STD), or long-term disability (LTD) were compared between matched treated and untreated cohorts over the variable post-period using paired t-tests or Chi-squared tests and two-part hurdle models.

Results: Treated and untreated absence, STD, and LTD analyses included n = 1,909, n = 20,065, and n = 20,318 employees respectively. NMV/r treatment was associated with significantly fewer lost workdays PPPM from absence (mean[SD]: 2.06[2.37] vs. 2.22[2.49], p < 0.05), STD (0.41[2.17] vs. 0.52[2.42], p < 0.001), and LTD (0.02[0.061] vs. 0.04[0.79], p < 0.05) in descriptive analyses. Marginal means from combined two-part hurdle models confirmed treated employees had 5% fewer absence days (mean ratio[95% CI]:0.95[0.91, 0.99]), 17% fewer STD days (mean ratio[95% CI]:0.83[0.77, 0.88]), and 27% fewer LTD days (mean ratio[95% CI]:0.73[0.69, 0.78]) compared to untreated patients (p < 0.01).

Limitations: The study sample was derived from large self-insured employers; results may not generalize to small employers or the uninsured and do not reflect other types of productivity loss (e.g. presenteeism).

Conclusion: Within this sample of high-risk employees, NMV/r therapy was associated with reduced societal burdens following COVID-19 infection.

Aims: Iron deficiency anemia (IDA) is among the most common extraintestinal sequelae of inflammatory bowel disease (IBD). Intravenous iron is often the preferred treatment in patients with active inflammation with or without active bleeding, iron malabsorption, or intolerance to oral iron. The aim of the present study was to evaluate the cost-utility of ferric derisomaltose (FDI) versus ferric carboyxymaltose (FCM) in patients with IBD and IDA in Norway.

Materials and methods: A published patient-level simulation model was used to evaluate the cost-utility of FDI versus FCM in patients with IBD and IDA from a Norwegian national payer perspective. Iron need was modelled based on bivariate distributions of hemoglobin and bodyweight combined with simplified tables of iron need from the FDI and FCM summaries of product characteristics. Patient characteristics and disease-related quality of life data were obtained from the PHOSPHARE-IBD trial. Cost-utility was evaluated in Norwegian Kroner (NOK) over a five-year time horizon.

Results: Patients required 1.64 fewer infusions of FDI than FCM over five years (5.62 versus 7.26), corresponding to 0.41 fewer infusions per treatment course. The reduction in the number of infusions resulted in cost savings of NOK 5,236 (NOK 35,830 with FDI versus NOK 41,066 with FCM). The need for phosphate testing in patients treated with FCM resulted in further cost savings with FDI (no costs with FDI versus NOK 4,470 with FCM). Total cost savings with FDI were therefore NOK 9,707. FDI also increased quality-adjusted life expectancy by 0.071 quality-adjusted life years (QALYs) driven by reduced incidence of hypophosphatemia and fewer interactions with the healthcare system.

Conclusions: FDI resulted in cost savings and improved quality-adjusted life expectancy versus FCM in patients with IDA and IBD in Norway. FDI therefore represents the economically preferable iron formulation in Norwegian patients with IBD and IDA in whom it is indicated.

Aims: Haemophilia is a rare genetic disease that hinders blood clotting. We aimed to review model-based cost-effectiveness analyses (CEAs) of haemophilia treatments, describe the sources of clinical evidence used by these CEAs, summarize the reported cost-effectiveness of different treatment strategies, and assess the quality and risk of bias.

Methods: We conducted a systematic literature review of model-based CEAs of haemophilia treatments by searching databases, the Tufts Medical Center CEA registry, and grey literature. We summarized and qualitatively synthesized the approaches and results of the included CEAs, without a meta-analysis due the diversity of the studies.

Results: 32 eligible studies were performed in 12 countries and reported 53 pairwise comparisons. Most studies analysed patients with haemophilia A rather than haemophilia B. Comparisons of prophylactic versus on-demand treatment indicated that prophylaxis may not be cost-effective, but there was no clear consensus. Emicizumab was generally cost-effective compared with clotting factor treatments and was always dominant for patients with inhibitors. Immune tolerance induction following a Malmö protocol was found to be cost-effective compared to bypassing agents, while there was no consensus for the other protocols. Gene therapies as well as treatment with extended half-life coagulation factors were always cost-effective over their comparators. Studies were highly heterogenous regarding their time horizons, model structures, the inclusion of bleeding-related mortality and quality-of-life impacts. This heterogeneity limited the comparability of the studies. 19 of the 32 included studies received industry funding, which may have biased their results.

Limitations: It was not possible to perform a quantitative synthesis of the results due to the heterogeneity of the underlying studies.

Conclusion: Differences in results between previous CEAs may have been driven by heterogeneity in modelling approaches, clinical input data, and potential funding biases. A more consistent evidence base and modelling approach would enhance the comparability between CEAs.

Background: Invasive pneumococcal disease (IPD), pneumonia (PNE), and otitis media (OM) are significant causes of morbidity among children in the United States (US). While studies have evaluated the economic burden of these conditions, recent data on episodic costs of IPD, PNE, and OM requiring hospitalization or ambulatory care only among US children by age and comorbidity profile are currently not available. This study was undertaken to address this evidence gap.

Methods: A retrospective observational cohort design and data (2015-2019) from Optum's de-identified Clinformatics^® Data Mart Database were employed. Episodes of IPD, all-cause PNE, and all-cause OM were ascertained on a monthly basis during the follow-up period and stratified by care setting (hospital vs. ambulatory); all-cause OM was alternatively stratified by disease severity (acute, persistent, tympanostomy tube placement) and, for acute/persistent, by complexity (simple, complex). Mean episodic costs of disease were estimated for children aged <1, 1-<2, 2-<6, and 6-<18 years, respectively, overall and by comorbidity profile (with vs. without ≥1 medical condition).

Results: Mean age-specific cost of IPD hospitalization ranged from $40,575-$95,607; IPD requiring care in an emergency department (ED), from $2,013-$5,606; and IPD requiring care in other ambulatory settings, from $619-$1,103. Mean cost of all-cause PNE ranged from $16,631-$21,429 for hospitalized cases; $2,462-$2,685 for ED cases; and $424-$473 for other ambulatory cases. Corresponding ranges for all-cause OM were $14,599-$16,341; $1,190-$2,083; and $253-$514. Children with (vs. without) comorbidities had higher mean costs of PNE episodes across all ages and care settings; mean cost of all-cause OM was largely invariant by comorbidity profile and was highest for episodes involving TTP.

Conclusions: Costs of IPD, all-cause PNE, and all-cause OM are high, particularly in the hospital setting. All-cause PNE, one of the most common causes of hospitalization for children, is particularly costly for children with comorbidities.

Aims: This study compared the six-month medical/pharmacy costs and healthcare resource utilization (HCRU) for knee OA patients undergoing intra-articular therapy with different classes of hyaluronic acid (HA) (by molecular weight) or corticosteroid (ICS).

Materials and methods: Patients across high molecular weight (HMW) HA, medium molecular weight (MMW) HA, low molecular weight (LMW) HA, and ICS therapy groups were matched from a U.S. claims database (Optum's de-identified Clinformatics Data Mart Database), with a final size of 6,234 patients per group. Adjusted six-month medical/prescription costs per patient per month (PPPM), and HCRU rates and costs, were determined. Secondary endpoints included complication rates and adjusted costs, new prescription analgesic use, and adjunctive/supplemental intra-articular treatment.

Results: Mean adjusted PPPM medical costs were highest for LMW HA ($527.14), followed by HMW HA ($469.35) and MMW HA ($441.97) (p < .001), and lowest for the ICS group ($240.26; p < .001). Office visit, arthrocentesis, and subsequent ICS/arthrocentesis rates and corresponding costs, as well as costs for any complications, decreased from LMW HA to MMW HA to HMW HA. The ICS group had greater arthrocentesis, subsequent ICS/arthrocentesis, and office visit costs versus MMW and HMW HA groups. The ICS group had higher rates of new prescription analgesic use (15.8% versus 11.7%-12.2%) and adjunctive ICS (21.8% vs. 9.4%-11.1%) and HA (14.1% versus 1.6%-5.3%) treatment than the HA groups. HMW HA had the lowest rates of adjunctive non-index HA treatment.

Limitations: Claims data contains limited clinical data and relied on the accuracy of coding of diagnoses and procedures.

Conclusions: Among HA products, HMW HA may provide greater short-term clinical and economic benefits. Additionally, intra-articular HA therapy may provide improved short-term clinical and economic results over ICS, in terms of lower rates of adjunctive intra-articular treatments, HCRU, and new prescription analgesic use. Complication rates were low reflecting the safety profiles of HA and ICS.

Aims: This study aimed to assess the public health impact and cost-effectiveness of gender-neutral vaccination (GNV) using a nonavalent vaccine (9vHPV) in Japan.

Methods: We used a published, validated dynamic transmission model to estimate the cases of, deaths from, quality-adjusted life years (QALYs) lost to, and costs of diseases associated with HPV genotypes included in the 9vHPV vaccine. These outcomes were modeled over a 100-year time horizon under different GNV and female-only vaccination (FOV) strategies. The primary analysis compared GNV to FOV at a female vaccination coverage rate (VCR) of 30% and male VCR of 15%. Scenario analyses assessed the effects of varying these VCRs, the age at vaccination, and the discount rate.

Results: In the base case, GNV averted an additional 2,070 female and 1,773 male deaths from HPV-associated cancers compared to FOV and was cost effective, with an incremental cost-effectiveness ratio (ICER) of 4,798,537 ¥/QALY from the payer perspective (direct medical costs) and 4,248,586 ¥/QALY from the societal perspective (including costs of lost work productivity). The ICER of GNV versus FOV was higher in scenarios with higher VCRs. However, the ICER could be reduced compared to the base case by implementing vaccination at <15 years of age to reduce the number of vaccine doses required or by reducing the discount rate to assign greater value to the long-term cancer prevention benefits of HPV vaccination.

Limitations: This study may be limited by inaccuracies in the model's input data and assumptions, as well as the exclusion of some societal costs, which may have underestimated cost-effectiveness.

Conclusions: Including boys and men in Japan's HPV vaccination strategy is predicted to provide additional public health benefits compared to FOV and to be cost effective, particularly while the female VCR remains low and if the full vaccine series is completed before age 15.