Journal of Medical Economics最新文献

Background: Von Willebrand Disease (vWD) is the most common congenital bleeding disorder, with an estimated prevalence in Spain of 0.01%. The aim was to assess the cost-utility of Haemate-P compared with present alternatives in the treatment of vWD in Spain.

Methods: A Markov model was developed in Microsoft Excel to estimate the cost-effectiveness of various treatments for vWD over a lifetime horizon. Transition probabilities among health states were based on age and number of bleeding events. Treatment strategies compared included Haemate-P, Fanhdi, and Wilate in long-term prophylaxis (LTP) or on-demand treatment (ODT). Costs and quality of life were measured based on patient age, treatment, and number of bleeding events incurred. Both costs and utilities were discounted at 3%. One-way and probabilistic sensitivity analyses were performed.

Results: When comparing LTP regimens, Haemate-P was less costly and numerically more effective than both Fanhdi (incremental costs of -€1,313,845; incremental quality-adjusted life-years [QALY] of 0.13) and Wilate (incremental costs -€2,233,940; incremental QALY 0.29). For ODT, Haemate-P was assumed to have equal effectiveness as Fanhdi and Wilate but reduced the costs by €696,857 and €1,145,780, respectively. Haemate-P prophylaxis was more effective and less costly compared with Haemate-P on-demand in the base case (incremental costs -€633,317; incremental QALY 0.90). Results were generally robust to sensitivity analyses.

Conclusions: In patients with severe vWD experiencing a high bleed rate, Haemate-P prophylaxis is a less costly and potentially more effective treatment strategy and Haemate-P is cost-saving among on-demand strategies.

Aims: Migraine leads to substantial healthcare utilization and associated costs. However, much higher costs are attributed to lost productivity. The impact of effective migraine treatment on these costs, at the individual level, has not been well established. Even less known is the impact of treatment failure. The objective of this study was to assess lost earnings as a measure of migraine-attributed burden among triptan non-responders in Denmark.

Materials and methods: We used data from the Danish National Prescription Register and Danish Income Statistics Register over the 27-year period 1995-2021. We identified 4,979 triptan non-responders (85.9% female) and matched them for sex, age and region of residence with 14,292 continuing users of triptans (triptan responders) and 13,592 individuals from the background population (triptan never-users). We then estimated average annual individual earnings, and compared those among triptan non-responders, from three years prior to and ten years after their last triptan redemption, with those among their matched triptan responders and triptan never-users over the same periods.

Results: Triptan non-responders earned significantly less than both their matched triptan responders and their matched triptan never-users. The earnings gap was evident even three years prior to the last triptan prescription (€4,344 and €4,356 respectively). This gap widened substantially over time, so that average cumulative earnings over the 14-year period of follow-up for each triptan non-responder were €93,684 less than those of responders and €99,485 less than those of never-users.

Limitations: There are uncertainties with regard to the reasons for triptan discontinuation (whether non-response or otherwise), and to lack of diagnostic confirmation of migraine.

Conclusions: Triptan non-response represents failure of currently available acute treatment options. It is associated with substantial and cumulative lost earnings, highlighting a disproportionate economic burden. These findings underscore the potential economic benefit of recognizing, and rectifying, unmet treatment needs in migraine management.

Introduction: A cell harvesting device for preparing non-cultured autologous skin cell suspension (ASCS) at the point-of-care is FDA-approved for repigmentation of stable depigmented vitiligo lesions in patients 18 years and older. The pivotal RSVP trial showed ≥80% repigmentation at Week-24 in 36% of lesions treated with laser ablation, ASCS, and narrowband ultraviolet B phototherapy compared to 0% with phototherapy alone (p = 0.012). The objective of this analysis was to evaluate the potential economic impact of laser ablation plus ASCS with phototherapy versus phototherapy alone for repigmentation of stable vitiligo lesions from a US payer perspective.

Methods: A 5-year decision-tree model was developed reflecting clinical pathways of adults with stable vitiligo lesions who had an inadequate response to prior topicals and phototherapy. Patients entering the model were treated with ASCS plus phototherapy or phototherapy alone and assessed for treatment response at Weeks-24 and 52 based on the RSVP trial's effectiveness endpoints. Durable response for Year-2 onwards was proxied by melanocyte-keratinocyte transplantation data. Model outcomes included per-patient total and incremental healthcare costs, treatment costs and total costs, cost per-patient per-month (PPPM), and cost per-patient per-year (PPPY). One-way sensitivity analyses assessed model result robustness.

Results: The cumulative total per-patient cost for ASCS plus phototherapy increased from $28,177 to $92,779 between Year-1 and Year-5. Phototherapy alone increased from $21,146 to $101,518 over the same period. Compared to phototherapy alone, ASCS plus phototherapy incurred $7,030 more total per-patient cumulative costs in Year-1 and $8,738 less by Year-5 (-$146 PMPM; -$1,748 PPPY). Breakeven occurred between Years 2-3. Results were most sensitive to changes in ASCS response at Weeks-24 and 52 and healthcare costs.

Conclusion: Among adults with stable vitiligo with prior inadequate response to topicals or phototherapy, ASCS treatment may lead to lower all-cause direct medical costs over 5 years compared to phototherapy alone.

Background: The prevalence of chronic kidney disease (CKD) in Thailand is high and kidney disease progression remains a problem. Empagliflozin has been known to be used to slow CKD progression, but its accessibility remains limited. This study aimed to assess the cost-utility of empagliflozin for CKD progression in Thailand.

Methods: A state-transition model was developed consisting of eight health states: five eGFR health states (G2, G3a, G3b, G4 and G5), dialysis, kidney transplantation and death. Empagliflozin 10 mg was assessed as an add-on treatment to standard of care (SoC). The efficacy of empagliflozin was derived from the EMPA-KIDNEY trial, while other inputs were obtained from a comprehensive literature review. The incremental cost-effectiveness ratio (ICER) per quality-adjusted life year (QALY) was calculated. A probabilistic sensitivity analysis (PSA) was performed to explore uncertainties.

Results: Empagliflozin could improve QALYs by 0.62 and 0.71 for patients with CKD without and with diabetes mellitus (DM) compared with SoC, respectively. However, it required higher total lifetime costs of 77,966 Thai baht (THB) and 59,454 THB for patients with CKD without and with DM, respectively. The ICER for CKD without DM was 126,201 THB/QALY, while the ICER for CKD with DM was 83,473 THB/QALY. The PSA indicated that empagliflozin had a 64.00% probability of being cost-effective for CKD without DM and an 89.18% probability for CKD with DM.

Limitations: An important limitation was that the treatment effects of empagliflozin were derived from the EMPA-KIDNEY, which was conducted in DM patients and assumed to be the same for non-DM patients because of the limited evidence in non-DM patients.

Conclusion: At the current willingness-to-pay threshold of 160,000 THB/QALY, empagliflozin was cost-effective for treating patients with CKD without or with DM.

In contrast to clinical effectiveness of resective epilepsy surgery (RES) for patients with drug-resistant epilepsy, societal costs of RES is still unclear. The aim of this study was to report on total societal costs up until two years after surgery and analyse the trend of post-surgical costs over time. Secondary objectives included assessing quality of life (QoL) changes and identifying determinants of post-surgical costs.Data were derived from the patients' entire medical history based on hospital files and accompanied by validated questionnaires before and 3-, 6-, 12-, and 24-months post-surgery to additionally include medical consumption outside of the hospital, productivity losses and gains, and QoL. To explore the trend of post-surgical costs over time and identify determinants of post-surgical costs, linear mixed effects and linear regression models were performed.The study included 44 patients. Mean complete costs from diagnostics and treatment strategies in the period before referral for pre-surgical evaluation up until two years after RES were €121,856 (Interquartile range=€76,058-€137,027). Post-surgical costs significantly decreased 12 months (mean 3-month difference=€-6,675, p = 0.000) and 24 months (mean 3-month difference=€-7,690, p = 0.000) after surgery compared to 3 months before surgery. Higher post-surgical costs were associated with a clinically relevant increase in disease-specific QoL after RES (p = 0.000), previous ketogenic diet (p = 0.005), RES in the left hemisphere (p = 0.014), previous RES (p = 0.007), and higher diagnostics and treatment strategies costs before referral for pre-surgical evaluation (p = 0.021). For disease-specific and generic QoL, 20 (45%) patients reached a clinically relevant QoL increase two years after surgery compared to before RES.In conclusion, RES leads to significant reduction in costs 2 years post-surgery. History of RES and ketogenic diet, clinically relevant disease-specific QoL increase, surgery in the left hemisphere, and higher costs of diagnostics and treatment strategies before referral for pre-surgical evaluation were significant determinants for higher post-surgical costs after RES.