Journal of comparative effectiveness research最新文献

Patient and public involvement in health technology assessment (HTA) has progressed from best practice to policy requirement, yet communication barriers persist. This perspective explores how plain language summaries (PLSs) and summaries of information for patients (SIPs) can enhance equity and transparency in HTA. Building on recent European regulatory developments and emerging research, it discusses the balance between accessibility, quality and feasibility. Generative artificial intelligence offers the potential to scale PLS and SIP production, but its responsible integration requires oversight, collaboration and a continued focus on equity and patient-centeredness within evolving HTA frameworks.

Aim: We compared the effects of nefecon, an oral targeted-release budesonide formulation, and sparsentan, an oral, dual endothelin-angiotensin receptor antagonist, on estimated glomerular filtration rate (eGFR) in patients with immunoglobulin A nephropathy, a leading cause of chronic kidney disease. Materials & methods: We conducted an anchored matching-adjusted indirect comparison (MAIC) using patient-level data from NefIgArd (NCT03643965; n = 364), a randomized (1:1) trial of nefecon plus optimized renin-angiotensin system inhibitor (RASi) therapy versus placebo plus RASi; and aggregate data from PROTECT (NCT03762850; n = 404), a randomized (1:1) trial of sparsentan versus irbesartan, an angiotensin receptor blocker. Mean absolute eGFR change and mean relative urine protein-to-creatinine and urine albumin-to-creatinine ratio changes from baseline at 9, 12 and 24 months (NefIgArd) or 36, 48 and 106 weeks (PROTECT) were analyzed using a mixed-effects model for repeated measures. A composite outcome (time to confirmed 40% eGFR reduction, end-stage kidney disease or all-cause mortality) was also included. An unanchored MAIC and network meta-analysis were used as sensitivity analyses. Results: The matching process reduced the effective sample for the NefIgArd trial from 364 to 208. Absolute eGFR change significantly favored nefecon over sparsentan at 9 months (mean difference, ml/min/1.73 m² [95% credible interval]: 5.7 [3.1-8.2]), 12 months (3.5 [1.0-6.0]) and 24 months (3.3 [0.0-6.5]). Differences in other outcomes were generally not statistically significant. Sensitivity analysis results were consistent with the main findings. Conclusion: In patients with immunoglobulin A nephropathy, nefecon plus optimized RASi may preserve kidney function to a greater extent than sparsentan.

Aim: This 2023 online survey assessed the management of patients with iron deficiency anemia/iron deficiency without anemia (IDA/IDWA) in Italy. Materials & methods: The study used the patient's perspective to examine care pathways used to manage IDA/IDWA in Italy, to raise clinician awareness, improve patient care (earlier diagnosis, initiate appropriate treatment) and outcomes (reduce recurrence/progression). The survey questioned iron deficiency (ID) diagnoses of patients, their sources of information and influence, their knowledge of ID, perceptions and fears surrounding treatment, unmet needs and expectations, and underlying causes of treatment compliance and persistence. Results: Of the 404 respondents (102 males and 302 females) who completed the survey, all were aged between 18 and 80 years. Almost all respondents (97.0%) experienced ID symptoms, most frequently fatigue, weakness and tiredness (71.0% of the total cohort; 80.1% females vs 44.1% males); 76.8% of all respondents regarded symptoms as 'bothersome'. Most respondents (70.5%) consulted a physician as the first action to treat ID (79.8% females vs 43.1% males); general practitioners were the main healthcare providers, consulted by 55.6% of all respondents. Most respondents (94.1%) were aware of potential ID recurrence, and 75.0% reported recurrent ID episodes since their diagnosis. Satisfaction with ID treatment was rated as average (7-7.7/10); 56.4% of all respondents stayed on ID treatment for as long as prescribed, and 41.1% did not fully comply with tablet intake, primarily because they 'felt much better' (64.8%). Conclusion: This survey identified high rates of self-reported ID symptoms and recurrent ID episodes among respondents. It highlights the importance of increasing awareness of ID and its consequences among healthcare practitioners and individuals in the general population, of shortening the time before diagnosis, and ensuring that patients continue treatment for the prescribed duration to resaturate serum iron levels.

Aim: This study aims to assess the cost-effectiveness of three major programmed death-ligand 1 (PD-L1) immunohistochemistry (IHC) testing assays in guiding immunotherapy for patients with advanced non-small-cell lung cancer (NSCLC) in China, and to provide empirical evidence on the selection of cost-effective diagnostics to support the immune checkpoint inhibitor monotherapy regimens for NSCLC patients. Materials & methods: From a healthcare system perspective, a decision tree model was constructed to simulate the cost and effectiveness of employing VENTANA PD-L1 (SP263) Assay, Dako PD-L1 IHC 22C3 assay, and Dako 22C3 antibody concentrate in advanced NSCLC patients in China. The cost-effectiveness of Ventana PD-L1 IHC SP263 assay compared with other testing methods was assessed through the incremental analysis. The robustness of the base case analysis results was validated by one-way sensitivity analysis and probabilistic sensitivity analysis. Results: The VENTANA PD-L1 (SP263) Assay, which can guide multiple immune checkpoint inhibitor monotherapies (e.g., atezolizumab and pembrolizumab) for advanced NSCLC patients, achieved a higher percentage of successfully diagnosed and treated, and a higher quality-adjusted life-year at a lower cost compared with Dako PD-L1 IHC 22C3 assay and Dako 22C3 antibody concentrate. The robustness of the base case results was confirmed by both one-way sensitivity analysis and probabilistic sensitivity analysis. Conclusion: The VENTANA PD-L1 (SP263) Assay is cost-effective compared with Dako PD-L1 IHC 22C3 assay and Dako 22C3 antibody concentrate for the immunotherapy treatment for advanced NSCLC patients in China.

In this update, we cover the evolving health technology assessment process in Australia and also examine how commercial health plans in the US influence patient access to treatments.

Aim: Pimavanserin (PIM) is the only US FDA-approved atypical antipsychotics (AAPs) for the treatment for hallucinations and delusions associated with Parkinson's disease psychosis (PDP). In addition to demonstrating symptom improvements in clinical trials, PIM appears to consistently show favorable outcomes among published real-world evidence (RWE) studies compared with off-label AAPs (e.g., quetiapine [QUE]). A comprehensive review of these RWE studies was conducted to summarize the overall benefits of PIM among PDP patients residing in community or nursing home/long-term care (NH/LTC) settings. Materials & methods: A literature review of published comparative RWE studies of PIM among PDP patients from 1 January 2017 to 1 April 2025 was conducted. Eligible studies examined the following outcomes in community and NH/LTC settings: clinical (e.g., falls, fractures), adherence (e.g., discontinuations), all-cause and psychiatric (psych)-related healthcare resource use (e.g., hospitalizations, emergency room (ER) visits, office visits, etc.), and mortality. Results: Sixteen RWE studies of PIM versus other-AAPs or QUE and PIM nonusers were included for review. In these studies, PIM had 20-37% lower all-cause and psych-related hospitalizations, 7-15% lower ER visits, significant delays to LTC admissions, lower rates of falls or fractures in NH/LTC settings and lower observed rates of mortality versus other-AAPs. Similar results were found for PIM versus QUE. Conclusion: PDP patients initiating PIM versus other-AAPs or QUE were associated with lower all-cause and psychiatric healthcare resource use burden, longer community-stays and delayed NH/LTC admissions. Additionally, PIM was associated with higher treatment compliance, fewer falls or fractures and lower overall mortality risk versus other-AAPs or QUE. PIM's favorable real-world profile versus other-AAPs or QUE across community and NH/LTC settings complement the favorable clinical trial findings.

In this update, we examine the growing potential of synthetic data in addressing data access challenges for health technology assessment. We explore studies evaluating synthetic data as external control arms in clinical trials and the application of synthetic data in health economic evaluation. Additionally, we review real-world evidence on the clinical impact of formulary restrictions in the US.

Aim: To describe real-world adherence, treatment utilization, vaso-occlusive crises (VOC) and economic outcomes in patients with sickle cell disease (SCD) with recurrent VOC treated with L-glutamine, voxelotor or crizanlizumab in the US. Materials & methods: In this retrospective study, patients with SCD with recurrent VOC who received L-glutamine, voxelotor, or crizanlizumab were identified from the Merative™ MarketScan^® Research Databases between 1 January 2015 and 30 September 2022. Eligible patients had ≥12 months continuous enrollment before and after the first chronic therapy claim (i.e., index date). Number of VOC, treatment utilization, healthcare resource utilization and healthcare costs were summarized for 12 months before (baseline) and after (follow-up) the index date. The proportion of days covered (PDC; i.e., proxy for adherence) for the index chronic therapy was measured during the 12-month follow-up period. Results: Overall, 440 patients initiated a recently approved chronic therapy (L-glutamine, n = 254; voxelotor, n = 110; crizanlizumab, n = 76) and met inclusion criteria. Mean (standard deviation [SD]) number of VOC during baseline and follow-up were similar for patients treated with any index therapy (n = 440; 7.21 [8.82] vs 7.27 [9.85]); this was similar across patients treated with L-glutamine, crizanlizumab, and voxelotor, respectively. Mean (SD) PDC for patients with any index therapy was 0.37 (0.29); results were similar across patients treated with L-glutamine, crizanlizumab, and voxelotor. Healthcare resource utilization during the 12-month baseline and follow-up periods were comparable. Mean (SD) total costs for patients initiating a recently approved chronic therapy increased by ∼50% or $38,111 during follow-up (follow-up, $118,235 [$177,125]; baseline, $80,125 [$120,950]; p < 0.001); most of the increased costs ($27,108 [71.1%]) were a direct result of recently approved chronic therapies. Conclusion: Patients initiated on L-glutamine, voxelotor or crizanlizumab had low adherence (based on PDC), continued to experience frequent VOC, and incurred higher healthcare costs mostly due to the costs of these therapies. This highlights the need for additional treatment options for patients with SCD with recurrent VOC.

Aim: The optimal management strategies for spontaneous intracerebral hemorrhage in the basal ganglia region are still controversial. The purpose of the present study is to evaluate the clinical benefits of craniotomy, minimally invasive puncture and drainage (MIPD) and conservative treatment in patients with basal ganglia hematoma. Materials & methods: This study retrospectively enrolled consecutive patients with hematoma ≥20 ml in the basal ganglia hemorrhage and onset to admission within 24 h. Primary outcome was the proportion of favorable outcome (modified Rankin scale score 0-3) at 3 months. Secondary outcomes included excellent outcome (modified Rankin scale score 0-2) and mortality at 3 months and 1 year. Sensitive analysis was performed in patients with hematoma ≥ 30 ml. Results: A total of 481 patients were included and divided into three groups: 146 received craniotomy, 211 underwent MIPD and 124 were managed with conservative treatment. The proportion of primary outcome was lower in the craniotomy (23.24%) than in the MIPD (35.41%) and conservative treatment (41.94%) groups. Among patients with hematoma ≥30 ml, MIPD has higher rates of favorable outcome at 3 months (37.32 vs 25.98%, p = 0.05) and 1 year (52.82 vs 40.16%, p = 0.04) compared with craniotomy; albeit. The short-term and long-term mortality was similar in three groups. Conclusion: MIPD was found to be associated with both short- and long-term favorable outcomes in patients with spontaneous intracerebral hemorrhage in the basal ganglia region. Moreover, neither MIPD nor craniotomy showed an association with increased risk of short- or long-term mortality.