Journal of comparative effectiveness research最新文献

{"title":"MOMENT registry: Patients with advanced non-small-cell lung cancer harboring <i>MET</i> exon 14 skipping treated with systemic therapy.","authors":"Michael Thomas, Petros Christopoulos, Wade T Iams, Julien Mazières, Alexis B Cortot, Nir Peled, Gabriele Minuti, Egbert F Smit, Francois Audhuy, Karin Berghoff, S Peter Eggleton, Frank Fries, Maike Hildenbrand, Peter Liu, Seyed Hamidreza Mahmoudpour, Christoph Menzel, Dina Oksen","doi":"10.57264/cer-2024-0127","DOIUrl":"10.57264/cer-2024-0127","url":null,"abstract":"<p><p><b>Aim:</b> <i>MET</i> exon 14 <i>(MET</i>ex14) skipping occurs in 3-4% of non-small-cell lung cancer (NSCLC) cases. Low frequency of this alteration necessitated open-label, single-arm trials to investigate MET inhibitors. Since broad MET biomarker testing was only recently introduced in many countries, there is a lack of historical real-world data from patients with <i>MET</i>ex14 skipping NSCLC receiving conventional therapies. Given the rarity of this population and limitations of existing real-world data sources, the MOMENT registry aims to prospectively collect uniform, comprehensive, high-quality data from patients with <i>MET</i>ex14 skipping advanced NSCLC treated in routine clinical practice, which can support clinical and regulatory decision making. <b>Patients & methods:</b> MOMENT is a multinational, non-interventional disease registry collecting data on patients with <i>MET</i>ex14 skipping advanced NSCLC receiving any systemic anticancer therapy. Newly diagnosed patients and those already receiving treatment are eligible. Patients with previous participation in a clinical trial can be included if they receive at least one subsequent therapy line in a routine clinical setting. Eligible systemic treatment includes all available anticancer therapies (approved, conditionally approved or provided through Early Access). Data collection includes biomarker testing results, demographics, baseline clinical characteristics, treatment details and effectiveness, safety information and imaging. Registry site inclusion is dependent on confirmation that local <i>MET</i>ex14 skipping detection methods are sufficient to confirm <i>MET</i>ex14 skipping status. MOMENT is currently active at more than 60 sites across Europe and North America and approximately 700 patients are expected to be enrolled within the next 4 years. The first patient was enrolled on 4 October 2022. After completion of data collection, MOMENT data can be shared with external parties to conduct non-interventional studies. <b>Discussion/conclusion:</b> The MOMENT registry collects comprehensive, high-quality real-world data from patients with <i>MET</i>ex14 skipping advanced NSCLC receiving systemic anticancer treatment in a routine clinical setting, to enable future studies informing regulatory decisions and optimal care for this rare population. <b>Clinical Trial Registration:</b> NCT05376891 (ClinicalTrials.gov); EUPAS47602 (EU PAS register no.).</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240127"},"PeriodicalIF":1.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11773919/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143006393","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Indirect treatment comparison of lanadelumab and a C1-esterase inhibitor in pediatric patients with hereditary angioedema.","authors":"Maureen Watt, Rachel Goldgrub, Mia Malmenäs, Katrin Haeussler","doi":"10.57264/cer-2024-0110","DOIUrl":"10.57264/cer-2024-0110","url":null,"abstract":"<p><p><b>Aim:</b> To compare the efficacy and safety of lanadelumab versus other approved long-term prophylaxis (LTP) treatments in patients with pediatric hereditary angioedema (HAE) aged <12 years. <b>Materials</b> <b>&</b> <b>methods:</b> A systematic literature review was conducted to identify studies of LTP in patients with HAE aged <12 years. Two studies met the inclusion criteria in an indirect treatment comparison of efficacy and safety data in pediatric HAE patients. These were for lanadelumab (SPRING, NCT04070326) and intravenous-C1-esterase inhibitor (C1-INH[IV], NCT02052141). A propensity score analysis used individual patient-level data from both studies in a logistic regression model to estimate inverse probability weights. To avoid convergence issues and an underpowered analysis due to the small sample size (n = 29), the base case was defined as Poisson regression analyses on monthly attack rate adjusting for one covariate (baseline attack rate). Model selection among unadjusted, adjusted and weighted regression models was conducted through the Akaike and Bayesian Information Criteria. <b>Results:</b> Lanadelumab 150 mg every 2 weeks (Q2W) reduced the monthly HAE attack rate by 82.1% versus C1-INH(IV) 1000 IU twice weekly (every 3-4 days [BIW]; rate ratio [RR], 0.1792 [95% CI: 0.0296-1.0853]) and by 88.9% versus C1-INH(IV) 500 IU BIW (RR: 0.1107 [95% CI: 0.0234-0.5239]). Treatment with lanadelumab Q2W reduced the risk of total adverse events by 56.2% versus C1-INH(IV) 1000 IU BIW (RR:0.4377 [95% CI: 0.1536-1.2469]) and by 66.0% versus C1-INH(IV) 500 IU BIW (RR: 0.3401 [95% CI: 0.1234-0.9371]). <b>Conclusion:</b> This exploratory analysis suggested a trend toward greater efficacy and fewer adverse events with lanadelumab 150 mg Q2W compared with C1-INH(IV) BIW 1000 IU and 500 IU in pediatric patients with HAE. Future studies could potentially assess larger samples over longer periods of time for the long-term preventative efficacy, safety and tolerability of lanadelumab and C1-INH(IV).</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240110"},"PeriodicalIF":1.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11773901/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143006388","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A real-world analysis of antidepressant medications in US veterans aged 60 years and older: a comparative analysis.","authors":"Ryan D Pittman, S Scott Sutton, Joseph Magagnoli, Tammy H Cummings","doi":"10.57264/cer-2024-0187","DOIUrl":"10.57264/cer-2024-0187","url":null,"abstract":"<p><p><b>Aim:</b> To compare the safety and efficacy of antidepressants (AD) among older adults with major depressive disorder (MDD) by assessing treatment change, augmentation and hospitalization rates. <b>Methods:</b> This retrospective study analyzed data from the Veterans Affairs (VA) database, including 142,138 patients aged ≥60 years diagnosed with MDD. Patients prescribed bupropion, citalopram, duloxetine, escitalopram, fluoxetine, mirtazapine, paroxetine, sertraline, or venlafaxine were included. Outcomes were treatment change, augmentation and hospitalization rates. Hazard ratios (aHRs) were calculated using sertraline as the reference. <b>Results:</b> Of the patients, 39.6% required augmentation, 18.1% changed antidepressant treatment and 13.3% were hospitalized. The corresponding incidence rate was 544, 124 and 122 events per 1000 person-years. Compared with sertraline, mirtazapine users had the highest AD change risk (aHR 1.34, 95% CI: 1.29-1.40), while duloxetine users had the lowest (aHR 0.87, 95% CI: 0.83-0.92). Duloxetine also had the lowest augmentation risk (aHR 0.89, 95% CI: 0.86-0.92). Mirtazapine users also had the highest risks of augmentation (aHR 1.15, 95% CI: 1.12-1.18) and hospitalization (aHR 1.14, 95% CI: 1.07-1.23). Bupropion had the lowest hospitalization risk (aHR 0.77, 95% CI: 0.71-0.84). <b>Conclusion:</b> Antidepressant choice significantly influences treatment outcomes in older adults with MDD. Duloxetine demonstrated the best profile with the lowest risks of AD change and augmentation, while mirtazapine posed the highest risks of all three outcomes. Personalized treatment strategies are crucial to improving outcomes in this population.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240187"},"PeriodicalIF":1.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11773886/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143006381","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Longer disease progression milestone-free time in people with amyotrophic lateral sclerosis treated versus not treated with intravenous edaravone: results from an administrative claims analysis.","authors":"James D Berry, Melissa Hagan, Jeffrey Zhang, Ying Liu, Malgorzata Ciepielewska","doi":"10.57264/cer-2024-0007","DOIUrl":"10.57264/cer-2024-0007","url":null,"abstract":"<p><p><b>Aim:</b> To estimate time-to-progression milestones in people with amyotrophic lateral sclerosis (PALS) treated versus not treated with intravenous (IV) edaravone (Radicava^® IV, Mitsubishi Tanabe Pharma America [MTPA], hereafter \"IV edaravone\") in a real-world setting. <b>Background:</b> IV edaravone is US FDA approved for the treatment of ALS and was shown in clinical trials to slow the rate of physical functional decline. <b>Patients & methods:</b> This retrospective observational analysis included PALS continuously enrolled in Optum's Clinformatics^® Data Mart between 8 August 2017 and 31 December 2021. Cases treated with IV edaravone and controls not treated with IV edaravone were propensity score matched for: age, sex, race, US region of residence, pre-index disease duration, insurance, riluzole prescription; and pre-index claims for cardiovascular disease, artificial nutrition/gastrostomy tube, noninvasive ventilation and all-cause hospitalization. The index date was the first IV edaravone claim for cases; for controls, the index date was randomly assigned after IV edaravone market availability. Restricted mean time lost was calculated for the following disease progression milestones: new use of canes/walkers/wheelchairs, artificial nutrition, noninvasive ventilation, invasive ventilation, speech-generating devices and hospice. <b>Results:</b> Cases (n = 395) were matched to controls (n = 395). Cases had less restricted mean time lost, indicating longer disease progression milestone-free time, for all disease progression milestones. From 0 to 24 months post index, more cases (n = 129) than controls (n = 103) reported no milestones and more controls (n = 232) than cases (n = 131) reported deaths. <b>Conclusion:</b> In a US-based real-world setting, IV edaravone-treated PALS had a longer time to disease progression milestone events and fewer deaths in 2 years compared with PALS not treated with IV edaravone.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240007"},"PeriodicalIF":1.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11773918/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143006391","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Use of individualized starting dose and niraparib hematologic adverse event management costs in ovarian cancer.","authors":"Whitney S Graybill, Ignace Vergote, Bhavana Pothuri, Maarit Anttila, David M O'Malley, Domenica Lorusso, Ashley F Haggerty, Michel Fabbro, John K Chan, Florian Heitz, Lyndsay J Willmott, Ilan Bruchim, Ying Zhuo, Purificación Estévez-García, Bradley J Monk, Hannelore Denys, Anja Knudsen, Anna V Tinker, Luis Manso Sánchez, Diane Provencher, Maria Pilar Barretina-Ginesta, John Hartman, Donna V Booth, Antonio González-Martín","doi":"10.57264/cer-2024-0133","DOIUrl":"10.57264/cer-2024-0133","url":null,"abstract":"<p><p><b>Aim:</b> To understand the impact of the niraparib individualized starting dose (ISD), compared with fixed starting dose (FSD), on the cost of hematologic adverse event (AE) management from a US payer perspective. <b>Methods:</b> The frequencies of grade ≥3 hematologic AEs that occurred in >1% of patients treated with niraparib were obtained from the primary analysis results of the phase III PRIMA/ENGOT-OV26/GOG-3012 trial. US unit costs for each grade ≥3 AE in the base case were obtained from the 2017 Agency for Healthcare Research and Quality Healthcare Cost and Utilization Project database; unit costs were adjusted to 2020 US dollars. AE management costs per patient were calculated by multiplying AE unit cost by the frequency of each AE by niraparib starting dose. Because AEs were assumed to occur independently of one another, costs were added to derive the total cost. <b>Results:</b> For niraparib, the estimated AE management cost per patient was lower for the ISD than the FSD for all hematologic AEs (FSD vs ISD: thrombocytopenia, $4701.87 vs $1921.89; anemia, $2784.00 vs $1760.59; platelet count decreased, $2103.47 vs $922.51; neutropenia, $2112.50 vs $1369.56; neutrophil count decreased, $1285.87 vs $770.38). The total mean calculated AE management cost per patient was $12,987.71 with the FSD and $6744.93 with the ISD. <b>Conclusion:</b> For niraparib, the cost of managing hematologic AEs in the US was reduced by almost half with the ISD compared with the FSD. The cost reduction and improvements in safety associated with the niraparib ISD support its use in clinical practice.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240133"},"PeriodicalIF":1.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11656343/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142785891","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"R WE ready for reimbursement? A round up of developments in real-world evidence relating to health technology assessment: part 17.","authors":"Paul Arora, Sreeram V Ramagopalan","doi":"10.57264/cer-2024-0212","DOIUrl":"10.57264/cer-2024-0212","url":null,"abstract":"<p><p>In this update, we discuss a position statement from the National Institute of Health and Care Excellence (NICE) on the use of artificial intelligence for evidence generation and publications reviewing the use of real-world data as external control arms. Finally, we discuss a number of recent studies investigating the real-world effectiveness of glucagon-like peptide-1 receptor agonists and whether these studies are informative for reimbursement decision making.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240212"},"PeriodicalIF":1.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11650383/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142729346","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Clinical and economic implications of focal dissection treatment following percutaneous transluminal angioplasty of the superficial femoral artery: an exploratory analysis based on the TOBA II Study.","authors":"Jan B Pietzsch, Benjamin P Geisler, Abigail M Garner, Anne M Ryschon, William A Gray, Masahiko Fujihara, Peter A Schneider","doi":"10.57264/cer-2024-0055","DOIUrl":"10.57264/cer-2024-0055","url":null,"abstract":"<p><p><b>Aim:</b> Percutaneous transluminal angioplasty (PTA) for peripheral artery disease (PAD) commonly leads to dissections which are associated with higher target lesion revascularization (TLR) rates. Clinical and economic consequences of dissection management in the femoropopliteal artery following PTA, and specifically the potential economic benefit of focal dissection repair using the novel Tack Endovascular System, remain unknown. <b>Methods:</b> A decision-analytic model was used to estimate 24-month clinical events, costs and quality-adjusted life year (QALY) gain for a Tack-supported versus status-quo PTA strategy. Patient and lesion characteristics and TLR rates were derived from the PTA cohort of the TOBA II clinical trial, an observational cohort, and literature. Cost-effectiveness was determined from a US payer and provider perspective separately for the non-severe (grade A or B), severe (grade C and higher) and the entire dissection cohort. <b>Results:</b> TLR rates were lower for the Tack-supported strategy compared with PTA (7.7 vs 27.4% in the non-severe, 13.9 vs 25.8% in the severe and 12.0 vs 26.3% in the entire dissection cohort). Cost and QALY differences were +$297/ + 0.0110 in the non-severe dissection cohort and -$1602/ + 0.0067 in the severe dissection cohort, resulting in an incremental cost-effectiveness ratio (ICER) of $25,622 in the non-severe cohort and dominance in the severe cohort and the entire cohort. <b>Conclusion:</b> Compared with a 'status-quo' approach, proactive focal stenting may lead to fewer reinterventions and improved quality of life. There appears to be a graded economic benefit of focal dissection treatment, being cost-effective in non-severe dissections and even cost saving in severe dissections.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240055"},"PeriodicalIF":1.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11656342/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142769478","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Hospital readmissions following catheter ablation for atrial fibrillation with THERMOCOOL™ STSF/ THERMOCOOL™ ST catheter with CARTO™ 3 system versus TactiCath™ catheter with EnSite™ system.","authors":"Alexandru I Costea, Rahul Khanna, Maximiliano Iglesias, Yiran Rong","doi":"10.57264/cer-2024-0075","DOIUrl":"10.57264/cer-2024-0075","url":null,"abstract":"<p><p><b>Aim:</b> Radiofrequency (RF) catheter ablation (CA) is a mainstay treatment for atrial fibrillation (AF). RF catheters with contact force (CF) sensing technology and electroanatomical mapping systems enable real-time assessment of catheter tip-tissue interface CF, facilitating individualized and precise CA. This study examined inpatient hospital readmissions in patients with AF treated with THERMOCOOL™ ST/ THERMOCOOL™ STSF catheter with the CARTO™ 3 System versus TactiCath™ catheter with the EnSite™ System. <b>Materials</b> <b>&</b> <b>methods:</b> Patients undergoing CA for AF between 1 July 2019 to 30 November 2021 were identified from the Premier Healthcare Database and grouped based on use of THERMOCOOL ST/STSF or TactiCath™. Study outcomes were all-cause, cardiovascular (CV)-, and AF-related inpatient readmission at 91-365-day post-CA. Inverse probability of treatment weighting of propensity scores balanced baseline patient, comorbidity and hospital characteristics. A weighted generalized estimating equation (GEE) model examined differences in readmission outcomes. <b>Results:</b> A total of 15,518 patients met inclusion criteria (THERMOCOOL ST/STSF, n = 13,001; TactiCath™, n = 2517). Patient characteristics were generally well-balanced after weighting. Patients treated with THERMOCOOL ST/STSF + CARTO 3 had a 20% lower likelihood of all-cause inpatient readmissions (7.8 vs 9.3%, chi-square p = 0.041; odds ratio [OR]: 0.80, 95% confidence interval [CI]: 0.66-0.96, GEE p = 0.019) and a 21% lower likelihood of CV-related inpatient readmission (5.2 vs 6.2%, chi-square p = 0.133, OR: 0.79, 95% CI: 0.62-0.99, GEE p = 0.043) in 91-365-days post-CA versus those treated with TactiCath™ + Ensite. No significant differences were observed for AF-related readmissions. <b>Conclusion:</b> Patients undergoing CA for AF treated with THERMOCOOL ST/STSF + CARTO 3 had a significantly lower risk of all-cause and CV-related inpatient hospital readmission versus those treated with TactiCath™ + Ensite.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240075"},"PeriodicalIF":1.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11656344/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142768186","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Characterization of medical device randomized controlled trials with adaptive designs.","authors":"Gui Su, Dan Shen, Dongyuan Deng, Qianqian Bai, Hui Xie","doi":"10.57264/cer-2024-0011","DOIUrl":"10.57264/cer-2024-0011","url":null,"abstract":"<p><p><b>Aim:</b> Adaptive designs are frequently used in drug randomized controlled trials (RCTs). However, their use in medical device RCTs remains unclear. We aimed to characterize medical device RCTs with adaptive designs. <b>Materials & methods:</b> We searched for adaptive RCTs in the following databases: ClinicalTrials.gov, International Clinical Trials Registry Platform and the International Standard Randomised Controlled Trial Number registry. Adaptive design keywords and medical device corporation names were used as terms to search the trial records registered between 1 January 2000 and 18 October 2024 in the databases. The annual number and proportions of adaptive trials were analyzed, and characteristics such as design type, sponsor, therapeutic area, trial stage and regulatory status were summarized. <b>Results:</b> Overall, 105 adaptive RCTs were identified from ClinicalTrials.gov, accounting for 2.112 per 1000 trials in 49,721 medical device clinical trials registered in ClinicalTrials.gov during the period. The average annual number of adaptive RCTs per 1000 clinical trials was the highest (8.55 ± 11.65) during 2005-2010, reduced to 3.33 ± 2.35 during 2011-2016, and significantly decreased to 1.29 ± 0.85 during 2017-2024 (p = 0.011). The most common adaptive designs were group sequential design (GSD, 50.5%), sample size reassessment (SSR, 17.1%) and investigating both superiority and non-inferiority (10.5%). Most RCTs were sponsored by the private sector (62.9%), conducted in Europe/North America (95.2%), in the field of heart disease (46.7%) and post-market trials (76.2%). Compared with pre-market RCTs, post-market RCTs showed more diverse adaptive designs such as response-adaptive randomization and adaptive enrichment. <b>Conclusion:</b> The average annual proportions of adaptive medical device RCTs in ClinicalTrials.gov has reduced in the last 10 years. The most-used adaptive designs in medical device RCTs are GSD, SSR and investigating both superiority and non-inferiority.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240011"},"PeriodicalIF":1.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11650385/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142800247","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Healthcare utilization and costs following molecular diagnostic testing among patients with vaginitis.","authors":"Azia Evans, Riddhi Doshi, Jason Yeaw, Katharine Coyle, Steven Goldberg, Elizabeth Wang, Maren S Fragala, Jairus Reddy","doi":"10.57264/cer-2024-0173","DOIUrl":"10.57264/cer-2024-0173","url":null,"abstract":"<p><p><b>Aim:</b> Vaginitis and other vaginal discharge syndromes lead to high healthcare utilization. Molecular tests like syndromic multiplex real-time (RT) polymerase chain reaction (PCR)-based tests are highly sensitive and specific at diagnosing the infectious causes of vaginitis. This study compared the healthcare resource utilization (HCRU) and direct all-cause healthcare costs among patients with vaginitis in the US receiving next-day syndromic multiplex RT-PCR tests with those receiving other PCR tests or no diagnostic test of interest. <b>Patients & methods:</b> This retrospective study utilized claims data from IQVIA PharMetrics^® Plus database to identify adult patients with a diagnosis for vaginitis (first claim = index) from January 2021 to April 2023, with 6 months of continuous enrollment prior to (baseline) and after index (follow-up). Pairwise comparisons were conducted between RT-PCR and 1:1 propensity matched Other PCR and No Test subcohorts for all-cause HCRU and costs during follow-up. <b>Results:</b> Each of the RT-PCR, Other PCR and No Test subcohorts included 1946 matched patients. Mean(SD) follow-up total cost was significantly lower for the RT-PCR than the No Test subcohort ($5607 [$15,122] vs $6680 [$20,751], p = 0.0023). Mean(SD) overall outpatient and other medical service costs were lower for RT-PCR versus Other PCR (outpatient: $2964 [$9666] vs $3174 [$7113], p = 0.0110; other medical: $1961 [$9244] vs $2099 [$6475], p = 0.0002) and No Test subcohorts (outpatient: $2964 [$9666] vs $4067 [$12,341], p < 0.0001; other medical: $1961 [$9244] vs $2973 [$11,685]; p < 0.0001). A lower proportion had any outpatient service HCRU in RT-PCR versus Other PCR subcohort (92.6% vs 94.2%, p = 0.0349). A lower proportion had any other medical service claim in RT-PCR versus Other PCR (78.3% vs 83.2%, p < 0.0001) and No Test subcohorts (78.3% vs 83.0%, p = 0.0001). Physician office, emergency room (ER), prescription use and costs were similar between the subcohorts. <b>Conclusion:</b> The use of syndromic multiplex RT-PCR diagnostics with next day test results in patients with vaginitis was associated with lower outpatient costs and total healthcare costs than those in the no test cohort over 6 months. These findings indicate that use of syndromic multiplex RT-PCR diagnostics may contribute to improved patient management compared with clinical diagnosis alone.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240173"},"PeriodicalIF":1.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11650384/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142750718","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}