Journal of comparative effectiveness research最新文献

{"title":"R WE ready for reimbursement? A round up of developments in real-world evidence relating to health technology assessment: part 14.","authors":"Benjamin D Bray, Sreeram V Ramagopalan","doi":"10.57264/cer-2023-0189","DOIUrl":"10.57264/cer-2023-0189","url":null,"abstract":"<p><p>In this latest update we highlight: a publication from the US FDA regarding the definitions of real-world data (RWD) and real-world evidence (RWE); a publication from academic researchers on a demonstration project for target trial emulation; a publication from the National Institute of Health and Care Excellence (NICE) on the 1 year anniversary of their RWE framework; and a publication from NICE and Flatiron Health on the utility of US RWD for initial UK health technology assessment decision making.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":"13 1","pages":"e230189"},"PeriodicalIF":2.1,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10842288/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139097976","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":"Real-world assessment of treatment inertia in the management of patients treated for major depressive disorder in the USA.","authors":"John J Sheehan, Chris LaVallee, Keshia Maughn, Santosh Balakrishnan, Jacqueline A Pesa, Kruti Joshi, Craig Nelson","doi":"10.57264/cer-2023-0091","DOIUrl":"10.57264/cer-2023-0091","url":null,"abstract":"<p><p><b>Aim:</b> Major depressive disorder (MDD) is a debilitating illness in which depressive symptoms may persist after treatment. Treatment inertia is the continued use of the same pharmacotherapy regimen when treatment goals are not met. This study assessed the frequency of treatment inertia among adult patients with MDD treated in a real-world setting. <b>Patients & methods:</b> This was a retrospective, observational study of patients with MDD identified in the Decision Resources Group Real World Evidence US Data Repository from January 2014 to June 2018. Patients (≥18 years) had an elevated Patient Health Questionnaire-9 (PHQ-9) score (≥5) following 8 weeks of stable baseline antidepressant use with/without mental-health outpatient therapy. Treatment inertia, modification and discontinuation were evaluated over a 16-week follow-up period (timeline based on the APA Practice Guidelines). The primary outcome was the proportion of MDD patients experiencing treatment inertia. <b>Results:</b> 2850 patients (median age, 55 years; 74% female) met the study criteria. Of these patients, 834 (29%) had study-defined treatment inertia, 1534 (54%) received treatment modification and 482 (17%) discontinued treatment. Use of mirtazapine (Odd ratio [OR]: 0.63; 95% confidence interval [CI]: 0.50-0.79), selective serotonin reuptake inhibitors (OR: 0.64; 95% CI: 0.54-0.75) or bupropion (OR: 0.71; 95% CI: 0.60-0.84) in the baseline period was associated with an increased likelihood of treatment modification versus not receiving treatment with these medications. Frequency of treatment inertia may differ among those who do not have a documented PHQ-9 score. <b>Conclusion:</b> Effective symptom management is critical for optimal outcomes in MDD. Results demonstrate that treatment inertia is common in MDD despite guidelines recommending treatment modification in patients not reaching remission.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e230091"},"PeriodicalIF":2.1,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10842298/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138176336","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":"Treatment patterns and economic burden of bacterial vaginosis among commercially insured women in the USA.","authors":"Eren Watkins, Clifton M Chow, Melissa Lingohr-Smith, Jay Lin, Candice Yong, Krishna Tangirala, Kevin Collins, James Li, Roy Brooks, Jennifer Amico","doi":"10.57264/cer-2023-0079","DOIUrl":"10.57264/cer-2023-0079","url":null,"abstract":"<p><p><b>Aim:</b> Bacterial vaginosis (BV) is a common vaginal dysbiosis associated with adverse clinical sequelae, most notably, increased risk of sexually transmitted infections (STIs). The aims of this study were to estimate the frequency of BV recurrence, treatment patterns, other gynecological (GYN) conditions, and the associated healthcare resource utilization (HCRU) and costs among commercially insured patients in the USA. <b>Patients & methods:</b> Female patients aged 12-49 years with an incident vaginitis diagnosis and ≥1 pharmacy claim for a BV medication (fungal treatment only excluded) were selected from the Merative™ MarketScan commercial database (2017-2020). During a minimum 12-month follow-up, additional treatment courses, treatment patterns, frequency of other GYN conditions, and HCRU and costs were assessed. Generalized linear models were used to identify baseline predictors of total all-cause healthcare costs and number of treatment courses. <b>Results:</b> The study population included 140,826 patients (mean age: 31.5 years) with an incident vaginitis diagnosis and ≥1 BV medication claim. During the follow-up, 64.2% had 1 treatment course, 22.0% had 2, 8.1% had 3, and 5.8% had ≥4; 35.8% had a BV recurrence (≥2 BV medication claims). The most commonly prescribed BV medication was oral metronidazole (73.6%). Approximately 12% (n = 16,619) of patients had a new diagnosis of another GYN condition in the follow-up; 8.2% had a new STI, which were more common among patients with ≥4 treatment courses (12.9%). During follow-up, total all-cause healthcare costs averaged $8987 per patient per year (PPPY) of which $470 was BV-related. BV-related healthcare costs increased from $403 PPPY among those with 1 treatment course to $806 PPPY among those with ≥4 with nearly half the costs attributed to outpatient office visits. <b>Conclusion:</b> BV recurrence among this population represented a substantial clinical and healthcare economic burden warranting improvements in women's healthcare.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e230079"},"PeriodicalIF":2.1,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10842271/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138803849","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 resource utilization, costs and treatment associated with myasthenia gravis exacerbations among patients with myasthenia gravis in the USA: a retrospective analysis of claims data.","authors":"Julia Pisc, Angela Ting, Michelle Skornicki, Omar Sinno, Edward Lee","doi":"10.57264/cer-2023-0108","DOIUrl":"10.57264/cer-2023-0108","url":null,"abstract":"<p><p><b>Aim:</b> There are limited data on the clinical and economic burden of exacerbations in patients with myasthenia gravis (MG). We assessed patient clinical characteristics, treatments and healthcare resource utilization (HCRU) associated with MG exacerbation. <b>Patients & methods:</b> This was a retrospective analysis of adult patients with MG identified by commercial, Medicare or Medicaid insurance claims from the IBM^® MarketScan^® database. Eligible patients had two or more MG diagnosis codes, without evidence of exacerbation or crisis in the baseline period (12 months prior to index [first eligible MG diagnosis]). Clinical characteristics were evaluated at baseline and 12 weeks before each exacerbation. Number of exacerbations, MG treatments and HCRU costs associated with exacerbation were described during a 2-year follow-up period. <b>Results:</b> Among 9352 prevalent MG patients, 34.4% (n = 3218) experienced ≥1 exacerbation after index: commercial, 53.0% (n = 1706); Medicare, 39.4% (n = 1269); and Medicaid, 7.6% (n = 243). During follow-up, the mean (standard deviation) number of exacerbations per commercial and Medicare patient was 3.7 (7.0) and 2.7 (4.1), respectively. At least two exacerbations were experienced by approximately half of commercial and Medicare patients with ≥1 exacerbation. Mean total MG-related healthcare costs per exacerbation ranged from $26,078 to $51,120, and from $19,903 to $49,967 for commercial and Medicare patients, respectively. AChEI use decreased in patients with multiple exacerbations, while intravenous immunoglobulin use increased with multiple exacerbations. <b>Conclusion:</b> Despite utilization of current treatments for MG, MG exacerbations are associated with a high clinical and economic burden in both commercial and Medicare patients. Additional treatment options and improved disease management may help to reduce exacerbations and disease burden.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e230108"},"PeriodicalIF":1.9,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10842297/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138803811","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":"Improved estimation of overall survival and progression-free survival for state transition modeling.","authors":"Peter C Wigfield, Bart Heeg, Mario Ouwens","doi":"10.57264/cer-2023-0031","DOIUrl":"10.57264/cer-2023-0031","url":null,"abstract":"<p><p><b>Aim:</b> National Institute for Health and Care Excellence guidance (Technical Support Document 19) highlights a key challenge of state transition models (STMs) being their difficulty in achieving a satisfactory fit to the observed within-trial endpoints. Fitting poorly to data over the trial period can then have implications for long-term extrapolations. A novel estimation approach is defined in which the predicted overall survival (OS) and progression-free survival (PFS) extrapolations from an STM are optimized to provide closer estimates of the within-trial endpoints. <b>Materials & methods:</b> An STM was fitted to the SQUIRE trial data in non-small-cell lung cancer (obtained from Project Data Sphere). Two methods were used: a standard approach whereby the maximum likelihood was utilized for the individual transitions and the best-fitting parametric model selected based on AIC/BIC, and a novel approach in which parameters were optimized by minimizing the area between the STM-predicted OS and PFS curves and the corresponding OS and PFS Kaplan-Meier curves. Sensitivity analyses were conducted to assess uncertainty. <b>Results:</b> The novel approach resulted in closer estimations to the OS and PFS Kaplan-Meier for all combinations of parametric distributions analyzed compared with the standard approach. Though the uncertainty associated with the novel approach was slightly larger, it provided better estimates to the restricted mean survival time in 10 of the 12 parametric distributions analyzed. <b>Conclusion:</b> A novel approach is defined which provides an alternative STM estimation method enabling improved fits to modeled endpoints, which can easily be extended to more complex model structures.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e230031"},"PeriodicalIF":2.1,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10842287/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138803838","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":"Risk of long-term care admissions among Medicare beneficiaries treated with pimavanserin or quetiapine for Parkinson's disease psychosis in USA: a retrospective administrative claims database analysis.","authors":"Krithika Rajagopalan, Nazia Rashid, Dilesh Doshi","doi":"10.57264/cer-2023-0114","DOIUrl":"10.57264/cer-2023-0114","url":null,"abstract":"<p><p><b>Aim:</b> Risk of long-term care (LTC) admission (LTCA) associated with atypical antipsychotic (AAP) use among patients with Parkinson's disease psychosis (PDP) is a major concern. However, no comparative studies have examined the differences in risk of LTC admissions between pimavanserin (PIM), the only FDA-approved AAP for PDP, and other off-label AAPs including quetiapine (QUE). <b>Objective:</b> To examine all-cause LTCA rates and risk among PDP patients treated with AAPs such as QUE or PIM. <b>Methods:</b> Analysis of Parts A, B and D claims (100% Medicare sample; 2013-2019) of Medicare beneficiaries with PDP that initiate ≥12-month continuous PIM or QUE monotherapy from 1 January 2014 to 31 December 2018 (i.e., index date) without any AAP use in the 12-month pre-index period was conducted. Outcome assessments among 1:1 propensity score-matched (31 variables - age, sex, race, region and 27 Elixhauser comorbidities) beneficiaries on PIM versus QUE included risk of all-cause skilled nursing facility stays (SNF-stays), LTC-stays, and overall LTCA (i.e., SNF-stays or LTC-stays). All-cause LTCA rates and LTCA risk were compared using logistic regression and cox proportional hazards models, respectively, controlling for demographics, comorbidities and co-existing-dementia or insomnia. <b>Results:</b> Of the matched sample (n = 842 for each group) from total sample (n = 9652), overall all-cause LTCA and SNF-stay rates were 23.2 and 20.2% for PIM versus 33.8 and 31.4% for QUE, respectively (p < 0.05, for each). Hazard ratio (95% CI) for risk of SNF-stay and overall LTCA was 0.78 (0.61, 0.98) and 0.80 (0.66, 0.97), respectively, for PIM versus QUE beneficiaries (p < 0.05, for each). <b>Conclusion:</b> The 20% lower risk of LTCA (i.e., greater delay) with PIM versus QUE in this analysis may suggest that PIM should be started early for the treatment of PDP.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e230114"},"PeriodicalIF":2.1,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10842290/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138803845","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":"Access in all areas? A round up of developments in market access and health technology assessment: part 2.","authors":"Alice Beattie, Catrin Treharne, Silvy Mardiguian, Sreeram V Ramagopalan","doi":"10.57264/cer-2023-0162","DOIUrl":"10.57264/cer-2023-0162","url":null,"abstract":"<p><p>In this latest update, we explore the Inflation Reduction Act (IRA) enacted by the US Congress in August 2022, with the Centers for Medicare and Medicaid Services (CMS) recently releasing the list of the first ten drugs it will negotiate prices on. We also cover the consequences of price controls and rigid value assessment in Germany which have led to the withdrawal of a number of medicines. It will be important to see how the IRA balances cost-saving with holistic value assessment, incentives for innovation and patient access to treatment.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e230162"},"PeriodicalIF":2.1,"publicationDate":"2023-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10734314/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"71424083","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":"Implementation of electronic patient-reported outcomes for symptom monitoring during cancer treatment: the importance of getting it right.","authors":"Ethan Basch, Kathryn Hudson, Gabrielle Rocque","doi":"10.57264/cer-2023-0157","DOIUrl":"10.57264/cer-2023-0157","url":null,"abstract":"","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e230157"},"PeriodicalIF":2.1,"publicationDate":"2023-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10734319/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"92154700","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":"Once-nightly sodium oxybate (FT218) improved symptoms of disrupted nighttime sleep in people with narcolepsy: a plain language summary.","authors":"Thomas Roth, Michael J Thorpy, Clete A Kushida, Matthew Horsnell, Jennifer Gudeman","doi":"10.57264/cer-2023-0133","DOIUrl":"10.57264/cer-2023-0133","url":null,"abstract":"<p><strong>What is this summary about?: </strong>This is a plain language summary of a published article in the journal <i>CNS Drugs</i>. Narcolepsy is a rare sleep condition. Most people with narcolepsy experience disrupted nighttime sleep and have poor quality of sleep. Sometimes these symptoms are not easily diagnosed as a symptom of narcolepsy. Sodium oxybate is an approved treatment for narcolepsy. The only version of sodium oxybate that was available until 2023 required people to take their sodium oxybate at bedtime and then again in the middle of the night. The US Food and Drug Administration (FDA for short) has approved a once-nightly bedtime dose of sodium oxybate (ON-SXB for short, also known as FT218 or LUMRYZ^™) to treat symptoms of narcolepsy in adults. These symptoms are daytime sleepiness and cataplexy, which is an episode of sudden muscle weakness. The once-nightly bedtime dose of ON-SXB removes the need for a middle-of-the-night dose of sodium oxybate. The REST-ON clinical study compared ON-SXB to a placebo (a substance that contains no medicine) to determine if it was better at treating symptoms of disrupted nighttime sleep associated with narcolepsy. This summary looks at whether; ON-SXB was better than placebo at treating symptoms of disrupted nighttime sleep.</p><p><strong>What were the results?: </strong>Compared to people who took placebo, people who took ON-SXB had fewer number of changes from deeper to lighter sleep stages and woke up less during the night. They also reported that they slept better at night and felt more refreshed when waking up in the morning. People with narcolepsy sometimes take alerting agents to help with sleepiness during the day, but alerting agents can cause difficulty sleeping at night. This study showed that people who took ON-SXB had better nighttime sleep even if they were taking alerting agents during the day. The most common side effects of ON-SXB included dizziness, nausea (feeling sick to your stomach), vomiting, headache, and bedwetting.</p><p><strong>What do the results mean?: </strong>A once-nightly bedtime dose of ON-SXB is a narcolepsy treatment option for people without the need for a middle-of-the-night dose of sodium oxybate.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e230133"},"PeriodicalIF":2.1,"publicationDate":"2023-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10734321/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"136397680","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":"Redesign of radiotherapy for prostate cancer: a proposal for universal healthcare systems.","authors":"Ana Paula Beck da Silva Etges, Luciana Rodrigues de Lara, Stella Lisboa Sapper, Ana Von Frankenberg Berger, Melissa Streck, Laise Zardo, Armani Linhares, Marina Nassif, Angélica Zanotto, Marta Nassif Pereira Lima, Rafael Vargas, Carisi Anne Polanczyk","doi":"10.57264/cer-2023-0023","DOIUrl":"10.57264/cer-2023-0023","url":null,"abstract":"<p><p><b>Aim:</b> This study was designed to recommend strategies to improve prostate patients' access to radiotherapy treatment in the Brazilian Unified Health System, along with a cost-tool to support radiotherapy care pathways' lead times and costs. <b>Methods:</b> Data was collected prospectively from patients with prostate cancer receiving radiotherapy in two Brazilian centers to provide data to apply design thinking and process reengineering techniques. The current status of the radiotherapy pathway was determined and the length of time taken for in-hospital activities was measured using data exported from ARIA^®. Interviews with patients were used to estimate their waiting periods. This provided the data used to provide recommended strategies and the cost tool based on time-driven activity-based costing. The strategies were classified according to priority. <b>Results:</b> Data from 47 patients were analyzed. The mean interval from diagnosis to start of radiotherapy was 349 days (SD581), and the mean interval from seeking medical attention to starting treatment was 635 days (SD629). Twelve strategies affecting in-hospital processes and 11 impacting patients' care pathways and experiences are recommended, mostly focused on system improvement opportunities. A time-driven activity-based costing monitoring using data extracted from ARIA was coded and can be used by centers as a cost assessment guide. <b>Conclusion:</b> This study uses reengineering and design techniques to introduce priority strategies to allow more efficient and patient-centered radiotherapy.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e230023"},"PeriodicalIF":2.1,"publicationDate":"2023-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10734317/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"71424085","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}