Journal of comparative effectiveness research最新文献

{"title":"A plain language summary of the perspectives of women who were interviewed about their experiences with uterine fibroids.","authors":"Elke Hunsche, Viatcheslav Rakov, Kayla Scippa, Brooke Witherspoon, Laura McKain","doi":"10.57264/cer-2023-0195","DOIUrl":"10.57264/cer-2023-0195","url":null,"abstract":"<p><strong>What is this summary about?: </strong>This summary describes what researchers learned during interviews of women with uterine fibroids and heavy menstrual bleeding (or period bleeding). At this time, little is known about how women perceive the impact of uterine fibroids on their lives and more information is needed. The goal of this study was to provide new information about the symptoms women have and how these symptoms affect their everyday lives. These interviews were done to better understand how uterine fibroid symptoms affect the lives of women in their own words.</p><p><strong>What were the results?: </strong>Thirty women from the United States, who had completed a clinical trial for a new treatment for heavy menstrual bleeding and uterine fibroids, agreed to be interviewed. The women described what their experiences with uterine fibroids were and the impact these experiences with uterine fibroids had on their lives before participating in the clinical trial. The most common symptoms of uterine fibroids the women described were heavy bleeding with their menstrual periods, pain in the pelvis or groin area, the passing of blood clots, and anemia (or low hemoglobin in red blood cells). Women said their symptoms affected them physically, emotionally, socially, and financially. They also said their symptoms made it hard to do daily activities, sleep, have a sex life, and go to work or school.</p><p><strong>What do the results mean?: </strong>Women who have heavy menstrual bleeding and uterine fibroids experience various uterine fibroid symptoms, and these symptoms affect most parts of the their lives.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e230195"},"PeriodicalIF":1.9,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11284807/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141158341","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":"RWE ready for reimbursement? A round up of developments in real-world evidence relating to health technology assessment: part 16.","authors":"Alejandra Castanon, Antonia Tsvetanova, Sreeram V Ramagopalan","doi":"10.57264/cer-2024-0095","DOIUrl":"10.57264/cer-2024-0095","url":null,"abstract":"<p><p>In this update, we discuss recent US FDA guidance offering more specific guidelines on appropriate study design and analysis to support causal inference for non-interventional studies and the launch of the European Medicines Agency (EMA) and the Heads of Medicines Agencies (HMA) public electronic catalogues. We also highlight an article recommending assessing data quality and suitability prior to protocol finalization and a <i>Journal of the American Medical Association</i>-endorsed framework for using causal language when publishing real-world evidence studies. Finally, we explore the potential of large language models to automate the development of health economic models.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240095"},"PeriodicalIF":1.9,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11284810/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141534563","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":"Changes in symptom burden and quality of life among women with uterine fibroids receiving relugolix combination therapy: a plain language summary.","authors":"Elizabeth A Stewart, Andrea S Lukes, Roberta Venturella, Yulan Li, Elke Hunsche, Rachel B Wagman, Ayman Al-Hendy","doi":"10.57264/cer-2023-0194","DOIUrl":"10.57264/cer-2023-0194","url":null,"abstract":"<p><strong>What is this summary about?: </strong>This is a summary of findings from two research studies (known as clinical trials). The studies looked at how well a medicine called relugolix combination therapy worked in women with heavy menstrual bleeding (heavy bleeding during a period) with uterine fibroids (noncancerous or benign growths in the uterus). In this analysis of the studies, researchers looked at how patients self-reported their uterine fibroid symptoms before and after taking relugolix combination therapy. Researchers also looked at how patients self-reported the impact of uterine fibroids on their health-related quality of life before and after taking relugolix combination therapy.</p><p><strong>What were the results?: </strong>Women took either relugolix combination therapy or placebo (a pill that contains no medicine) by mouth once daily for 24 weeks. Women completed the Uterine Fibroid Symptom and Quality of Life questionnaire (where \"quality of life\" refers to the women's health-related quality of life related to uterine fibroids) before, during, and after treatment. The questionnaire let researchers see if the women felt that relugolix combination therapy decreased the burden of uterine fibroid symptoms and improved the women's health-related quality of life related to uterine fibroids. More women said that they felt less distress due to their uterine fibroid symptoms and that their health-related quality of life related to uterine fibroids was better after taking relugolix combination therapy compared with women who took placebo.</p><p><strong>What do the results mean?: </strong>Relugolix combination therapy may lessen distress associated with uterine fibroid symptoms and improve health-related quality of life related to uterine fibroids.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e230194"},"PeriodicalIF":1.9,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11284808/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141457247","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":"Evaluation of inpatient and emergency department healthcare resource utilization and costs pre- and post-nusinersen for the treatment of spinal muscular atrophy using United States claims.","authors":"Cong Zhu, Craig Zaidman, Bora Youn, Angela D Paradis, Stephanie Raynaud, Bridget A Neville, Nicole B Johnson","doi":"10.57264/cer-2023-0187","DOIUrl":"10.57264/cer-2023-0187","url":null,"abstract":"<p><p><b>Aim:</b> Nusinersen, administered by intrathecal injection at a dose of 12 mg, is indicated across all ages for the treatment of spinal muscular atrophy (SMA). Evidence on real-world healthcare resource use (HRU) and costs among patients taking nusinersen remains limited. This study aimed to evaluate real-world HRU and costs associated with nusinersen use through US claims databases. <b>Patients & methods:</b> Using the Merative™ MarketScan^® Research Databases, patients with SMA receiving nusinersen were identified from commercial (January 2017 to June 2020) and Medicaid claims (January 2017 to December 2019). Those likely to have complete information on the date of nusinersen initiation and continuous enrollment 12 months pre- and post-index (first record of nusinersen treatment) were retained. Number and costs (US$ 2020) of inpatient admissions and emergency department (ED) visits, unrelated to nusinersen administration, were evaluated for 12 months pre- and post-nusinersen initiation and stratified by age: pediatric (<18 years) and adult (≥18 years). <b>Results:</b> Overall, 103 individuals treated with nusinersen were retained: 59 were pediatric (mean age [range]: 9 [1-17] years), and 44 were adults (30 [18-63] years). Inpatient admissions decreased by 41% for pediatrics and 67% for adults in the 12 months post-treatment versus the 12 months pre-treatment. Average inpatient admission costs per patient for the pediatric cohort decreased by 63% ($22,903 vs $8466) and by 79% ($13,997 vs $2899) for the adult cohort when comparing the 12 months pre-index with the 12 months post-index period. Total ED visits and ED visit costs decreased by 8% and 35%, respectively, for the overall cohort over the 12-month period pre- and post-index. <b>Conclusion:</b> Using US claims databases, nusinersen treatment in pediatric and adult patients was associated with reductions in HRU and costs over a 12-month period post-treatment initiation relative to the pre-treatment period.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e230187"},"PeriodicalIF":1.9,"publicationDate":"2024-07-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11225157/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141498196","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":"Phosphodiesterase type 5 inhibitors as treatment for erectile dysfunction: a webinar-based poll unveiling perceptions of healthcare professionals.","authors":"Maher S Moazin, Abdulaziz Baazeem, Ayman Al-Bakri, Adel Al Dayel, Ashraf Amir, Saud Al Sifri, Mona Reda, Fahad Bashraheel, Abdullah Alfakhri, Yousef Hamdy, Fayez Elshaer","doi":"10.57264/cer-2023-0155","DOIUrl":"10.57264/cer-2023-0155","url":null,"abstract":"<p><p><b>Aim:</b> Erectile dysfunction (ED) is marked by a recurring incapacity to achieve or uphold a satisfactory erection during sexual activities. The study aims to increase awareness about male reproductive health, dispel misconceptions about ED and encourage physician-patient discussions. <b>Materials & methods:</b> A live online poll was conducted during a 3-day webinar titled \"Turning the Tide of Men's Health\" organized by Viatris™ in collaboration with the Saudi Society of Family and Community Medicine and attended by healthcare professionals (HCPs) from diverse specialties. The attendees voluntarily responded to nine poll questions on adherence to ED medication, use of phosphodiesterase type 5 inhibitors (PDE5is) as cure versus management of ED and patients' challenges. The responses to the poll questions were recorded and assessed to understand the perceptions of HCPs. <b>Results:</b> The poll garnered 10,423 responses from 5831 attendees on the second day of the webinar. The key findings included HCPs' perceptions that PDE5is contribute to ED management not complete cure. The respondents acknowledged that adherence to ED medications might decline on days without planned sexual activity, and long-term adherence on a daily PDE5i is exhibited by a relatively modest percentage of ED patients. The consensus among respondents was that PDE5is do not enhance or generate sexual desire, and the daily schedule of ED treatment may be burdensome for some patients. <b>Conclusion:</b> The findings from this poll offer insights into the perspectives of HCPs regarding the usage of PDE5is to treat ED. Responders of the poll generally agreed that PDE5is can help manage ED without affecting desire, though adherence may be lower on days without planned sexual activity. Furthermore, most respondents acknowledged that adhering to a daily pill regimen posed a greater burden than waiting for the medication to take effect.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e230155"},"PeriodicalIF":1.9,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11225304/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141076178","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 among nursing home residents with Parkinson's disease psychosis: an analysis of Medicare beneficiaries treated with pimavanserin or other-atypical antipsychotics.","authors":"Krithika Rajagopalan, Nazia Rashid, Daksha Gopal, Dilesh Doshi","doi":"10.57264/cer-2024-0038","DOIUrl":"10.57264/cer-2024-0038","url":null,"abstract":"<p><p><b>Aim:</b> Real-world healthcare resource use (HCRU) burden among patients with Parkinson's disease psychosis (PDP) treated with pimavanserin (PIM) versus other atypical antipsychotics (other-AAPs) including quetiapine (QUE) in long term care (LTC) and nursing home (NH) settings are lacking. This analysis examines HCRU differences among residents in LTC/NH settings who initiate PIM versus QUE or other-AAPs. <b>Methods:</b> A retrospective analysis of LTC/NH residents with PDP from the 100% Medicare claims between 1 April 2015 and 31 December 2021 was conducted. Treatment-naive residents who initiated ≥6 months continuous monotherapy with PIM or QUE or other-AAPs between 04/01/16 and 06/30/2021 were propensity score matched (PSM) 1:1 using 31 variables (age, sex, race, region and 27 Elixhauser comorbidity characteristics). Post-index (i.e., 6 months) HCRU outcomes included: proportion of residents with ≥1 all-cause inpatient (IP) hospitalizations and emergency room (ER) visits. HCRU differences were assessed via log binomial regression and reported as relative risk ratios (RR) and 95% confidence intervals after controlling for dementia, insomnia and index year. <b>Results:</b> From a total of PIM (n = 1827), QUE (n = 7770) or other-AAPs (n = 9557), 1:1 matched sample (n = 1827) in each cohort were selected. All-cause IP hospitalizations (PIM [29.8%]) versus QUE [36.7%]) and ER visits (PIM [47.3%] versus QUE [55.8%]), respectively, were significantly lower for PIM. PIM versus QUE cohort also had significantly lower RR for all-cause IP hospitalizations and ER visits, respectively, (IP hospitalizations RR: 0.82 [0.75. 0.9]; ER visits RR: 0.85 [0.8. 0.9]). PIM versus other-AAPs also had lower likelihood of HCRU outcomes. <b>Conclusion:</b> In this analysis, LTC/NH residents on PIM monotherapy (versus QUE) had a lower likelihood of all-cause hospitalizations (18%) and ER (15%) visits. In this setting, PIM also had lower likelihood of all-cause HCRU versus other-AAPs.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240038"},"PeriodicalIF":1.9,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11225156/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141288182","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":"Comparing prospectively assigned trial and real-world lung cancer patients.","authors":"Brigham Walker, Herman E Ray, Ping Shao, Claudio D'Ambrosio, Craig White, Mark S Walker","doi":"10.57264/cer-2023-0176","DOIUrl":"10.57264/cer-2023-0176","url":null,"abstract":"<p><p><b>Aim:</b> To evaluate the comparability of a probable clinical trial (CT) cohort derived from electronic medical records (EMR) data with a real-world cohort treated with the same therapy and identified using the same inclusion and exclusion criteria to emulate an external control. <b>Methods:</b> We utilized de-identified patient-level structured data sourced from EMRs. We then compared patterns of overall survival (OS) between probable CT patients with those drawn from non-contemporaneous real-world data (RWD) using a two-sided log-rank test, hazard ratios (HRs) using a Cox proportional-hazards model and Kaplan-Meier (KM) survival curves. Each regression estimate was calculated with a corresponding 95% confidence interval. We additionally conducted multiple matching methods to assess their relative performance. <b>Results:</b> Median (standard deviation) OS was 10.2 (0.7) months for the RWD arm and 11.3 (1.3) for the probable CT arm with a Log rank p-value equal to 0.4771. OS in both cohorts is longer than the reported CT median OS of 9.2 (0.6). The HRs generated under all five assessed matching methods (including without adjustment) were not statistically significant at the 95% confidence level. <b>Conclusion:</b> Our results suggest, with caveats noted, that survival patterns between real-world and CT cohorts in this NSCLC setting are not statistically significantly different.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e230176"},"PeriodicalIF":1.9,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11225159/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141087722","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":"Six-minute walk test as clinical end point in cardiomyopathy clinical trials, including ATTR-CM: a systematic literature review.","authors":"Jose Nativi-Nicolau, Ali Yilmaz, Noel Dasgupta, Richard Macey, James Cochrane, Judith Peatman, Catherine Summers, Jennifer Luth, Ronald Zolty","doi":"10.57264/cer-2023-0158","DOIUrl":"10.57264/cer-2023-0158","url":null,"abstract":"<p><p><b>Aim:</b> The six-minute walk test (6MWT) is a common measure of functional capacity in patients with heart failure (HF). Primary clinical study end points in cardiomyopathy (CM) trials, including transthyretin-mediated amyloidosis with CM (ATTR-CM), are often limited to hospitalization and mortality. <b>Objective:</b> To investigate the relationship between the 6MWT and hospitalization or mortality in CM, including ATTR-CM. <b>Method:</b> A PRISMA-guided systematic literature review was conducted using search terms for CM, 6MWT, hospitalization and mortality. <b>Results:</b> Forty-one studies were identified that reported 6MWT data and hospitalization or mortality data for patients with CM. The data suggest that a greater 6MWT distance is associated with a reduced risk of hospitalization or mortality in CM. <b>Conclusion:</b> The 6MWT is an accepted alternative end point in CM trials, including ATTR-CM.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e230158"},"PeriodicalIF":1.9,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11234454/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141310769","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":"First in class, best in class or a wild card: who will dominate the anti-obesity medication market?","authors":"Emma Collins, Alice Beattie, Sreeram V Ramagopalan, Jonathan Pearson-Stuttard","doi":"10.57264/cer-2024-0044","DOIUrl":"10.57264/cer-2024-0044","url":null,"abstract":"","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240044"},"PeriodicalIF":1.9,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11225158/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141161300","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":"Evaluating the robustness of an AI pathfinder application on eligibility criteria in multiple myeloma trials using real-world data and historical trials.","authors":"Rana Jreich, Hao Zhang, Zhaoling Meng, Fei Wang","doi":"10.57264/cer-2023-0164","DOIUrl":"10.57264/cer-2023-0164","url":null,"abstract":"<p><p><b>Background:</b> Eligibility criteria are pivotal in achieving clinical trial success, enabling targeted patient enrollment while ensuring the trial safety. However, overly restrictive criteria hinder enrollment and study result generalizability. Broadening eligibility criteria enhances the trial inclusivity, diversity and enrollment pace. Liu <i>et al.</i> proposed an AI pathfinder method leveraging real-world data to broaden criteria without compromising efficacy and safety outcomes, demonstrating promise in non-small cell lung cancer trials. <b>Aim:</b> To assess the robustness of the methodology, considering diverse qualities of real-world data and to promote its application. <b>Materials/Methods:</b> We revised the AI pathfinder method, applied it to relapsed and refractory multiple myeloma trials and compared it using two real-world data sources. We modified the assessment and considered a bootstrap confidence interval of the AI pathfinder to enhance the decision robustness. <b>Results & conclusion:</b> Our findings confirmed the AI pathfinder's potential in identifying certain eligibility criteria, in other words, prior complications and laboratory tests for relaxation or removal. However, a robust quantitative assessment, accounting for trial variability and real-world data quality, is crucial for confident decision-making and prioritizing safety alongside efficacy.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e230164"},"PeriodicalIF":1.9,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11225521/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141310768","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}