| 1. |
- Abuloha, Sumaya, et al.
(författare)
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A Review of the Cost-Effectiveness Evidence for FDA-Approved Cell and Gene Therapies.
- 2024
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Ingår i: Human gene therapy. - 1557-7422.
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Tidskriftsartikel (refereegranskat)abstract
- Cell and gene therapy innovations have provided several significant breakthroughs in recent years. However, cell and gene therapies often come with a high upfront cost, raising questions about patient access, affordability, and long-term value. This study reviewed cost-effectiveness analysis studies that have attempted to assess the long-term value of FDA-approved cell and gene therapies. Two reviewers independently searched the Tufts Medical Center Cost-Effectiveness Analysis Registry to identify all studies for FDA-approved cell and gene therapies per January 2023. A data extraction template was used to summarize the evidence in terms of the incremental cost-effectiveness ratio expressed as the cost per Quality-Adjusted Life-Year (QALY) and essential modeling assumptions, combined with a template to extract the adherence to the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) checklist. The review identified 26 CEA studies for seven cell and gene therapies. Around half of the base-case cost-effectiveness results indicated that the cost per QALY was below $100,000-$150,000, often used as a threshold for reasonable cost-effectiveness in the US. However, the results varied substantially across studies for the same treatment, ranging from being considered very cost-effective to far from cost-effective. Most models were based on data from single-arm trials with relatively short follow-ups, and different long-term extrapolations between studies caused large differences in the modeled cost-effectiveness results. In sum, this review showed that despite the high upfront costs, many cell and gene therapies have cost-effectiveness evidence that can support long-term value. Nonetheless, substantial uncertainty regarding long-term value exists because so much of the modeling results are driven by uncertain extrapolations beyond the clinical trial data.
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| 2. |
- Hansson, Emma, 1981, et al.
(författare)
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Systematic review of cost-effectiveness in breast reconstruction: deep inferior epigastric perforator flap vs. implant-based breast reconstruction.
- 2024
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Ingår i: Journal of plastic surgery and hand surgery. - 2000-6764. ; 59
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Tidskriftsartikel (refereegranskat)abstract
- There are several techniques for reconstructing breasts after mastectomy, but little scientific evidence for which technique is superior. The aim of this systematic review was to compare the cost-effectiveness of implant-based and autologous reconstruction and to evaluate the overall certainty of evidence, as well as the quality of reporting of the included studies.Studies investigating the cost-effectiveness of breast reconstruction with a deep inferior epigastric perforator (DIEP) flap compared to implant-based reconstruction, meeting criteria defined in a PICO (population, intervention, comparison, and outcome), were included. Medline, PubMed, Embase, Cochrane library, CinahL, EconLit, and NHS EED databases were searched. The Grading of Recommendations Assessment, Development and Evaluation (GRADE) approach was used to assess the certainty of evidence, and the Consolidated Health Economic Evaluation Reporting Standard (CHEERS) 2022 was used to evaluate the quality of reporting.A total of 256 abstracts were retrieved from the search, and after scrutiny, seven studies were included. The findings of this present systematic review should be interpreted with caution as the overall certainty of evidence is low (GRADE ƟƟОО). The included studies suggest that DIEP-flaps are cost-effective compared with implant-based breast reconstruction when the applied cost-effectiveness thresholds of $50,000 to $100,000 per quality-adjusted life years are used. It is noteworthy that no high level evidence exists regarding cost-effeciency, to support recommendations and decision in breast reconstruction. Methodological issues that can be improved in future studies are presented.
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| 3. |
- Johansson, Naimi, 1988-, et al.
(författare)
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Place or patient as the driver of regional variation in healthcare spending – Discrepancies by category of care
- 2024
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Ingår i: Social Science and Medicine. - : Elsevier. - 0277-9536 .- 1873-5347. ; 342
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Tidskriftsartikel (refereegranskat)abstract
- We study how much regional variation in healthcare spending is driven by place- and patient-specific factors using a random sample of 53,620 regional migrants in Sweden. We find notable differences depending on the category of care, with place-specific factors having a significantly larger impact on specialized outpatient care compared to inpatient and pharmaceutical care. The place effect is estimated to 75% of variation in specialized outpatient care, but 26% or less in variations in inpatient care, and 5% in prescription drug spending. We also find that the empirical estimator has a substantial impact on the estimates of the place-specific effect. The results based on the traditional approach in the literature with two-way fixed effects and event-study models produce much larger estimates of the place-specific effect compared to results based on recently developed heterogeneity-robust models. For total healthcare spending, the traditional two-way fixed effects model estimates a place effect of 78%, while the heterogeneity-robust estimator finds a place effect around 10%. This finding indicates that previous results in this literature, all based on traditional two-way fixed-effects regressions, should be interpreted with care.
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| 4. |
- Labori, Frida, et al.
(författare)
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The impact of stroke on spousal and family income : a difference-in-difference study from Swedish national registries
- 2024
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Ingår i: Topics in Stroke Rehabilitation. - : Taylor & Francis. - 1074-9357 .- 1945-5119. ; 31:4, s. 381-389
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Tidskriftsartikel (refereegranskat)abstract
- AimTo investigates the financial consequences in the overall population spouses of persons with stroke in Sweden as well as for subgroups based on spouses age, sex and modified Rankin Scale (mRS) of the person with stroke.MethodsThe study population consists of spouses aged <= 60 during the year of their partner's stroke event. Each spouse was matched to four reference individuals. This longitudinal registry data covers spouses and a reference population between 2005 and 2016. We use difference-in-differences to estimate the impact on individual income from paid work, disposable individual income, and disposable family income.ResultsThe primary analysis shows a small and statistically insignificant decrease on spouses' individual income from paid work and disposable individual income. In the subgroup analysis based on mRS, the largest effect is seen in mRS 4-5, where spouses' individual income from paid work and disposable individual income increases after their partner's stroke. Further, younger female spouses' individual income from paid work decreases by 1 614 EUR (p = 0.008) on average.ConclusionThe financial consequences are small in the overall population of spouses. However, for some subgroups, younger women, and spouses of persons with stroke and mRS 4-5, the financial consequences are more prominent.
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| 5. |
- Ramadani, Royasia Viki, et al.
(författare)
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Effects of the COVID-19 pandemic on healthcare utilization among older adults with cardiovascular diseases and multimorbidity in Indonesia: an interrupted time-series analysis.
- 2024
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Ingår i: BMC public health. - : BioMed Central (BMC). - 1471-2458. ; 24:1
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Tidskriftsartikel (refereegranskat)abstract
- The COVID-19 pandemic has disrupted healthcare utilization globally, but little is known about the effects among patients with cardiovascular diseases (CVDs) and other multimorbidities. This study analyzed the impacts of COVID-19 on healthcare utilization for patients aged 30 years and older with cardiovascular diseases (CVDs) with or without other chronic disease comorbidities in Indonesia.We designed a retrospective cohort study based on the Indonesian National Health Insurance (NHI) sample data from 2016-2020. We defined healthcare utilization as monthly outpatient and inpatient visits related to chronic diseases at the hospital and primary healthcare levels per 10,000 NHI members. We used interrupted time series analysis to evaluate how the healthcare utilization patterns had changed due to the COVID-19 pandemic.Overall, hospital outpatient visits decreased by 39% when the pandemic occurred (95% Confidence Interval (CI): 0.48,0.76), inpatient visits by 28% (95% CI: 0.62,0.83), and primary healthcare visits by 34% (95% CI:0.55, 0.81). For patients with CVDs and multimorbidity, hospital outpatient and inpatient visit rates were reduced by 36% and 38%, respectively and primary healthcare visits by 32%. Some insignificant differences in the reduction of out-and inpatient visits were observed across diagnosis groups and regions.Healthcare utilization among patients with chronic diseases decreased significantly during COVID-19 and consistently across different chronic diseases and regions. To cope with the unmet needs of healthcare utilization in the context of the pandemic, the healthcare system needs to be strengthened to cater to the needs of the population-at-risk, especially for patients with CVDs and multimorbidity.
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| 6. |
- Ramadani, Royasia Viki, et al.
(författare)
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The impact of multimorbidity among adults with cardiovascular diseases on healthcare costs in Indonesia : a multilevel analysis
- 2024
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Ingår i: BMC Public Health. - : BioMed Central (BMC). - 1471-2458. ; 24:1
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Tidskriftsartikel (refereegranskat)abstract
- Background: Cardiovascular diseases (CVDs) are the leading cause of death in Indonesia, accounting for 38% of the total mortality in 2019. Moreover, healthcare spending on CVDs has been at the top of the spending under the National Health Insurance (NHI) implementation. This study analyzed the association between the presence of CVDs with or without other chronic disease comorbidities and healthcare costs among adults (> 30 years old) and if the association differed between NHI members in the subsidized group (poorer) and non-subsidized households group (better-off) in Indonesia. Methods: This retrospective cohort study analyzed the NHI database from 2016–2018 for individuals with chronic diseases (n = 271,065) ascertained based on ICD-10 codes. The outcome was measured as healthcare costs in USD value for 2018. We employed a three-level multilevel linear regression, with individuals at the first level, households at the second level, and districts at the third level. The outcome of healthcare costs was transformed with an inverse hyperbolic sine to account for observations with zero costs and skewed data. We conducted a cross-level interaction analysis to analyze if the association between individuals with different diagnosis groups and healthcare costs differed between those who lived in subsidized and non-subsidized households. Results: The mean healthcare out- and inpatient costs were higher among patients diagnosed with CVDs and multimorbidity than patients with other diagnosis groups. The predicted mean outpatient costs for patients with CVDs and multimorbidity were more than double compared to those with CVDs but no comorbidity (USD 119.5 vs USD 49.1, respectively for non-subsidized households and USD 79.9 vs USD 36.7, respectively for subsidized households). The NHI household subsidy status modified relationship between group of diagnosis and healthcare costs which indicated a weaker effect in the subsidized household group (β = -0.24, 95% CI -0.29, -0.19 for outpatient costs in patients with CVDs and multimorbidity). At the household level, higher out- and inpatient costs were associated with the number of household members with multimorbidity. At the district level, higher healthcare costs was associated with the availability of primary healthcare centres. Conclusions: CVDs and multimorbidity are associated with higher healthcare costs, and the association is stronger in non-subsidized NHI households. Households’ subsidy status can be construed as indirect socioeconomic inequality that hampers access to healthcare facilities. Efforts to combat cardiovascular diseases (CVDs) and multimorbidity should consider their distinct impacts on subsidized households. The effort includes affirmative action on non-communicable disease (NCD) management programs that target subsidized households from the early stage of the disease.
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| 7. |
- Svensson, Mikael, 1980, et al.
(författare)
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Analyses of quality of life in cancer drug trials - a review of measurements and analytical choices in post-reimbursement studies
- 2024
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Ingår i: BMC Cancer. - : BioMed Central (BMC). - 1471-2407. ; 24:1
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Forskningsöversikt (refereegranskat)abstract
- Objectives: For drugs reimbursed with limited evidence of patient benefits, confirmatory evidence of overall survival (OS) and quality of life (QoL) benefits is important. For QoL data to serve as valuable input to patients and decision-makers, it must be measured and analyzed using appropriate methods. We aimed to assess the measurement and analyses of post-reimbursement QoL data for cancer drugs introduced in Swedish healthcare with limited evidence at the time of reimbursement. Methods: We reviewed any published post-reimbursement trial data on QoL for cancer drugs reimbursed in Sweden between 2010 and 2020 with limited evidence of improvement in QoL and OS benefits at the time of reimbursement. We extracted information on the instruments used, frequency of measurement, extent of missing data, statistical approaches, and the use of pre-registration and study protocols. Results: Out of 22 drugs satisfying our inclusion criteria, we identified published QoL data for 12 drugs in 22 studies covering multiple cancer types. The most frequently used QoL instruments were EORTC QLQ-C30 and EQ-5D-3/5L. We identified three areas needing improvement in QoL measurement and analysis: (i) motivation for the frequency of measurements, (ii) handling of the substantial missing data problem, and (iii) inclusion and adherence to QoL analyses in clinical trial pre-registration and study protocols. Conclusions: Our review shows that the measurements and analysis of QoL data in our sample of cancer trials covering drugs initially reimbursed without any confirmed QoL or OS evidence have significant room for improvement. The increasing use of QoL assessments must be accompanied by a stricter adherence to best-practice guidelines to provide valuable input to patients and decision-makers.
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| 8. |
- Baldvinsdóttir, Bryndís, et al.
(författare)
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Adverse events during endovascular treatment of ruptured aneurysms : A prospective nationwide study on subarachnoid hemorrhage in Sweden
- 2023
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Ingår i: BRAIN AND SPINE. - : Elsevier. - 2772-5294. ; 3
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Tidskriftsartikel (refereegranskat)abstract
- Introduction: A range of adverse events (AEs) may occur in patients with subarachnoid hemorrhage (SAH). Endovascular treatment is commonly used to prevent aneurysm re-rupture.Research question: The aim of this study was to identify AEs related to endovascular treatment, analyze risk factors for AEs and how AEs affect patient outcome.Material and methods: Patients with aneurysmal SAH admitted to all neurosurgical centers in Sweden during a 3.5-year period (2014-2018) were prospectively registered. AEs related to endovascular aneurysm treatment were thromboembolic events, aneurysm re-rupture, vessel dissection and puncture site hematoma. Potential risk factors for the AEs were analyzed using multivariate logistic regression. Functional outcome was assessed at one year using the extended Glasgow outcome scale.Results: In total, 1037 patients were treated for ruptured aneurysms. Of which, 715 patients were treated with endovascular occlusion. There were 115 AEs reported in 113 patients (16%). Thromboembolic events were noted in 78 patients (11%). Aneurysm re-rupture occurred in 28 (4%), vessel dissection in 4 (0.6%) and puncture site hematoma in 5 (0.7%). Blister type aneurysm, aneurysm smaller than 5 mm and endovascular techniques other than coiling were risk factors for treatment-related AEs. At follow-up, 230 (32%) of the patients had unfavorable outcome. Patients suffering intraprocedural aneurysm re-rupture were more likely to have unfavorable outcome (OR 6.9, 95% CI 2.3-20.9).Discussion and conclusion: Adverse events related to endovascular occlusion of a ruptured aneurysm were seen in 16% of patients. Aneurysm re-rupture during endovascular treatment was associated with increased risk of unfavorable functional outcome.
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| 9. |
- Chauca Strand, Gabriella, 1995, et al.
(författare)
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Cancer Drugs Reimbursed with Limited Evidence on Overall Survival and Quality of Life : Do Follow-Up Studies Confirm Patient Benefits?
- 2023
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Ingår i: Clinical drug investigation. - : Springer. - 1173-2563 .- 1179-1918. ; 43:8, s. 621-633
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Tidskriftsartikel (refereegranskat)abstract
- Background and ObjectiveCancer drug costs have increased considerably within healthcare systems, but many drugs lack quality-of-life (QoL) and overall survival (OS) data at the time of reimbursement approval. This study aimed to review the extent of subsequent literature documenting improvements in OS and QoL for cancer drug indications where no such evidence existed at the time of reimbursement approval.MethodsDrug indications with claims of added therapeutical value but a lack of evidence on OS and QoL that were reimbursed between 2010 and 2020 in Sweden were included for review. Searches were conducted in PubMed and ClinicalTrial.gov for randomized controlled trials examining OS and QoL.ResultsOf the 22 included drug indications, seven were found to have at least one trial with conclusive evidence of improvements in OS or QoL after a mean follow-up of 6.6 years. The remaining 15 drug indications either lacked subsequent randomized controlled trial data on OS or QoL (n = 6) or showed no statistically significant improvements (n = 9). Only one drug demonstrated evidence of improvement in both OS and QoL for its indication.ConclusionsA considerable share of reimbursed cancer drug indications continue to lack evidence of improvement in both OS and QoL. With limited healthcare resources and an increasing cancer burden, third-party payers have strong incentives to require additional post-reimbursement data to confirm any improvements in OS and QoL.
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| 10. |
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