| 1. |
- Campbell, C., et al.
(författare)
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Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy
- 2020
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Ingår i: Journal of Comparative Effectiveness Research. - : Becaris Publishing Limited. - 2042-6305 .- 2042-6313. ; 9:14
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Tidskriftsartikel (refereegranskat)abstract
- Aim:Assess the totality of efficacy evidence for ataluren in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD).Materials & methods:Data from the two completed randomized controlled trials (ClinicalTrials.gov: NCT00592553; NCT01826487) of ataluren in nmDMD were combined to examine the intent-to-treat (ITT) populations and two patient subgroups (baseline 6-min walk distance [6MWD] >= 300-<400 or <400 m). Meta-analyses examined 6MWD change from baseline to week 48.Results:Statistically significant differences in 6MWD change with ataluren versus placebo were observed across all three meta-analyses. Least-squares mean difference (95% CI): ITT (n = 342), +17.2 (0.2-34.1) m, p = 0.0473; >= 300-<400 m (n = 143), +43.9 (18.2-69.6) m, p = 0.0008; <400 m (n = 216), +27.7 (6.4-49.0) m, p = 0.0109.Conclusion:These meta-analyses support previous evidence for ataluren in slowing disease progression versus placebo in patients with nmDMD over 48 weeks. Treatment benefit was most evident in patients with a baseline 6MWD >= 300-<400 m (the ambulatory transition phase), thereby informing future trial design.
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| 2. |
- Makady, Amr, et al.
(författare)
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Practical implications of using real-world evidence (RWE) in comparative effectiveness research : learnings from IMI-GetReal
- 2017
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Ingår i: Journal of Comparative Effectiveness Research. - : Becaris Publishing Limited. - 2042-6305 .- 2042-6313. ; 6:6, s. 485-490
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Tidskriftsartikel (övrigt vetenskapligt/konstnärligt)abstract
- In light of increasing attention towards the use of real-world evidence (RWE) in decision making in recent years, this commentary aims to reflect on the experiences gained in accessing and using RWE for comparative effectiveness research as a part of the Innovative Medicines Initiative GetReal Consortium and discuss their implications for RWE use in decision-making.
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| 3. |
- Martin, Andrew, et al.
(författare)
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Measures to improve angiotensin receptor blocker prescribing efficiency in the UK : findings and implications
- 2014
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Ingår i: Journal of Comparative Effectiveness Research. - : Becaris Publishing Limited. - 2042-6305 .- 2042-6313. ; 3:1, s. 41-51
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Tidskriftsartikel (refereegranskat)abstract
- Background: Generic losartan provides an opportunity to enhance angiotensin receptor blocker (ARB) prescribing efficiency, with all ARBs essentially being similar. Initially, there was limited activity in NHS Bury (UK). This changed in March 2011 with therapeutic switching and other measures encouraging the prescribing of losartan following generics to enhance its utilization versus patented ARBs. Aim: This study aims to assess the impact of multiple measures on losartan utilization, its price and total ARB expenditure. Methods: An interrupted time series analysis was performed. Utilization was measured as prescription items dispensed, typically 28 days. Results: No immediate change in losartan utilization was observed following generics. This changed after the multiple initiatives with losartan accounting for 65% of all single ARB items dispensed by the study end. ARB expenditure was 59% below prestudy levels by the study end, which was helped by a 92% reduction in expenditure per item for losartan. Annual net savings from the program were estimated at just under GB 290,000 pound, which is over eight-times the cost of implementation. Conclusion: Multiple measures can enhance prescribing efficiency. Health authorities cannot rely on a spillover' effect from other classes in order to affect changes in physician prescribing habits.
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| 4. |
- McDonald, C. M., et al.
(författare)
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Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients
- 2022
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Ingår i: Journal of Comparative Effectiveness Research. - : Becaris Publishing Limited. - 2042-6305 .- 2042-6313. ; 11:3
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Tidskriftsartikel (refereegranskat)abstract
- Aim: We investigated the effect of ataluren plus standard of care (SoC) on age at loss of ambulation (LoA) and respiratory decline in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD) versus patients with DMD on SoC alone. Patients & methods: Study 019 was a long-term Phase III study of ataluren safety in nmDMD patients with a history of ataluren exposure. Propensity score matching identified Study 019 and CINRG DNHS patients similar in disease progression predictors. Results & conclusion: Ataluren plus SoC was associated with a 2.2-year delay in age at LoA (p = 0.0006), and a 3.0-year delay in decline of predicted forced vital capacity to <60% in nonambulatory patients (p = 0.0004), versus SoC. Ataluren plus SoC delays disease progression and benefits ambulatory and nonambulatory patients with nmDMD. ClinicalTrials.gov: .
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| 5. |
- Mercuri, E., et al.
(författare)
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Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study
- 2020
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Ingår i: Journal of Comparative Effectiveness Research. - : Becaris Publishing Limited. - 2042-6305 .- 2042-6313. ; 9:5, s. 341-360
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Tidskriftsartikel (refereegranskat)abstract
- Aim: Strategic Targeting of Registries and International Database of Excellence (STRIDE) is an ongoing, multicenter registry providing real-world evidence regarding ataluren use in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). We examined the effectiveness of ataluren + standard of care (SoC) in the registry versus SoC alone in the Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study (DNHS), DMD genotype-phenotype/-ataluren benefit correlations and ataluren safety. Patients & methods: Propensity score matching was performed to identify STRIDE and CINRG DNHS patients who were comparable in established disease progression predictors (registry cut-off date, 9 July 2018). Results & conclusion: Kaplan-Meier analyses demonstrated that ataluren + SoC significantly delayed age at loss of ambulation and age at worsening performance in timed function tests versus SoC alone (p <= 0.05). There were no DMD genotype-phenotype/ataluren benefit correlations. Ataluren was well tolerated. These results indicate that ataluren + SoC delays functional milestones of DMD progression in patients with nmDMD in routine clinical practice. ClinicalTrials.gov identifier: NCT02369731. ClinicalTrials.gov identifier: NCT02369731.
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| 6. |
- Merseburger, Axel S., et al.
(författare)
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Treatment costs for advanced prostate cancer using luteinizing hormone-releasing hormone agonists: a solid biodegradable leuprorelin implant versus other formulations
- 2015
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Ingår i: Journal of Comparative Effectiveness Research. - : Becaris Publishing Limited. - 2042-6305 .- 2042-6313. ; 4:5, s. 447-453
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Tidskriftsartikel (refereegranskat)abstract
- Aim: To compare treatment costs with alternative luteinizing hormone-releasing hormone (LHRH) agonist preparations and determine whether a leuprorelin solid implant is associated with potential cost savings. Patients & methods: A hypothetical population of 1000 prostate cancer patients was apportioned between the three most commonly-prescribed LHRH agonist preparations. Differentiated annual costs for 1- and 3-monthly formulations were calculated for France, Germany, Italy, Spain, the UK (EU5) and Sweden, and compared with the leuprorelin solid implant. Results: Compared with alternative formulations, leuprorelin solid implants had potential annual cost savings/1000 patients of Euro353,000 (EU5) and Euro699,000 (Sweden; 1-month formulations), and Euro259,000 (EU5) and Euro300,000 (Sweden; 3-month formulations). Conclusion: The leuprorelin solid implant was associated with potential cost savings compared with the most commonly used LHRH agonist preparations.
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| 7. |
- Muntoni, F., et al.
(författare)
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Ataluren use in patients with nonsense mutation Duchenne muscular dystrophy: patient demographics and characteristics from the STRIDE Registry
- 2019
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Ingår i: Journal of Comparative Effectiveness Research. - : Becaris Publishing Limited. - 2042-6305 .- 2042-6313. ; 8:14, s. 1187-1200
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Tidskriftsartikel (refereegranskat)abstract
- Aim: Strategic Targeting of Registries and International Database of Excellence (STRIDE) is an ongoing, multicenter registry providing real-world evidence regarding ataluren use in patients with nonsense mutation Duchenne muscular dystrophy (DMD) in clinical practice (NCT02369731). Here, we describe the initial demographic characteristics of the registry population. Patients & methods: Patients will be followed up from enrollment for >= 5 years or until study withdrawal. Results & conclusion: As of 9 July 2018, 213 DMD boys were enrolled from 11 countries. Mean (standard deviation) ages at first symptoms and at study treatment start were 2.7 (1.7) years and 9.8 (3.7) years, respectively. Corticosteroids were used by 190 patients (89.2%) before data cut-off. Mean (standard deviation) ataluren exposure was 639.0 (362.9) days. Six patients withdrew. STRIDE is the first drug registry for patients with DMD and represents the largest real-world registry of patients with nmDMD to date.
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| 8. |
- Vroman, Heleen, et al.
(författare)
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Continuous vital sign monitoring in patients after elective abdominal surgery: a retrospective study on clinical outcomes and costs
- 2023
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Ingår i: Journal of Comparative Effectiveness Research. - : Becaris Publishing. - 2042-6305 .- 2042-6313. ; 12:2
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Tidskriftsartikel (refereegranskat)abstract
- Plain language summary What is this article about?Continuous vital sign monitoring assists in identifying deteriorating patients outside intensive care. This study analyses changes in clinical outcomes and costs before and after implementation of a clinical vital sign monitoring device in postsurgical patients on a general ward in a Dutch hospital. What were the results?Results show that after implementation, patients were less likely to be admitted to the intensive care unit, had shorter length of stay and had lower hospital stay costs. Aim: To assess changes in outcomes and costs upon implementation of continuous vital sign monitoring in postsurgical patients. Materials & methods: Retrospective analysis of clinical outcomes and in-hospital costs compared with a control period. Results: During the intervention period patients were less frequently admitted to the intensive care unit (ICU) (p = 0.004), had shorter length of stay (p < 0.001) and lower costs (p < 0.001). The intervention was associated with a lower odds of ICU admission (odds ratio: 0.422; p = 0.007) and ICU related costs (odds ratio: -662.4; p = 0.083). Conclusion: Continuous vital sign monitoring may have contributed to fewer ICU admissions and lower ICU costs in postsurgical patients. Tweetable abstractA retrospective study of clinical outcomes and costs compared with a control period indicated that implementation of continuous vital sign monitoring in postsurgical patients in a general hospital in The Netherlands may have contributed to fewer intensive care unit admissions and lower intensive care unit costs.
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