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Article
Open AccessIssues, Challenges and Opportunities for Economic Evaluations of Orphan Drugs in Rare Diseases: An Umbrella Review
There are significant challenges when obtaining clinical and economic evidence for health technology assessments of rare diseases. Many of them have been highlighted in previous systematic reviews but they hav...
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Article
Open AccessCost Effectiveness of Difelikefalin Compared to Standard Care for Treating Chronic Kidney Disease Associated Pruritus (CKD-aP) in People with Kidney Failure Receiving Haemodialysis
Chronic kidney disease-associated pruritus (CKD-aP) is associated with an increased risk of depression, poor sleep and reduced health-related quality of life. Two phase III studies (KALM-1 and KALM-2) of difel...
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Article
Living Health Technology Assessment: Issues, Challenges and Opportunities
Health technology assessments (HTAs) are typically performed as one-off evaluations and can potentially become out-of-date due to the availability of new data, new comparators, or other factors. Recently, livi...
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Article
Open AccessENTIMOS: A Discrete Event Simulation Model for Maximising Efficiency of Infusion Suites in Centres Treating Multiple Sclerosis Patients
Improved multiple sclerosis (MS) diagnosis and increased availability of intravenous disease-modifying treatments can lead to overburdening of infusion centres. This study was focused on develo** a decision-...
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Article
Open AccessCost effectiveness of nusinersen for patients with infantile-onset spinal muscular atrophy in US
Patients with infantile-onset spinal muscular atrophy (SMA), a rare, genetic neuromuscular disease, do not achieve key motor function milestones (e.g., sitting) and have short life expectancy in the absence of...
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Article
Open AccessStakeholder involvement in Multi-Criteria Decision Analysis
This brief perspective highlights the importance of decision maker buy-in and ownership through stakeholder engagement in the co-construction of the multi-criteria decision analysis (MCDA) model. A brief histo...
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Article
Open AccessIncorporating MCDA into HTA: challenges and potential solutions, with a focus on lower income settings
Multicriteria decision analysis (MCDA) has the potential to bring more structure and transparency to health technology assessment (HTA). The objective of this paper is to highlight key methodological and pract...