-
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...
-
Article
Open AccessFlexor Injury Rehabilitation Splint Trial (FIRST): protocol for a pragmatic randomised controlled trial comparing three splints for finger flexor tendon repairs
Without surgical repair, flexor tendon injuries do not heal and patients’ ability to bend fingers and grip objects is impaired. However, flexor tendon repair surgery also requires optimal rehabilitation. There...
-
Article
Open AccessTroponin in acute chest pain to risk stratify and guide effective use of computed tomography coronary angiography (TARGET-CTCA): a randomised controlled trial
The majority of patients with suspected acute coronary syndrome presenting to the emergency department will be discharged once myocardial infarction has been ruled out, although a proportion will have unrecogn...
-
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...
-
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Health technology assessments (HTAs) are typically performed as one-off evaluations and can potentially become out-of-date due to the availability of new data, new comparators, or other factors.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...
-
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-...
-
Article
Open AccessCost-effectiveness of out-of-hospital continuous positive airway pressure for acute respiratory failure: decision analytic modelling using data from a feasibility trial
Standard prehospital management for Acute respiratory failure (ARF) involves controlled oxygen therapy. Continuous positive airway pressure (CPAP) is a potentially beneficial alternative treatment, however, it...
-
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...
-
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...
-
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...
-
Article
Open AccessThe ACUTE (Ambulance CPAP: Use, Treatment effect and economics) feasibility study: a pilot randomised controlled trial of prehospital CPAP for acute respiratory failure
Acute respiratory failure (ARF) is a common and life-threatening medical emergency. Standard prehospital management involves controlled oxygen therapy and disease-specific ancillary treatments. Continuous posi...
-
Article
Open AccessThe RAPID-CTCA trial (Rapid Assessment of Potential Ischaemic Heart Disease with CTCA) — a multicentre parallel-group randomised trial to compare early computerised tomography coronary angiography versus standard care in patients presenting with suspected or confirmed acute coronary syndrome: study protocol for a randomised controlled trial
Emergency department attendances with chest pain requiring assessment for acute coronary syndrome (ACS) are a major global health issue. Standard assessment includes history, examination, electrocardiogram (EC...
-
Article
Open AccessUsing MCDA to generate and interpret evidence to inform local government investment in public health
Smoking is the single biggest cause of preventable death in the Uited Kingdom (UK) and is a major cause of coronary heart disease, some cancers, and respiratory disease, including chronic obstructive pulmonary...
