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The objectives of the assessment report were: To assess as far as available data from randomised controlled trials (RCTs) would allow, whether the five technologies named above (alone or in combination) are clinically effective when used within their licensed indications for the treatment of adults with PAH for whom CCBs are inappropriate or no longer effective compared to supportive treatment (and/or intravenous iloprost), and whether the clinical effectiveness differs significantly between PAH of various causes.

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Make sure to read all the important safety information that comes with your treatment. Start our free online consultation for the best treatments specifically tailored to you Get a doctor-backed and personalised recommendation Join more than 100,000 happy customers who have used Manual Included under terms of UK Non-commercial Government License. A service of the National Library of Medicine, National Institutes of Health. NIHR Health Technology Assessment programme: Executive Summaries. Pulmonary arterial hypertension (PAH) is a diverse group of diseases with similar pathophysiology and clinical presentation.

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It is characterised by a progressive increase of pulmonary vascular resistance, leading to right ventricular heart failure and premature death. PAH can occur with no identifiable cause. This was previously referred to as primary pulmonary hypertension (PPH) but was renamed as idiopathic PAH (IPAH). PAH is also commonly associated with various conditions including connective tissue disease (CTD-APAH) and congenital heart disease (CHD). Symptoms of PAH include dyspnoea (breathlessness), fatigue, chest pain, syncope (fainting) and oedema, which can result in loss of capacity to perform exercise and eventually activity of daily living. To assess whether the clinical effectiveness differs significantly between the technologies (alone or in combination) if head-to-head RCTs exist.

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To assess whether each of the five technologies are cost-effective when used within their licensed indications for treating adults with PAH for whom CCBs are inappropriate or no longer effective compared to supportive treatment.

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It therefore has a devastating impact on both the quality and duration of patients' life. PAH is a rare disease with an estimated incidence of two to four cases per million per year, which approximates 100 to 200 new cases in England and Wales per year. Until the 1990s, PAH was managed by supportive treatments, which include anticoagulation therapy, diuretics, oxygen and digoxin that mainly aim at controlling symptoms. In addition, calcium channel blockers (CCBs) were found to be effective for treating a small proportion of patients with PAH. More recently, sildenafil tablet manforce 50 mg new technologies specifically licensed for treating PAH have become available in the UK. Further data were obtained from dossiers submitted to the National Institute for Health and Clinical Excellence (NICE) by the manufacturers of the technologies.

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RCTs of longer than one week duration that compared any of the five technologies (alone or in combination) to placebo, supportive care, any other technologies (alone or in combination) and/or non-licensed drugs in adult PAH patients were included.

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These include intravenous epoprostenol, inhaled iloprost, and three oral treatments: bosentan, sitaxentan and sildenafil. The licenses differ between the technologies in terms of type of PAH and severity of disease measured by functional class (FC). These technologies are believed to not only relieve symptoms but also to potentially modify disease progress. Once initiated the technologies are given repeatedly and only when inevitably the disease progresses are additional treatments or (more rarely) switching considered. The costs for these technologies vary but are very high (≈£12–£400 per patient per day, list price of drug only). Primary analysis included data from FCIII patients (and FCIV patients for epoprostenol) for licensed doses only. A systematic review of published studies on the costs and cost-effectiveness of the technologies in PAH, and a review of the dossiers submitted to NICE by the manufacturers of the technologies were undertaken.

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In addition, model-based economic evaluations of the cost-effectiveness of the technologies from the perspective of the UK National Health Service (NHS) and Personal Social Service (PSS) were carried out. A total of 20 RCTs, most of good quality, were included in this assessment.

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The majority had durations of 12 to 18 weeks and compared one of the technologies added to supportive treatment versus supportive treatment alone.

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