Details of HTA project
Last updated: 15 July 2008 - Next update due: 22 July 2008
Research type: |
NICE Technology Assessment Report (TAR) |
Project title: |
Clinical and cost effectiveness of epoprostenol, iloprost, bosentan, sitaxentan, and sildenafil for the treatment of pulmonary arterial hypertension in adults: a systematic review and economic evaluation |
Project ref: |
05/24/01 |
Cost: |
This project has been commissioned by the HTA programme on behalf of the National Institute for Health and Clinical Excellence on a call-off contract basis. |
Chief Investigator : |
West Midlands Health Technology Assessment Collaboration (WMHTAC), University of Birmingham |
Start Date: |
February 2007. |
Publication date: |
January 2009. This project is at the editorial review stage. Delays in the review process can cause the forecast publication date to be delayed. |
Plain English Summary |
Pulmonary arterial hypertension (PAH) is a rare, progressive but severe condition. It involves elevated pressure in the arteries that carry blood from right heart to the lung. PAH may occur without any identifiable reasons (idiopathic PAH). It may also be associated with a diverse group of diseases such as certain diseases of the collagen tissues and congenital heart disease. If not treated, PAH leads to heart failure and eventually death. Various interventions have been used to treat patients with PAH. Some of them are for treating symptoms and conditions that are frequently associated with PAH, such as diuretics (water tablets) for swelling of limbs, anticoagulants for preventing clots in the blood vessels, inhaled oxygen to increase oxygen level in the blood, and digoxin to strengthen heart beats. As these treatments do not alter the elevated blood pressure in the lung, they are regarded as 'supportive treatments'. In addition, a group of drugs called calcium channel blockers, which are used to treat hypertension, have been found to work in a minority of patients with PAH. More recently, newer drugs have been developed that target at different potential mechanisms underlying the development of PAH. Five of these drugs have been licensed in the UK; epoprostenol, iloprost, bosentan, sitaxentan and sildenafil. The aim of this technology assessment is to evaluate whether these five drugs are effective for treating PAH, and whether the use of these drugs in addition to supportive treatments is cost-effective (good value for money) for the National Health Services (NHS). The key components of the report are: -A systematic review of randomised controlled trials (RCTs) that investigated the effectiveness of these drugs in PAH. Variations in the effectiveness between the drugs and/or between different PAH populations will be explore if evidence from RCTs permits. -A systematic review of published studies on the costs and cost-effectiveness of these drugs in PAH. -A review of the dossiers submitted to the National Institute for Health and Clinical Excellence (NICE) by the manufacturers (GlaxoSmithKline, Schering Health Care, Actelion Pharmaceuticals, Encysive and Pfizer). -A focused, model-based economic evaluation. This involves the use of mathematical and statistical methods to synthesise information on treatment costs and the impact of the treatments on patients' quality and duration of life. |
Abstract: |
-Whether epoprostenol, iloprost, bosentan, sitaxentan and sildenafil, when used within their licensed indications, are clinically effective and cost effective compared to supportive treatments (see section 4.4) in adults with PAH for whom calcium channel blockers are inappropriate or no longer effective. -Whether the interventions being considered are clinically more effective, or more cost-effective, in patients with certain subcategories of pulmonary arterial hypertension according to Venice 2003 clinical classification (see Appendix 1). -Whether significant differences in clinical and cost effectiveness exist between the interventions being considered (either used alone or in combination) when compared to each other and/or intravenous iloprost. It is likely that the assessment report will be able to address only some of the issues surrounding these decision problems for the following reasons: (1) While the Venice 2003 clinical classification provides a significantly improved framework for the diagnosis and management of PAH, it is worth highlighting that patients with PAH represent diverse populations that vary greatly in aetiology, disease progression, and prognosis. Cases being grouped under each of the Venice subcategories can still be heterogeneous in term of severity, the choice and response to treatment and prognosis. For example, within the Venice subcategory 1.3.1, scleroderma has distinct features that may warrant it being considered separately from other forms of connective tissue diseases. (2) The five interventions being considered in this technology appraisal have different routes of administration, demand on patients' self-management, speediness of action, adverse effect profile and contraindications. The selection of treatments is highly dependent on the nature of the underlying condition, clinical circumstances and patient ability and acceptance. The choice of treatment and appropriate comparators will therefore be dependent on these factors. (3) PAH is a rare condition. The number of patients included in clinical studies is relatively small. There is unlikely to be sufficient data to allow meaningful comparison between many of the subcategories of PAH and between different treatments (or combinations of treatments). Bearing these in mind, the assessment group intends to undertake a systematic review of randomised controlled trials (RCTs) and a review of industry submissions to establish the underlying evidence base that is available to answer the above decision problems and to highlight issues that are unlikely to be addressed due to paucity of evidence. A model-based economic evaluation will then be carried out to address refined and focused decision problem(s) that take into account the availability of evidence, the appropriateness of combining different populations of PAH in terms of underlying cause (e.g. whether the model can include all PAH populations or the modelling can be reasonably done only for a specific population according to the evidence), disease severity (e.g. it may be necessary to model patients in functional class III and IV separately), and the most appropriate place in the treatment pathway for each of the interventions being considered (e.g. oral treatments would not be considered as alternative, competing interventions against intravenous epoprostenol for patients in NYHA/WHO functional class IV). THE HTA PROGRAMME COMMISSIONED THIS TECHNOLOGY ASSESSMENT REPORT ON BEHALF OF THE NATIONAL INSTITUTE FOR HEALTH AND CLINICAL EXCELLENCE |
NRR* number, if applicable: |
N0484190637 (*National Research Register) |
Project Protocol: |
Project protocol not available |
URL of this page: |
http://www.hta.ac.uk/1621 |
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