This document is for consultation only and does not constitute the Institute's final guidance to the National Health Service (NHS). However, if implemented without any revisions, these proposals will significantly limit the prescribing options for pulmonary hypertension. This will be of particular concern to those patients in the more advanced stages of primary pulmonary hypertension (PPH), which is a very rare, incurable and relentlessly progressive disease. In the event that their disease progresses to the point where oral therapy can no longer control symptoms or cannot be tolerated, patients with PPH would have no alternative disease-specific drug treatment available to them on the NHS.
Primary pulmonary hypertension is defined by NICE as an ultra-orphan disease (i.e. very rare, occurring in less than 1,000 people in the UK)(i) and before the advent of targeted therapies for PPH, when only symptomatic treatments were available, the median survival after diagnosis was only 2.8 years.
In UK clinical practice, patients with PPH normally begin with symptomatic treatment alone. As the disease progresses, the patient will require disease-specific oral therapies and will eventually need a prostanoid in order to control the advancement of the disease and maintain quality of life. Prostanoids can be inhaled or given by intravenous infusion. The appraisal consultation document from NICE proposes that patients are to be denied access to prostanoid therapy and that other therapeutic choices are limited to one first line oral therapy (sildenafil) and two alternative oral therapies (bosentan and sitaxentan), for patients in whom sildenafil is contraindicated or poorly tolerated.
Ventavis(R) (inhaled iloprost) has a vital role in bridging the gap between oral therapy alone and the need for continuous IV prostanoids (e.g.
epoprostenol). It is not intended as a replacement for oral therapies.
Ventavis(R) offers advantages over IV therapy in respect to convenience and tolerability and avoids both tachyphylaxis (large increases in dose, therefore cost, over time) and the risks associated with the IV route of administration.
The appraisal of Ventavis(R) by NICE omits two important considerations:
1. It does not evaluate the subgroup in which Ventavis(R) is actually used in current UK clinical practice (i.e. in patients who have failed oral agents and otherwise require IV epoprostenol).
2. It does not take into account either the ultra-orphan status of PPH or the ultra-orphan drug status of Ventavis(R) (by EMEA criteria(ii)), even though a draft discussion paper produced by NICE in 2006(i) suggests there is a case for not applying the normal cost-effectiveness thresholds in the appraisal of ultra-orphan diseases and specifically references iloprost in PPH as an example.
About Pulmonary Hypertension
Pulmonary hypertension is a rare, long-term disease that affects people of all ages and races. The cause of it is unknown and there is no cure.
However, there are several treatments which may help and information is provided on these later.
"Pulmonary" means the disease is in the lungs. "Hypertension" is another word for high blood pressure. In pulmonary hypertension it is harder for blood to flow to the lungs and pressure builds up in the pulmonary artery.
Pressure is measured in millimetres of mercury or mm Hg. In healthy people who are resting, the pressure in the pulmonary artery is about 14 mm Hg. In pulmonary hypertension, pressure in the pulmonary artery rises above 25 mm Hg. This means the heart has to work harder and pump faster than normal to get blood to the lungs and the rest of the body.
The heart muscle and right ventricle may get bigger than normal, to cope with the extra work. With time the right ventricle may become weak and unable to pump enough blood. Eventually the heart may fail completely.
Pulmonary hypertension can make the patient feel tired. Although, many patients ignore the tiredness at first because they think it is a result of not being particularly fit. They are likely to have difficulty in breathing or feel like they are not getting enough air. This is called dyspnoea or shortness of breath. The patient may experience dizzy spells and fainting, which is called syncope. The lips and skin may appear bluer than normal (cyanosis) and sometimes the patient will experience pain in their chest (angina).
These are all common symptoms of pulmonary hypertension that happen because there is not enough blood going to the lungs collecting oxygen for the rest of the body. Also, ankles or legs may swell (oedema) because blood is not being pumped around the body properly, so fluid collects in the legs and ankles, especially if the patient stays on their feet for a long time.
(i) Appraising Orphan Drugs, National Institute for Health and Clinical Excellence, 16 March 2006.
(ii) COMP Report to the Commission in relation to Article 10 of Regulation 141/2000 on orphan medicinal products, European Medicines Agency (EMEA),
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