Rare disease treatment advocates criticise NHS delays in commissioning

pharmafile | May 24, 2016 | News story | Medical Communications, Sales and Marketing NHS, commissioning 

Medical professionals are calling on NHS England to speed up access to treatment for patients with a rare genetic disease that causes premature heart disease and early death.

Homozygous familial hypercholesterolaemia (HoFH) is a rare, inherited disease characterised by highly elevated levels of low density lipoprotein cholesterol (LDL-C) in the blood, which leads to premature and progressive cardiovascular disease and early death. Currently, only around 100 people in the UK are diagnosed and treated with HoFH, with the average life expectancy only 18 years.

Lojuxta (lomitapide) was approved by the EU in July 2013 in combination to other lipid lowering therapies but it is still not available on the NHS. Made by Aegerion Pharmaceuticals, the drug has yet to make it past the commissioning stage and will now potentially not reach patients until April 2017 at the earliest.

Several advocates have urged access to this treatment sooner. Dr Robert Cramb, director of pathology and consultant chemical pathologist at University Hospital Birmingham, says: “As clinicians, we are aware of clinical outcomes improving in other countries that use best treatment and we find it difficult to explain these points to our patients for who we wish best clinical care. It is disappointing that current delays in NHS England commissioning limit our ability to treat this very small number of patients effectively.”

Professor Gilbert Thompson, emeritus professor of clinical lipidology at Imperial College London comments: “Additional therapy with recently licensed drugs such as evolocumab and lomitapide is needed if EAS targets for desirable LDL cholesterol levels are ever to be achieved in these very high risk patients. I urge NHS England to commission both these additional HoFH treatments without further delay.”

Sean Murray

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