NHS under fire for delaying access to rare disease cholesterol drug

England’s National Health Service budget holders has come under fire for delaying access to a drug for a rare genetic disease which begins in childhood and causes dangerously high levels of blood cholesterol.

Homozygous familial hypercholesterolaemia (HoFH) has a prevalence of only three per million population, and there are less than 100 people in the UK diagnosed and treated with the condition.

Prognosis is poor, as patients can develop cardiovascular disease in early childhood, and without treatment average life expectancy is just 18 years.

The concerns raised about NHS England’s decision making add to growing disquiet among the industry and patient groups about how the health service budget holder makes decisions on specialised medicines.

An NHS England meeting next month will decide on whether to recommend funding for drug treatment. Experts in HoFH are calling for NHS England to make Amgen’s Repatha (evolocumab) and Aegerion’s Lojuxta (lomitapide) available.

Aegerion and specialist doctors are calling on NHS England to include Lojuxta in its Clinical Priorities Advisory Group (CPAG) meeting next month, which will recommend specialised services treatments to fund, or to issue an in year policy.

But publication of a policy proposition on Lojuxta has been delayed, and it looks likely that it will not be considered by the CPAG.

Lojuxta was approved in Europe in July 2013 as an adjunct to other lipid lowering therapies (HoFH) but is still not available on the NHS. Repatha was approved last year. According to a statement released by Aegerion, patients treated with apheresis have a life expectancy of 45-48 years, compared with the UK average of 81.5 years.

Professor Gilbert Thompson, emeritus professor of clinical lipidology at Imperial College, London, noted recently published data show lipoprotein apheresis and/or high dose statins plus Merck, Sharp and Dohme’s Ezetrol (exetimibe) increase life expectancy to a limited extent.

However if European Atherosclerosis Society targets for LDL-C are to be achieved, Thompson said recently approved drugs such as Repatha and Lojuxta need to be available.

He said: “I urge NHS England to commission both these additional HoFH treatments without further delay.”

Professor James Scott, professor of medicine and cardiovascular medicine at Imperial College London and Hammersmith Hospital said the benefits of apheresis and Lojuxta would be “a big deal for patients.”

Aegerion UK general manager, Julian Trimming, said: “Aegerion has followed all the processes set out by NHS England and hope they can propose a solution for lomitapide to be included in this year’s funding.”

The CPAG makes recommendations on funding priorities, based on feedback from Clinical Reference Groups within the health service. Final decisions on funding for specialised services are taken by the Specialised Commissioning Committee, a sub group of the NHS England board.

NHS England says the list price of the drug is very high – upwards of £200,000 and that the drug doesn’t cure the condition. It says this means it would represent a “significant and recurring call” on its specialised commissioning budget.

Many in the pharma industry are concerned by the fairness and transparency of how NHS England runs its Prioritisation Framework. Furthermore, a newly updated decision-making system is likely to make funding even harder to secure, reflecting an unprecedented squeeze on NHS budgets.  A wave of new specialised drugs, including cancer and rare disease drugs are adding even greater pressure on budgets. Last month a report by the National Audit Office found growth in spending on specialised services presents an ‘ongoing risk to NHS financial stabiliity’.

“The lack of a functional NHS England process to commission vital treatments for rare diseases is not fit for purpose and the continuing delays in access to lomitapide proposed by NHS England would limit our ability to provide an optimal care to our patients, especially when such treatments are accessible elsewhere,” noted Dr Handrean Soran, from Central Manchester University Hospitals NHS Foundation Trust.

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