Leading experts on an inherited condition causing dangerously high levels of cholesterol have come together to pile pressure on NHS England to finally commission Aegerion Pharmaceuticals' lomitapide after years of delay.
Homozygous familial hypercholesterolaemia (HoFH) is 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.
It is a very rare condition with an estimated prevalence of 3/million population and, based on recently conducted surveys, it is estimated there are less than 100 people in the UK currently diagnosed and treated with HoFH. Without treatment, the average life expectancy is just 18 years.
Lomitapide works differently to treatments like statins and PCSK9-inhibitors, and boosts standard treatment for HoFH including apheresis. In clinical trials, the drug reduced levels of LDL-C by an additional 40-50 percent and enabled 51 percent to achieve the target of <2.5mmol/L at least once in weeks 0-26 and 74% in weeks 0-246. Almost 50% of patients were able to either stop or reduce frequency of apheresis, further highliting its effectiveness.
Lomitapide was cleared by the European Commission in July 2013 as an adjunct to other lipid lowering therapies for the treatment of adults with HoFH, but the drug is still not available to patients in England because of continuing delays in NHS England issuing a policy for 2016 commissioning.
NHS England is in the process of developing a commissioning policy, but lomitapide would still need to pass a policy prioritisation round via the Clinical Priorities Advisory Group process before patients can benefit. HoFH experts fear that inclusion in this year's CPAG meeting "is now in question, and likely to be delayed until next year", further delaying access for patients.
"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.