Impact case study database

An estimated 2,500,000 people in the world have multiple sclerosis (MS Trust), which affects >100,000 people in the UK and is the commonest neurological cause of disability in adults. University of Cambridge research has led to the following impacts:

Impact on policy/guidelines

In 2014, Coles provided evidence and gave expert advice during the NICE alemtuzumab approval process [A]. Chief Executive Sir Andrew Dillon said, “We are very pleased to be able to recommend alemtuzumab for adults with relapsing-remitting multiple sclerosis. Evidence has shown that alemtuzumab is more effective and less expensive than current similar treatments for those with severe relapsing-remitting MS. The NICE Committee heard from clinical specialists and patients during the appraisal process who described alemtuzumab as a revolutionary treatment for some people, allowing them to live their lives as they had before their diagnosis” [B].

Alemtuzumab is now prominent in national treatment guidelines for multiple sclerosis in the UK, including the Association of British Neurologists guidance (listed as a high-efficacy drug under recommendations for starting disease-modifying treatment), the NHS England treatment algorithm, the European Committee for Research and Treatment of Multiple Sclerosis and the European Academy of Neurology guideline and the American Academy of Neurology Practice [C].

In 2019 new safety concerns about alemtuzumab emerged, and the European Medicines Agency temporarily restricted the use of alemtuzumab pending a review. Coles advised Sanofi and gave evidence to the Committee for Medicinal Products for Human Use in December 2019, following which the restrictions were lifted. The new recommended guidance was issued in February 2020 for safe use of alemtuzumab [D]

Impact on health

If multiple sclerosis is not treated, or lower efficacy drugs are used, most patients accumulate disability. However, treatment with alemtuzumab can lead to disability improvement. Data from the latest extension of the pivotal trials (n=c.800, reported at the 2019 European Committee for Treatment and Research in Multiple Sclerosis meeting) shows that nine years after starting alemtuzumab, 49% of patients have improved disability [2].

The most important outcome for people with multiple sclerosis is prevention of the secondary progressive phase of the disease, which occurs 15-25 years after disease onset. Cambridge research showed that first-line treatment with alemtuzumab lowers the risk of developing secondary progressive disease over 8 years from 41%, if untreated, to 21%. Of patients treated with alemtuzumab or two other powerful drugs (fingolimod, natalizumab), 16% had entered the secondary progressive phase at 9 years, compared to 27% on interferon-beta. Therefore, of the approximately 25,000 patients currently treated with alemtuzumab, 2,750 people will have been prevented from developing secondary progressive multiple sclerosis, compared to standard treatment. In accord with this, quality of life measures improve with alemtuzumab treatment of multiple sclerosis (see below) [E].

Alemtuzumab is very convenient for patients, who require five daily infusions at baseline and three daily infusions a year later. Over the eight years since its use in patient treatment, approximately half of the 369 patients originally enrolled in trials have required no further treatment [F]. There are potentially serious side-effects of alemtuzumab, but most can be managed safely.

As of November 2019, over 25,000 people with MS have been treated with alemtuzumab [Sanofi] and the drug is licensed and reimbursed over 70 countries worldwide [G]. The greatest use is in Europe and Northern America (as these are the regions with the highest incidence of multiple sclerosis). The UK is the highest prescribing country of alemtuzumab, pro rata.

Commercial impact/impact on economy

In 2012 Campath was acquired by Sanofi, a global pharmaceutical company. In September 2013, alemtuzumab was licensed in the UK since when it has been licensed in over 70 countries, including the EU and United States [F]. Since licensing, annual sales of alemtuzumab have steadily increased, generating net sales income between 1^st January 2014 and 31^st December 2019 of EUR1.859 billion [H].

Alemtuzumab, and the other high efficacy drugs, double the chance of improvements in amount of work, work attendance and work productivity by people with multiple sclerosis [I]. Alemtuzumab is an expensive drug, but because it is given so infrequently and has such a prolonged action, its cost compares favourably to drugs of a similar class. In Austria, alemtuzumab use saves EUR 60-100,000 in drug costs per person treated over ten years. In the US, between USD 450-600,000 is saved for each person treated over 20 years [J].

In 2014, NICE judged that alemtuzumab was the most cost-effective treatment for MS compared with other available immunosuppressant drugs, giving incremental cost gain - of GBP13,600 - 24,500 per quality-adjusted life year (QALY) compared with glatiramer acetate and GBP8,900 per QALY compared with fingolimod, both alternative MS treatments[A]. The Norwegian Institute of Public Health found that “over a 20-year time horizon, alemtuzumab dominated all other alternative treatments, i.e. it was both more effective and less costly” [J]; the same conclusion was reached by Hamidi et al [J]. In Italy, over the lifetime of someone with multiple sclerosis, alemtuzumab is more cost effective than similar drugs and also leads to an incremental benefit of 1 QALY per person [J]. Finally, a similar study in the US showed lower cost and higher QALY gains from alemtuzumab, compared to any other MS drug [J].