16 November 2021

NICE Does Not Recommend Elusulfase Alfa for NHS Use Following Evaluation of New Evidence

NICE has today (12 November 2021) published draft guidance for public consultation which does not recommend elosulfase alfa (also called Vimizin and made by BioMarin) for the treatment of mucopolysaccharidosis type 4A (also known as MPS 4A and Morquio A syndrome).

Today’s draft guidance follows an evaluation of new evidence on the use of elosulfase alfa through research and the collection of ‘real-world’ data as part of a managed access agreement which has made the treatment available on the NHS since 2015.

Clinical trial evidence and data from the managed access agreement shows some long-term benefits with elosulfase alfa treatment which suggest the condition becomes stable.

However, the independent committee concluded that the company’s cost-effectiveness modelling is not robust, or plausible, particularly because it does not adequately capture disease progression. They were also concerned about how the ‘real world’ data was used to model long-term wheelchair use, how the quality-of-life values of patients and carers were captured and the assumptions made about body weight and drug dose.

Despite offering additional quality adjusted life years (QALYs) and therefore being assessed against a maximum threshold up to £300,000 per QALY, the concerns around the modelling and its high cost mean the cost-effectiveness estimates for elosulfase alfa are much higher than NICE considers acceptable for highly specialised technologies.

Meindert Boysen, NICE deputy chief executive and director of the Centre for Health Technology Evaluation, said: “We are disappointed not to be able to recommend elosulfase alfa for use within the NHS. The length of the managed access agreement has been extended twice to allow the company more time for its submission. But despite this, there are still significant concerns with the company’s analysis and modelling. It is important to recognise this recommendation is not intended to affect those people who have begun treatment with elosulfase alfa on the NHS before this guidance was published.

“We will continue to work with the company to try to address these concerns ahead of the next committee meeting in January 2022.”

Affecting around 88 people in England, mucopolysaccharidosis type IVa is an extremely rare, life-limiting, inherited lysosomal storage disease. People born with the disease lack an enzyme – N-acetylgalactosamine-6-sulfatase – that breaks down large sugar molecules (glycosaminoglycans) the body’s cells can’t use.

The resulting accumulation of glycosaminoglycans in the cells of tissues and organs causes a wide range of symptoms that typically appear in early childhood and worsen over time. These include joint and skeletal abnormalities, hearing and vision loss, heart valve disease, pain, fatigue, and progressive loss of endurance leading to increasing dependence on wheelchairs.

MPS IVa leads to reduced life expectancy - the average life expectancy in people with this condition is about 25 years - primarily because of respiratory failure and heart problems (63% and 15% of deaths respectively).