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In provisional draft guidance published today (5 August) the National Institute for Health and Clinical Excellence (NICE) does not recommend that a drug, licensed to treat people who have a particular type of multiple sclerosis, should be prescribed on the NHS. This is because there are uncertainties over its clinical effectiveness and, based on the available evidence, it would not be a cost effective use of NHS resources.

While clinical trials have shown that fingolimod (also called Gilenya) can reduce the number of relapses in some people who have highly active relapsing-remitting multiple sclerosis (RRMS), it is unclear how much the drug may help the specific groups of people for whom it is licensed - i.e. adults with RRMS who experience at least one relapse in a year despite being treated with beta interferons, and adults with rapidly evolving severe RRMS who experience two or more disabling relapses regardless of their treatment. The evidence submitted by its manufacturer (Novartis Pharmaceuticals UK) mainly looked at a subgroup of the former.

As well as this, NICE's independent committee of experts could not determine how much fingolimod reduces the rate of relapses compared with some of the treatments already available. This is because Novartis only submitted evidence which compared fingolimod with a placebo, and fingolimod with a specific type of a beta interferon (beta interferon 1a, also called Avonex) that is not believed to be widely prescribed in the NHS.

NICE's independent committee believes that Novartis should have submitted a comparison of fingolimod with other beta interferons for people with highly-active RRMS, as well of fingolimod with natalizumab (Tysabri, Biogen) for the subgroup of people with rapidly evolving severe RRMS. NICE has advised the NHS to consider natalizumab as a treatment option for people with rapidly evolving severe RRMS since 2007. For NICE to recommend a drug for use on the NHS, the evidence has to demonstrate that it is a good use of resources; e.g. that it works at least as well or better than treatments already available and that it is cost effective.

Professor Carole Longson, Director of the Health Technology Evaluation Centre at NICE said: “While it's important that people with multiple sclerosis have treatment options, NICE has to ensure that the NHS provides treatments that bring benefits that are value for money. This is so that everyone who uses the NHS can receive the best care possible.

“Unfortunately our independent committee wasn't given sufficient evidence to show that fingolimod could reduce relapses considerably better than the other treatments currently being used. Based on the available clinical evidence and economic analysis, our independent committee concluded that fingolimod would not be effective good use of NHS resources.

“We encourage healthcare professionals, people with MS and our other relevant third parties to comment on our committee's provisional recommendation so that they can contribute to the development of this guidance.”

Those wishing to comment on NICE's draft recommendations have until 26 August 2011 to do so. NICE's independent committee will meet again in October to review the comments received. NICE then expects to publish its final guidance for the NHS in December 2011.

NICE's final guidance will determine whether or not the NHS is legally obliged to allocate funding for fingolimod in RRMS. Until NICE issues final guidance, decisions on the funding of fingolimod should continue to be made locally by NHS bodies.

Notes to Editors

About the draft guidance (appraisal consultation document)

1. For further information about the appraisal consultation document of “Fingolimod for the treatment of relapsing-remitting multiple sclerosis”, visit: http://guidance.nice.org.uk/TA/Wave20/71. Contact the press office for embargoed copies.

2. Highly active relapsing-remitting multiple sclerosis (RRMS) is generally defined by at least one relapse in previous year while on therapy (such as with beta interferon) and at least nine T2-hyperintense lesions detected by brain MRI or at least one gadolinium-enhancing lesion.

3. Rapidly evolving severe RRMS is generally defined by two or more disabling relapses in one year, and one or more gadolinium-enhancing lesions detected by brain MRI or a significant increase in T2 lesion load compared with a previous MRI.

4. Fingolimod (Gilenya, Novartis Pharmaceuticals UK) is an oral medicine for highly-active relapsing remitting multiple sclerosis. In multiple sclerosis, white blood cells (called lymphocytes) attack the coating of the nerve cells which help messages from the brain travel to the rest of the body. As these cells are damaged, people experience symptoms such as numbness and tingling, blurred vision, mobility and balance problems, and muscle weakness and tightness. Fingolimod works by preventing the lymphocytes from attacking nerve cells in the brain and spinal cord. This is why it is called a “disease modifying” medication, but it is not a cure.

5. The recommended dosage for fingolimod is 0.5mg once a day. 28 capsules cost £1470. This is equivalent to an annual cost of approximately £19,196 per person. Costs may vary in different settings because of negotiated procurement discounts. The manufacturer estimated in its base case that the Incremental Cost Effectiveness Ratio (ICER) for fingolimod compared to Avonex is £55,600 per QALY gained. The committee believed this ICER to be highly uncertain and underestimated.

6. NICE has previously recommended natalizumab for the treatment of adults with rapidly evolving severe relapsing-remitting multiple sclerosis. For further information, visit: www.nice.org.uk/TA127

7. In 2002, NICE published guidance which did not recommend the use of beta interferons or glatiramer acetate for relapsing remitting multiple sclerosis. For further information about this technology appraisal, visit: www.nice.org.uk/TA32. After this guidance was issued, the Department of Health agreed a patient access scheme with manufacturers, which encouraged these disease-modifying treatments to be offered to patients on the NHS under certain conditions. For further information about this patient access scheme, please contact the Department of Health press office.

About NICE

8. The National Institute for Health and Clinical Excellence (NICE) is the independent organisation responsible for providing national guidance and standards on the promotion of good health and the prevention and treatment of ill health

9. NICE produces guidance in three areas of health:

• public health - guidance on the promotion of good health and the prevention of ill health for those working in the NHS, local authorities and the wider public and voluntary sector
• health technologies - guidance on the use of new and existing medicines, treatments, medical technologies (including devices and diagnostics) and procedures within the NHS
• clinical practice - guidance on the appropriate treatment and care of people with specific diseases and conditions within the NHS

10. NICE produces standards for patient care:

• quality standards - these reflect the very best in high quality patient care, to help healthcare practitioners and commissioners of care deliver excellent services
• Quality and Outcomes Framework - NICE develops the clinical and health improvement indicators in the QOF, the Department of Health scheme which rewards GPs for how well they care for patients

11. NICE provides advice and support on putting NICE guidance and standards into practice through its implementation programme, and it collates and accredits high quality health guidance, research and information to help health professionals deliver the best patient care through NHS Evidence.