In its final guidance, out today (25 April), NICE, the healthcare guidance body recommends that the NHS provides fingolimod (Gilenya, Novartis) - the first pill-based medicine to help reduce the number of relapses - for some adults who have highly active relapsing-remitting multiple sclerosis (RRMS).

RRMS is the most common type of the condition, estimated to affect around 27,500 people in England and Wales, and is characterised by periods when symptoms worsen and then improve. Treatments to manage relapses are generally administered by injection, whereas fingolimod is administered orally making it innovative.

Of those who have RRMS, NICE specifically recommends fingolimod for adults who have an unchanged or increased relapse rate or ongoing severe relapses compared to the previous year, despite taking beta interferons. People with RRMS who are currently receiving fingolimod but whose disease does not meet these criteria should be able to continue treatment until they and their doctors consider it appropriate to stop.

The positive recommendation only applies if Novartis (the manufacturer) provides the drug at a confidential discounted price, as proposed in its patient access scheme.

Professor Carole Longson, Director of the Health Technology Evaluation Centre at NICE said: "Our committee concluded that fingolimod is a valuable new therapy for highly active relapsing-remitting multiple sclerosis. We are pleased to recommend fingolimod as a treatment option for the specific patient population for whom it has been demonstrated to be cost effective, providing Novartis applies its proposed discount. Multiple sclerosis can be a disabling condition and so we hope that this novel treatment will help to reduce relapses for these people."

Ends

Notes to Editors

About the final guidance

1. The final guidance, Fingolimod for the treatment of relapsing-remitting multiple sclerosis, can be found here: www.nice.org.uk/TA254

2. 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.

3. The recommended dosage for fingolimod is 0.5mg once a day. The list price for 28 capsules is £1,470. This is equivalent to an annual cost of approximately £19,196 per person (without the patient access scheme).

4. The most plausible incremental cost-effectiveness ratio (ICER) for fingolimod, taking into account the patient access scheme, is likely to be between £25,000 and £35,000 per QALY gained for the specified population. The committee recognised that including all of the benefits of fingolimod which may not be adequately captured in the QALY calculation (as suggested by the manufacturer and the patient experts) could decrease the ICER to a level that would be considered a cost-effective use of NHS resources. For further information about how NICE measures cost effectiveness, please visit the cost effectiveness webpage.

5. Patient Access Schemes can be proposed by pharmaceutical companies during the development of NICE's technology appraisals in order to make drugs more affordable for the NHS. Manufacturers agree these directly with the Department of Health. Novartis has requested that the size of its proposed discount for fingolimod remains confidential.

6. NICE's technology appraisal will apply to NHS settings in England and Wales. In March 2012, the Scottish Medicines Consortium (SMC) published advice which does not recommend fingolimod for use in NHS Scotland for the treatment of highly active relapsing remitting multiple sclerosis in adults.

7. As with all of NICE's technology appraisal guidance, the recommendations regarding the use of fingolimod for highly active RRMS are in accordance with its marketing authorisation. In March 2011, the European Medicines Agency licensed fingolimod for RRMS for:

• Adults with high disease activity despite treatment with a beta interferon. These patients may be defined as ‘those who have failed to respond to a full and adequate course (normally at least one year of treatment) of beta-interferon. Patients should have had at least one relapse in the previous year while on therapy, and have at least nine T2-hyperintense lesions in cranial magnetic resonance imaging (MRI) or at least one gadolinium-enhancing lesion. A “non-responder” could also be defined as a patient with an unchanged or increased relapse rate or ongoing severe relapses, as compared to the previous year'.
• Adults with rapidly evolving severe relapsing-remitting multiple sclerosis defined by two or more disabling relapses in 1 year, and with one or more gadolinium-enhancing lesions on brain MRI or a significant increase in T2 lesion load as compared to a previous recent MRI.

8. In 2007, NICE recommended natalizumab (Tysabri, Biogen) as a possible treatment for people with rapidly evolving severe relapsing-remitting multiple sclerosis. For further information, please visit: www.nice.org.uk/TA127.

9. In 2002, NICE 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 NICE published this guidance, the Department of Health agreed a risk sharing 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 press office at the Department of Health.

About NICE

1. The National Institute for Health and Care 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

2. 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

3. 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

4. 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.