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Additional Evidence Means First Pill for MS Could Soon be Recommended for Some Patients

In its final draft guidance, out today (16 March), NICE, the healthcare guidance body outlines its plans to recommend fingolimod, the first pill-based medicine to help reduce the number of relapses for some adults who have a particular type of multiple sclerosis. This positive recommendation is a change from NICE's previous draft guidance from December and follows a public consultation during which the manufacturer (Novartis) and clinicians provided additional information and analyses.

The draft document (called a final appraisal determination) recommends fingolimod (Gilenya) as a treatment option for some adults who have highly active relapsing-remitting multiple sclerosis (RRMS); this is a type of the condition characterised by periods when symptoms worsen and then improve.

Specifically, NICE provisionally recommends the treatment for adults who have an unchanged or increased relapse rate or ongoing severe relapses compared to the previous year, despite them taking other drugs such as beta interferons. The provisional recommendation relates to a subgroup of the population for which fingolimod is licensed.

Professor Carole Longson, Director of the Health Technology Evaluation Centre at NICE said: "The latest draft guidance from our committee recommends the NHS-use of fingolimod for a specific group of adults who have highly active relapsing-remitting multiple sclerosis. Following new information provided during the consultation, the analyses show that for these people, treatment with fingolimod will be a cost effective option for the NHS in this group of people with multiple sclerosis, if Novartis provides the drug at a discounted price, as proposed in its patient access scheme.

"We have published the latest version of our draft guidance on our website so that interested parties can highlight any factual errors or appeal against our final draft recommendations."

Registered stakeholders wishing to appeal NICE's final appraisal determination have until 5pm on Friday 30 March to do so. If no appeals are received, NICE expects to publish its final guidance for the NHS in April 2012.

Until NICE issues final guidance, decisions on the funding of specific treatments should continue to be made at local NHS levels.


Notes to Editors
About the draft guidance (final appraisal determination)
1. Further information about the final appraisal determination of Fingolimod for the treatment of relapsing-remitting multiple sclerosis can be found here: Embargoed copies are available on request from the NICE press office.

2. After the first draft document (called an appraisal consultation document, ACD) was published in August 2011, Novartis submitted a proposed discount (Patient Access Scheme) for consideration. However despite this, NICE's independent committee concluded that fingolimod still did not present a cost effective option for the NHS, and so provisionally was not able to recommend the drug again in its second ACD, published in December 2011. During the second consultation period, Novartis revised its analyses for a subgroup of the licensed population. Following this, NICE's committee now believes fingolimod represents a cost effective treatment option for a subgroup of the licensed population. The subgroup is adults with highly active RRMS, whose relapses have increased or stayed the same compared to the previous year, despite them taking beta interferons. People currently receiving fingolimod whose disease does not meet these criteria should be able to continue treatment until they and their clinician consider it appropriate to stop. This is reflected in the final appraisal document, published today (16 March).

3. 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.

4. 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).

5. The final most plausible Incremental Cost Effectiveness Ratio (ICER) for fingolimod is likely to be between £25,000 and £35,000 per QALY gained for the specified population. Previously, in the ACD2 (i.e. before Novartis revised its analyses) this was estimated to be least £40,000 per QALY gained, which was why NICE was provisionally unable to recommend it. Generally if a treatment costs more than £20,000-30,000 per QALY, then it is not considered to be a cost effective option for the NHS. For further information about how NICE measures cost effectiveness, please visit the cost effectiveness webpage.

6. 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.

7. NICE's technology appraisal will apply to NHS settings in England and Wales. On Monday 12 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.

8. 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.
9. 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:

10. 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: 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 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

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.

This page was last updated: 15 March 2012


Posted: March 2012