NICE 2001/007
Issued: 04 February 2002
PRESS RELEASE
NICE issues guidance on drugs for multiple sclerosis
The National Institute for Clinical Excellence has issued guidance on the use of beta interferon and glatiramer acetate for multiple sclerosis (MS) to the NHS in England and Wales.
The guidance states that:
- on the balance of their clinical and cost effectiveness neither beta interferon nor glatiramer acetate is recommended for the treatment of MS in the NHS in England and Wales.
- because people with MS currently receiving these drugs could suffer loss of well being if treatment was stopped at a time they did not anticipate, they should continue treatment, until they and their consultant decide it is appropriate to stop. This also applies to people who have been part of clinical trials (regardless of whether they have received a placebo or active therapy), and to women who stopped taking the drugs because of pregnancy and want to start again.
- the Department of Health and the National Assembly for Wales along with the manufacturers should consider ways to obtain these medicines for the NHS in a manner which could be considered to be cost effective.
Lord Hunt, the Parliamentary Under-Secretary of State, informed The Lords last month that the Department of Health and the National Assembly for Wales had responded to the Institute's recommendation and had entered into discussions with manufacturers and others.
Andrew Dillon, Chief Executive of NICE said, "Although this has been a particularly challenging appraisal, which has taken much longer than anyone involved would have wished, the outcome is good, both for people with MS and for the NHS as a whole."
When asked if he felt that the Department of health had undermined the Institute's guidance Andrew Dillon was clear "Obviously not. It's only because of the work that we have done that the government and the manufacturers have been able to develop this scheme. The government's position is clearly supportive of the robust and transparent process the Institute follows in developing its guidance."
On average, NICE technology appraisals take around 12 months to complete. Some appraisals take longer when they need to be extended to allow additional evidence to be gathered or if the evidence has to be re examined after an appeal. In this case, both happened.
The Institute recognises the importance of its decisions to those with MS and their families and carers. We believed that it was important that it was better to make the decision on the best available evidence rather than making potentially the wrong decision without all the facts. It was particularly important to commission an independent economic model.
Professor Sir Michael Rawlins, Chair of NICE said, "These medicines have caused debate amongst the medical community worldwide. The Institute has been faced with an extremely challenging and complex task. Having heard two separate appeals on this appraisal the Board is confident that the Appraisal Committee has carried out the appraisal properly."
Ends
Notes for editors
Background
1. The following documents relating to this appraisal are available on the NICE website at www.nice.org.uk:
- The Institute's Guidance
- ScHARR additional economic modelling report
- Appeal decision
- Final Appraisal Determination (November 2001)
- Provisional Appraisal Determination (August 2001)
- Original assessment report
- Key dates and activities in this appraisal
2. The Institute's process allows for submissions from manufacturers, groups that represent patients and professional organisations. It also provides for periods of consultation, feedback, and an appeal should it be required.
3. The Institute's provisional and final determinations on beta interferon and glatiramer acetate for MS (published in August and October 2001 respectively, encouraged the Department of Health and the National Assembly for Wales to consider, with manufacturers, a strategy which might enable any or all of these medicines to be acquired cost effectively by the NHS. Requests for further information on the Government's negotiations with manufacturers should be directed to the Department of Health's and National Assembly for Wales' press offices.
4. The consultees in this appraisal are:
- Association of British Neurologists
- Aventis Pharma Ltd.
- Biogen Ltd
- Chartered Society of Physiotherapy
- Department of Health
- MS Trust
- Multiple Sclerosis Society of Great Britain and Northern Ireland
- National Assembly for Wales
- Neurological Alliance
- Royal College of General Practitioners
- Royal College of Nursing
- Royal College of Physicians
- Schering Health Care Ltd
- Serono Pharmaceuticals Ltd.
- Teva Pharmaceuticals Ltd.
The Guidance
The guidance recommends that:
1. On the balance of their clinical and cost effectiveness neither beta interferon nor glatiramer acetate is recommended for the treatment of multiple sclerosis (MS) in the NHS in England and Wales.
2. It is likely that patients currently receiving beta interferon or glatiramer acetate for MS, whether as routine therapy or part of a clinical trial, could suffer loss of well being if their treatment is discontinued at a time they did not anticipate. Because of this, all NHS patients who are on therapy at the date of publication of this Guidance should have the option to continue treatment until they and their consultant consider it is appropriate to stop, having regard to the criteria established for withdrawal from treatment in the Guidelines of the Association of British Neurologists published in January 2001. This also applies to all participating patients at the conclusion of a clinical trial (irrespective as to whether they had received placebo or active drug) and women whose therapy has been interrupted by pregnancy.
The Appraisal Committee considered that:
- the Department of Health, the National Assembly for Wales and manufacturers, might usefully consider what actions could be taken, jointly, to enable any of the four medicines appraised for this guidance to be secured for patients in the NHS in England and Wales, in a manner which could be considered to be cost effective. Unless further evidence emerges which reveals a significant improvement in their clinical effectiveness, the cost-effectiveness of these medicines can only be improved if there is a significant reduction in the total cost of their acquisition by the NHS in England and Wales.
The Committee further noted that:
- the uncertainty surrounding the definition of which patients benefit and to what extent, together with the ability of the NHS to identify a total potential patient population for which these medicines might most beneficially be purchased, are factors which could be considered relevant in any discussions between the Department of Health and the National Assembly for Wales and manufacturers on ways in which these medicines could be acquired cost effectively. The Committee noted that the results of the additional economic modelling commissioned by the Institute revealed that interferon beta-1b (Betaferon, Schering) is, currently, the most cost effective of the four products appraised for this guidance.
5. When appraising a technology the Institute considers both its clinical and cost effectiveness. In this appraisal the cost effectiveness was a very important factor in the decision but it was by no means the only one. NICE has carefully considered all of the evidence about the effectiveness of the beta interferons and glatiramer in people with MS As well as looking at the results available from clinical trials, it has heard evidence from health professionals, including neurologists, and from patients, their carers and the organisations that represent them.
6. MS is a disease that affects people over a very long period (often 20 years or more), but at the moment there is no firm evidence available about the effects of beta interferon or glatiramer acetate on the disease in the long term. Because of this, NICE had to look at 'models' of the cost effectiveness of the drugs. These models make estimates of how the drugs might work in the long term. The further ahead a model looks, the harder it is to be sure about what it tells us.
7. During 2000, NICE considered several models of the cost effectiveness of beta interferon and glatiramer acetate, which had been produced by the manufacturers of the drugs. New models were submitted during the appraisal and these were also considered. NICE was advised that there were 'flaws' with all of them all and so the Institute decided to commission a new, independent model, ultimately giving the work to a consortium of UK universities, based on ScHARR, the School of Health and Related Research at Sheffield University. NICE provided the patient groups, the professionals and the manufacturers with the opportunity to review and comment on the model.
The Appeal
8. The Final Appraisal Determination was sent to the consultees on Monday 29th October 2001 and was published on the NICE website on Friday 2nd November 2001. The consultees had until 14th November 2001 to lodge an appeal against the guidance if they believed that the Institute:
- had not acted fairly and in accordance with its procedures
- had made a decision that was perverse in light of the evidence presented
- had exceeded its powers
9. Appeals were lodged by the following appellants:
- Teva Pharmaceuticals and Aventis Pharma Ltd jointly
- Biogen Ltd and Schering Health Care jointly
- Serono Pharmaceuticals Ltd ("Serono")
- The Neurological Alliance
- The Royal College of Nursing ("RCN")
- The Multiple Sclerosis Society ("MS Society")
- The Multiple Sclerosis Trust ("MSRT")
The Appeal Panel convened on 26th November 2001 to consider these appeals. The Appeal Panel comprised Professor Sir Michael Rawlins (chair of the Appeal Panel and chair of the Institute), Mrs Mary McClarey and Ms Mercy Jeyasingham (non-executive directors of the Institute's Board), Mrs Gill Donovan (patient representative) and Dr Peter Brock (industry representative). All the appellants were represented at the appeal hearing with the exception of the Neurological Alliance. The MS Society communicated the views of the Neurological Alliance on its behalf.
Related guidance
10. The Institute has been commissioned to produce a full clinical guideline on the management of MS. The planned publication date of this guidance is January 2003.
Information on NICE
11. Copies of the full guidance and supporting documentation will be available on the NICE web site (www.nice.org.uk) from midday on Monday 4th February 2002.
12. Health professionals are expected to take the Institute's guidance fully into account when exercising their clinical judgement for individual patients. This guidance does not, however, override the individual responsibility of health professionals to make appropriate decisions in the circumstances of the individual patient, in consultation with the patient and/or guardian or carer. In the case of this appraisal, the Institute's guidance should be read in the context of the circulars issued by the Department of Health and National Assembly for Wales.
13. The National Institute for Clinical Excellence (NICE) is a part of the NHS. Part of its work is technology appraisals. The Institute produces guidance for both the NHS and patients on medicines, medical equipment and clinical procedures based on evidence of clinical and cost effectiveness. Each appraisal takes an average 12 months to complete and involves the manufacturers of the technology, groups that represent patients/carers and healthcare professionals.
14. NICE promotes clinical and cost effectiveness through its technology appraisals, clinical guidelines and audit tools. The Institute supports the work of those who make the complex treatment decisions - doctors, nurses, and other health professionals. The needs of the patient are central to NICE's work, and the Institute has forged strong links with patient groups and representatives.
15. Topics for the NICE work programme are selected by the Department of Health and the National Assembly for Wales. NICE advises the NHS on how these technologies can best be used. It is also responsible for the production of national clinical guidelines, promoting best practice throughout the NHS. To support and assess the implementation of such guidelines, audit tools are produced for use in the clinical setting.
