EXECUTIVE SUMMARY

  1.  The British Society for Rheumatology (BSR) is a medical society committed to advancing knowledge and practice in the field of rheumatology. We aim to improve awareness and understanding of arthritis and other musculoskeletal conditions and work at national and local level to promote high quality standards of care for people with these conditions. We have around 1,400 members in the UK and overseas; the majority of these are consultant and trainee rheumatologists. BSR also has a number of members who are allied health professionals, primary care workers, scientists and others working in the field of Rheumatology.

  2.  As an organisation BSR has participated in over 25 NICE technology appraisals.

  3.  Submissions to NICE, by all stakeholders, are becoming more detailed and sophisticated.

  4.  Scepticism is growing about the health economic analysis that is used by NICE.

  5.  The lack of accessibility to the analyses is unhelpful.

  6.  NICE health economics focus on the costs to the NHS without taking societal and employment costs into account. This skews the focus of the analysis from the perception of patients and their carers.

  7.  Much of health economic analysis is beyond the understanding of the general public (and many professionals), and this raises the suspicion that NICE has a purely rationing function.

  8.  Stakeholders such as BSR and patient representative organisations do not have the resources or the manpower to mount several appraisals simultaneously.

  9.  Because of the thoroughness of the evidence considered, the number of stakeholders even in single technology appraisals, and the appeals process, the speed of publishing guidance is very slow.

  10.  Because the process is slow, and new evidence is emerging all the time, it seems perverse that NICE will not consider new evidence that is pertinent to the issues at hand.

  11.  SIGN and NICE duplicate a great deal of work. It would be logical to look at ways avoiding unnecessary duplication and the sharing of evidence and therefore speeding up the process.

  12.  NICE has not eliminated postcode prescribing or poor implementation of NICE guidance.

Why are NICE's decisions increasingly being challenged?

  13.  Stakeholders are increasingly sophisticated in their submissions, improving their data, and performing their own health economic analyses. It is no longer just the pharmaceutical industry that can mount robust defences of the cost-effectiveness of their drugs. For example, BSR established a Biologics Register to collect data on patients with rheumatoid arthritis. This was done with the encouragement of NICE as it is a useful tool to support their guidance. The Biologics Register has enabled the BSR to commission an independent health economic analysis of the cost effectiveness of anti-TNF? therapy in rheumatoid arthritis (performed in Sheffield[49]).

  14.  The results demonstrated incremental cost efficiency ratios that were substantially lower than those of the Birmingham Assessment Group. This appears to be a recurrent theme that generates scepticism about the process. Health economic analyses commissioned from Assessment Groups are invariably more expensive than most other analyses, and lack transparency[50],[51].

  15.  The lack of accessibility of these analyses has been highlighted as a serious problem.[52] This is the principal reason cited by those seeking a judicial review on the NICE decision on drugs for Alzheimer's disease.[53] The Assessment Group has access to other economic analyses at the start of an appraisal, but other stakeholders do not have reciprocal access to their work, which is unjust3.

  16.  Currently the BSR is engaged in the appraisal process for anti-TNF drugs in ankylosing spondylitis (AS). The Liverpool Assessment group has generated a health economic model with which the BSR has profound difficulties, with assumptions about the disease that do not bear any relationship to the disease as we recognise it. However, the lack of transparency has made their economic modelling impenetrable. Because health economics is the central pillar of technology appraisals, the unsatisfactory accessibility of the Assessment groups' work will continue to fuel challenges to decisions. It is at least a positive step that the AS health economic model has now been referred to the Decisions Support Unit for further assessment.

  17.  Because re-appraisals may not take place for three to four years (or even longer in the case of guidelines), stakeholders who feel that appraisals or guidelines are incorrect will feel obliged to put a great deal of effort into trying to ensure contentious elements are modified. This is in an attempt to avoid patients being denied access to appropriate treatment in the intervening years.

  18.  NICE health economics focus on the costs to the NHS without taking societal costs into account. The cost to the state of social services care, invalidity benefit and loss of tax revenue from a person of working age who has had to give up their employment must be considered, otherwise the focus of the analysis is skewed from the perception of patients and their carers.

Is public confidence in the Institute waning?

  19.  Much of health economic analysis is beyond the understanding of the general public (and many professionals). However the decisions as reported always list the cost-benefit analysis as a key driver, and this raises the suspicion that NICE has a purely rationing function.

  20.  Members of the press have raised populist arguments criticising the decisions of NICE (eg Clare Rayner and Thomas Stuttaford), and patient representative bodies protest against NICE decisions. Seeing angry patients and their carers carrying condemnatory placards and handing in large petitions leaves a powerful emotional image. The possibility that the Alzheimer's society may mount a legal challenge to the NICE decision on availability of drugs for this condition results in bad publicity for the Institute5.

  21.  NICE claims not to have a cut-off for costs per QALY in the decision-making process, but its decisions point clearly to a ceiling for an incremental cost effectiveness ratio of £30,000/QALY. Above this a drug will only be supported if it is innovative, if there are particular features of the condition and population receiving the technology, and sometimes with reference to wider societal costs and benefits.[54] It is unclear how this figure was reached, which spawns the suspicion that it is arbitrary. It has been argued that deciding affordability is not a role of NICE in setting a threshold above which a technology will not be accepted, and this should be a role for parliament.[55]

  22.  Decisions mean that some drugs may be less available to NHS patients than they are in other countries, inviting the public to query why this is the case. For example, NICE eligibility criteria for anti-TNF? drugs for rheumatoid arthritis are pitched at a level that means that far fewer patients have access to these drugs than is the case in other European countries and the US.[56] For example, a recent survey showed that 47.9% of Norwegian and 41.3% of Danish rheumatoid arthritis patients currently on anti-TNF would not meet the NICE criteria.[57] It is also true that some drugs rejected by NICE have been approved in Scotland.

  23.  Although patient representatives are invited to Appraisal Committee meetings, in our experience their presence is cosmetic, and does not influence the committee's final decisions.

  24.  Although NICE claims to be independent,[58] , there are clear examples where it appears that there has been political influence (eg the intervention of the Health Secretary to fast-track the assessment of Herceptin after a politically embarrassing court case, and B-interferon where the government intervened with a risk sharing special purpose scheme with the drug company[59]). This raises the suspicion among healthcare professionals that political interference might take place at other levels of the process, despite the reassurances.

  25.  Because of the delay between initiating a technology appraisal and the final determination, patterns of treatment of uncertain cost effectiveness can become established. The longer they persist the harder they become to reverse. For example, some patients for whom biologic agents have been prescribed for ankylosing spondylitis, and who have done very well, may be denied ongoing access to their treatment if the Final Appraisal Determination bears any resemblance to the Appraisal Consultation document. Understandably, the withdrawal of effective therapy will be very unpopular with these patients and their clinicians and will undoubtedly result in local political pressure and adverse publicity.[60], [61]

  26.  The general public may not be aware of the strategy that NICE has to explain its decisions and processes. Much of the strategy is laid out on their website, however this has a limited audience. As a consequence the process can have an unnecessary air of mystery which does little to give confidence to the wider public. NICE may engage with patients' representative groups, but the wider public gets its information mainly from the media, and then only on issues which have grabbed the headlines.

The NICE Evaluation Process

  27.  Stakeholders such as BSR and patient representative organisations do not have the resources or the manpower to mount several appraisals simultaneously. These processes are incredibly time-consuming for all concerned.

  28.  Any individual or organisation that does not have grounding in health economics will find the arguments very difficult to follow.

  29.  Smaller organisations are effectively excluded from processes if they rely on volunteers and have no paid staff.

The Speed of Publishing Guidance

  30.  Because of the thoroughness of the evidence considered, the number of stakeholders even in single technology appraisals, and the appeals process, the speed of publishing guidance is very slow. For example, the NICE re-appraisal of anti-TNF was started in October 2004 with the consultation on the draft scope. The appeal will be heard in early April 2007. This means that by time the Guidelines are published, over two and a half years will have passed.

The Appeal System

  31.  Because the process is slow, and new evidence is emerging all the time, it seems perverse that NICE will not consider new evidence that is pertinent to the issues at hand. In the two and a half years of waiting for the NICE re-appraisal of anti-TNF therapy a great deal of new evidence has emerged that could have informed the process.

Comparison with the Work of SIGN

  32.  SIGN are considerably quicker at publishing guidelines. SIGN and NICE duplicate a great deal of work. It would be logical to look at ways of avoiding unnecessary duplication and the sharing of evidence and therefore speeding up the process, especially as in some instances the two bodies have reached contradictory conclusions.

The Implementation of NICE Guidance

  33.  NICE guidance is poorly implemented. This remains a key problem for BSR. A survey of rheumatologists revealed that 46% had limited access for their eligible RA patients to anti-TNFa four years after the initial NICE guidelines, with funding issues being the main reasons for lack of compliance from their PCTs.[62] Although implementation of appraisals and guidelines is not a direct responsibility of NICE, the authority and purpose of NICE is undermined if its work is ignored or funding of approved products is unobtainable.

  34.  In 2005 (following the publication of NICE guidance on the topic) BSR, working with the Arthritis and Musculoskeletal Alliance (ARMA), commissioned a survey of 148 consultant rheumatologists. They were asked whether they were able to prescribe anti-TNFa therapy to all rheumatoid arthritis patients they identified, in accordance with NICE guidance, and if not what was the main barrier to prescription. 31% of rheumatologists were unable to prescribe for all patients they identified, with most saying that funding was the main barrier to prescription. The results showed that no improvement had been made since the same survey was undertaken in 2003 (before NICE guidance).

  35.  There are implementation problems with all guidelines that are not unique to NICE. A study of the implementation of NICE guidance came to the conclusion that NICE guidance is more likely to be implemented where "there is strong professional support, a stable and convincing evidence base, and no increased or unfounded costs, in organisations that have good systems for tracking guidance implementation and where the professionals involved are not isolated[63]". We are sure that the same could be concluded about the implementation of any guidelines. If guidelines court controversy, their uptake will be patchy. If opinion leaders, professional bodies and associations are critical of guidance it is unlikely to be accepted.

  36.  Many of the problems associated with poor implementation are often caused by a lack of ownership. There is still confusion between local and national powers, and despite a number of attempts to resolve these issues the problems still persists. A clear implementation strategy would help this.

  37.  NICE has a tendency to focus on new drugs that add to the budgetary demands of funding authorities. If they also assessed older drugs and technologies for which there was no good evidence to support their perpetuation, the release of funds might make acceptance of new drugs and technologies more palatable[64]. Canvassing the opinions of NHS staff on appropriate topics for consideration by NICE might increase the chances of cost-saving initiatives being considered, increase the ownership of the process for NHS workers, and increase implementation of guidance.

British Society for Rheumatology

March 2007

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