Select Committee on Health Minutes of Evidence


Memorandum by the Association of the British Pharmaceutical Industry (NC 118)

SUMMARY OF KEY POINTS

  The ABPI fully supports the objectives of NICE. It has the potential to play a key role in improving the quality of care in the NHS but NICE cannot achieve its objectives on its own.

  The pharmaceutical industry has been in regular dialogue with NICE to resolve many issues and progress has been made. However, the process leading to selection of technologies for appraisal and implementation of guidance post NICE review are issues that cannot be addressed without action from the Department of Health and NHS.

  Analysis of prescribing data shows there are still wide variations in uptake of medicines that have been appraised by NICE: postcode prescribing still persists. Overall increases in uptake have rarely resulted from NICE guidance.

  NICE's own estimates of increased uptake in its guidance predicted a total annual addition of £93 million. This figure compares with an actual increased uptake of, at most, £32 million, i.e. only a third. There is also evidence that predicted savings accruing from NICE decisions have not been realised.

  In addition, NICE is leading to reduced access to modern effective treatments through a phenomenon known as "NICE blight".

  A major consideration is the NHS's capacity to comply with NICE guidance and limitations of NHS ability to redesign and adapt services to meet many priorities.

  The Government's recent announcement requiring HAs and PCOs to fund NICE recommendations is implicit confirmation that they not currently being implemented satisfactorily. While we very much welcome this move as a step in the right direction, additional sufficient funding needs to be made available to enable such compliance if "Peter is not going to be robbed to pay Paul", diverting funding from other important areas of clinical need.

  ABPI believes that NICE is being asked to focus too much on technology appraisals and with little impact as a result. We believe that the development of sound, evidence-based clinical guidelines, which address the full range of care for particular conditions, will improve the quality of NHS care far more than a narrow assessment of the cost-effectiveness of individual interventions and will have far greater influence.

  Recommendations made by NICE in some circumstances are being made too early on the basis of immature evidence. NICE is currently relying heavily on a methodology that is based on a one-size-fits-all measure of cost effectiveness. In practice this has a profound impact on patients, who can be denied treatment on the basis of theoretical assumptions.

  Perceptions prevail among many stakeholders that NICE is not independent of Ministerial intervention and is not yet perceived as a genuine force for improving the quality of NHS care rather than as a tool to control costs.

  ABPI believes that NICE, as an important area of health policy, should evolve with the help of an ongoing, systematic and independent review.

1.  THE ASSOCIATION OF THE BRITISH PHARMACEUTICAL INDUSTRY

  1.1  The Association of the British Pharmaceutical Industry (ABPI) represents the majority of the companies in Britain engaged in the research, development, manufacturing and supply of prescription medicines. The ABPI brings together companies producing such medicines, whether branded or generic, many smaller organisations involved in pharmaceutical and bio-pharmaceutical R&D, and those with an interest in the pharmaceutical industry operating in the UK. ABPI member companies manufacture and supply more than 80 per cent of the medicines prescribed through the NHS and are major exporters to countries all over the world.

  1.2  The pharmaceutical industry in the UK is a world leader in medicines research, and patients in the UK have the potential to benefit from this research by the early introduction of new medicines developed in their own country.

2.  THE ROLE OF NICE

  2.1  Annex 1 responds to the specific questions posed by the Select Committee. The ABPI fully supports the objectives of NICE to encourage faster uptake of effective new treatments, to promote more equitable access to treatments, to improve the use of NHS resources and to promote the longer-term interests of the NHS in the development of innovative treatments for the future. NICE has the potential to have a key role to play in improving the quality of care in the NHS. The ABPI has sought constructive dialogue with both NICE and the Department of Health (DH) to support NICE in achieving its objectives.

  2.2  NICE has an important and difficult job to do. The postcode lottery of care still exists, and patients in the UK have some of the poorest access to modern effective treatments in Western Europe. The Wanless Report made some unflattering international comparisons, concluding that the UK lags behind other countries in the adoption and diffusion of new technologies, and confirming that the uptake of new drugs is at best half of that in Germany and a third of France. Another study, jointly undertaken by the ABPI and DH, published in December 2001, shows that in comparison with 12 countries in Europe, North America and Asia, new medicines in the UK achieve only about a quarter of the average level of international use one and three years after launch. These facts highlight the need for NICE to encourage the uptake of medicines in the UK.

  2.3  NICE needs to be seen in context, as part of a complex web of interweaving factors. NICE cannot achieve its objectives on its own.

    —  The policy framework within which NICE operates is determined by Ministers, Parliament and the National Assembly for Wales (NAW).

    —  Ministers and the NAW determine the work programme for NICE technology appraisals and clinical guidelines.

    —  NICE's ability to develop guidance and guidelines on clinical and cost effectiveness is also affected by:

      —  the quality of the evidence base

      —  other regulatory assessments - medicines pass through several hurdles before becoming available to patients, including assessment of safety, quality and efficacy by the Medicines Control Agency (MCA).

      —  other regulations, for example the pharmaceutical price regulation scheme.

    —  Having received NICE guidance, it is the role of local NHS organisations to develop supportive infrastructure, allocate funding and enable implementation by clinicians.

  2.4  Many of the observations below, therefore, identify weaknesses in the overall system, of which NICE is but a part, rather than specific criticisms of NICE. The process leading up to selection for technology appraisal and the post NICE review implementation of guidance both have weaknesses that are beyond the remit of NICE yet both have a critical influence on the value and effectiveness of NICE itself. The pharmaceutical industry has been in regular dialogue with NICE to resolve issues within its own remit and progress has been made on many fronts but some remaining issues cannot be rectified without action from the DH and NHS.

  2.5  ABPI believes that NICE is being asked to focus too much on technology appraisals and with little impact as a result. We believe that the development of sound, evidence-based clinical guidelines, which address the full range of care for particular patients in areas of high morbidity and mortality, will improve the quality of NHS care far more than a narrow assessment of the cost-effectiveness of individual interventions and will have far greater influence. This concords with Professor Kennedy's recommendation in his Bristol Inquiry report that NICE manages a comprehensive programme of standard setting through the development of clinical guidelines.

3.  "NICE BLIGHT"

  3.1  The very existence of NICE is leading to reduced access to modern effective treatments through a phenomenon known as "NICE blight". There are two kinds of blight. The first is when Health Authorities (HAs) and Primary Care Organisations (PCOs) hold back decisions on funding of a product on the NICE work programme pending a decision by NICE, which is often issued months or even years after grant of a licence. The second is when HAs/PCOs hold back funding for new treatments that are not short-listed for consideration by NICE on the assumption that they will at some stage be the subject of an appraisal. This clearly runs against the objective of NICE to bring patients faster access to modern treatment.

  3.2  NICE blight is having a profound effect on patients. Two examples illustrate a much bigger problem. The first of these relates to a treatment, Visudyne, for age-related macular degeneration, an aggressive disease that is the most common cause of blindness in the elderly. Poor vision not only leads to loss of quality of life and independence, it is also a major cause of falls in the elderly, and blindness consumes considerable health and social care resources. Some 4000 patients per year could benefit from treatment; yet despite the facts that Visudyne has been available in the UK for 16 months, that 100 hospitals have the ability to offer Visudyne treatment, and that the DH has issued guidance that NHS patients should be offered Visudyne before NICE guidance is published, only 500 patients have received treatment. Novartis, the licence holder for Visudyne, estimates that 9000 patients in the UK will have been denied the opportunity to have their vision saved by the time NICE's guidance is published.

  3.3  Another important example of blight is in the area of breast cancer. Herceptin has been licensed and available to UK patients since September 2000. It represents a major advance for the treatment of a particularly aggressive form of breast cancer. In combination with chemotherapy, it can induce disease remission and extend survival, and does so in a way that enhances the quality of the life remaining to patients. NICE guidance is still awaited. Since its launch, Herceptin has only been used in a very small number of hospitals in the UK (with significant postcode prescribing as a result). Even some of the largest cancer centres do not make it routinely available to patients. A reason given by Health Authorities and Primary Care Organisations for non-funding of Herceptin is that they are waiting for NICE guidance.

  3.4  These examples illustrate that the very existence of a NICE technology appraisal process is having an effect opposite to the very purpose of NICE. ABPI would like to recommend that a robust mechanism be put in place to ensure that patients are not denied access either because a treatment has not been subjected to NICE appraisal or because an appraisal has not yet been published.

4.  IMPLEMENTATION OF NICE GUIDANCE

  4.1  Even once NICE guidance is issued, implementation is poor. Further detail on this is provided in response to the Select Committee's questions in Appendix 1 (Question 3) and in the examples shown in Appendix 2. ABPI would be happy to share additional data with the Committee.

  4.2  A major consideration is the NHS's capacity to comply with NICE guidance. Clinicians and managers are being deluged with directions and guidance on a daily basis, and have many competing priorities, such as implementation of National Service Frameworks, organisational change, and managing day-to-day patient care. We would recommend that the Health Select Committee consider the practical issues around implementation and the limitations NHS organisations face in their ability to redesign and adapt services to meet many priorities. This is especially true in the field of cancer, where lack of staff is often given as a reason for denying patients modern treatments.

  4.3  If NICE is to recommend individual technologies, adequate money needs to be provided to cover the funding of all aspects of compliance with NICE guidance. The Secretary of State is seeking to address non-compliance by the issue of Directions set out in Statutory Instrument 2001/74. This is a step in the right direction, but unless there is adequate funding an inevitable outcome of the new directions will be that "Peter is going to be robbed to pay Paul", diverting resources from other important areas of clinical need. Whilst postcode prescribing may reduce in areas covered by NICE, it is likely to increase in areas not subject to NICE guidance.

5.  THE TECHNOLOGY APPRAISALS PROCESS

  5.1  While a change of focus should be the priority issue as described in 2.5 above, NICE has been instructed by the DH to concentrate the majority of its resources on new medicines. ABPI has had discussions with the DH and NICE on the process for the selection of topics for NICE appraisal, the decision on when is the best time to conduct an appraisal, and NICE's appeals process. We await the outcome of those discussions. We have also made good progress through regular meetings with NICE to highlight areas where process improvements can be made.

  5.2  ABPI believes that NICE should only appraise a new medicine where there is an adequate evidence base to do so. At launch, the evidence base for a new medicine is founded on a highly scientific clinical trials process, the results of which are used as the basis for the granting of a product licence by the licensing authorities, which make a rigorous assessment of quality, safety and clinical efficacy. At this stage in a medicine's life, there is a detailed understanding of its use only in a scientifically restricted patient population.

  5.3  NICE is examining clinical effectiveness. Clinical efficacy and clinical effectiveness are different; the latter is a broader assessment of whether a medicine helps people and is usually demonstrated by examining evidence on the medicine in routine clinical use—use that is prohibited until a product licence is granted. In many cases, therefore, the true effectiveness of a medicine cannot be assessed at the time of launch when there has been little or no use in a "natural" clinical setting. To fill this gap, NICE generally uses economic modelling to estimate effectiveness on the basis of efficacy data. This is not an invalid approach; however, it is important to remember that the quality of a model is only as good as the assumptions upon which it is based, which by definition are more uncertain the less that is known about the product's use. This is particularly true where innovative medicines lead to significant changes in the design and delivery of care.

  5.4  NICE rightly believes that it can only have the confidence of its stakeholders if it delivers authoritative guidance, which is based upon robust evidence. But frequently NICE is being asked to appraise treatments before it is possible to have an adequate effectiveness evidence base, with some decisions therefore based on health economic models for which assumptions are highly uncertain.

  5.5  NICE and the DH are in a difficult situation: ABPI understands that the NHS needs information on new products at the time of launch, but we are concerned that recommendations made by NICE in some circumstances are being made too early on the basis of immature evidence. We would recommend that for those treatments where an appraisal at launch is inappropriate, an information bulletin be issued by a respected body skilled in doing this work. Our recommendation would be that a format is adopted, based on the current output of the National Prescribing Centre, and that the medicine is made fully available to the NHS while better evidence is gathered.

  5.6  Issuing definitive guidance on a medicine too early can have damaging consequences - it can deny opportunities for discovery of valuable therapeutic potential in the form of new uses. Indeed, as Gelijns et al commented in the New England Journal of Medicine in 1998 (NEJM, 1998, Vol 339, No 10, pp693-698), "Unanticipated uses of diagnostic and therapeutic interventions are often identified many years after their introduction. Indeed, widespread use is often an essential precondition for the identification of new applications, and clinical practice itself is thus a particularly important source of medical innovation." Examples of important breakthroughs attributed to medicines initially used for other indications are many, and include ACE Inhibitors and beta blockers in the treatment of heart failure and acute myocardial infarction, and antibiotics for the treatment of peptic ulcer disease.

  5.7  ABPI, DH and NICE have agreed that a "one size fits all" approach to the timing of an appraisal is not appropriate. We have recommended that a dialogue between the manufacturer and a reformed Technologies Advisory Group take place on when is the best time to appraise on a case-by-case basis. ABPI and the DH have worked together to develop a checklist of factors that could form the basis of this discussion. Our proposals for a process to undertake this dialogue, and the checklist, are given in Annex 3. We look forward to hearing how the DH and NICE intend to incorporate these proposals into their processes.

6.  DEFINING COST-EFFECTIVENESS

  6.1  Determining cost effectiveness is a challenging task. We would like to suggest that the Committee consider whether the methods used to determine cost effectiveness, and which drive recommendations as to whether a treatment should be funded by the NHS, are appropriate. By assessing medicines at launch, and basing many of its decisions upon models that are based on theoretical assumptions for newly developed medicines that have not yet found their place in clinical practice, NICE is likely to reach false conclusions. NICE is currently relying heavily on a methodology that is based on a one-size-fits-all measure of cost effectiveness, an Incremental Cost Effectiveness Ratio (ICER) based upon a cost per Quality of Life Year gained (QALY), which attempts to apply the same parameters to a wide range of health interventions and subjective outcomes such as quality of life. While such a measure has a role, it is but one of a number of parameters. Over-reliance on this single measure may have a profound impact on patients, who can be denied treatment on the basis of theoretical assumptions.

  6.2  QALY values can change over time, as more is learned about the optimum use of a medicine. The best example of this is erythropoietin (EPO), a treatment for patients with kidney failure, which improves red blood cell count, reduces anaemia, reduces the need for blood transfusions and improves patients' well being. At the time of EPO's launch in 1990, the estimated cost per QALY was £103,000, a value which does not represent cost effectiveness under NICE's methodology. Over time, management of kidney failure using EPO has changed, as more has been learned about the drug and benefits, including improved quality of life for patients, have been found. Current estimates of cost per QALY for EPO show a dramatic reduction from the initial value to £3,000-9,000. If usage of EPO had been rejected or highly restricted at its launch both patients and NHS would have been the losers.

7.  SELECTION OF TOPICS FOR NICE WORK PROGRAMME

  7.1  The DH has acknowledged that the current procedures to select topics for NICE appraisal are unsatisfactory and not transparent. In a presentation at the annual NICE conference in December 2001, a DH speaker indicated that current selection criteria are vague and open to broad interpretation, and the selection process, including the reasons topics are chosen, needs to be made clearer to stakeholders. ABPI concurs with these views and believes that the skills base of the body that makes recommendations to Ministers on topic selection (the Technologies Advisory Group (TAG)) needs to be more appropriate for this complex task. It is even unclear who sits on TAG. We believe that a broad range of stakeholders should be represented, including industry. We have discussed these points with the DH (see Annex 3) and look forward to hearing their views.

8.  ONGOING REVIEW OF NICE

  8.1  ABPI believes that NICE, as an important area of policy, should evolve with the help of an ongoing, systematic and independent review.

  8.2The review should be open and inclusive of all stakeholders, with appropriate consultation. It should examine whether NICE is achieving its objectives, the impact it is having on its stakeholders, and should make tangible recommendations for improvement in consultation with those affected. This should not detract from ongoing improvement in the working of NICE's processes. Such a review should also examine implementation of NICE guidance by HAs/PCOs once the new arrangements under SI 2001/74 have bedded down.

  8.3  We would recommend that the review suggest appropriate performance indicators, so that the progress of NICE against its objectives can be continuously monitored, and that the review should answer the following questions:

    —  Is the existence of NICE adding value to healthcare provision and benefits to patients?

    —  Were the technologies chosen for appraisal of most relevance to improving patient care?

    —  Has NICE eliminated post-code prescribing?

    —  Is there evidence of increased post-code prescribing in areas not covered by NICE guidance?

    —  How does the uptake of medicines appraised by NICE compare with the recommendations in NICE guidance?

    —  How does the uptake of innovative medicines in the UK compare with those in comparable European countries?

    —  Does NICE have the confidence of its stakeholders, including patients, professionals, the wider NHS and the pharmaceutical industry?


 
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