Select Committee on Health Minutes of Evidence

Memorandum by Campaign for Effective and Rational Treatment (CERT) (NC 23)


  The drug approval process in the UK is governed by the Medicines Control Agency and by the European Medicines Evaluation Agency. These assess new drugs in terms of safety and efficacy. However, the role of the National Institute for Clinical Excellence (NICE) is uncertain, with its added value to the statutory processes being unclear. Errors and inefficiency in the application of health economics by NICE appear to decrease any added value.

  CERT sees NICE as a short-term political solution to the rationing of new health technologies within the NHS. Only by reforming NICE can the Government live up to its promise to provide an improved health service. In its present state, NICE acts as a barrier to reform. Any credible assessment body must be separate from the Government and the Department of Health if it is to be seen as truly independent and credible.


  To date it is unclear whether NICE has achieved the key goals envisaged in a First Class Service, thus:

    —  is providing clear and credible guidance;

    —  has ended confusion by providing a single national focus;

    —  is providing guidance that is locally owned and acted on in the right way;

    —  is actively promoting interventions with good evidence of clinical and cost-effectiveness that patients have faster access to treatment known to work.

  NICE appears to be actively hindering widespread introduction of pharmaceutical interventions in its extremely lengthy and flaw-ridden processes.

  NICE uses health economics to be able to arrive at decisions on cost effectiveness. This application of health economics is fundamentally flawed by the organisation's apparent inability to understand the data presented by stakeholders. Likewise the material that NICE collects through its own offices is equally problematic.

  The NICE process as it stands can add up to three years before a drug becomes available for use. The lengthy and flawed appraisal process, the inevitable round of appeals that occur following problematic NICE decisions, and the further delays as new drugs only become available once their cost is covered in the next Health Authority spending cycle, only serves to limit the availability of vital new therapies.

  Whilst NICE guidance remains clear, the credibility of most of the organisation's appraisals for oncology agents that are still in the appeal process, is negligible. At first sight, it would appear that NICE has issued many recommendations that should have improved patients' access to new and innovative treatments. However for those familiar with the process and the delays that have beset a number of appraisals, this apparent success has come about in spite of that process.

  NICE activities only serve to increase confusion around provision of new treatments. Health Authority decision-making manipulates and uses NICE rulings to prevent full treatment provision. In some instances, Health Authorities have made drugs available to the very few whilst claiming full provision. Some Health Authorities provide funding for treatment for agents still under review by NICE whilst other Authorities do not, thereby perpetuating the accusations of inequitable access within the NHS.


1.  Flexibility and delay

  It is clear from recent appraisals that NICE has little or no flexibility in assessing the advantages of a treatment regime over existing therapies. In a recent assessment of a "salvage therapy" for cancer (ie a treatment that is used after all other treatment options have failed), NICE initially attempted to refuse the new treatment saying it should be referred to a randomised, controlled clinical trial, despite the fact that it would have been unethical to do so.

  The NICE appraisals process needs more flexibility - with its first full appraisal, NICE set the precedent of splitting the guidance on the use of taxanes for ovarian and breast cancer. The appraisal of drugs for colorectal cancer has been the subject of successful appeals against the proposal by NICE to limit the first-line combination use of these agents to clinical trials. The proposal by NICE to support the use of monotherapy in second-line treatment of colorectal cancer is understood to be uncontroversial, yet NICE have refused to separate out this section of the guidance. As a result, thousands of patients with colorectal cancer continue to be denied access to drugs that could extend their survival by several months.

  NICE also appears to move slowly. The whole of the appraisal process is extremely laborious, taking at least 11 months from commencement to completion. However, experts have commented that some appraisals of existing therapies could be made in an afternoon by those that understand the particular field of medicine and the evidence base from the relevant clinical studies.

  There have been unplanned and unexplained delays in some of the appraisals conducted by NICE. For instance, the appraisal of Herceptin has been inexplicably delayed by six months, even though this drug can have a dramatic effect on the survival of women who have an aggressive form of breast cancer. How many women with breast cancer have died prematurely as a result of this delay alone?

  There have also been unacceptable delays in announcing the result of appeals and for setting dates for reassessment by the Appraisals Committee following successful appeals. For example, in the appraisal of treatments for colorectal cancer, the appeal was held on 23 May 2001, yet it took until 2 July - 5 weeks and 5 days - for NICE to announce that this appeal had been successful. Furthermore, the next Appraisals Committee meeting to review the draft Guidance was held on 6 November; a delay of almost six months. What was the reason for such an inordinately long delay and how many patients with colorectal cancer have died prematurely as a result of this delay?

  There have also been delays in uncontested or uncontroversial parts of an appraisal. This may not matter when NICE appraises new treatments before the approval of a licence (although there have been very few of these parallel reviews conducted), but it is causing enormous problems for treaters who are already using the medicines.

  Health Authorities are also telling doctors that they will refuse funding for drugs that doctors are already using until NICE recommendations are published. This is not, however, a problem with NICE but rather a criticism of the Health Authorities using the NICE process as an excuse to curtail funding. In some cases, doctors have been using the drugs for several years before they were referred to NICE for appraisal. Despite having been proven to be more effective than alternative treatments during a NICE appraisal, Health Authorities are exerting pressure to limit or prevent their use. Doctors are placed in a very difficult position, perhaps even having to tell patients that they can no longer treat them with their drug/s of choice.


    —  It is essential that time-scales for appraisals are established to minimise delays.

    —  The results of appeals should be announced promptly.

    —  Re-assessment following a successful appeal should be conducted without undue or unnecessary delay.

    —  Where appropriate, NICE should look to make more use of fast track procedures, particularly for treatments that are believed to improve survival.


  The method by which NICE accepts or refuses the evidence and suitability of stakeholders appears arbitrary.

  Lip service is given to incorporating the knowledge, concerns and expertise of specialist health charities and patient advocacy groups. NICE has failed to identify relevant patient advocacy groups as being stakeholders in a particular appraisal despite their expertise in representing the patient population affected. Specialist doctors are also marginalised, with either gross under-representation or a failure to represent at all the concerns of the expert clinical community on the NICE assessment panel.

  The problems of expert witness's representation in the review process are further compounded by systematic failures in the administrative ethos of NICE. We are aware of several organisations and individual experts who have not received correspondence from NICE inviting them to participate. For example, NICE did not contact the two specialist colorectal cancer patient advocacy groups in the recent appraisal of colorectal drugs.

  These problems of process may reflect problems of resourcing of NICE. CERT shares the concern of other stakeholders that patients' experience should play a greater role in the NICE appraisal process.

  NICE do not let health charities and patient advocacy groups hear how their evidence is taken into account by the Appraisals Committee in its deliberations, yet such transparency would go a long way to reassure stakeholders of their contribution.


    —  It is in NICE's interest to ensure that their processes and procedures always stand up to scrutiny. To ensure this, full consideration must be given to stakeholders' evidence in a much more inclusive way than at present.

    —  NICE could ensure that the deliberations of the Appraisals Committee are open and transparent to all stakeholders.

    —  Many patient groups that possess some of the most relevant patient-centred evidence are being denied the opportunity to state the case on behalf of patients and their families. Full account should be made by NICE of all those patient-centred groups who have an interest in a therapeutic area.

    —  Furthermore, there are many other professional organisations that work with the medical profession who can provide a general overview of the position within health care. Similarly these organisations should not be denied the opportunity to present evidence to NICE without compelling reasons.


  This is perhaps one of the crucial areas in the operation of NICE.

  At the moment, a single expert is recruited by NICE to advise an Appraisals Committee comprised of clinicians and other individuals with no experience in the specific clinical area. But how is the expert identified, and how does NICE ensure that the clinician represents the expert opinion on current best practice? Other problems relate to the Appraisal Committee trying to interpret clinical trials in an area that is not their speciality, rather than having expertise in the area being appraised.

  We have heard of occasions when NICE have telephoned experts to ask whether they would be available to advise the Appraisals Committee on a particular date in two or three weeks time. When the experts have said that they were not available on a particular date, NICE have simply advised that they would find someone else. This leads people to the conclusion that NICE are working at short notice through a list of people to see who might be available on a particular date, rather than identifying the experts in the field with sufficient warning to ensure their attendance.

  Medical consultants have said that they find it "staggering" that treatments are being appraised by clinicians who have no working understanding of the clinical area they are investigating.

  The medical profession remains critical of a system that uses non-expert clinicians in its appraisals. Many clinicians have said they will simply ignore NICE recommendations if they run contrary to their own experience and practice.

  The failure to use experts of suitable standing, and the use of clinicians who do not operate within the therapeutic area under investigation, has led to NICE being described as inadequate, unrepresentative and lacking credibility within the medical profession.

  Unless NICE uses experts who are widely recognised, its credibility within the medical profession will remain suspect.

  Furthermore, the over-emphasis on the role of health economics undermines the clinical credibility of decision-making.


    —  Consideration should be given to using a panel of experts to advise the Appraisal Committee, rather than just a single clinician.

    —  NICE must ensure that it uses experts of a suitable standing to advice the Appraisal Committee.

    —  NICE must also ensure that the experts have appropriate input into the appraisal procedure.


  A number of stakeholders have reported many appraisals to be problematic.

  Some stakeholders have reported substantial errors in the evaluation reports on which the Guidance is based. It is worrying that the quality of evaluation reports and of the draft Guidance that is developed is variable or inconsistent.

  Other errors in the evaluation reports have been as great as two orders of magnitude with a complete reversal of the result stated.

  NICE arbitrarily decided to extend the appraisal to include use of a drug for unlicensed indications even when the drug is not being used for that purpose, and this has delayed the appraisal of the licensed indication.

  The following provide examples of some of the problems encountered, but are not by any means an exhaustive list.

COX-2 Inhibitors:

    —  Experts reported that there were numerous factual errors in the Assessment Report, and particularly on the two coxibs where more clinical information exists - this was in stark contrast with the data on the two older drugs that was mainly accurate. Experts questioned whether different people had prepared the Report for the older and the newer drug assessments. Despite substantial errors being pointed out, no attempt was made to correct the Assessment Report. When an Assessment Report is patently inaccurate, it is difficult to see how the draft appraisal that is developed from it can itself be accurate, and as a result the credibility of the Guidance is undermined.

    —  Several patient groups invited to submit to NICE never received a letter.

    —  NICE insisted on grouping four drugs together for the appraisal process, even though there is evidence to suggest that the two older agents may still cause the dangerous gastrointestinal adverse events that the Guidance is designed to stop, and these two agents also lack evidence from clinical studies demonstrating that they reduce GI adverse events. NICE appears to have lost sight of the original objective - to reduce the number of people suffering dangerous GI adverse effects that result in 12,000 hospitalisations and over 2,000 deaths a year.

Taxanes in Ovarian Cancer

    —  NICE substantially underestimated the number of women who would be eligible for and benefit from taxanes in ovarian cancer. They had estimated that approximately a third of women with ovarian cancer would be eligible for treatment. When investigated by CERT, the reference used by NICE was found to be based solely on a local estimate from a single oncologist who it is understood does not even treat ovarian cancer. The feedback to CERT from experts in the field was that 60-80 per cent of women with ovarian cancer would be eligible and benefit from taxane treatment, and this figure was therefore supplied to NICE. The final Guidance reflected this feedback.

Verteporfin in Age-related Macular Degeneration (AMD)

    —  NICE extended the scope of the appraisal on verteporfin to include patients with wet Age-related Macular Degeneration (AMD) associated with occult choroidal neovascularisation (CNV). This was outside the current licensed indications and in spite of assurances from the Department of Health that "treatments would only be considered in relation to their licensed indications".

    —  The inclusion within the appraisal of verteporfin for wet AMD associated with occult CNV was not discussed during the consultation rounds with stakeholders on the remit and scope of the appraisal. Needless to say, the input to the appraisal by the stakeholders would have differed if this "off-label" indication had been raised.

    —  Patients with wet AMD associated with predominantly classic subfoveal CNV will therefore continue to be denied effective treatment with verteporfin pending the licence approval for the other indication.

  The NICE appraisal process appears to lack any quality control or quality assurance procedures to prevent errors in either the Assessment Report or draft Guidance from being issued.


    —  NICE should adopt appropriate quality procedures into the appraisal process, but without introducing delay.


5.1  Resources within NICE

  It is in all stakeholders' interest that NICE not only works properly but is seen to work properly, and is fair and open in its decision-making process. It is thought by many that NICE lacks the proper resources to fully gain the confidence of the medical profession and patients.

  Some of the variability in appraisals may reflect insufficient resources at NICE. The health technology groups commissioned to develop the evaluation reports are paid only around £14,000. Considering that this is a three-month project requiring detailed analysis of all the relevant clinical data, it is to be expected that at this level of funding the bulk of the work is being performed by less-experienced staff, rather than by experts with detailed knowledge of the relevant area. Assessments are carried out by a limited number of academic units in England and Wales. These units are comprised of health economists, with little or no input from the clinical community. There is a degree of reflexivity within this academic community, with personnel moving from one unit to another repeating policy learned at their previous institution. No use has been made of health technology resources outside of the UK.

5.1  Resources within stakeholder groups

  Many of the patient advocacy groups and research charities, particularly those that are heavily reliant on voluntary work, lack the resources and expertise to respond to the opportunity that a NICE appraisal presents to their community. Those involved in broad areas of interest (such as the elderly) receive numerous requests from NICE to submit evidence, but are unable to respond due to the sheer volume of work involved. Ideally, such funding should be independent from NICE. One potential source could be the National Lottery.


    —  Consideration should be given to ensure that an appropriate level of funding is available for NICE to meet the projected workload and ensure adequate quality.

    —  Consideration should also be given for funding that is independent of NICE to allow patient advocacy groups and research charities to respond to the requests for submissions and participate as stakeholders.


  NICE consistently disregards ethics in its appraisals. It appears to have little concern for the ethical dilemmas which specialists such as oncologists encounter in their working day. For instance, a different emphasis is placed on survival times for different tumour types. The breast cancer lobby has successfully exerted significant pressure on available funds and political focus. Colorectal cancer does not have such an active lobby; consequently funding and political activity are less. If three months' survival benefit from a taxane used to treat advanced breast cancer is sufficient enough to be recommended by NICE, then why shouldn't a similar survival advantage be sufficient to gain a recommendation from NICE for the drugs used to treat patients with advanced colorectal cancer?

  As another example, in non-Hodgkins' lymphoma, NICE recommended entry of patients into a randomised, controlled clinical trial despite the fact that the treatment under appraisal is a salvage therapy making an RCT unethical.

  Rather than tackle the ethical issues directly, NICE recently announced that they were to set up a panel of members of the public as a "Citizen's Council" to advise it on, amongst other things, ethical issues. The Council will not include anybody in patient advocacy groups, those working in healthcare, or even those working in ethics. CERT is concerned that the panel is simply a way of enabling NICE to take unethical decisions whilst having a group of the public without any expertise in ethics to take the blame.

  The concern that many stakeholders had regarding NICE was that it was originally established by the Department of Health to take unpalatable decisions that Ministers did not want to make themselves in order to avoid being tarnished in the public's eyes. Having found how difficult some of these decisions are to substantiate, NICE now appears to be delegating these same questions to another group—the Citizen's Council—in order that NICE itself avoids criticism.

  It is unclear whether the Citizen's Council will have access to information from sources other than NICE in order to reach their decisions, or whether their deliberations will be open and transparent. If NICE is the only source of information, there is a clear risk that the Council could be fed with unbalanced information in order to reach a particular decision.


    —  The Appraisals Committees should include an ethicist.

    —  The role of the Citizen's Council should be examined to ensure it cannot be used by NICE as an unskilled group that can be fed unbalanced information in order to reach a particular decision. Where possible patient representatives knowledgeable in clinical trial design, health economics and patient advocacy should be recruited.


  The NHS lacks an open and transparent means by which stakeholders can easily assess whether particular treatments are subject to inequitable access across the NHS. Most stakeholders, particularly the patient advocacy groups, charities and professional organisations, have to rely on anecdotal information on access to particular treatments, although there are commercial sources of information available which are actively used by both the Department of Health and industry. An open and transparent mechanism of monitoring would allow the NHS, Department of Health and stakeholders to identify problem areas and respond at a local level rather than waiting for a horror story to emerge through the media.

  CERT welcomes the announcement from Lord Hunt (5 Dec 2001, "New Statutory Obligations for the NHS to fund treatments recommended by NICE"). We believe it is important that the Health Select Committee ask how these recommendations are to be enforced.

  Some patients receiving drugs NICE have recommended are still experiencing delays in treatment due to Health Authority funding problems. These can extend beyond the end of the financial year in which NICE Guidance is issued by (eg breast cancer, MND, Alzheimer's disease). The situation appears to be slowly getting better, but it is still unclear why there is such a disparity between those Health Authorities who can immediately find the money from increased Government investment in the NHS, whilst others take much longer and require greater amounts of pressure exerted on them. It is a pity that those Health Authorities who do the job well don't get credit, and those who fail aren't held to account.

  It is unclear at this stage how the shift of responsibility from Health Authorities to Primary Care Trusts will impact the funding process and the uptake of NICE Guidance.


    —  There should be open and transparent monitoring of the use of treatments recommended by NICE.

    —  Consideration should be given to how the new statutory obligations are to be enforced.

    —  There should be open and transparent monitoring of the transition of responsibility from Health Authorities to Primary Care Trusts upon the uptake of NICE Guidance.


  The general perception of NICE is that it is a child of the Department of Health. Many of its staff have come from the Department, and this wholesale importing of personnel can only lead to a culture that "follows the Whitehall party line". There is at least a perception that the culture is dominated by the Department of Health, whereas the work of independent personnel would be welcomed by stakeholders.

  Furthermore, should any of the stakeholders believe that there are problems with the draft Guidance developed by NICE, the main route of recourse is through the appeal procedure. However, the appeal board itself includes two members of NICE—the chairman and a non-executive director—which does little to reassure stakeholders and observers that the process is truly independent and credible.


    —  CERT calls for the active recruitment of senior personnel from overseas, industry and non-Department of Health-aligned academia. Particularly welcome would be the employment of health economic departments from overseas, facilitating an increase in the number of assessments possible at any one time. This would also minimise the bias of a UK academy that favours a non-interventionist therapeutic policy.

    —  The appeal board should be truly independent from NICE and the Department of Health.

January 2002

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