Select Committee on Health Minutes of Evidence

Memorandum by the National Cancer Alliance (NC 95)



    —  The NCA welcomes the formation of NICE and the rationale behind its work and thinks following ongoing reviews of its remit and work it can only improve.

    —  We have some concerns regarding a lack of clarity regarding NICE's overall remit and terms of reference. This is reflected in the questions asked by the Health Committee inquiry, which do not relate to what we understand to be the terms of reference of NICE.

    —  Topic selection, and the NHS implementation of guidance, though not part of NICE's remit, need to be more carefully thought through, particularly in relation to the impact on NHS prioritisation of resources and the potential for sealing in inequity of access, rather than addressing it.

    —  We have a number of concerns regarding some of its methods and evaluations processes for assessing Health Technologies.

    —  We are concerned about the cost-effectiveness and economic evaluation methodology is not transparent or appropriate.

    —  We are also concerned that linking cost-effectiveness to clinical effectiveness in the one body evaluations could lead to mistrust in the outcomes of both evaluations.

    —  The purpose and type of patient involvement needs to be clarified and properly resourced.


  1.1  The apparent criteria used to select the Topics for appraisal or guideline development are opaque.

  1.2  The adverse impact of the topic selection process and outcome (which topics have and haven't been selected) has clearly been under-estimated. This affects patients and the NHS in two key ways:

    (a)  "NICE Blight".

    It seems that many local health organisations (Trusts, GPs, HAs, Commissioners) feel they must await NICE guidance on a particular Health Technology Assessments (HTAs), or guideline before agreeing to funding or prescribing (even if the appraisal is due to take place some time in the future). This is clearly an absurd stance to take. The DOH should issue firm instructions to health ecomonies and providers that this is not an acceptable approach.

    (b)  Skewing of NHS funding resources and clinical priorities.

    The Minister has instructed the NHS that from January of this year, they have a statutory obligation to fund NICE approved HTAs—but out of existing resources and cost-envelopes. This is to be welcomed in some respects, as it deals with the implementation and "NICE-But No Teeth" issue. However, this ruling (inevitable though it has been) causes other—un-thought-through scenarios and problems, most significant of which relate to the skewing of funding priorities to those interventions which have been "Nice-d". This means that other interventions or service-improvements (whether for the same condition or different condition) will necessarily get less funding. This is the case, whether or not the NHS has received ring-fenced funding to support the extra costs of the intervention.

1.4  An illustration

  The government has promised additional monies for cancer services modernisation. In 2001-02 an additional "hypothecated" £255 million was allocated to Health Authorities for specified areas of cancer services improvement. It was suggested by the DoH that out of the £255 million, an indicative £170 million should be "sub-hypothecated" in order to cover the possible costs of the 13 cancer drugs which NICE had been asked to appraise as quickly as it could (in year). This meant that only £85 million remained to fund all other major improvements in cancer services, including meeting the very ambitious waiting times target for people the GP suspected might have cancer; the urgent need to increase the number of cancer specialist cancer multi-disciplinary teams; the need to develop palliative and terminal care services; etc.

  The total costs of making these absolutely vital service improvements would obviously be far greater than the costs of the 13 NICE drugs, and would benefit many more patients in a number of ways. However, the mandate was clearly laid out by the DoH that HA's needed to ensure that they prioritised the funding of these high-cost drugs, once approved by NICE. This is important in that it effectively seals in inequity of resource allocation in one disease group—effectively for ever (the Saff agreements for 2001-02 were required to specify the total projected spend on the Cancer NICE approved drugs, both for 2001-02 and into the future as carry forward sums).

  1.5  The NCA, of course, is delighted that hypothecated, or ear-marked additional and growth resources are being allocated to fund improvement in cancer, and is also pleased that 13 cancer drugs were put as a priority for NICE appraisal. However, we would have liked there to be a more sophisticated discussion about the fair and equitable allocation of resources from the cost-envelope at both the national and local level.

  1.6  So, should a person with lung cancer have the choice of how to spend the £5,000 it costs for a cycle of for the NICE approved drugs (which gives them an average of three months extension of life). They could, for example, perhaps choose to be able to ensure they have a nurse looking after them at home, who knows how to manage the last difficult phases of a lung cancer death (some people get this, most do not). But on a population basis, the funding required to be "held back" to fund Gemcitobene, etc has effectively meant that the appointment of sufficient numbers of lung cancer specialists has had to be given a lower priority.

  1.7  The skewing of resource allocation because of the NICE process will of course be as polarised across the conditions and service-types as within them.


    —  Topics selected has already had an adverse effect on NHS priorities and access to equitable services. In some ways it is sealing in inequity - rather than improving it.

  THIS IS NOT the FAULT OF NICE - but crucially relates to it's work programme.

    —  The criteria and process of topic selection needs to be transparent and should cover a wide spectrum of conditions.

    —  Guidance should be issued to to the NHS that it cannot await NICE guidance before making interim decisions on appropriate interventions. Business as usual should apply

    —  The number of topics addressed by NICE needs to be dramatically increased—at least in the short-term.


  2.1  There seem to be three main areas of activity for NICE: Health Technology Appraisals, Guidelines and Audit.

  2.2  However, there seems to be ongoing confusion about the differences and similarities between these strands of work, how they link together and more importantly about the processes and methodolgies used and their final status.

The process and methodologies used

  2.3  For the final guidance (HTA or Guidelines) to be credible, whether with the public, patients, or the NHS, the process of assessment or development needs to be highly robust, transparent, and if at all possible, above criticism.

  2.4  It is recognised that NICE has been "learning on the job", and should be congratulated for sharing its learning and being open about the changes and developments it has made, and continues to make, to its appraisals and guideline development processes.

  2.5  However, there remain a number of concerns about the process and methods used by NICE in both its appraisal process and guidelines development processes. In particular:

    (1)  a general lack of clarity about the methodology and process used in the appraisals and the guidelines development.

    (2)  allegedly inappropriate differences in approach to assessing and weighing the evidence and the impact on the final decision-making process between different appraisals

    (3)  the reportedly inadequate quality of the evidence-base available, how NICE handles this when making a final recommendation, including the availability of relevant evidence (whether they be high-quality RCTs, or high-quality quality of life measures)

    (4)  the lack of clarity and perceived differences in the handling and impact of the patient-"witness"/ representative "qualitative input, including taking into account "patient-values", quality of life (usually never systematically and objectively measured).

    (5)  the difference in approach to "patient-involvement" between the guidelines and the HTAs.

    (6)  the lack of clarity and transparency regarding the whole methological approach and relevance to the cost-effectiveness appraisal-side of the work—and the impact of this on the final guidance.

    (7)  the lack of transparency and perhaps honesty regarding the approach to the links to having to assess "value-for-money", costs to the NHS, cost-effectiveness assessment (sometimes using QALYS, sometimes not—often with questionable outcome), and affordability.

    (8)  the impact of the influence of the different stakeholders, whether government, industry, patient-groups, academics, the NHS itself, on the process of evaluation remains unclear. This links also to the topic selection issue.


  3.1  The difference in "status" between NICE issued/ recommended HTAs and Guidelines in terms of the requirement of the NHS to implement NICE's recommendations (ie HTAs approved by NICE have to be funded by Health Authorities/—soon to be PCTs, whilst Guidelines do not) is questionable, confusing and could indeed increase inequity of access to best quality care.

  3.2  Why are guidelines not going to have to be statutorily implemented in the same way as HTAs? Is hints loudly at this really a matter of affordability in disguise. It makes no sense, especially when the guidelines are far more relevant to the overall costs and comprehensive management of a particular problem or condition.


  4.1  Although this is currently NICE's remit, mixing the "does it work, or work-enough?" evaluation process with the "is it worth they money/ is there something equally helpful for less money/ can or should we afford it" causes problems to the process and outcomes of both elements of assessment.

  4.2  Firstly, so-called cost-effectiveness measures are still very crude, hotly debated can only be used in some, restricted circumstances. So-called "cost-effectiveness" gets quickly entangled with "value-for money, and affordability issues and the broader rationing and ethical prioritisation debate.

  4.3  Patients, patient-groups, clinicians and indeed the NHS, think that the cost-effectiveness assessments will have an impact on the final outcome of the NICE approval process, either positively or negatively. So it is widely assumed that if an intervention is deemed to be "too expensive" (and it is unclear what this "too-expensive" is and how it is arrived at, as QALYS are used in only half the assessments, and in some cases perhaps inappropriately so), then even if it proven to be "clinically effective" (ie has more benefits than dis-benefits for patients), then it still won't be approved.

  This means that people are suspicious of the purity and objectivity of the process.

  4.4  It may be that there should be a separation between the assessment of "clinical-effectiveness" and of cost-effectiveness", the former undertaken by NICE—systematically, objectively appraising and assessing the clinical and scientific evidence base for interventions, guidelines, etc—whilst another body or organisation should be established to undertake the work on costs—which should include a more robust understanding of what what the costs and/or prices are and why, whether they represent value for money, what they are worth (financially and in health-benefit terms), etc.

  4.5  This separation of the two processes or functions of NICE, would mean that hitherto hidden and opaque decision-making processes regarding funding and resourcing of health care interventions, (including costs, links to effectiveness and affordability, etc) would be brought into the open, shared and understood.

  4.6  Currently, the mixing up of the clinical and cost-effectiveness processes pollutes the outcomes of both, especially when the methods are unclear or questionable.


  5.1  Apart from the above point on the need to separate the clinical evaluation from the cost-effectiveness assessment, we are concerned that the approach to health economic assessment in the NICE process has not been consistent. A review needs to be undertaken of the methodologies and measures used by NICE, and the applicability and use for different circumstances.


  6.1  The establishment of the new patient-involvement "unit" is to be welcomed. However, this does not really address the key problem, namely that small voluntary organisations, especially those made up of, or run by "real-patients", rather than patient-representatives or health professionals, simply do not have the resources or time to be able to properly contribute to the NICE process.

  6.2  NICE needs to resource the relevant involvement of real patients and carers and those organisations or groups which truly represent patient-views and experiences.

  6.3  NICE should also be much clearer about whom it is "consulting" in relation to the "voluntary sector" or so-called patient-advocacy groups and for what purpose. Many large national health-related charities have no formal patient-constituency and have no formal mechanisms in which to hear the views or represent the views of patients. Many raise money for research and support health professionals' research work (often in partnership with the pharmaceutical industry). Other's are effectively "service-providers" in their own right—be that professional information and advice services, or other more direct care-provision. These are therefore not "patient-groups".

  6.4  Many of the smaller patient-groups (and probably some of the larger) have received offers of help and support from the pharmaceutical industry in the formation of their submissions to NICE. Some have turned these offers down, others may have accepted help in tangential ways. This draws into question the legitimacy and value of the patient-input and the independence of the overall consultation and submission process.


  7.1  The NCA greatly welcomes the establishment of a national body undertaking the national development of clinical guidelines, protocols and evaluation of interventions. This, amongst other initiatives aimed at addressing inappropriate variation, and inequity of access to relevant and best treatment is to be strongly supported.

  7.2  The eventual outputs of NICE, having a national focus and producing a national body of work, with and on behalf of local health professionals, will, in theory, be able to help address inequity.

  7.3  However, there are a number of limiting factors already alluded to above, including the number of topics being appraised and the rate of appraisals being so limited as to increase inequity; the problems associated with the need to be constantly updating assessments and guidelines.

  7.4  The NHS implementation issue and the differing status of the guidelines and HTAs has been discussed above. NICE is working on improving its dissemination and publicity process but it does not have the remit or role of overseeing implementation. However, if the Guidelines—arguably the more important part of NICE's work—are not given equal status (in terms of statutory implementation obligation) there is danger they will remain in the territory of Confucius—wise thoughts from the mountain!


    —  Topic selection needs to be reviewed, as does the spread of topics, number and rate of appraisal

    —  The NHS should be given clear guidance of how to handle the situation whilst awaiting NICE guidance

    —  Guidelines should have equal implementation status as the HTAs

    —  The nature and type of the clinical evidence needs to be "fit for the purpose"—ie RCTs are important in some circumstances and qualitative evidence is A Grade evidence in others

    —  The weighting of the evidence needs to be more transparent.

    —  The overall methodology, process and above all rationale needs to be clearer and more transparent

    —  The cost-effectiveness assessments need to be undertaken by a separate body. NICE should concentrate on the clinical-effectiveness agenda—which is hard enough

    —  A different body should be set up to use the clinical effectiveness information and work in a different way on the cost-effectiveness agenda and the links to NHS costs and affordability—even rationing.

    —  There should be a review of the cost-effectiveness measures used and how they can and cannot influence final cost-effectiveness decisions

    —  Patient-groups—especially small ones should be given resourced properly to be able to input effectively to the process

    —  "Patient-groups" need to be honest about who they represent and how they do this—the may not be real patient groups. NICE needs to be sure about which groups it works with for what purpose.

January 2001

previous page contents next page

House of Commons home page Parliament home page House of Lords home page search page enquiries index

© Parliamentary copyright 2002
Prepared 8 July 2002