Select Committee on Health First Report

Conclusions and recommendations

1.  We note that it is not the role for Ministers to directly or indirectly seek to influence the NICE decision-making process. (Paragraph 68)

2.  It is clear that the environment in which NICE operates has changed considerably since the Institute was established in 1999. It is also clear that there is a vital role for NICE in the rationing of healthcare and in encouraging best clinical practice. In the future the role of NICE will be ever more important and demanding with new expensive drugs and a slower rate of growth in NHS expenditure. There remains, however, concern about aspects of how NICE does its job. (Paragraph 79)

3.  It seems to us appropriate that topics are selected for interventional procedures, clinical guidelines and public health guidance. It is not appropriate, however, to limit technology appraisals to selected, often new and expensive, products. Instead, as we recommend below, all new drugs should be assessed. (Paragraph 94)

4.  Witnesses were concerned that NICE's focus on acute treatments, in particular medicines, could skew NHS spending towards selected new and expensive (NICE-approved) drugs for acute illness. (Paragraph 95)

5.  In our previous report we recommended that NICE give more emphasis to examining old technologies to encourage disinvestment. This the organisation has failed to do as fully as we expected. Its statement that few interventions have absolutely no benefit may be true but is irrelevant. Many treatments currently used are not cost-effective as many studies attest. NICE should adopt a similar standard of cost-effectiveness in assessing such treatments as it uses in its technology appraisals. The organisation must now give more emphasis to disinvestment. One approach would be to undertake more MTAs, which would reveal the existing treatments that provide poor value for money. (Paragraph 96)

6.  We heard much criticism of the use of QALYs. Some of the criticisms seem to be the special pleading of disappointed parties. It is vital that a method which allows comparison of the benefits and costs of different treatments for different conditions is used in cost-effectiveness evaluations. However, it is also vital that the system is accurate and reflects the real costs to society and the benefits to patients. We recommend that:

  • Research is undertaken to follow up specific guidance to see whether the predictions of the cost-effectiveness analysis are borne out in practice;
  • Wider benefits and costs, such as costs borne by carers and social care services, be more fully incorporated into NICE's assessment. We were told that this would have to be a decision for Parliament. (Paragraph 120?)

7.  NICE does not have all the information it needs to assess and compare treatments. First, while access to EMEA documents and other changes have improved NICE's access to information, it still does not have access to all the relevant information which is available. Secondly, clinical trials undertaken by pharmaceutical companies understandably focus on generating data about the drug's efficacy and safety, which is required for the licensing process; such trials are not usually designed to generate the type of data on cost-effectiveness which NICE requires. Third, in some areas, without commercial sponsors, notably public health and many physical and psychological therapies, there is little research about the cost-effectiveness of different interventions. (Paragraph 143)

8.  We recommend that NICE be granted the right to see all the evidence the MHRA uses when making its decisions. We appreciate that this would mean that there would be some commercial-in-confidence material that NICE could not make public when it published its guidance. (Paragraph 144)

9.  We welcome the fact that both NICE and drug companies are aware that they need to collaborate closely to ensure that clinical trials are undertaken with the needs of NICE appraisal in mind. The Government should encourage all countries in which large-scale clinical trials take place to adopt a similar policy. We support the mandatory registration of all clinical trials so that the results of all negative trials are accessible. We recommend that NICE assesses and reports the quality of the research it receives. (Paragraph 145)

10.  More publicly funded research should be undertaken to assist the development of public health guidance and other areas without commercial sponsors. (Paragraph 146)

11.  Many witnesses thought that too few experts with the relevant detailed expertise were involved in the process of producing guidance. Since they have a permanent membership, Appraisal Committees are unlikely to have such experts. They do consult experts, but this is unsatisfactory because such experts appear for the day alone. We therefore recommend that Appraisal Committees appoint specialist advisers, without voting rights, to work with the Committee throughout consideration of a technology appraisal or clinical guideline. This will improve guidance and ensure public and patient confidence in the system. Decisions about which experts should be appointed should remain the responsibility of NICE following consultation with the appropriate clinical bodies. (Paragraph 156)

12.  The wide consultation which takes place during the development of NICE guidance is greatly valued. While we agree that it is difficult for some organisations to respond within the often brief time limits, we recognise that a long consultation period would slow the guidance production time further. Nevertheless, the situation would be improved if NICE were to give interested stakeholders greater warning of forthcoming consultations, to allow them to organise their resources in time to respond effectively. (Paragraph 168)

13.  Some consultees complain that their views are ignored. We understand that NICE does not have the resources to respond individually to each consultee. NICE could, however, issue a standard response to inform every consultee how it will respond and setting out how the system works. (Paragraph 169)

14.  We note the pressure to change the grounds for appeal, but consider changes might cause more problems than they solved. Allowing additional evidence at the appeal stage would extend the process significantly, and might discourage companies from producing high quality trial data at the time of first assessment. It also might risk more "gaming" appeals. We make recommendations in the next section which we expect will lead to fewer appeals being brought in the first place. (Paragraph 184)

15.  A shorter, less in-depth initial evaluation of medicines at an early point would be useful. It is important that clinicians have access to independent information about new therapies as soon as they are available. However, a quick, in-depth, fully consultative evaluation for all new medicines by the time of launch is not possible. We therefore recommend that NICE should examine all new medicines for their indications as set out in the marketing authorisation. Assessment should be carried out during the period between licensing and launch. It should be brief and published prior to, or at the time of, launch. There should be no formal appeal process and only limited consultation. These brief assessments should be followed by a larger scale multiple technology appraisal for selected products (an MTA or STA as appropriate) at a later date, when more evidence is available. The technology appraisal should include current levels of consultation. The guidance issued at this later stage should be definitive, over-riding that issued earlier. (Paragraph 200)

16.  Since providing an evaluation of all drugs at launch will be a more rough and ready process, it would be inappropriate to use the same threshold range as the full assessment. One of the aims of the new process is to ensure that treatments which are obviously cost effective are available at an earlier stage than at present. We therefore recommend that a threshold below the current range be used in these early assessments. This could be raised for individual products in special circumstances, for instance where no other treatment exists. At the time of the full assessment, the cost per QALY threshold could increase. (Paragraph 201)

17.  The threshold or ceiling NICE employs (measured in pounds sterling per QALY) to decide whether a treatment is cost-effective, and so should be available in the NHS, is not based on empirical research. Nor is the threshold directly related to the NHS budget, since the threshold has remained constant while the budget has increased hugely since 1999. (Paragraph 239)

18.  The threshold used by NICE does not take into account the funding decisions made by PCTs generally. For interventions not assessed by NICE, PCTs appear to use thresholds which vary from treatment to treatment but for the most part seem to be lower than the NICE threshold. (Paragraph 240)

19.  Many PCTs struggle to afford to implement NICE technology appraisals, as well as clinical guidelines. As more interventions are evaluated it is feared that the position will become unsustainable. Funding is essentially ring-fenced for technology appraisals, leaving PCTs little room for manoeuvre in their budgets to reflect local needs and priorities. (Paragraph 241)

20.  A number of steps were proposed by witnesses to alleviate the situation. To improve coordination between NICE and PCTs, we support the wider use of implementation consultants, who would provide information both from NICE to the PCTs and from the PCTs to NICE. (Paragraph 242)

21.  There must be incentives for clinicians to be very careful about the use of expensive drugs. We recommend that current exclusion of high-cost drugs from the payment by results tariff be reviewed. (Paragraph 243)

22.  It is difficult for individual PCTs to decide which areas to prioritise and in which to reduce spending when their expenditure rises as a result of new NICE guidance. In the absence of NICE guidance on disinvestment, we recommend that groups of PCTs should work together to determine appropriate areas of spending in consultation with the public. Such groups should also examine existing treatments to determine which are not cost-effective. (Paragraph 244)

23.  While the measures listed above would mitigate the problems PCTs face, the fundamental problem which has to be addressed, according to several witnesses, is NICE's cost-effectiveness threshold. Given the uncertainties, for example about the thresholds used by PCTs, we are not in a position to decide authoritatively whether the current threshold, or threshold range, is appropriate. We recommend that more work similar to that undertaken by Professor Smith and colleagues at York University takes place on the thresholds used by NICE. We are encouraged that NICE has commissioned its own research in this area. (Paragraph 245)

24.  During the inquiry, doubt was cast on whether NICE alone should continue to determine the level of the threshold. We consider the present situation is unsatisfactory. We recommend that a separate body, with representation from NICE, the Department, PCTs and others should set the level, or range, to be used. NICE's threshold should be closely linked to that used by PCTs. The threshold should also relate to the size of the NHS budget. The new body should decide whether orphan drugs continue to be treated differently from other treatments. (Paragraph 246)

25.  Demand for NHS services will always exceed the ability to meet it. Not every treatment can be provided to every person. NICE has a vital role to play in the rationing arrangements and, working with Government, should make clear to the public how and why such decisions are made. (Paragraph 247)

26.  There need to be additional measures to improve the implementation of clinical guidelines. There should be more help for PCTs to implement guidelines. We recommend that the Department ensure that PCTs are aware of the assistance that is available and develop other ways of helping PCTs to plan and prioritise clinical guidelines. (Paragraph 294)

27.  Better measurement of guidance implementation is also needed. Self-assessment is not enough. We recommend that the Healthcare Commission conduct more in-depth inspections of this element of practice. (Paragraph 295)

28.  Improvements to the system of evaluating medicines and greater involvement of experts in the technology appraisal and guideline development processes should also result in guidance that is more acceptable to clinicians. (Paragraph 296)

29.  We also recommend greater involvement of Royal Colleges and other professional organisations in ensuring implementation. For instance, the approval of trusts as training organisations could be linked to uptake of guidance. Elements of clinical guidelines, particularly those covered by technology appraisals, such as risk assessment of VTE patients, should be mandatory. (Paragraph 297)

30.  To combat public confusion over the status of technology appraisals and other types of guidance, we recommend:-

  • Recommendations made following technology appraisals should be referred to as 'NICE directives'; and
  • Everything else should be referred to as guidelines or guidance. (Paragraph 298)

31.  Greater involvement of PCTs in NICE assessments and a re-examination of the NICE cost per QALY threshold, which we recommend above, would produce guidance which NHS organisations find more affordable. (Paragraph 299)

32.  Given that discussions between the Government and the pharmaceutical industry on future drug pricing arrangements are already underway, we do not make any firm recommendations on how a future system should operate. However, we agree with the Government that better mechanisms are needed to ensure that the NHS pays a fair and affordable price for medicines. Any change to the system of medicines pricing is likely to have profound consequences for NICE and the Institute should be involved in any changes that might affect how it works. Moreover, it should be funded for the alterations in practice it might be required to make. (Paragraph 343)

33.  We recommend that risk-sharing schemes be used with caution. They should not be used as a catch-all in cases of uncertainty over a drug's benefit. The Department must bear in mind the evidence that will be foregone in such cases. Uncertainty would be better addressed by the careful design and performance of a publicly funded randomised controlled clinical trial. Better use should be made of NICE's 'only in research' recommendation in this regard. (Paragraph 344)

34.  The short evaluation of all medicines at launch, which we recommended earlier, could be established in such a way that negotiations on drug pricing could be incorporated into the process. The NICE evaluation process could also take account of potential improvements in subsequent data about clinical and cost effectiveness, and its consequences for product pricing. (Paragraph 345)

35.  To improve the evaluation process, we recommend that:

  • All drugs be assessed at the time of licensing, so that clinicians can prescribe useful and cost-effective products as soon as they are launched;
  • There be more emphasis on disinvestment;
  • Our last report on NICE recommended that the legislation be changed to accommodate the need to ensure that assessments of products take account of the wider benefits to society; we make the same recommendation here;
  • NICE have access to the same material used by the licensing body, clinical trials be registered and there should be closer working between NICE and the industry to enable these early assessments to take place. (Paragraph 349)

36.  The affordability of NICE guidance and the range, measured in cost-per-QALY, it uses to decide whether a treatment is cost-effective is of serious concern. The threshold it employs is not based on empirical research and is not directly related to the NHS budget, nor is it at the same level as that used by PCTs in providing treatments not assessed by NICE, which tends to be lower. Some witnesses, including patient organisations and pharmaceutical companies, thought NICE should be more generous in the cost per QALY threshold it uses, and should approve more products. On the other hand, some PCTs struggle to implement NICE guidance at the current threshold and other witnesses argued that a lower level should be used. However, there are many uncertainties about the thresholds used by PCTs. Accordingly we cannot authoritatively at this stage recommend a change in NICE threshold. Nevertheless, we recommend that it be reviewed. We do recommend that an independent body determine the threshold used when making judgements of the value of drugs to the NHS. (Paragraph 350)

37.  To improve the implementation of NICE guidance we recommend:

  • More help for PCTs to implement guidance;
  • Better assessment of the level of uptake;
  • That PCTs should play a larger role in the development of guidance;
  • Better use of experts in the development of guidance;
  • A change in the terminology used by NICE, to clarify to patients what they can and cannot expect by right from their local NHS organisation; and
  • That some elements of clinical guidelines should be made mandatory. (Paragraph 352)

38.  We recommend that risk-sharing schemes be used with caution. They should not be used as a catch-all in cases of uncertainty over a drug's benefit. Uncertainty would be better addressed by the careful design and performance of a publicly funded randomised controlled clinical trial. Better use should be made of NICE's 'only in research' recommendation in this regard. (Paragraph 354)

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