EXECUTIVE SUMMARY

—  The increasing number of challenges to NICE's decision making should be seen and understood in a wider context.

—  Challenge should not always be seen as a bad thing. Nor should legal action if the case is testing an important principle. The courts are there to define the parameters within which public bodies can act.

—  No organisation, including NICE, should have the power to commit resources without also having the budgetary responsibility. The mandatory nature of the technology guidance should be removed at the very least.

—  As currently constructed NICE's decision making and the mandatory requirement for PCTs to implement its guidance on individual technologies (mainly drugs) creates an ethical dilemma that needs to be resolved. It also creates a distortion of clinical and service priorities.

—  NICE does and can do useful work on behalf of the NHS. Although there are problems, most see a role for NICE.

—  Although NICE's decision-making may come under criticism caution must be exercised in making recommendations which create a situation that even further undermines priority setting.

GENERAL COMMENTS

  1.  This response is confined to NICE's technology appraisal programme, the implementation of which is mandatory for primary care trusts.

THE COMMITTEE'S AREA OF INTEREST

Why are NICE's decisions increasingly being challenged?

  I believe there are three main reasons for this.

Misperceptions About the Role of NICE

  2.  The need to consider "value for money" in the NHS is accepted but this is not always linked with "rationing".

  3.  When NICE was established, it was "sold" to the public as an organisation which would "end postcode rationing". In my working day I regularly come across the view of NICE as "defender" of patient access.

  4.  The public has a poor understanding of the "effectiveness" of treatments and the concept of "cost-effectiveness". This is true also of many people working in the NHS, including healthcare professionals. In addition we have not yet reached the tipping point at which the need for priority setting is not only fully accepted but also faced openly and squarely.

  5.  Confusion exists over the role of licensing and how this relates to the role of NICE. For many licensing is seen as the stamp of approval for a drug (although few policy makers feel that licensing provides adequate information on which to properly judge the value of a drug).

  6.  Given this, many individuals find it perplexing that doctors have the freedom to prescribe a drug but that the NHS does not have to pay for it.

  Given the above, it is not surprising that a "no" decision from NICE comes as a shock to patients and the public.

Challenges are an expression of general social trends

  7.  NICE and primary care trusts (PCTs) are the organisations tasked with making explicit decisions about the availability of services in the NHS. This task, by definition, is going to create controversy and invite challenge. Anger is a common emotion associated with loss. Those who have had a long term involvement with priority setting understand that having to deal with fear, anger, denial and bargaining is a part of their role.

  8.  With respect to legal challenge, NICE is not unique in experiencing threats of legal challenges and at an increasing rate. This is part of a trend in readiness to use the courts. During most of my time as a consultant in public health (about 15 years) there were about 5 significant court cases related to funding issues. In contrast, in the last year there have been at least 5 cases that I am aware of.

  9.  The recent high number has, in part, been encouraged by the very unusual precedent one judge has set by requiring the PCT to fund treatment before the case was heard.

  10.  As a result most commissioners expect an ongoing rise in the number of legal challenges. It is not surprising that NICE finds itself defending its decisions in court. Most of us working in this area are only surprised that it has taken this long.

  11.  Legal challenge should not be seen as a bad thing or a result of bad decision making. True, for an organisation to lose a case because of poor process is neither good for the organisation nor for the NHS. This is to be avoided. However, it is important sometimes to use the courts to test principle and define more clearly the parameters within which public bodies operate. Unlike local authorities, the NHS and the Department of Health has always been extremely court adverse. This is frequently counter-productive.

  12.  A greater readiness to challenge (even when there is no real threat of actual legal action) also stems from a belief that the courts supports patients' right to treatment, which is does not. Health is often talked about as a basic human right. One can understand the sentiment but clearly this cannot be the case. When need is greater than the resource available which individual has a health need has "right to be met" and which does not?

  13.  The perception that it is wrong to deny treatment is fuelled by the fact that PCTs frequently step down when there is a real threat of legal action. This is interpreted as an acknowledgement of the PCT being "in the wrong". This is often not the case. PCTs are most likely to step down for one of four reasons (or a combination of them):

—  Fear of the court process and hostile publicity.

—  PCT manpower is stretched at the best of times and judicial review is very labour intensive. Some see defending a court case as creating a major diversion of their organisation's main purpose.

—  A reluctance to put the PCT at financial risk or concern over the fact that court costs have to come out of the same fund which is used for patient services.

—  PCTs are frequently subjected to covert political pressure to overturn their first decision not to fund.

  14.  Finally, there are a number of wider social changes and forces that increase the likelihood of challenge:

—  There is a well-described changing dynamic between both the public and public authorities and also patients and the NHS. This change is being promoted by the "patient as consumer" model of healthcare and increased access to information.

—  The Pharmaceutical Industry are becoming ever more sophisticated in the ways they can fuel demand for drugs.

—  The media plays a major role in creating demand and indeed dissatisfaction with public services—some of it justified and some of it not.

—  Politicians themselves also play a role. For example, the message that is frequently conveyed is that if a treatment is effective it will be either be freely available on the NHS. This is constantly reinforced yet clearly this cannot be the case when demand and need are greater than the resources available.

  15.  Public policy makers also need to be mindful of the information that is available to patients. The World Wide Web has completely transformed the information available to the individual and this has great potential for democratising Society. However it must be appreciated that there is a great deal of poor information and misinformation on the web. For example—a drug which "extends life by 4 weeks" is often described as one which "increases survival". Clearly these are two very different things. On more than one occasion I have talked to a patient or a carer following a decision not to fund some treatment, only for them to learn that the treatment will neither cure nor provide a remission. Not only to I find it appalling that in these instances patients have been so ill-informed but worse, that they should learn of a more realistic prognosis from a stranger, in such a stressful circumstance.

  16.  We cannot perhaps be surprised at the lack of good information available to the public and the patient. Interpreting the complexities of trial data is often beyond even individuals such as myself, and most certainly many practising clinicians. Presenting research data in a way that is accurate and meaningful to the lay person is a real challenge that we have not yet fully resolved.

NICE's basis for decision making make it particularly susceptible to challenge

  17.  Although most people are supportive of the need to take cost-effectiveness into consideration when committing resources, very few PCTs would support it as the ONLY basis for making a decision. For many involved in priority setting a treatment has to be cost-effective in order for it to be considered for funding. Its priority must then be determined by comparing its value with that of other competing needs.

  18.  Decision making basis only on cost-effectiveness creates problems on a number of fronts, some of which are dealt with in section 3 below. This method of allocating resources drives the decision maker to agree to fund a treatment when it lies below the threshold and decline funding when it is above. In this case the threshold is the QALY threshold set currently at an indicative figure of £30,000.

  19.  This basis for decision making sets up the environment for hot debate about whether the QALY is £29,999 or £30,001 (I know that NICE are not that rigid but this is for illustrative purposes only). If other factors and principles are used in decision making then the precise figure is less important—a rough figure is often good enough.

  20.  The situation is made worse of course, because in deriving a cost-effectiveness figure for a QALY assumptions have to be made. This is true of whether NICE is developing the costing model or whether the Pharmaceutical Industry is. Needless to say because assumptions are, just that, assumptions such decision making is likely to make it vulnerable to challenge and an endless debate about whether this figure is correct or that. Never mind that the Pharmaceutical Industry are considered by experts to frequently use overly optimistic assumptions, and therefore inflate the benefits—controversy has been created and a seed of doubt has been placed in the public's mind. Other problems with this type of decision making leads us to the next section.

The Committee's area of interest 3: Does NICE's evaluation process disadvantages particular patient groups by the process?

  21.  In short the answer is yes. This is related to both the fact that NICE's uses largely cost-effectiveness to determine funding and how NICE is used to commit the resources of PCTs.

  22.  Because NICE uses only a limited range of factors when coming to a decision—its decisions are often at complete odds with those of PCTs. The automatic assumption that this is because they come to better conclusions can be brought into question and from the PCTs perspective NICE does not say "no" often enough.

  23.  Most importantly this situation presents a real ethical problem, because the NHS now has two decision makers, committing a single budget, but making decisions in very different ways and using very different values. This is illustrated below. NICE's high threshold for saying no is also unsustainable.

  24  NICE is in the position of not needing to worry about the NHS budget, it is not required to look at affordability nor the real opportunity costs that might result from its decisions. It does not need to consider the priority of the treatment vis-a"-vis other competing needs. Its decision making is also incompatible with programme budgeting which those most knowledgeable about priority setting see as a key way forward in priority setting.

  25.  Fairness, in allocating resource, at the very least demands that everything gets a chance to be considered along side everything else. The mandatory nature of the technology guidance means certain drugs are treated preferentially—regardless of whether they are the most important and valued service to fund at any point in time. Indeed, it is often the controversial treatments that are referred to NICE. it is wrong to allow drugs and other individual interventions to queue jump priority setting processes.

  26.  The fact that NICE guidance is mandatory, however well intentioned this move was, has resulted in a serious distortion of health service priorities. To illustrate this, when I was working in Worcestershire I tried for over 5 years to establish a number of palliative care units in the County. At that time the County only had 5 beds for a population of 500,000. Year after year funding earmarked for beds was diverted to fund mandatory guidance by NICE. In some instances these drugs extended life by a matter of only a few weeks. When the treatment had done its work patients still then needed palliative care. It is completely perverse to have a situation where it is mandatory to fund a drug of marginal benefit (and no PCT would invest in treatment extending life by only 1-2 months by choice) yet a service a basic as palliative care beds are not. If the NHS is to consider mandatory services, which eradicates postcode variation, then it needs to look to the basics—not the latest "wonder drug" which very rarely fulfils this description. Yet we NEVER have had a discussion about what provision must not vary across country.

  27.  One might of course ask why would NICE support treatment that extends life by only a few weeks? The answer is related to point 18 above. It has to be understood that the QALY needs interpretation, which under the current system, NICE would find difficult to do. When it is estimated that a drug has a cost effectiveness £20,000 per QALY for example—it would be difficult for NICE to refuse to support its use. However this figure does not distinguish between whether one patient gets one year of quality adjusted life or whether 365 patients get one day each.

SUMMARY, CONCLUSIONS AND RECOMMENDATIONS

  28.  The increasing number of challenges to NICE's decision making should be seen and understood in a wider context.

  29.  Challenge should not always be seen as a bad thing. Nor should legal action if the case is testing an important principle. The courts are there to define the parameters within which public bodies can act.

  30.  No organisation, including NICE, should have the power to commit resources without also having the budgetary responsibility. The mandatory nature of the technology guidance should be removed at the very least.

  31.  As currently constructed NICE's decision making and the mandatory requirement for PCTs to implement its guidance on individual technologies (mainly drugs) creates an ethical dilemma that needs to be resolved. There are a number of options for resolving this ethical problem: remove the mandatory requirement, harmonise its decisions in line with PCTs decisions (which would significantly drive down the threshold) or give it a cash limited budget within which it has prioritise the treatments it considers.

  32.  We should not throw the baby out with the bath water. NICE does and can do useful work on behalf of the NHS. Although there are problems, most see a role for NICE—not the least of which is assessing evidence. Many PCTs would like to see this role retained and for NICE to rely less on Industry modelling (which has been the most recent requirement placed on NICE). Indeed NICE should have the freedom to demand information from Industry, to consider all sources of evidence and look at unlicensed protocols and unlicensed drugs. It should also have the freedom to demand better evidence including requiring treatment to be provided in the context.

  33.  On the other hand, if it must have a policy making role then its decisions need to come into line with those of PCTs who have a better grasp of the total needs of populations and services. NICE's horizons, in reality, are very limited.

  34.  Although NICE's decision-making may come under criticism caution must be exercised in making recommendations in order to avoid creating a situation that even further undermines priority setting. To react to the uncomfortable truth that Society cannot afford everything it wishes by pushing it underground merely adds injustice by placing the burden of rationing onto those with the least voice and those without big Industry interest. Making priority setting invisible is the worst of all scenarios.

  35.  Finally—I would make a plea to politicians, on behalf of those making difficult decisions on Societies behalf (both NICE and PCTs), to find ways to support those undertake the difficult task of priority setting. Providing leadership to create a debate about priority setting could most usefully do this. Until we can accept that not all needs can be met we will continue to have a distortion in health service priorities and, ironically, fail to get value for money overall.

Dr Daphne Austin

Consultant in Public Health Medicine

West Midlands Specialised Services Agency

March 2007