1.  WHY NICE'S DECISIONS ARE INCREASINGLY BEING CHALLENGED

  2.  This arises out of several factors.

  3.  First, the appeal process cannot take into account up to date evidence so the decision is out of date and irrational to those consultants expert in this field who actually have to treat patients.

  4.  Second, the methodology of decision making at NICE involves people who may be accomplished at reviewing evidence and compiling a report but are not necessarily the best in their field and not all "cutting edge" experts in for example oncology.

  5.  Third, the mechanics of decision making involve a contentious concept which is termed a "QALY". This is a rough attempt to provide some objective approach to a complex matter of quality of life and the number of years of that quality of life which can be achieved as a statistical median for the proposed drug or treatment under review. QALY does not always provide the only or best way of assessing this. It is the measure of cost effectiveness but it can mislead in particular difficult and complex cases involving terminally ill patients or those whose life is already affected by a particular condition and who are not looking at a cure as such. The statistics of QALY also can lend themselves to decision made not on the basis of an individual's prospect of successful treatment but on an overall assessment which is to the disadvantage of particulars groups of patients. For example, there will be those who with these particular drugs of Avastin and Erbitux would on the evidence pull through to operable status. These will be discarded because the majority would not reach that stage. This has to be wrong and nonsensical.

  6.  Fourth, the QALY cost of £20,000 (£30,000 in oncology) appears to move upwards in some cases and not at all in other cases. It also has not been increased to reflect high inflation in the medical sector let alone kept up with inflation in terms of the RPI for a number of years (2000).

  7.  An example of why a QALY has sometimes little or no meaning in a terminal bowel cancer patient's treatment can be given from direct personal experience.

  8.  It is admitted from the outset that Bowel Cancer is not as "sexy" a subject as Breast cancer might be to the publicity conscious but the following example is worth considering as I believe everyone can relate to this.

  9.  A patient with advance bowel cancer might commonly have an operation to remove part of their lower bowel or a stent inserted.

  10.  Let us consider the lucky patient with a stent. This stent is a narrow tube (one finger width diameter) which allows their stools or motions to pass, otherwise these would block up due to the size of tumour in the bowel. In order for the stool to pass, however, it must be almost liquid in consistency and large doses of laxative have to be prescribed. This causes embarrassment, misery and cramps. It can create a mood of depression. It also means that the type and range of food that can be eaten is very much reduced, with a ban on say broccoli, greens, tinned tomatoes and a host of other foods.

  11.  With the benefit of "normal" chemotherapy, the bowel tumour will hardly ever (less than 1% chance) reduce in size sufficiently so as to allow the stent to be removed (or remove itself). In the case of first line prescription of Avastin (Bevacizumab) in conjunction with "normal" chemotherapy, to a patient who had a stent fitted in September 2006, the bowel tumour reduced by half in volume after 3Ö months which meant that the stent left the body, and the patient was and is able to pass normal motions in the normal way. The patient is now able to eat normally. This means that the patient is able to eat anti oxidant food (broccoli, greens, tinned tomatoes) and able to build the body back up to increase antibodies against infection. The quality of life in simply being able to live normally in this way cannot ever be underestimated.

  12.  In another patient who was unable to walk or move or carry items or care for his teenage son who had water on the brain requiring a stent operation to relieve the pressure on the brain. With the advantage of Erbitux, in combination with Irinotecan chemotherapy which had previously been given alone and failed to work, as third or last line of treatment, the patient's tumours in liver and lung have reduced to such an extent that he is now able to move around and look after his son who would otherwise be in the care of the social services.

13.  WHETHER PUBLIC CONFIDENCE IN THE INSTITUTE IS WANING, AND IF SO WHY

  14.  To my mind and experience over the past eight months, it has waned and is waning rapidly.

  15.  Just to state that I became involved in the process of applying for the "banned" biological agent drugs Avastin and Erbitux for advanced bowel cancer treatment through a close relative being diagnosed with advanced bowel cancer in July 2006.

  16.  By the time diagnosis had occurred and a consultant oncologist advised treatment in August 2006, NICE had brought in their interim determination making the "Not Recommended" decision in both Avastin (Bevacizumab) first line chemotherapy and Erbitux (Cetuximab) third line use. It should be noted that the patient had paid privately to see the consultant as the time delay in the NHS would have meant waiting several weeks longer for that decision on treatment.

  17.  Following that recommendation for Avastin, the patient applied for, was refused and appealed the decision of the PCT to refuse Avastin first line use in combination with "normal" chemotherapy Oxaliplatin and 5FU. The appeal was refused on baseless grounds. The decision was then voided, and a fresh decision made again to refuse Avastin. That was appealed and again refused and only when judicial review proceedings were notified to the PCT was a decision made to approve Avastin in first line use. The basis of refusal was always the NICE "not recommended" interim determination. The basis of appeal was the exceptional grounds that in this particular case, the patient could not tolerate 100% of the side effects associated with the "normal" chemotherapy.

  18.  The basis for refusal in all subsequent appeals has always been the NICE recommendations.

  19.  The delay occasioned by this refusal and appeal process meant that effective combination treatment involving Avastin could not begin until October 2006. That was 3Ö months after diagnosis. By then the CEA count had risen to 4,500.

  20.  The combination of Avastin with "normal" chemotherapy has meant the bowel tumour reduced in size by half and through lack of contact with the tumour, the stent removed itself and this patient can presently enjoy a relatively normal life in terms of bowel motions anyway.

  21.  The delay in treatment however, meant the patient lost her job. She would normally have been working and paying taxes. Instead she is in receipt of income support, housing benefit, and a car has been provided. She would have preferred to have been on her own two feet and working.

  22.  It is a story repeated with variations many thousands of times each year across the UK. In fact 30,000 times for bowel cancer. Each patient has on average five close relatives who are of voting age. These are in the main people who paid taxes for a health service to benefit not only themselves but those who cannot work.

  23.  Some patients have been able to benefit from a PCT deciding to provide primary care on the NHS ("normal" chemotherapy) whilst the patient pays privately to take the drug involved whether it is Avastin or Erbitux.

  24.  Some PCT's refuse to allow this mix confusing themselves with the rules on "Public/Private" mix. Although the patient would be entitled to seek judicial review of this refusal of primary care, the patient is usually too ill, and too impoverished by buying one off drugs privately to be able to seek judicial review.

  25.  It would be very useful to clarify the position so that more PCT's can understand that taking drugs privately does not mean the patient cannot have primary care on the NHS. It is not like a hip replacement situation, the patient is not asking for any NHS input in taking the drug privately. The patient by analogy will be able to take healthy foods privately without risking abandonment by the NHS of primary care and indeed patients often take both alcohol and smoke tobacco without losing primary NHS care.

  26.  It is anticipated that eventually there will be a case brought by way of judicial review to challenge this refusal of a PCT to provide primary care whilst the patient is taking either Avastin of Erbitux privately.

  27.  Since that first appeal, I have assisted pro bono a further 18 patients in their applications mainly referred via individual consultants and Bowel Cancer UK.

  28.  I cannot continue to do this indefinitely. I have published the results of my efforts and provided guidance for lay people and lawyers. I have referred my material en bloc to the Bar Council pro bono unit.

  29.  It would be helpful if a person could be paid to coordinate advice and representation nationally across all PCT's on behalf of patients without relying on ad hoc charity work.

  30.  The table at the end of these submissions sets out the date of my application/appeal for exceptional funding; the patient (anonymised); the type of drug sought; the area of PCT; whether the appeal was successful/the time period of delay in deciding from first application/whether the PCT allows them to have normal chemotherapy on the NHS whilst taking the drug privately. Please note the delay includes time previous to my involvement.

  31.  Each patient has expressed the frustration at being refused a drug that can help them, not knowing the avenue of appeal or even that it existed, of not being helped by anyone in bringing an appeal, and at the same time, having to receive debilitating treatment for a life threatening illness.

  32.  Each carer has expressed the view that they wished to be able to take all steps they could to know that they had done as much as possible to help their loved ones.

  33.  In those appeals, only four have been fully successful, a further three have come to a private public arrangement with the PCT and the remainder have had their treatment limited.

  34.  Obviously, there will be other cases which probably deserve to be brought to a PCT appeal which have not been brought.

  35.  The pattern of success appears to indicate a postcode lottery system at work. Indeed one patient even indicated that he would be better visiting relatives in Italy to obtain the Erbitux.

  36.  I have been in contact with oncologists around the world and it is sobering to think that in Guatemala the public doctors there view with amazement the UK's refusal to treat with Erbitux third line.

  37.  Treatment in France, Germany, Spain and Italy all outstrip the UK's level of use and effectiveness on treatment. This is due in part to their use of these drugs.

  38.  On instruction from Bowel Cancer UK and Cancer Back Up I appeared at the NICE appeal in regard to Avastin and Erbitux headed by Professor Sir Michael Rawlins on 27 November 2006.

  39.  NICE in the appeal on 27 November 2006 expressed the view that not all clinicians might be as professional in their approach to prescribing these drugs as the two experts who were called to give evidence on behalf of the appeal.

  40.  It has been my experience in all 18 appeals that the clinicians have in the main been reluctant to prescribe drugs which they and the patients know can cause side effects, reluctant to prescribe until the right moment, and cautious in regard to whom they would lend their support, bearing in mind some patients just could not physically endure these additional biological agents. In other words, they have erred on the side of caution and have not taken an unprofessional approach to prescription. Indeed the figures for prescription of Erbitux in Wales in a small way are consistent with that picture in that the numbers being prescribed per year dropped slightly from 35 to 30 following the Welsh Assemblies decision to ignore the NICE recommendation.

  41.  As a result of stories like these those related to the unfortunates make decisions to enrol themselves on private medical insurance schemes. They lose their interest in themselves supporting and promoting a universal health scheme that does not provide sensible solutions to their life's problems.

  42.  People see the present restrictions from NICE as being a means to claw back money in NHS spending that has been wasted on inflated wages and wasted on computer systems.

  43.  The basis of most NICE "not recommended" decisions appears to people to be based on a lack of proof of effectiveness when the standard of that proof is set too high for what are new and groundbreaking treatments.

  44.  Also NICE look at the case as if everyone on the drugs would continue on the drugs for a full course of treatment whereas the new evidence rejected by NICE in Erbitux was to the contrary.

  45.  The perception of NICE is that it is a body which fails to trust expert clinical opinion.

  46.  Far from protecting the NHS, NICE decisions are seen as undermining the effectiveness of the NHS.

47.  NICE'S EVALUATION PROCESS, AND WHETHER ANY PARTICULAR GROUPS ARE DISADVANTAGED BY THE PROCESS

  48.  The evaluation process involves QALY. Those disadvantaged are patients whose condition is not likely to result so much in curing the patient's condition or in saving of life but prolonging life and those whose quality of life is not amenable to a quantification of level but is likely to be variable through the proposed course of treatment.

  49.  It most certainly disadvantages those exceptional patients who may be able to respond so well that they are able to have surgery.

50.  THE APPEAL SYSTEM

  51.  The present "appeal" system is not an appeal system in the sense of it having the power to hear new evidence. This is not necessarily wrong in law. I take the strong view that it is wrong in practice and leads to more problems than it solves.

  52.  The present system of appeal is based on the model of a judicial review.

  53.  The only legal avenue of appeal from the final decision of the appeal panel is itself by way of judicial review.

  54.  It thus follows that once a determination is made, there is no appeal which involves any new evidence at all, neither on appeal to NICE or on appeal by way of judicial review from NICE.

  55.  In fact the date on which the decision becomes out of date is in fact much earlier than that because there is a "cut off" date for the admission of evidence set long before NICE makes any reported determination.

  56.  The present appeal committee's job is a review only of the material which was before the original decision maker at the date of decision.

  57.  As such it only relates to checking if the internal procedures of that original decision were correct.

  58.  There are different ways of conducting an "appeal". Some can be rehearing, some can be as this process is, review and others can be reconsiderations.

  59.  I would not recommend rehearing.

  60.  The present chosen method of limited review has advantages and disadvantages.

61.   Advantages

  62.  The advantages are that the appeal committee do not have to do anything but look at the original material and even simpler than that, look at only those parts that fit into the limited criteria for these "due process" appeals. It makes the appeal committee's task easier. In addition it prevents an appeal being brought as and when new evidence comes into being. It prevents the original decision being challenged (and even re challenged) and implemented simply because a new relevant fact has emerged. This enables a decision to in theory be promulgated without too much delay.

63.   Disadvantages

  64.  The disadvantages closely mirror the advantages.

  65.  The disadvantages principally relate to the peculiar nature of oncology. It is to do with the fact that under this method of appeal, the decision can be very quickly out of date with the best practices in the real world.

  66.  There exists in the professional world of oncology a known and knowable consensus of expert professional clinical opinion which takes account of up to date information on the results of the myriad of ongoing medical trials.

  67.  What NICE decision subtly does is stifle "hands on" practice with new drugs. Apart from clinical trials, it is actual practice that eventually lets good results filter down as best practice. Without access to the new drugs the UK will always be second best and playing catch up to other countries. It is as if we are allowing NICE to state we need a perfect world and a perfect basis for a positive decision before approving any new treatment.

  68.  It also means that drug companies will not provide the UK's best clinicians with their drugs so that the best clinicians may be drawn to practice elsewhere around the world.

  69.  From the moment the closure date for admitting evidence passes in the original decision makers process, the clock is running for the next piece of clinical trial information. By the time the original NICE decision maker has reported the determination, a substantial period of time has usually elapsed during which time relevant but inadmissible evidence about recent results in clinical trials has occurred. That schism between the real world and the determination can become wider once an appeal is lodged and heard and findings made on the appeal. With this further delay, further material can become available in the real world and the original decision, albeit in proper procedural format and albeit "right" on the original material, can lead NICE to make recommendations that any sensible person at the time of the final appeal, would not follow.

70.   Improvements

  71.  Obviously, to keep the time from last evidence being admitted to determination and final appeal as short as possible would be beneficial.

  72.  The problems with that are that there are what are known as "reasonable" time periods of submission of evidence, sending it to the interested parties for comment, reports from expert committees to NICE, conclusions and report drawing, time period for notice of appeal, time period for setting down the appeal as the original committee will want to respond to the appeal notice, and then time for the appeal committee to consider and report back. There will always be delay. Where any of those processes can be taken out of the "process equation" or whether any particular time period can be shortened is not something I can accurately comment upon.

  73.  As a general observation, there remains an obligation for fairness in procedure such that it would appear that none of the above steps can be removed without causing unfairness and that the time limits set for each step (which cumulatively add up to so much time) cannot reasonably be cut much further without missing the opportunity for sensible evidence to be given and commented upon. There does not appear to me at least much scope for improving the time delay aspect of a decision.

  74.  There does appear to be another simpler way forward in improving the whole appeal process.

75.   A fair appeal

  76.  A fair appeal process in the circumstances of rapidly changing oncology would allow in evidence "appertaining to the material facts as at date of decision" to be adduced at an appeal. This is a phrase common enough in legal parlance and is understood and defined by the Courts.

  77.  A proper appeal in my view would involve a reconsideration of the facts on the basis of the best available evidence.

  78.  The present system of decision making and review is one in which there is no opportunity to have relevant and material up to date facts considered in any appeal.

  79.  Judicial review of its very nature prevents new evidence. The evidence is limited to that which was before the original body.

  80.  Even if one were to successfully argue before a judicial review that the evidence before the appeal panel should have been taken into account, it would still not enable the judicial review body to come to its own conclusions on those facts. It could only refer the matter back for a fresh decision.

  81.  That really would prevent promulgation of decisions and create a "never ending" system of decision making.

  82.  The danger of a "never ending" system of decision making in changing the present terms of reference for a NICE appeal is met by the wording of the new procedural rule which would apply to admitting any new evidence which would apply when an appeal body reconsidering the matter looked at what evidence to take into account.

  83.  That wording would be similar to that used in appeals in other legal fields such as immigration.

  84.  "The appeal body may take into account evidence of material facts which appertained as at the date of decision".

  85.  These other appeal bodies continue to function and do not suffer from a "never ending" process due to admitting new evidence because the new evidence is restricted to the relevant material issues and is not allowed to widen the debate or bring up new issues.

  86.  Thus the rule of admissibility is a discretionary one and not an absolute right to new evidence, the person seeking to place it before the appeal body has the onus of establishing it as relevant and material and as is the present position, the appeal body would take legal advice on whether it should be admitted.

  87.  The appeal process presently is limited to an essentially "due process" review; this means the merits of the decision are not reviewed. This artificial limit is not necessary. It means that when coupled to excessive delay in the process, the decision can become out of step with the reality of rapid technological advances. The fact that the appeal panel themselves are experts is somewhat wasted by their attendance on what is little more than observing that the original panel went through the right process and having made proper assumptions on the then available evidence, came to a decision by a logical and reasonable process.

  88.  It would be useful upon reconsideration on available relevant material evidence would be if the appeal panel could form their own view as to the recommendation.

  89.  In human rights law, under article 8, the right to a private and family life, the appeal authority must consider the facts as they are before them on the day of the appeal. This to my mind is akin to a rehearing and not necessary for NICE appeal committees and has many obvious disadvantages.

90.  THE IMPLEMENTATION OF NICE GUIDANCE, BOTH TECHNOLOGY APPRAISALS AND CLINICAL GUIDELINES (WHICH GUIDANCE IS ACTED ON, WHICH IS NOT AND THE REASONS FOR THIS)

  91.  The NICE guidelines are followed by all PCT's I have been involved with. Unfortunately they are not followed in the same way by all PCT's. Some apply the guidelines as absolute rules which take precedence over clinical decisions by the consultant. Some treat them as the deciding factor when compared to clinical decisions. Only a few apply them properly as matters to be taken into account when looking at all the evidence and for them not to override clinical decisions in a particular case. Some PCT's decide without legal assistance and others take legal assistance. The level and quality of legal assistance sought varies enormously. The PCT's really do only get what they pay for in that respect.

  92.  It would be useful for there to be a National Guidance Manager who could oversee and help apply guidance for PCT's.

93.  THE SPEED OF PUBLISHING GUIDANCE

  94.  For the above reasons it can be seen that there is considerable delay built into the system. However, it is not so much the speed or lack of it but the way in which the decision makers go about their decision which is flawed. Increasing the speed will to my mind only increase the level of judicial reviews which will occur.

95.  COMPARISON WITH THE WORK OF THE SCOTTISH INTERCOLLEGIATE GUIDELINES NETWORK (SIGN)

  96.  I have left this to last as I have no direct knowledge of this save to say that two PCT's have based their initial decision to refuse on the fact that the Scottish board had decided to make a "no" recommendation. One of these PCT's overturned the decision (Derby) and another did not (Uxbridge).

CONCLUSIONS

  1.  It would be very useful to clarify the position so that more PCT's can understand that taking drugs privately does not mean the patient cannot have primary care on the NHS.

  2.  It would be helpful if a person could be paid to coordinate advice and representation nationally across all PCT's on behalf of patients without relying on ad hoc charity work.

  3.  Obviously, to keep the time from last evidence being admitted to determination and final appeal as short as possible would be beneficial.

  4.  A fair appeal process in the circumstances of rapidly changing oncology would allow in evidence "appertaining to the material facts as at date of decision" to be adduced at an appeal.

  5.  A proper appeal in my view would involve a reconsideration of the facts on the basis of the best available evidence.

  6.  It would be useful upon reconsideration on available relevant material evidence would be if the appeal panel could form their own view as to the recommendation.

  7.  It would be useful for there to be a National Guidance Manager who could oversee and help apply guidance for PCT's.

Peter Telford

21 March 2007