Colin Ross v West Sussex Primary Care Trust
 EWHC B15 (Admin) (temporary reference)
Case No: CO/8257/2008
IN THE HIGH COURT OF JUSTICE
QUEENS BENCH DIVISION
Royal Courts of Justice, Strand, London, WC2A 2LL
10th September 20087
B e f o r e :
HIS HONOUR JUDGE S P GRENFELL
THE QUEEN on the application of Colin ROSS (Claimant)
- and -
WEST SUSSEX PRIMARY CARE TRUST (Defendant)
Mr Richard Clayton QC and Mr Vikram Sachdeva (instructed by Irwin Mitchell) for the claimant
Mr Martin Forde QC and Mr Matthew Barnes (instructed by Mills & Reeve) for the defendant
Hearing dates: 4th and 8th September 2008
Crown Copyright ©
His Honour Judge Grenfell:
1. The claimant, Colin Ross, a multiple myeloma sufferer, challenges the decision of the defendant, West Sussex Primary Care Trust (the "PCT"), not to fund the relatively new cancer drug, Lenalidomide, in a three drug combination. His clinical situation since the early part of this year has been that previous treatments of Velcade and latterly Thalidomide have resulted in peripheral neuropathy to the extent that it had to be stopped. That meant that, unless he could be treated with Lenalidomide, the only option was palliative treatment. It is accepted that without the chance of attempting the life extending properties of Lenalidomide in conjunction with one or two other drugs, which are themselves uncontroversial, his life expectancy is limited to the progress of the myeloma. The decision was taken by the PCT's Review Panel on 28th May 2008, and confirmed on appeal on 23rd June and finally reviewed and confirmed on the 22nd August.
2. The application for Judicial Review of the decision was made on the 1st September when I directed that the application for permission with the full hearing to follow if granted be heard on the 4th. On the 4th September counsel indicated their acceptance that the grant of permission was inevitable. Accordingly I heard the arguments, supported by their written detailed grounds and submissions, on the 4th and 8th September. I record my appreciation of the co-operation and speed of counsel in mustering and mastering their detailed arguments in the short time available.
3. Lenalidomide has not yet been assessed by the National Institute for Health and Clinical Excellence ("NICE") which is expected to make a decision in January 2009.
4. No one can completely put aside the human element of a case like this. Nevertheless, it cannot be allowed to dictate the result. My approach has to be to decide whether or not the decision can be successfully challenged on clear and laid down principles.
5. Guidance comes from Sir Thomas Bingham MR in R v Cambridge Health Authority ex parte B  1 WLR 898 , in which he observed at 905B:
" … the courts are not, contrary to what is sometimes believed, arbiters as to the merits of cases of this kind. Were we to express opinions as to the likelihood of the effectiveness of medical treatment, or as to the merits of medical judgment, then we should be straying far from the sphere which under our constitution is accorded to us. We have one function only, which is to rule upon the lawfulness of decisions. That is a function to which we should strictly confine ourselves." and at page 906D to F:
"I have no doubt in a perfect world any treatment which a patient, or a patient's family, sought would be provided if doctors were willing to give it, no matter how much it cost, particularly when a life was potentially at stake. It would however, in my view, be shutting one's eyes to the real world if the court were to proceed on the basis that we do live in such a world. It is common knowledge that health authorities of all kinds are constantly pressed to make ends meet. They cannot pay their nurses as much as they would like; they cannot provide all the treatments they would like; they cannot purchase all the extremely expensive medical equipment they would like; they cannot carry out all the research they would like; they cannot build all the hospitals and specialist units they would like. Difficult and agonising judgments have to be made as to how a limited budget is best allocated to the maximum advantage of the maximum number of patients. That is not a judgment which the court can make."
6. Auld LJ in R v North West Lancashire Health Authority  1 WLR 977 at 991E to F stated the principle:
"As illustrated in the Cambridge Health Authority and Coughlan cases, it is an unhappy but unavoidable feature of state funded health care that Regional Health Authorities have to establish certain priorities in funding different treatments from their finite resources. It is natural that each Authority, in establishing its own priorities, will give greater priority to life-threatening and other grave illnesses than to others obviously less demanding of medical intervention. The precise allocation and weighting of priorities is clearly a matter of judgment for each Authority, keeping well in mind its statutory obligations to meet the reasonable requirements of all those within its area for which it is responsible. It makes sense to have a policy for the purpose – indeed, it might well be irrational not to have one - and it makes sense too that, in settling on such a policy, an Authority would normally place treatment of transsexualism lower in its scale of priorities than, say, cancer or heart disease or kidney failure."
7. These principles are of particular application to the present case against the background of the information that other PCTs have granted funding for Lenalidomide where others have not. It follows that each PCT is entitled, provided it does so rationally, logically and lawfully, to set its own policy for making such difficult decisions.
8. The general background in respect of the drug Lenalidomide is as follows. It is structurally similar to Thalidomide, but more potent, and largely without the side effect of peripheral neuropathy. Lenalidomide is freely available in mainland Europe. The recommendation in a position paper of the British Committee for Standards in Haematology is that where side effects prevent the administration of Thalidomide, Lenalidomide should be given. In a recent survey, I am told, 60% of the applications made for exceptional funding of Lenalidomide in England and Scotland have succeeded. The relevance of this information is to dispel any ideas that the prescription of Lenalidomide is somehow breaking new ground. The London Cancer New Drugs Group Briefing of May 2008 estimates that between 1.6 and 2.8 patients per 100,000 population could receive Lenalidomide every year. The significance of these factors is that a decision on this application in the claimant's favour is unlikely open any 'flood gates' of further applications, each of which are in any event likely to be considered on its own merits.
9. On 17th September 2007, Dr Stern, a Consultant Haematologist, made an application to the Surrey, West Sussex and Hampshire Cancer Network Chemotherapy Group for a new drug policy and guideline in respect of the use of lenalidomide in combination with dexamethasone for the treatment of patients with multiple myeloma who had received at least one prior therapy. That application set out that lenalidomide had been launched commercially in the UK following the grant of a licence by the European Medicines Agency on 19th June 2007 and approval by the Medicines and Healthcare Products Regulatory Agency.
10. The Chemotherapy Group on 27th November 2007 concluded:
"The current evidence was not felt to be adequate to support the standard use of lenalidomide in patients failing to respond to thalidomide based regimens ie, as a third or fourth line option. This will need a separate application/business case as it is clearly a significant service development with potentially substantial associated costs as this would be a further treatment option which would apply to many more myeloma patients.
"Application supported as an alternative second line option for patients who have responded to thalidomide but who develop unacceptable neuropathy. Details to be included in Horizon Scanning for the PCTs as a formal business case will be required."
11. On 13th December 2007, the Surrey, West Sussex and Hampshire Cancer Network Haematology Tumour Group considered the use of Lenalidomide. and noted:
"… that the network chemotherapy group had given support for this treatment option for patients unable to receive thalidomide as a standard alternative due to side effects. SS noted that this did not include patients unresponsive/resistant to thalidomide – a proportion will show sensitivity to lenalidomide. It was agreed that the network chemotherapy group should be asked to reassess this specific subgroup as a separate application."
12. No application has been made by the Cancer Networks for funding approval for lenalidomide. In fact, it does not appear that the Cancer Networks, other than their sub groups, have reached a concluded position on the funding of lenalidomide. Accordingly, the process of considering funding lenalidomide has not yet reached the second stage of the process.
13. It is however, worth observing that in what is termed the 'hierarchy of evidence' in support of a new drug the stage of randomised controlled trials comes 2nd to highest. This assumes importance when the evidence presented to the PCT in respect of clinical efficacy is considered.
14. I take the factual background from the Statement of Facts.
15. The Claimant was first diagnosed with multiple myeloma in 2004, and has been treated with a variety of chemotherapy treatments. He responded to all of them, but is now on his third relapse.
16. His most recent treatment was composed of Thalidomide, but by February 2008 the side effects of Thalidomide, and in particular
peripheral neuropathy, were becoming too debilitating to tolerate, and the drug was stopped.
17. The only alternative treatment is Lenalidomide, which, as I have indicated, is largely without the side effect of peripheral neuropathy.
18. The way in which the PCT approaches new drugs is against the background of their Operational Plan, in which the PCT allocates funds on an annual basis, considering both clinical effectiveness, and cost effectiveness, against the needs of its population and the limited resources available. There is no challenge to the obvious result of limited resources that, where additional expenditure is allocated to one area, savings or economies have to be made in others.
19. New cancer drugs are emerging. Ms Braysher, the PCT's Deputy Chief Executive, deals with the process for taking funding decisions in respect of these new drugs in her evidence. I take the key points in the process to be as follows.
20. When a new cancer drug emerges, the first step in the process of making a decision about funding is detailed consideration of the drug by the local Cancer Networks, which in the case of this PCT are the Sussex Cancer Network, the Surrey, West Sussex and Hampshire Cancer Network, and the Central South Coast Cancer Network.
21. Once a Cancer Network has reached a clear view as to whether a new drug or treatment is clinically suitable for one or more specific cancers in defined circumstances, the Network makes a case to the PCT for its inclusion in the authorised care pathways for those cancers.
22. In those circumstances, the PCT will look at the evidence of clinical effectiveness of the drug, consider its cost, and look to see what health outcomes can be expected for the requested investment.
23. In doing so, the Defendant follows the National Institute for Health and Clinical Excellence ("NICE") approach of considering the Incremental Cost Effectiveness Ratio ("ICER") of the drug, on the basis of the costs of each Quality Adjusted Life Year ("QALY") that the drug is likely to provide. When dealing with interventions with an ICER below £20,000, the decision by NICE to recommend will usually be based on the cost effectiveness calculation. When dealing with interventions in excess of £20,000, and in particularly in excess of £30,000 per QALY, NICE will require advisory bodies to make an increasingly stronger case for supporting the intervention as an effective use of NHS resources (see the NICE Guide to the Methods of Technology Appraisal, at paragraphs 6.2.22 – 6.2.25). It is important to note that this is not set as a limit or threshold, but as its name indicates a guide.
24. Where the drug does not require significant additional expenditure, it may be made available during the course of the year. However, if it requires a large amount of expenditure, it will be dealt with in the annual Operational Plan process, and be judged against the Defendant's other financial priorities, for the reasons outlined in Ms Braysher's evidence.
25. Where funding is sought for treatments which are not routinely commissioned pursuant to the Operational Plan, the application is considered under the Defendant's Individual Cases Policy ("ICP"). Cases are initially dealt with by a Review Panel, constituted of at least five members, with at least two clinically qualified members, and one lay member. There is a right of appeal to an Appeal Panel, constituted of at least three members, with at least one clinical member. The grounds of appeal are procedural impropriety, irrationality, and illegality. Where there is new evidence, the case will be remitted by the Appeal Panel back to the Review Panel. Panel members receive specific training before sitting, including considering and evaluating clinical evidence, and the Defendant's accountability for public funds.
26. The starting point when dealing with the ICP is that it allows for cases to be funded exceptionally, outside of the routine commissioning pursuant to the Operational Plan. Accordingly, the ICP is dealing with exceptional cases. As explained by Professor Griffiths, an expert with impressive experience in the field of Public Health, at paragraph (e) of his report:
"Every time that the PCT agrees to fund an exceptional allocation it has to find the resources from somewhere and that must mean reducing what it spends on things that have already been agreed as priorities. Such expenditure can only be justified if it offers better value for the population's health than would have been delivered by the operational plan.
"As a consequence of these approaches the PCT requires three questions to be answered: Is this a truly exceptional case that justifies deviation from the agreed plan? Does the requested treatment work? Is it cost effective? Only if the answer to all three questions is yes can any additional expenditure be justified and even then there will be an opportunity cost requiring something else to be cancelled."
27. Mr Forde submits that there are very few cases where the patients are in genuinely exceptional circumstances, and the vast majority of applications are for medical interventions that the clinician or patient believes would be beneficial, but which fall outside of the carefully planned and agreed care pathways. Applications for funding are always difficult and almost always invoke considerable sympathy. However, those sitting on the panel are acutely aware that if the procedures are not operated properly, there will be a shift of resources to the patients making applications to the Review Panel, and others in similar circumstances, and a shift of resources away from groups of patients that they cannot identify (the evidence of Dr John Bull, Consultant Physician and member of the Panel.)
28. This approach is reflected in the guidance on exceptionality contained in the document setting out the Terms of Reference for panels considering applications made pursuant to the ICP, as follows:
"(v) There is no complete definition of the conditions which are likely to come within the definition of an exceptional case. The word 'exception' means 'a person thing or case to which the general rule is not applicable'. The following however are examples of exceptions in the present context:
• In order for funding to be agreed there must be some unusual or unique clinical factor about the patient that suggests that they are:
o Significantly different to the general population of patients with the condition in question
o Likely to gain significantly more benefit from the intervention than might be expected from the average patient with the condition.
• The fact that a treatment is likely to be efficacious for a patient is not, in itself, a basis for an exemption.
• If a patient's clinical condition matches the 'accepted indications' for a treatment that is not funded, their circumstances are not, by definition, exceptional.
It is for the requesting clinician (or patient) to make the case for exceptional status. Social value judgments are rarely relevant to the consideration of exceptional status.
(vi) The Individual Case Panel can NOT make a decision to fund a patient where by doing so a precedent would be set that establishes a new policy (because the patient is not, in fact, exceptional, but representative of a group of patients). In cases where the Individual Case Panel feels strongly evidence has been provided in support of a particular health technology they should make a recommendation for further consideration by the Operational Plan process, but individual funding of the specific case must be refused by the Individual Case Panel."
29. It now seems more likely than not that this, the second version to be disclosed, was the current version.
30. The Legal Framework
31. The general duty and power of the Secretary of State is defined by sections 1, 2 and 3(1) of the National Health Service Act 2006. The Defendant's establishment as a Primary Care Trust continues in force pursuant to s.18(1) of the 2006 Act. The Defendant was established as a Primary Care Trust by Order made pursuant to section 16A of the National Health Service Act 1977. The primary duties of Primary Care Trusts are set out in section 15 of the 1977 Act "to administer the arrangements made in pursuance of this Act for the provision of primary medical services ..." The general duties of the Secretary of State under section 2 of the 1997 Act were delegated to Primary Care Trusts (and Strategic Health Authorities) as from 1 October 2002 by Regulation 3 of the National Health Service (Functions of Strategic Health Authorities and Primary Care Trusts and Administration Arrangements) (England) Regulations 2002 SI No. 2375. The general duties of the Secretary of State under s.3 of the 2006 Act remain delegated to Primary Care Trusts and Strategic Health Authorities, by virtue of s.7(1) of the 2006 Act read together with Regulation 3 of the National Health Service (Functions of Strategic Health Authorities and Primary Care Trusts and Administration Arrangements) (England) Regulations 2002 (SI 2002/2375) (as amended).
32. The Role of the Secretary of State is set out in HSC 1999/176. Thus the PCT has a duty to commission medical services as it considers necessary to meet the healthcare needs of the local population as a whole and within allocated resources, and to do any other thing which will facilitate, or is conducive or incidental to the discharge of such a duty.
33. It is a matter for the PCT how it allocates its resources, so long as it does so reasonably, which will involve difficult and agonizing judgments as to how a limited budget is best allocated to the maximum advantage of the maximum number of patients (R v Cambridge Health Authority ex parte B  1 WLR 898 at 906D).
34. In reaching these decisions, the PCT should consider the nature and seriousness of each type of illness, and the effectiveness of various forms of treatment (R v NW Lancashire HA ex parte A  1 WLR 977 at 991 E – G). However, it would be very difficult, if not impossible, to challenge a decision not to fund treatment that took into account financial restraints and the particular circumstances of the individual patient (R on the application of Rogers v Swindon NHS PCT  EWCA Civ 392 at paragraph 58).
35. It is lawful for the Defendant to have a policy not to fund a treatments save in exceptional circumstances, so long as it is possible to envisage, and the decision maker does envisage, what such exceptional circumstances might be, as the policy will otherwise be in practice a complete refusal of assistance, and unlawful because it is justified not as a complete refusal, but as a policy of exceptionality (Rogers at paragraph 62).
36. The court's role is not to be an arbiter of the merits of cases of this kind, nor is it to express opinions as to the likelihood or the effectiveness of medical treatment or on medical judgment, but is strictly limited to ruling upon the lawfulness of decisions (R v Cambridge limits the ability of the courts to review decisions of this kind). Nevertheless, where life and death decisions are involved, the courts must submit the decision making process to rigorous scrutiny. See Rogers at paragraphs 56 and 57 in the judgment of the court:
"56 There is little, if any, dispute between the parties as to the correct approach at common law in a case of this kind. In R v Ministry of Defence, Ex p Smith  QB 517 , 554, Sir Thomas Bingham MR accepted a submission (as it happens by Mr Pannick) as to the correct approach to irrationality: "The court may not interfere with the exercise of an administrative discretion on substantive grounds save where the court is satisfied that the decision is unreasonable in the sense that it is beyond the range of responses open to a reasonable decision-maker. But in judging whether the decision-maker has exceeded this margin of appreciation the human rights context is important. The more substantial the interference with human rights, the more the court will require by way of justification before it is satisfied that the decision is reasonable in the sense outlined above."
"In this case there is an issue between the parties as to whether article 2 of the Convention is engaged but, whether article 2 is engaged or not, the case is concerned with a decision which may be a life or death decision for the claimant. In these circumstances, as we think Mr Havers accepted, it is appropriate for the court to subject the decision to refuse funding for the treatment (and thus in practice the treatment) to rigorous scrutiny.
"57 In giving it that scrutiny, it is important for the court to have in mind that a critical feature of the circumstances of this case is that, as the judge put it in para 58 of his judgment, this is not a case about the allocation of scarce resources. The judge quoted in this regard the following well-known observations of Sir Thomas Bingham MR in R v Cambridge Health Authority, Ex p B [to which I have already referred."
37. At paragraph 62 Sir Anthony Clarke MR dealt with the lawfulness of a policy of exceptionality:
"In [R v North West Lancashire Health Authority, Ex p A  1 WLR 977] Auld LJ stresses that a policy which allows for exceptions in undefined exceptional circumstances is not unlawful " provided that the policy genuinely recognises the possibility of there being an overriding clinical need and requires each request for treatment to be considered on its individual merits" . As we see it, that means that a policy of withholding assistance save in unstated exceptional circumstances (in the case addressed by Auld LJ, and no doubt in this case also, overriding clinical need) will be rational in the legal sense provided that it is possible to envisage, and the decision-maker does envisage, what such exceptional circumstances might be. If it is not possible to envisage any such circumstances, then the policy will be in practice a complete refusal of assistance: and irrational as such because it is sought to be justified not as a complete refusal but as a policy of exceptionality."
38. Whether a mistake of fact is unlawful was held by Carnwath LJ in E and R v Secretary of State for the Home Department  QB 1044, paragraph 63, to be satisfied by a combination of five factors: (i) an erroneous impression created by a mistake as to, or ignorance of, a relevant fact; (ii) the fact was "established", in the sense that, if attention had been drawn to the point, the correct position could have been shown by objective and uncontentious evidence; (iii) the claimant could not fairly be held responsible for the error; (iv) although there was no duty on the PCT to do the claimant's work of proving his case, all the participants had a shared interest in co-operating to achieve the correct result; (v) the mistaken impression played a material part in the reasoning.
39. The test which I have to apply is as follows. The PCT has acted irrationally if it reached a conclusion which no reasonable authority could have made; the more substantial the interference with human rights, the more the court will require by way of justification before it is satisfied that the decision is reasonable; the Courts must subject their decision to anxious scrutiny because the Claimant's life is at stake.
40. Finally, the approach to the decision of panels such as those who made the decisions under review cannot be as strict as to a decision of a court of law.
41. The Facts
42. On 31st March 2008 the Claimant's treating Consultant Haematologist Dr Faith Davies, an internationally recognised authority on myeloma based at the Royal Marsden Hospital, applied for funding for Lenalidomide, in combination with Dexamethasone (in accordance with the international studies on Lenalidomide published in the New England Journal of Medicine in November 2007 which led to its authorisation), plus a further drug, Cyclophosphamide. This latter drug had been found to be a useful addition to Lenalidomide and Dexamethasone by Dr. Davies and her team, their results being published in the British Journal of Haematology on 5 March 2007. This is the small retrospective review referred to in the 28th May Review Panel minutes.
43. Dr. Davies considered that the application was urgent, and sought a reply within a maximum of 2 weeks.
44. The evidence in support of the application was:
"Two recently published (New England Journal of Medicine) randomised phase 3 trials demonstrate lenalidomide and dexamethasone is superior to dexamethasone for patients with relapsed disease. High responses are seen in patients who have previously had velcade and thalidomide therapy, even if they were resistant to these drugs. The RMH experience to date suggests combining with cyclophoshamide may improve the response rate. The data would also suggest that peripheral neuropathy symptoms are not aggravated. We would aim to give 3-4 courses and assess response at that time. If there was no response we would stop therapy. If the patient was responding we would re-discuss with the PCT."
45. In addition, the final draft of the London Cancer New Drugs Group report on lenalidomide, dated May 2008, and the final draft of the Scottish Medicines Consortium advice on lenalidomide, dated April 2008, were made available to the Panel.
46. In the event, the PCT's Review Panel for 'Patients with Individual Needs' met to consider the application on 28th May 2008. The decision letter dated 28th May 2008 was sent to Dr. Davies on 5th June.
47. The decision letter gave the following reasons for rejecting funding:
i) There is no robust evidence for use of Lenalidomide in combination with Dexamethasone and Cyclophosphamide as evidence for the LDC combination in this setting to one small retrospective review.
ii) Lenalidomide has a very high cost in relation to the potential benefit.
48. There are two versions of the minutes of the meeting of 28th May 2008. The first was consistent with the above two reasons stated, which appeared to cover clinical efficacy and cost effectiveness. The second indicated that the Panel had considered whether there was a case for an exception, but did not feel that there was. It is surprising, in the light of Dr Bull's evidence in this application particularly to the effect that the first thing the Panel did as required in all cases was to discuss whether the Claimant's case was exceptional, that this did not appear as part of the decision letter of the 28th May; that despite the accepted inexperience of the minute taker that there was no mention of this; that, even after the addition of matters omitted from the minutes that the discussion of exceptionality does not appear until towards the end of the minutes. There is a natural concern that this aspect of the application may not have assumed the prominence that a retrospective view of the discussions may be.
Nevertheless, I am prepared to accept that the second version of the minutes is correct.
49. The Claimant appealed.
50. Dr Davies, in her letter to the PCT on 16th June was unclear and wrote, identifying the key point on exceptionality:
"… it is not clear from your letter whether you considered our patient's exceptional circumstances that of delibilitating peripheral neuropathy, making treatment with either Bortezomib or Thalidomide inappropriate."
51. In her letter she emphasised the importance of the Dimopoulos and Weber randomised controlled studies the results of which had been published as demonstrating "very impressive response rates." She then made reference to the Royal Marsden experience of combining not only dexamethasone with Lenalidomide but also cyclophosphamide. Her reference to there only being a limited data set, confirmed by another study presented at the European Haematology Association, was simply a reference to the addition of cyclophosphamide. This did not in any sense water down the supporting evidence for Lenalidomide for a patient such as the Claimant. Unfortunately, the Panel appears to have mistaken this for an indication that there was no robust evidence for the use of Lenalidomide, when in fact the identity of the additional combination drug did not make a significant difference to the central question whether the main drug, Lenalidomide, was itself clinically effective.
52. Unfortunately, this misconception appears to have remained to the end.
53. On the 23rd June 2008 the PCT Appeal Panel upheld the decision of the Review Panel, for the following reasons:
i) All published evidence reviewed for this case was considered in terms of clinical effectiveness and cost effectiveness. The Appeal Panel felt that the Review Panel were correct in their statement that Lenalidomide is not cost effective in relation to the benefit of the treatment.
ii) It was noted that the referral from the Royal Marsden proposed 3 – 4 courses of treatment to determine whether there was a response and if not they would stop treatment.
iii) It was noted that the Review Panel had taken into account the neuropathic pain issue but did not feel that these were exceptional circumstances.
iv) The Appeal Panel did not feel that the Review Panel's decision was irrational and they felt that procedurally the Review Panel were correct.
54. In view of the fact that Dr Bull did not revise the minutes of the 28th May until early August, the only information the appeal panel had on the Review Panel's views on exceptionality was the last sentence of the notes which read:
"On the question of exceptionality which is based on the patient having neuropathic pain the Panel did not feel that this was exceptional."
55. In fact, the additional paragraph added by Dr Bull added very little:
"The Panel concluded that a cohort of patients will have such side effects resulting in pain. They therefore did not feel that this case was an exception."
56. It is a central part of the Claimant's case that the Panel's deliberations and ultimately their decision proceeded on a fundamental misunderstanding of the case being advanced by Dr Davies for bringing this case within the PCT's own exceptionality policy. The claimant's case has been refined in oral argument to level criticism of the policy itself insofar as it is argued that the attempts to define exceptionality are more likely to mislead a review panel than assist it to apply the policy; that, further, the policy itself is unlawful because properly interpreted it required a test of exceptionality which went beyond the natural and ordinary meaning of the word 'exception' in effect, a test of uniqueness, which in turn would severely restrict the panel's discretion. The fundamental misunderstanding, it is submitted, is that the Claimant was intolerant to his previous treatments of Velcade and Thalidomide; that it was not just that he suffered neuropathic pain, but rather the peripheral neuropathy was such that rendered further treatment impossible. It was clear from the application that the claimant was unable to continue with his treatment.
57. Further submissions were sent to the PCT by Irwin Mitchell, the Claimant's solicitors, on the 15th August 2008, including a report by Professor Karol Sikora, a world authority on cancer treatment. That report expressed the view that the recommended treatment was clinically indicated for the Claimant and that his case was exceptional within the meaning of the PCT's policy, on both versions of the Terms of Reference which were disclosed.
58. There then followed an exchange of correspondence with further information and clarification of the Claimant's case as between Irwin Mitchell and Mills and Reeve, the PCT's solicitors.
59. On the 22nd August 2008 a fresh Review Panel considered funding Lenalidomide and Dexamethasone both with and without the addition of Cyclophosphamide, and decided to reject funding. A letter of the same date contained the following reasons for rejecting funding:
i) There is no new evidence to suggest that the Claimant's case is exceptional. A cohort of patients will have side effects of neuropathic pain with Bortezomib and Thalidomide and therefore this fact is insufficient to make this case exceptional. The Panel noted that social circumstances are not taken into account as per the policy.
ii) The evidence suggests that while there is a chance that there is some clinical effectiveness this would be small. (While the study is very small and therefore the subgroup analysis difficult it suggests that those who have previously received Thalidomide gained less benefit).
iii) The evidence indicates that the proposed treatment is not cost effective per QALY.
60. It follows that it is the decision of the 22nd August which is essentially the subject of judicial review, although in realty the reasoning of the panel is inextricable linked to the 28th May and 23rd June decisions.
61. It is common ground that the PCT cannot be criticised for failing to give reasons.
62. The Claimant's case is summarised in Messrs Clayton's and Sachdeva's written summary, that the decision to refuse the Claimant life prolonging treatment is unlawful on the following grounds:
a) the policy itself is unlawful;
b) the decision proceeded on the basis of material errors of fact; and
c) the decision was irrational in its approach to (i) clinical efficacy; (ii) exceptionality; and (iii) cost effectiveness; and
d) the PCT unlawfully fettered its discretion.
63. The PCT's case is in short as follows: (i) the issue of funding lenalidomide was at an early stage, with no suggestion by the Cancer Networks that the evidence supporting funding was so compelling that it should be fast tracked; (ii) accordingly, and for the reasons set out above, it could only be funded in individual cases on the basis of exceptionality; (iii) the Claimant's case was plainly not exceptional amongst fellow sufferers from multiple myeloma who are intolerant of thalidomide; and, (iv) in any event, the efficacy of lenalidomide, and its cost effectiveness, were not such as to warrant funding.
64. Mr Forde submits that the Panels reached decisions that were within their discretion; that they were not irrational; that they were entitled in the end to prefer the expert evidence of Professor Griffiths and to reject that of Professor Sikora as providing in effect no new evidence.
65. Both counsel enlarged their submissions in the course of argument, but time does not permit me to set them out in detail. I propose to deal with the various points in turn.
66. The structure of the decision under review contains three parts: exceptionality; clinical efficacy; cost effectiveness. I consider each in turn, although I am persuaded that the considerations of clinical efficacy and cost effectiveness are interrelated.
67. On a view of the matter in general, one of the practical problems that faces a Review Panel of a PCT is that very often, as in the present case, its members will be faced with an urgent consideration of funding which inevitably places them under pressure. I note that in the first version of the minutes of the 22nd August Review Panel the following introductory statement headed "Issues raised by the Panel" appeared which did not find its way into the revised minutes, but which clearly reflected the Panel's concerns.
"It was noted that he Panel had not had much time to read and consider the additional information. They also felt rushed into reconvening to reconsider this case, especially considering that the Appeal Panel letter of response was sent on the 23rd July 2008. The feeling of the Panel was that they would have preferred to be better prepared and to meet face-to-face." (the Review took place by telephone conference)
68. In purely human terms it is not surprising if on occasions a Panel may fall into logical error which leads to a decision that on legal analysis is viewed as irrational. I for one do not doubt the good faith of the members of each of the panels that reviewed the decision in question. I say this because concern was expressed by Mr Forde as to the terms of the criticism levelled at them by Mr Clayton during his submissions. Sympathy in this matter is not entirely one sided. I sympathise with the pressure that faced the 22 August Panel and the less than satisfactory circumstances in which they had to consider these important issues.
69. Professor Sikora considers that the Review Panel should have contained an suitably qualified expert in oncology. On the other hand, both Dr Bull and Professor Griffiths make the point that the constitution of PCT Review Panels provides a proper balance of professional and lay input. It is suggested that it would be an unreasonable use of expert resources to require a specialist in the precise field under scrutiny in every application for funding that comes before a PCT.
In my view, there is a danger of becoming involved in hyperbola here. The answer is that the PCT's policy on the constitution of its Review Panels is, in my judgment, reasonable, because the policy allows for the seeking of appropriate expert advice where thought necessary. Such advice, in my view, would almost certainly be in the form of a written report along the lines of Professor Sikora's report in this case. One of the matters that I have to consider is whether the original Review Panel should have sought such advice before reaching their decision and whether the subsequent Review Panel adopted a rational and logical approach in concluding that Professor Sikora's report provided no new evidence.
70. In this regard, Ms Braysher makes the point that as a PCT it does not have the analytical resources that NICE has and that, therefore, it has to make decisions "without, in the main, having world leading experts to advise its members." In my view, it is all the more important that its Reviewing Panel should, where necessary, seek such expert advice, and, where available to its members, take particular care to understand and to analyse critically such advice.
71. In my judgment, the original Review Panel failed to appreciate the clear need for expert advice from a specialist in the field of oncology such as Professor Sikora. In his report he highlights the failure to understand the essential points, in particular, as to when a cancer patient can be regarded as exceptional; the significance and weight to be attached to the Dimopoulos and Weber randomised controlled studies and to the London New Drugs Group Briefing in May 2008, both of which made a clear case of clinical efficacy for "the use of Lenalidomide (in combination with dexamethasone) in patients at any disease stage where Thalidomide treatment is indicated, but is not tolerated because of peripheral neuropathy …" (my emphasis), making, in Professor Sikora's words the statement in the first decision letter that there was "no robust evidence for the use of Lenalidomide in combination with dexamethasone and cyclophosphamide" as "patently incorrect", making the point which the Panel does not appear to have unerstood without expert assistance that the Royal Marsden study with regard to adding cyclophosphamide was simply beneficial.
I agree with Mr Clayton that there could be no indication that the addition of cyclophosphamide could make any difference to the robustness of the evidence that was available for the use of Lenalidomide in combination with dexamethasone for a patient who otherwise would benefit from Thalidomide but for his intolerance due to peripheral neuropathy. Professor Sikora on the question of clinical efficacy and cost effectiveness pointed out that the Panels had not taken into account the savings in not providing the other life prolonging drugs to which the Claimant had become intolerant.
72. Mr Forde has criticised Professor Sikora for giving an opinion on the PCT's exceptionality policy as being out of his field of expertise. In my view, Professor Sikora did demonstrate, as part of his expertise in the field of oncology and haematology, that the consideration of which patients satisfy the various tests of exceptionality is within his expertise. I do not regard his criticism of this PCT's policy on exceptionality as trespassing on my province and found his reference to the simpler versions usually found as helpful in considering whether the exceptionality policy of this PCT as submitted by Mr Clayton, was unduly restrictive and, therefore, unlawful.
73. Professor Sikora, in my view, correctly pointed out that Lenalidomide, on a proper calculation, comes below the NICE 'threshold' of £30,000.
74. Finally, his reference to the possibility of Lenalidomide giving the Claimant nearly 3 further years of life was derived from and based on data presented at the British Society of Haematology in April 2008, see the Ishak report demonstrating on average nearly 3 further years.
75. I find it surprising that, if proper consideration was given to Professor Sikora's report, it could be said that there was no new evidence. His expert evidence was new and, in my judgment, went a long way to explain where the Review Panel and Appeal Panel had failed to understand the essential case presented so clearly in the first place and then re-emphasised by Dr Davies.
76. The important facts in this case are that the Claimant responded well to Thalidomide, but that he developed intolerable peripheral neuropathy so that he could no longer be treated with anything but palliative treatment. It seems clear to me, from my advantageous position of being able to review the decisions of each reviewing panel with the assistance of expert evidence and counsel's detailed arguments, that each panel equated the examples of patients being resistant to Thalidomide and of patients merely developing unpleasant side effects to those for whom the side effects amounted to intolerable peripheral neuropathy. In my view, the distinction between these three examples was of vital importance when considering whether or not the application for funding in the Claimant's case was exceptional.
77. It seems to me that the PCT in attempting to formulate a fair policy as to what could amount to an exceptional case introduced an unnecessary element of confusion into what is a relatively straightforward concept. Whilst the PCT correctly starts with the dictionary definition of 'exceptional' the attempts to illustrate its application created more difficulties to the reader who had to apply the policy. This, in my view, is to the extent that it becomes difficult, if not impossible, to envisage who and in what circumstances might be successful in overcoming this hurdle in respect of the drug Lenalidomide. It is no coincidence that no application for funding of Lenalidomide has been approved by this PCT.
78. The crucial point, as I accept, is that the policy states that an application "must" be refused where a patient is "representative of a group of patients"; that the inevitable result of the policy is that if a patient is representative of a cohort of patients, then he is automatically refused treatment; that a 'group' must extend to at least two patients, otherwise it would not satisfy the definition of what the minimum number for a 'group' would be; that the focus on 'other patients' explains the references in the decision letters and minutes to whether the Claimant shared characteristics which were similar to other patients; that it is crystallised in the Detailed Grounds of Resistance paragraph 31:
"As set out in the documents, it was decided that, as a patient with multiple myeloma who suffered from well recognised neuropathic side effects as a result of taking thalidomide, whether responding well or not, he was not exceptional within the meaning of the policy, but came within a class of patients."
79. I am satisfied, therefore, that the PCT policy is unlawful because it is a contradiction as defined by its own terms because:
i) it is not a policy for exceptional cases because a person is automatically disqualified if he can be likened to another: in order to qualify, a patient must show in effect that he is unique, rather than merely exceptional in the ordinary sense of the word as being 'of the nature of or forming an exception; out of the ordinary course, unusual, special.' (Oxford Dictionary);
ii) in practical terms, in a case such as this Claimant's it is impossible to show uniqueness, so the policy is incapable of fulfilment because (i) it will always be possible for another patient to emerge who is appropriately comparable; and (ii) the comparison depends on how widely a label is drawn by the PCT eg whether the Claimant should be compared to any cancer patient who suffers unpleasant side effects or to something more specific;
iii) in my view, it is impossible to envisage circumstances other than those where the applicant shows that his or her circumstances are unique, whereas in a simple policy of exceptionality as encountered by Professor Sikora and set out in his report a reviewing panel would have no difficulty in applying the ordinary meaning of 'exceptional'.
80. I have seen some examples of drugs that have been funded under the exceptionality policy of this PCT, which, in my view, tends to support Mr Clayton's submission that the policy is really one of uniqueness rather than exceptionality.
81. The way in which the policy on exceptionality was framed, in my judgment, first of all led the Review and Appeal Panels into error simply on the ground that they clearly thought that, because other patients could find themselves in the Claimant's position, therefore, he did not come within the exceptionality. In any event, the Panels proceeded on a fundamental misapplication of the term 'exceptional', misdirecting themselves that it was not exceptional to suffer 'unpleasant' side effects from Thalidomide and therefore, failing to appreciate and to understand properly the vital distinction that was made clear in the application that it was the claimant's intolerance to the peripheral neuropathy which made him an exception, as indeed Professor Sikora relying on his wide experience in this field confirmed.
82. For these reasons, in my judgment, the decision to refuse funding on the ground of exceptionality was logically flawed and on the evidence before the panels ought to have been upheld on an ordinary reading of the term 'exceptional.'
83. I accept Mr Forde's submission that the panels were correct to consider clinical efficacy and cost effectiveness. In my view, it would make no sense for a PCT simply to ignore clinical efficacy and cost effectiveness once a patient comes within the category of exceptional circumstances. However, it seems to me that, once an exceptional case is made out, particularly where matters of extending life are concerned, that logically and rationally a PCT should take a less restrictive approach to cost effectiveness than when considering the case for funding a drug as part of its Operational Plan.
84. The second hurdle, therefore, was clinical efficacy. The important part of the Panel's decision in this respect is that they did not rule out supporting treatment on the grounds of clinical effectiveness, expressing only their reservations as to the quality of evidence presented to support effectiveness. That meant that the application would have cleared the second hurdle.
85. Nevertheless, in my judgment, the failure of any of the Panels to understand the strength of the evidence in favour of treating a patient such as this Claimant with Lenalidomide in whatever combination of supplementary drugs, was replicated through each of the decisions and probably stemmed from a failure to understand the significance of the fact that the randomised control studies had to 'unblind' the patients who were being given the placebo: first, because Lenalidomide was proving so successful and it would have been unethical not to have offered it to the placebo patients; second, it skewed the statistical results. This, I accept, led to a fundamental misunderstanding of the results which showed a one year survival rate of 82% in the Lenalidomide pateients compared to the 75% in the control group, simply because, as the London New Drugs Group briefing explained, 47% of patients crossed over to Lenalidomide. To this extent, in my judgment, the Review Panel proceeded on a mistake of fact which satisfies the test in E (above).
86. In my judgment, the Review Panels ought to have upheld the clear case on clinical efficacy presented to them by Dr Davies in the original application, her letter of the 16th June and Dr Boyd's letter of the 1st August.
87. In looking at cost effectiveness, the Panels accepted the figures submitted by the manufacturer which yielded a cost per QALY of £28,980.
88. The Panel considered the cost of providing Lenalidomide to all patients suffering from myeloma would inevitably "a lot greater" on the basis that some would only have a partial response and some would have no response. However, in taking this into account it seems to me that the Panel were looking ahead and beyond its Patient Individual Needs policy to considerations that would have to be applied were Lenalidomide to be commissioned by the PCT in the future.
89. The question I have to ask myself is whether the Panel asked themselves the correct questions when considering the cost effectiveness of treating an exceptional patient with Lenalidomide.
90. I can see how the Panel fell into error when considering cost effectiveness. Of course, the possibility and potential expense of a year's course of treatment were relevant considerations for the reasons Mr Forde advanced. If the treatment were found to be effective for this patient after the 4 cycles envisaged, then the human consideration of stopping treatment at that stage or after 6 cycles, if it were extended, might result in funding for substantially longer than the funding sought by the Royal Marsden in this application. However, the error came in considering that the cost per QALY would be substantially in excess of the £28, 980 figure on the basis of the Dimopoulos and Weber randomised control trials, which resulted in 60% of patients showing response, but only 15% showing a full response.
91. I accept Mr Clayton's and Mr Sachdeva's submissions that the views expressed by the Panels were such that no reasonable authority could reach in terms of its decision that the Claimant's case was not cost effective because:
(a) they misunderstood the effectiveness of Lenalidomide: with the result that they could not rationally assess its 'cost effectiveness';
(b) they failed to understand that the actual sums sought were for 4 cycles, with the potential for a total of 11 cycles if the Claimant responded to Lenalidomide; that, if he did not respond, the treatment would probably not be continued so that further cost would not arise;
(c) they misunderstood the median survival advantage figure of 9.4 months, and erroneously appeared to apply to a double discount for the 60% partial response rate and the 15% full response rate;
(d) they failed to take account of the saving to the PCT of not having to provide the expensive life prolonging treatment which had been given to the Claimant within its Operational Plan before he developed his intolerance to it, which could have been another ground for holding that his circumstances were exceptional to the Plan;
92. As I have indicated, in order for the Claimant to proceed beyond the 3 to 4 cycle stage, as I understand the submission made by Dr Davies, he would have to be showing a satisfactory response. It follows that I accept Mr Clayton's and Mr Sachdeva's submissions that, as explained by Dr Bull, who chaired both the 28th May and 22nd August Panels, the Panel found themselves in a double counting exercise, in which account was taken of those patients who would not have a response of one year's additional life. Logically, any additional cost would only arise in the event that the Claimant was responding to the treatment, which would make it both clinically and cost effective.
93. For the sake of completeness, although not decisive the Panel concluded that Lenalidomide was "not supported" by the local Sussex Cancer Network. It was clear that its sub group had indicated its support. It was just that the Network had not yet reached the stage of supporting it, which, in my view, gives an entirely different impression. Further, although it appears to me that the Panels misdirected themselves when applying the policy as to whether or not social circumstances should have been taken into account, by applying it too strictly, I accept Mr Forde's submission that the PCT was entitled to avoid where possible taking decisions purely based on such considerations.
94. I conclude, therefore, that the decision of the PCT was one which no reasonable authority could have made on the application before it. It follows that the decision must be quashed.
95. Although consideration of relief does not form part of this judgment, in my judgment, the urgency of the situation coupled with the delays that occurred between the original submission of the application for funding dated 31st March 2008 and now indicate that there should be some mechanism, in the light of my judgment, for commencing treatment at the earliest time. This is a case which has a number of harsh realities. Should the PCT on considering the application afresh in accordance with the guidance given in this judgment nevertheless conclude that funding should not be given, then the claimant has to recognise that his treatment might well have to cease. I make these observations not to anticipate any decision on relief, but rather to suggest how the question of relief might be approached, in the unusual circumstances of this case, should it be accepted that treatment could commence on the strict understanding that, if the PCT properly directing itself in accordance with the reasoning of my judgment, nevertheless reached a contrary decision, that the treatment would have to cease, however harsh that may seem.
Note 1 Secretary of State's duty to promote health service 1(1) The Secretary of State must continue the promotion in England of a comprehensive health service designed to secure improvement— (a) in the physical and mental health of the people of England, and (b) in the prevention, diagnosis and treatment of illness. (2) The Secretary of State must for that purpose provide or secure the provision of services in accordance with this Act. (3) The services so provided must be free of charge except in so far as the making and recovery of charges is expressly provided for by or under any enactment, whenever passed.
Note 2 Section 2(1) The Secretary of State may— (a) provide such services as he considers appropriate for the purpose of discharging any duty imposed on him by this Act, and (b) do anything else which is calculated to facilitate, or is conducive or incidental to, the discharge of such a duty.
Note 3 Section 3(1) The Secretary of State must provide throughout England, to such extent as he considers necessary to meet all reasonable requirements— … (c) medical, dental, ophthalmic, nursing and ambulance services, … (e) such other services or facilities for the prevention of illness, the care of persons suffering from illness and the after-care of persons who have suffered from illness as he considers are appropriate as part of the health service, (f) such other services or facilities as are required for the diagnosis and treatment of illness.
Note 4 1. “Summary 2. This HSC… 3. iii asks NHS bodies to continue with local arrangements for the managed introduction of new technologies for any interventions which are not referred to NICE, or where NICE’s guidance is not available at the time the technology first becomes available. 4. … 5. The overall context 6. ... 5. If a new intervention is not referred to NICE, this does not imply any judgment on whether the intervention(s) in question are clinically or cost effective. NHS bodies should continue to use exi[s]ting arrangements to access the publicly available evidence and to determine local policies for the managed entry of the new intervention. The same principle should apply if an intervention has been referred to NICE but guidance is not yet available at the point which the new intervention is first introduced…”