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Re: Final Appraisal Determination - Immunosuppressive therapy for kidney transplant in adults (review of technology appraisal guidance 85) The British Kidney Patient Association is appealing against the FAD for both adults and children and young people on the grounds that the recommendation is unreasonable in the light of the evidence submitted to NICE. The BKPA is the leading kidney patient support charity whose aim is to improve their quality of life. I and my 2 colleagues have all experienced dialysis and are only able to be here today because we have received and maintained our kidney transplants. We want the committee to consider how the recommendations apply to the entire group of patients, and look at the subgroups who will not be able to benefit from the FAD. We want you to look at how they have been defined and what the disbenefit will be to them. For each group, the comparator is dialysis. The wastage, knock-on costs to the system and quality of life have not been adequately considered in the FAD. Reaction to standard medications 1) For basiliximab the comparator used was a placebo or no induction, not the inability to receive a transplant. There is no consideration for the 20% you document who will be unable to tolerate or for whom it will be ineffective. 2) For MMF you have very little to say about the 10% for whom the GI reaction will be so intense it will risk loss of transplant and mean people cannot leave to the house due to uncontrollable diarrhoea. My colleague Patrizia can discuss this and the MMF subgroup with some authority. 3) For prolonged release Tacrolimus where is the consideration for the subgroups who will struggle to comply with complex drug regimes and who have variable immunosuppressant levels, for whom this formulation was developed. My colleague Nick has been working with children and young people recently and can give more information on this point. 1 For every one of the sub-groups affected by the FAD, there is a significant impact and little evidence of it being evaluated. Quality of life impact of dialysis The quality of life for an individual and their family unable to have a transplant because of this determination will be very difficult. The symptom burden is high, including sleeplessness, pain, itching, thirst, nausea, anorexia, aching bones, poor memory, anxiety. Transplantation resolves much of this. Dialysis is not an effective alternative treatment to transplantation. In order to keep on living, patients have to comply with many many restrictions, must drink no more than 0.5 litres of fluid a day, and even a banana could bring on heart failure. Additionally they must go to hospital 3 days a week for about 4 hours of treatment which plus travel time means people are out for up to 8 hours at a time, many without food all day long. This continues until death. Harm from lost transplant Recommendation 1.4 does not take into account the harm in each subgroup of a lost transplant. For people in these groups there may also be additional physical harm caused by the unsuccessful surgery and a high incidence of depression. Increased mortality impact Survival on dialysis is limited compared with survival with a transplant. This has again not been considered but is of absolute significance to us. The recommendation does not take into account the increased mortality and additional hospitalisation of those who will be unable to access transplantation and are taken off the transplant waiting list because alternative treatments are not available. Vascular access for dialysis will also give out over time leading to death. Risk of death 19x that of regular population at 35-39, 2.5x at 85+. Economic impact Recommendation 1.4 has failed to take into account the resultant reduction in future possible transplants, which would lead to more dialysis. The costs of dialysis are estimated by NHSBT at £30,000 pa, excluding some drugs and hospital transport (transport, by the way, can cost as much as the dialysis 2 treatment itself). We estimate that the appraisal will affect between 485-730 patients a year, being the sub-groups of 20-30% who are unable to tolerate the recommended drugs and will develop rejection or would no longer be suitable to have a kidney transplant. This is £14.5-£22m per annum, every year, for the rest of their lives. A further 485-730 patients would be added to the numbers on dialysis and the bill every year. Where are the plans for additional dialysis capacity to cope with these numbers? The costs of failed transplant surgery at £17,000 per operation and watage of a donated scarce resource are also not considered in the FAD to date. There is a further economic impact in the inability of most dialysis patients to return to the workplace and the consequent reliance on social care and benefits to support them to live. Over time, it is less expensive to maintain someone with a transplant than to maintain them with dialysis. The Appraisal Committee acknowledge that there are limitations in the available evidence and of the consequent clinical and cost-effectiveness analysis. Nevertheless the risks in this process are disregarded as are the many sub-groups who will not be able to have or maintain transplants. Please change the recommendation from ‘do not recommend’ to ‘cannot make a recommendation’. 3 Conclusion The BKPA asks the Committee to change its wording from ‘Rabbit ATG, prolonged-release tacrolimus, mycophenolate sodium, sirolimus, everolimus and belatacept are not recommended to prevent organ rejection in adults having a kidney transplant’ to ‘It is not possible to make recommendations on the use of rabbit anti-human thymocyte immunoglobulin, prolonged-release tacrolimus, mycophenolate sodium, and sirolimus to prevent organ rejection in adults having a kidney transplant’. We are asking NICE to retain an effective alternative treatment path for patients who will otherwise remain on dialysis or die. This approach is entirely consistent with NICE’s wording in the note in the following paragraph that ‘The Appraisal Committee was unable to make recommendations on these technologies for…biopsy proven nephrotoxicity…’ The BKPA would like to state that the impact of this recommendation from NICE on the sub-groups of the small heterogeneous population with kidney failure has not been fully considered by NICE; and to ask that it reconsider the wording of the technology appraisal. We are not asking for the earth but simply want to see these drugs used and do not believe that the wider implications of quality of life, harm of failed surgery and economic impact have been considered. The result of this determination, if it proceeds, will seriously affect the future of transplantation in this country. There is a long and complex path to obtaining an organ. We wish NICE to adopt the alternative approach. 4