Download link to appeal document - British Kidney Patient Association

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