NKF Campaigns Zone:
Transplantation & Live Donors

On 22 January 2004, the National Institutue for Clinical Excellence (NICE) published a Final Appraisal Determintation (FAD) of its report on Immunosupressive Therapy for Renal Transplantation. A copy of this report can be downloaded from this website by clicking here (PDF, 175 Kb).

The NKF has submitted to following appeal:

IMMUNOSUPPRESSIVE THERAPY FOR RENAL TRANSPLANTATION
APPEAL AGAINST
FINAL APPRAISAL DETERMINATION
BY
National Kidney Federation 

30^th January 2004

To: Dr Carole Longson
c/o Nina Pinwell
NICE
Mid City Place,
71 High Holborn,
London,
WC1V 6NA

Dear Dr Longson,

The National Kidney Federation is a charity run by Kidney Patients for kidney patients. It currently represents 37,500 patients suffering end stage renal failure, of which 20,000 have had a transplant and 17,500 are awaiting a transplant opportunity. Of those 17,500 approximately 5,500 are on the Transplant waiting list.

Clearly the NKF has a very deep interest in the findings of your appraisal which is why we have sent you submissions at every stage of the process to date. We have always argued that the drugs in use are far from perfect and that what is required is continued experimentation and development of these drugs, above all we want nothing ruled out in order that doctors can continue to switch patients from one therapy to another as and when the medical condition of the patient deems this appropriate. Our survey of 2,500 patients, carried out in 2001 confirmed that this switching is taking place all the time with the majority of patients – the aim being to protect the patient and protect the enormously valuable transplanted organ.

The Perversity of the FAD

We have been alarmed to read in the Final determination the following two sentences

1. Mycophenolate mofetil is recommended as an option as part of an immunosuppressive regimen ONLY where there is proven intolerance to calcineurin inhibitors, particularly nephrotoxicity leading to risk of chronic allograft dysfunction or during periods of very high risk of nephrotoxicity, necessitating minimisation or avoidance of the calcineurin inhibitor
2. Sirolimus is recommended as an option as part of an immunosuppressive regumen ONLY in cases of proven intolerance to calcineurin inhibitors (including nephrotoxicity) necessitating complete withdrawal of these treatments.

We find this judgement to be utterly perverse and likely to defeat the entire object of giving patients immunosuppressant treatment. We believe that if this were to be insisted upon, many of the transplanted kidneys (a very rare and valuable gift from another human being) will be lost as a result and the patient forced back onto dialysis, or even worse.

Our reasoning

1. The only drugs you are permitting to be used immediately following the Transplant operation are the nephrotoxic ones – the very drugs which can damage the kidney they are trying to protect
2. Whilst your appeals time span has not given the NKF time to carry out a further written survey of our patients, we have done a straw poll, and believe that more than 50% of our Transplanted patients are taking Mycophenolate mofetil as part of their treatment at this time. We are alarmed that your FAD will not just affect a minority of our members, but will affect adversely the majority.
3. Our transplanted patients are badly affected by the side effects of todays immunosuppressant drugs, many die from heart failure and cancer. We are in desperate need of new drugs which may prove to be free of these harmful effects. Sirolimus is seen to be one of these new drugs, but it has not been available long enough for many of our patients to find out yet whether they will benefit. If your FAD is to be followed, clinicians will not be able to prescribe this as a first use drug, and patients will have to be subjected to the drugs with the known risks right from the very beginning of their treatment. We believe this to be a very retrograde step that will take away one of our greatest hopes for the future
4.  Your FAD, does allow the use of the above two drugs once intolerance to cacineurin inhibitors has been proved, but we would argue that this is much to late to make the switch to these other drugs. The NKF is very well aware that the speed with which the new organ begins to function and its performance in the early days (certainly during the first year) is a very good indicator as to whether the organ will survive in the long term and whether it will serve the patient well for a long time. We believe strongly that by denying access to these two drugs at the beginning, you are likely to consign many of the transplanted organs to failure when they might have been saved. We are very distressed by this, as we are finding it increasingly difficult to persuade the public to donate organs in the first place, particularly the relatives of the deceased. We do not relish the prospect of wasting unnecessarily many of the donated organs so generously given.

The Appeal hearing

As the Chief Executive officer of the NKF, I am neither a patient, nor a medical expert, and as such you may feel that this written submission is sufficient for your needs and that my attendance at your appeal would not provide anything additional to the matters stated here, however, the NKF is very alarmed by your decision, and I am instructed to use every endeavour to request you to alter your opinion, therefore if you do wish me to attend and give evidence, then I am more than willing to do so. Any such invitation should be addressed to

Yours sincerely,

Timothy F Statham OBE

Chief Executive
National Kidney Federation