NICE Lenalidomide appraisal

NICE – Lenalidomide Appraisal – ACD – June 2014

NICE has issued their latest decision regarding the use of lenalidomide in del 5q MDS patients.
Unfortunately, this is still negative, but it is not yet NICE’s final decision.

Despite the introduction of a Patient Access Scheme, as well as further data and evidence of use of the drug amongst further patients in the UK and abroad, NICE is still unable to come to a definite decision regarding the real cost-effectiveness of the drug in this small group of patients who require frequent blood transfusions.

The number of MDS patients in England, Wales and Northern Ireland who would be likely to benefit from the drug is very small – estimated at only 400 a year.  These are patients whose quality of life could be tremendously improved by taking this drug, which could potentially free them of the need for blood transfusions, frequent hospital visits and infections.

In the Patient Access Scheme, the manufacturer offers the drug free of charge to the NHS after 26 cycles of treatment.  It is estimated that – on average – about 31.9% of del 5q transfusion dependant patients are alive and well at this particular time-point, based on the data available to date.
This same scheme is already in use in Multiple Myeloma, where it works well.

One of the issues cited by the NICE appraisal committee is the validity of this 31.9% survival figure.
Other health economists and Health Technology experts at the Scottish Medicines Consortium (SMC) have approved the cost-effectiveness of this drug – based on exactly the same data.

MDS experts have yet to be consulted officially in this appraisal, as to date only the manufacturer and NICE experts were asked for detailed additional information.

NICE also fully recognises the burden of transfusions – and have even issued recent guidelines that recommends the use of treatments such as EPO for treatment associated anaemia.

But despite all of this evidence, NICE is still questioning some of this data, running different treatment scenarios, for which there simply is not always enough data for long-term projections in time.  In NICE terms, these cost-effectiveness calculations end up with uncertain answers.

With ever smaller, and more specific groups of cancer patients,  and limited amounts of data, both in terms of length of use and overall number of patients, the “uncertainty” around the economic models that NICE has to use is bound to increase.  With this in mind, it is not surprising that NICE was able to approve only 1 cancer treatment in this past year.

The SMC in Scotland have been able to approve the use of the drug for patients, without the need for a patient access scheme, as their del 5q patient population is extremely small – and the overall burden on the health budget extremely low.
Having witnessed the SMC Committee deliberate and debate, we are very aware of the difficulties regarding the assessment of cost-effectiveness, but fortunately, the state of patient data as it stands now was deemed to be sufficient for this groups of Scottish physicians and professionals to realise the benefits overall, without having to resort to cost models which might be too complex for such small groups of patients.

Trying to eliminate all uncertainty may take even more years , during which many del 5q patients will be forced to remain on transfusions and unable to enjoy a reasonable QOL, whilst living – and possibly also working – with the disease.

MDS UK, in their joint submission with Leukaemia Care has repeatedly offered NICE to validate clinical practice data, through the UK MDS Forum of experts at MDS Centres of Excellence, but the NICE process of appraisal does not enable non-pharma consultees to provide additional input, outside standard ACD comments.
Only the pharmaceutical consultees are invited to provide extra evidence outside the ACD process.

MDS UK is now urging NICE to engage more fully with both patient groups and MDS experts – to verify real-time clinical practice in the UK and abroad – in order to reduce the “uncertainty” and get a realistic, independent picture of the treatment results.  In parallel – we also urge the manufacturer to see where and how the patient access scheme could be altered to make the drug available to all in the UK – as soon as possible.

For Northern Ireland, we have the support and assistance of MP David McIlveen (North Antrim), who recently spoke in the Northern Ireland Assembly about the issue of access to treatment for people affected by Rarer Cancers:

Private Members’ Business – Cancer Drugs: Funding
Topic: The difficulties faced by patients obtaining funding for cancer drugs; concern about the varying policy positions throughout the UK and Ireland regarding the accessibility of cancer drugs; calls on the Minister of Health, Social Services and Public Safety to undertake a review into the practices in Northern Ireland to provide greater clarity for patients trying to obtain funding for cancer drugs.

Comment extracted:
“Mr McKinney: Thank you. I welcome the intervention. It is a point that I will expand on in just a moment.

In England, as we are aware, a cancer drugs fund (CDF) is in operation that provides direct access for cancer sufferers to cancer drugs. In Scotland, just this month, the peer-approved clinical system has been put in place. That has replaced the IFR model and the clear inequalities in access that it was producing. In Wales, the All Wales Medicines Strategy Group is in place to appraise medicines before NICE. Consequently, that, too, improves access for cancer patients. However, a recent study by Bristol University uncovered that cancer sufferers in England are seven times more likely to receive the drug than those in Wales. There are varying journeys towards greater access to cancer drugs in the UK. We, however, are not yet at the starting line. When the SDLP has asked the Minister about that inequality, he has deflected it by bringing up the viability of the IFR process or even the issue of prescription charges or welfare reform.
The Rarer Cancers Foundation and others have undertaken research about the cost of a CDF model here. It ranges between £5 million and £6 million. That is significantly less than the amounts needed in England, and it is feasible, we argue, if the Minister wants it to be. It is also important to note that, due to sophisticated molecular testing, improved diagnosis is available. Clinicians are available to accurately pinpoint which drug will work for a particular individual. So a cancer drugs fund model will cost more but will be much more refined and accurate than initially imagined.
Please see link to full transcript here:
Hansard 6 May REVISED (2)

As a reminder – lenalidomide is currently available through the Cancer Drug Fund in England, but not in Wales or Northern Ireland.
It is also available as a standard treatment in Scotland.
The full NICE consultation can be found here:
NICE-Lenalidomide Consultation papers

 

MDS UK – 10/06/14

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