NICE gives preliminary approval for lenalidomide20 Aug. 2014
20/08/14 – NICE has now finally issued their FDA (Final Draft Guidance) for the use of lenalidomide (Revlimid) for del 5q MDS patients.
NICE is now recommending the use of this drug within the NHS.
This comes as a very welcome decision, after almost 18 months, requiring 5 meetings and the introduction of a patient access scheme.
The manufacturer proposed the patient access scheme, which provides a price discount: the NHS receives the drug for free for any patients remaining on the drug after 26 cycles.
This particular appraisal was especially difficult, due to the relatively small trial data available – and the lack of long term data for these patients.
This is however a common problem for all rare diseases – and an issue that will come up many times in future when appraising similar drugs in similar patient populations.
This time, the manufacturer was fortunately able to supplement real-life data – to satisfy NICE’s cost-effectiveness queries – and to help reach a satisfactory conclusion for patients.
In our joint submission with Leukaemia CARE and Rarers Cancers Foundation, MDS UK supported the evidence regarding the improvement in Quality of Life – and anecdotal evidence regarding survival past 26 weeks.
But what was truly needed was MDS clinical experts able to participate in the discussions.
We still believe that the NICE process should be improved further by inviting clinical experts to take part in every subsequent appraisal meeting – especially when the decisions are highly complex.
Relying purely on the presence and additional data of pharmaceutical companies is limiting.
The presence of clinical experts at the meetings would provide independent, unbiased opinions and would help to clarify any queries instantly – which can otherwise lead to misinterpretations during the appraisal discussion.
MDS UK, Leukaemia CARE and Rarer Cancers Foundation therefore welcome NICE’s decision – but maintain our strongest request to alter the NICE process – to include experts DURING each public meeting.
You can read our previous entries on this topic on our News column (use the Search facility on our website) – or in our newsletters.
Please also find NICE’s full press release below:
NICE proposes to recommend lenalidomide for myelodysplastic syndromes
NICE has issued final draft guidance recommending lenalidomide (also known as Revlimid and marketed by Celgene) as an option, for treating myelodysplastic syndromes.
Myelodysplastic syndromes (MDS), which are diagnosed in around 2000 people each year in England, are a group of bone marrow disorders characterised by the underproduction of one or more types of blood cells due to dysfunction of the marrow. MDS can lead to life threatening disease including acute myeloid leukaemia (AML), as well as anaemia and increased risk of bleeding and infections.
This appraisal focused on the use of lenalidomide for treating people with a specific type of MDS that is characterised by a chromosomal abnormality called an isolated deletion 5q cytogenetic abnormality. At the moment the main treatment option for people with the particular kind of MDS considered in this appraisal is best supportive care including regular blood transfusions.
Commenting on the final draft guidance, Sir Andrew Dillon, NICE chief executive, said: “The committee heard from clinical experts that lenalidomide is an effective therapy. Celgene – who market lenalidomide – worked with us to provide enough evidence to make it possible for us to recommend it for this group of people.
“Celgene provided a revised analyses and further information on their proposal for a reduction in the cost of the drug to the NHS (patient access scheme).”
Celgene’s patient access scheme involves the NHS paying for lenalidomide treatment for up to 26 monthly cycles. The company will provide the drug free of charge for those people who receive more than 26 monthly cycles.
The draft guidance is now with consultees, who have the opportunity to appeal against it. Until NICE issues final guidance, NHS bodies should make decisions locally on the funding of specific treatments.
Notes to Editors
Explanation of terms
1. Lenalidomide holds a marketing authorisation for treating transfusion-dependent anaemia caused by low or intermediate-1 risk myelodysplastic syndromes associated with an isolated deletion 5q cytogenetic abnormality when other therapeutic options are insufficient or inadequate.
About the guidance
NICE technology appraisal 218 guidance recommends azacitadine as a treatment option for patients with intermediate-2 or high risk MDS who are not eligible for haematopoietic stem cell transplantation, which is a different population to the one considered in this appraisal.
The main treatment option currently available for people with low- or intermediate-1-risk MDS associated with an isolated deletion 5q cytogenetic abnormality when other therapeutic options are insufficient or inadequate is best supportive care, which involves regular red blood cell transfusions.
The Committee concluded that lenalidomide is a clinically effective treatment for people with low- or intermediate-1-risk MDS associated with an isolated deletion 5q cytogenetic abnormality when other therapeutic options are insufficient or inadequate, because it was associated with a statistically significant improvement in transfusion independence and health related quality of life compared with placebo. The Committee agreed that it was plausible for lenalidomide to indirectly improve overall survival by improving transfusion independence.
The Committee noted that the revised company’s base-case incremental cost-effectiveness ratio (ICER) for lenalidomide compared with best supportive care with the patient access scheme was £25,300 per QALY gained. The Committee noted that the patient access scheme was not a simple discount and that would only benefit those on treatment after 26 cycles. The Committee agreed that because the proportion of people on treatment beyond 26 cycles was uncertain so were the potential cost savings from the patient access scheme, and noted that if the proportion of people who reached 26 cycles was less than 27%, the ICER would be greater than £30,000 per QALY gained.
The Committee agreed that the ICER was uncertain, because of the patient access scheme, but accepted that a commitment from the company to publish data on the proportion of people on treatment beyond 26 cycles would provide reassurance that lenalidomide for treating MDS associated with an isolated deletion 5q cytogenetic abnormality was recommended as a cost-effective use of NHS resources.
Lenalidomide is available in 21-day packs of 10 mg and 5 mg capsules at net prices of £3780 and £3570 respectively. The cost of a 28-day cycle of treatment with 10 mg of lenalidomide (excluding VAT) is £3,780. Celgene has agreed a standard patient access scheme with the Department of Health, in which the NHS pays for lenalidomide treatment for up to 26 monthly cycles. The company subsequently provides free of charge lenalidomide for those people who receive more than 26 monthly cycles.
NICE appraisal document: