This decision should see a significant breakthrough in the fight for patient access to the ground-breaking AL amyloidosis treatment.
Commenting on the successful appeal, Sarah Ellson, co-head of the Regulatory group at Fieldfisher, said: "This landmark victory highlights the crucial importance of equitable access to life-saving treatments and we're pleased to have worked with Myeloma on achieving this outcome."
Shelagh McKinley, Director of Research and Advocacy at Myeloma UK, said:
"We are overjoyed with the outcome of this appeal, which is a significant milestone in our fight for patients' rights and access to vital treatments. Fieldfisher's expertise in regulatory matters in the healthcare sector played a significant role in achieving this outcome and we thank them for their guidance throughout this challenging journey".
Associate, Chloe Williams was also involved in the case.
NICE recommended in an appraisal letter to the NHS that they should not use cancer drug, daratumumab to treat a rare incurable disease, light chain (AL) amyloidosis. Following the recommendation, Myeloma submitted a formal appeal against the decision on 16 December 2022. Jansen Pharmaceuticals separately appealed the decision on the same day.
Myeloma appealed the recommendation made by NICE on the grounds that NICE failed to liaise and seek guidance from haematologists (the treating physicians for AL amyloidosis patients) and as a result, without the input of clinical experts NICE had failed to act fairly when providing its recommendation.
NICE use cost-effective analysis as a type of economic evaluation that examines the costs and health outcomes when assessing whether to recommend a medicine to the NHS. This is known as an incremental cost-effectiveness ratio (ICER). An ICER is calculated by comparing the cost of two different medications divided by the difference in their effect. NICE use the Quality Adjusted Life Year metric as a measure of health outcomes. NICE attributed a low score of £20,000 per (QALY) ICER threshold to daratumumab, meaning that in the view of NICE, daratumumab is not a cost-effective medicine.
Fieldfisher, on behalf of Myeloma, challenged the ICER value that NICE attributed to daratumumab. The appeal detailed how NICE failed to consider all the necessary factors required to establish an ICER value. It was therefore argued that NICE had not acted in an objective manner when determining the ICER value. The appeal made clear that, as stated under the guide to the technology appraisal and highly specialised technology process 2013, cost-effectiveness should not be the sole basis for decision-making when determining whether to recommend a medicine to the NHS.
The appeal outlined other factors that should have been considered in the decision-making process; for example, Myeloma research has shown that daratumumab has been used internationally with life-changing results. Consequently, the appeal argued that if the NHS could not provide daratumumab, it could cause AL amyloidosis patients to suffer and would mean that they are not receiving an international standard of treatment.
The appeal panel heard from consultant haematologists Professor Ashutosh Wechalekar and Dr Mamta Garg, speaking on behalf of Myeloma, who reinforced the importance of consulting with clinicians at every step of the appraisal process. The panel heard the moving testimony from Michael Jameson, a patient with AL amyloidosis, who demonstrated the importance of daratumumab for AL amyloidosis sufferers.
The NICE appeal panel held on 27 April 2023 agreed with Myeloma that NICE had failed to act fairly on the following grounds:
a) NICE failed to act fairly by not seeking the advice of haematologists at every stage of the process;
b) NICE failed to act fairly by failing to allow the National Amyloidosis Centre to nominate its own clinical expert for committee meetings; and
c) NICE had not acted fairly when applying criteria for determining an acceptable ICER value under the Methods Guide 2013.
The appeal panel made a series of recommendations to the Committee requiring them to review and revise their recommendations in light of the successful appeal points.
Read the full decision.
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