Tue 01 Aug 2023

Good news for people living with amyloidosis

I recently had the enormous privilege of helping Myeloma UK with their invaluable work. The client is the only charity in the UK dealing exclusively with the incurable blood cancer myeloma and related conditions.

Our client got in touch at the end of 2022 asking for help with appealing against a recommendation made by the National Institute of Health and Care Excellence (NICE). The recommendation was that daratumumab (aka Darzalex) should not be used in combination with other drugs by the NHS to combat a form of amyloidosis (namely untreated systemic amyloid light-chain amyloidosis also known as AL amyloidosis). This condition which affects around 500 people every year is currently incurable and can lead to organ failure.

My role involved advising the client on preparing the potential grounds of appeal they had identified. Whilst the science was complex (at least for someone like me whose relevant qualifications extends no further than a couple of Highers), the clients did a great job explaining this. I, in turn, was able to advise the client about appellate principles and how they applied to the matter before us. Together we were able to draft an appeal which sought to argue that: (i) the assessment preceding the recommendation involved failures to act fairly in terms of reg. 9(3)(a)(i) of the relevant regulations; and (ii) the recommendation was unreasonable in light of the evidence before the decision-maker in terms of reg. 9(3)(b). Janssen Pharmaceuticals also appealed against the decision.

The Appeal Panel's decision was issued on 28 June. Happily, it granted the appeal, accepting a number of the client's arguments. In an attempt to simplify, without trivialising, the Panel decided (i) the original decision was not sufficiently clear about the weight attached to the rarity of the condition when determining cost-effectiveness of the treatment and so this should be reconsidered alongside the other factors in NICE's 2013 Guide to the methods of technology appraisal; (ii) advice from a specialist haematologist, or specialists at the National Amyloidosis Centre, ought to be obtained to help determine the effect of daratumumab on overall survival; and (iii) clinical data about survival rates used for the purpose of economic modelling should be reconsidered and how this data is used clarified. The matter was remitted to the Appraisal Committee for re-appraisal.

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