General Updates | The impact of the withdrawals of crizanlizumab and voxelotor on patients with SCD in the UK

Well, first of all, the withdrawal of crizanlizumab and voxelotor was a shock to the sickle cell system in this country, and that is very, very clear. So in the crizanlizumab case, it was for reasons unconnected to safety, but more efficacy, how effective the drug was. And it was the regulators who did the withdrawal. In the Oxbryta voxelotor case, it was the company, because of deaths in sub-Saharan Africa, that withdrew it. Voluntarily, they withdrew it. And that was impactful. 

Now, you have to understand why it’s very impactful and negative. And the reason for that, and I must stress this, the reason for that is that decades and decades and decades there’s been one standard treatment for sickle cell, hydroxycarbamide, which was actually never invented for sickle cell, but is effective, safe, and makes a difference. That’s what’s been available. So in the spirit of hope, when these new treatments come along, people were saying, oh, this will make a substantial difference. What has happened is we’ve gone backwards. We haven’t got forwards. We haven’t gone forwards at all. We’ve gone backwards. We’ve produced two new treatments, which are no longer available. And certainly, not for everybody, but for some patients on those treatments, they were doing well. I can’t say everybody was doing well, but for some, they were doing well. So that’s a shock, a disappointment, and actually one that goes to the heart of trust. Think about the LISTEN survey for a moment and the findings from the LISTEN survey about people participating in clinical trials. What is the incentive, the motivation for people to participate in clinical trials when new treatments that come out are withdrawn for different reasons. So you have to make those connections. You have to make those connections. So I think that it is very much a huge disappointment to the sickle cell community. But I must also say that there will be a degree of scepticism about new drugs coming on the market. You know, so it’s just extraordinary, and I think that that would be our position on the withdrawal and the impact of crizanlizumab. 

On early access and early access to new novel treatments, there are two things I would say. First of all, it’s pointless having new treatments that are not going to be effective. Why? We’ve just had two that have been withdrawn, and so if the endpoints are the same, it would suggest to me that the complexities of this condition, in terms of drug development, are not yet fully understood. So that’s the first thing that I would say. 

The second thing that I would say, of course, it will help to address some health inequalities. But for sickle cell, new novel treatments is not the only tool to address health inequalities. There are health inequalities that go across the system, whether that’s accident and emergency, whether it’s about education of healthcare professionals when people are on medical law. So there’s a range of other health inequalities, and those health inequalities go back to what I said about the Sickle Cell Improvement Programme and the cliff edge. So there’s connections with these things. So novel treatments, yes, as long as they’re safe and effective, in contrast to what we’ve seen before, and some way of sort of building back trust with organisations like ourselves and the community and healthcare professionals, because it’s not only patients who were disappointed about the withdrawals, it was healthcare professionals as well. So I think the connection about the broader range of health inequalities is linked back to the earlier point about sickle cell improvement. It’s pointless. Remember what I said? It’s pointless starting an improvement program that is funded non-recurrently and then not being able to sustain that going forward. That alone will increase health inequalities, not decrease them.

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