Aducanamab: keeping hope informed

A new drug to treat dementia, called aducanumab, has been licensed for use in the USA, but should it be approved in this country too? Toby Williamson warns against placing too much faith in “Big Pharma” and questions the relationship between the business world and dementia care

Reports of a new drug that research indicates is successful for treating dementia always attracts a lot of public interest. This has certainly been the case with the recent announcement from the United States that a drug called aducanumab has been approved for medical use by the Food and Drug Administration (FDA) to treat people with early stage Alzheimer’s disease.

The FDA is the equivalent of the UK’s Medicine and Healthcare products Regulatory Authority (MHRA) and the European Medicines Agency (EMA), which decide if medicines work and are safe. Aducanumab has not yet been approved for use in this country, but the MHRA is considering whether to license it for sale here too.

The announcement has generally been welcomed in the UK. Alzheimer’s Society describes it as a “promising step in the quest to find dementia treatments” and Alzheimer’s Research UK (ARUK) talks about it in terms of “reason for hope”. The Society does point out that there are still grounds for caution because the drug has only been tested with people in the early stages of Alzheimer’s disease and more research may be required to provide additional evidence to show the benefits for this group of people. However, ARUK has written to the health secretary asking for the process of assessing the drug for approval in the UK to be accelerated.

Most people would agree that the story of aducanumab sounds like good news. But there are justifiable grounds for being more sceptical or even concerned about it. Pharmaceutical companies are very large commercial enterprises – “Big Pharma” – and they have to invest millions of pounds in researching new drugs. Naturally, they want the drugs to be safe and effective for the benefits that they will bring, and it’s partly thanks to this that effective vaccines for Covid were so quickly developed.

But they also want drugs to work because it enables them to recoup the costs of the research and for their future profits if a drug is licensed for use. And this is one of the concerns about aducanumab. Trials of the drug initially showed it to be ineffective and research into the drug was halted in 2019. But later that year the manufacturer Biogen said it had reanalysed the data from the original trials and subsequently found aducanumab to work when given in higher doses, though this only occurred in one of the two trials. A larger dose, of course, means that Biogen makes more money from selling the drug.

One might be less sceptical if Biogen had announced that aducanumab was effective given in smaller doses. Treatment using the drug costs around $56,000 a year in the US and Biogen says it will maintain this price for four years. In 2020, a panel of independent experts for the FDA concluded that there was a lack of good evidence that aducanumab was effective, there were potential safety issues associated with the drug, and the reanalysis of the research data that Biogen carried out contained a number of flaws.

A number of FDA members who reviewed the drug did not vote for its approval for use. When the FDA announced it was giving aducanumab the go-ahead, three members resigned, and a senator called for the organisation’s director to be replaced. This aspect of the aducanumab story has not been so widely reported in the UK.

An elusive quest

Many people affected by dementia are desperate that effective treatments for the diseases that cause the condition are found, and this search is a holy grail for Big Pharma and their shareholders. But as with a number of other long-term health conditions and disabilities, to date, this quest has proved elusive. As a comparison, in the case of schizophrenia the side effects of some drugs have proved far more harmful than the treatment benefits they are supposed to bring.

While total pessimism is unlikely to be a helpful response, the hope for a drug like aducanumab must be tempered by reality and the controversial history of its development. And even if it does prove effective, only a minority of the 900,000-plus people currently living with dementia will benefit by the time it is approved here.

The aducanumab story also reminds us of the imbalance in dementia research. Despite the costs of developing new drugs, the actual process is relatively simple compared to doing research into psychosocial interventions for dementia involving psychology, social care, meaningful activities, end of life care and other interventions. Whether they are aimed at reducing symptoms, alleviating distress, responding therapeutically to behaviour that challenges, or improving quality of life and wellbeing, all these interventions involve human interactions which can often be harder to measure and research, but they are potentially just as important as research into new drugs. Yet most funding for investigating new ways of treating people with dementia still goes into drugs research.

Furthermore, for many people affected by dementia, life can be a daily struggle to get the support they need from services which are often under-resourced, inappropriate to meet their needs, not person-centred, or simply not available. In 2020 Biogen reported full year revenues of $13.4 billion (£9.7 billion) with an estimated profit margin of nearly 30%.

Compare that with UK government spending on dementia: around £100 million on research and £14.6 billion on health and social care.

Rather than having expectations raised too high, perhaps what we should really be hoping for are more questions to be asked about the way pharmaceutical companies operate, and the relationship we want between big business and supporting people affected by dementia.

Toby Williamson is an independent health and social care consultant