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Brexit leaves rare disease patients in the lurch

Thursday, 19 April, 2018 - 14:19

Of all of the areas likely to be adversely affected by Brexit, medicine and healthcare remain among the most precarious. Although all patients are liable to feel the effects of the UK’s departure from the single market, none will be harder hit than those suffering from rare diseases.

The fragility of their position has been highlighted by the case of Dr Nick Sireau, who quit his job in 2010 to work full-time on finding a cure for alkaptonuria, the debilitating bone disease which afflicts his two sons. With Brexit on the horizon, Dr Sireau now fears that the fallout from the separation might impede his sons’ ability to access the treatment he has been working so hard to discover and which could significantly enhance their quality of life.

Brexit threatens promising drug

Alkaptonuria, also known as AKU, is an incredibly rare degenerative disease which corrodes the bones and cartilage of the human body, turning them black and compromising joint functionality. In the UK, AKU affects an estimated 88 people. In his search for the miracle cure that would save his children’s lives, Dr Sireau co-founded the AKU Society. After campaigning tirelessly for a decade – including giving up his job to devote himself to the cause full-time – he and his colleagues successfully secured €6 million from the European Commission in 2012 to conduct clinical trials testing the drug’s efficacy.

The trials are now in the fifth year of a six-year study and the results thus far have been promising. If the final outcome is as positive as is predicted, the AKU Society intends to apply to the European Medicines Agency for its approval. However, Dr Sireau now fears that the UK’s impending separation from the EU might mean that it will no longer fall under the EMA’s jurisdiction, which would result in the AKU Society having to secure a separate license for the drug in Britain – thus slowing down the process significantly.

Par for the course for NHS

Unfortunately, the Sireau quandary is not an isolated incident when it comes to the NHS. The body’s National Institute for Health and Care Excellence (NICE) has something of a track record regarding the review of breakthrough medicines, as has most recently been observed with the wonder drug Spinraza. Approved by the US Food and Drug Administration (FDA) in late 2016 and by the EMA the following June, Spinraza is currently the only known treatment for spinal muscular atrophy (SMA), a debilitating and often fatal condition which affects around 1,300 Britons.

Despite the remarkable potential shown by the drug, NICE repeatedly delayed its appraisal of Spinraza until the beginning of this year. North of the border, the Scottish Medicines Consortium (SMC) met earlier this month to discuss the future of the drug with regards to NHS Scotland. Specialist charity Muscular Dystrophy UK has urged the SMC to approve it as soon as possible in order to give a lifeline to those who need it most, though a decision isn’t due to be published until May 7th – nearly a year after the EMA issued its approval, and too late for some patients whose conditions have continued degenerating in the meantime.

Bigger storm clouds on the horizon

What’s worse, this laboriously slow process is likely to be endangered even further by the upheaval caused by Brexit. The NHS itself is sure to be hit by any tightening of immigration laws, with a 96% decrease in European nurses registering for UK practices already apparent since the referendum in 2015. Since the NHS relies on EU nationals for 10% (or 150,000 professionals) of its workforce, the split could be potentially disastrous. When it comes to rare diseases, however, dwindling personnel numbers are just the tip of the iceberg.

For starters, the uncertainty surrounding UK borders could pose problems for the 82 million patient packs which travel back and forth between Britain and the EU each week. Unless a robust replacement framework is devised and a seamless transition implemented, patients could face lengthy delays in receiving vital medicines. As mentioned above, those delays could be lengthened further by the need to obtain separate licensing permits caused by the potential division in regulatory legislation. All of this adds up to an even longer waiting list than the one NHS patients currently face.

Collaboration beneficial for all concerned

As if that wasn’t bad enough, the very research efforts which discover these new treatments could themselves become threatened. Functioning apart from the EMA, the UK’s Medicines and Healthcare Products Regulatory Association (MHRA) might find it difficult to continue to participate in continent-wide studies – the Brexit Health Alliance (BHA) has already warned that 750 UK-led studies could be jeopardised. By their very nature, rare diseases require multinational cooperation; without sufficient subject numbers to provide meaningful data, the UK could become deprived not only of the benefits of participation, but the possibility of even conducting its own trials.

Of course, this sword will cut both ways. The NHS is involved in 23 of 24 European Reference Networks (ERNs) and leads six of them. At present, the MHRA leads 35% of safety monitoring schemes for EMA medicines and 40% of highly complex therapy evaluation. The expertise and ability of the UK medicinal and healthcare sector should not be underestimated, and both Britain and the bloc will lose out if the pair can’t hammer out a workable deal.

Rare diseases most at risk

The biggest losers, of course, will be the patients themselves – and none more so than those suffering from rare diseases. The case of Dr Sireau and his children has highlighted the very real danger that Brexit will only serve to make a bad system even worse, with the BHA flagging the particular dangers faced by cancer patients and rare disease sufferers in this unpalatable scenario. With those issues in mind, it’s vital that lawmakers work expediently to ensure that the UK doesn’t throw out the supreme medical benefits offered by the EU along with the bathwater.


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