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Speed up access to life-changing drugs

As the rest of Europe competes to rehome the European Medicines Agency (EMA) from London, minds at home are finally focusing on what that means for access to new treatments in a post-Brexit Britain.

The warnings have been clear: without an effective relationship with the EMA, there is a risk that Britain will fall to the back of the queue when it comes to accessing new drugs.

And there are a lot of new treatments that could prolong and change lives, particularly for rare conditions, that are close to coming to market.

Thrashing out a post-Brexit drug access plan is vital. But regardless of what happens with Europe, the government is failing to implement its own report recommendations that will speed up access and prevent the delays that have stopped treatments from reaching the people who need them.

The Accelerated Access Review[1] was commissioned by the government to help avoid delays caused by the typical drug approval process, which can take years. The UK has its work cut out with speeding up drug access. Translarna, the first drug to treat some children with the life-limiting Duchenne muscular dystrophy, took three years to be made available to patients in the UK, long after it was available in other European countries. Duchenne muscular dystrophy and other muscle-wasting conditions do not allow such delays without a risk of serious repercussions for people’s health and mobility. We know people are ready to head abroad to avoid delays in accessing treatments if the life of their child is at risk.

The government says it is committed to the report’s findings — first published last year — a commitment repeated in the general election manifesto of the Conservative Party, but we are yet to even see a published response to it. We do not want this to become the ’Delayed Access Review’.

Key among the report’s recommendations is the need for a commercial unit at NHS England that can take on experienced negotiating teams of drug companies to get the taxpayer a good deal. Translarna was eventually provided to the eligible boys after the NHS and the National Institute for Health and Care Excellence (NICE) struck a special deal with the drug company to provide the drug for the next five years, while considering whether to fund it in the long term based on clinical effectiveness. But could the delivery have been sped up — or delivered at a lower price — with a commercial unit working to make these tough conversations happen sooner rather than later?

The new commercial unit is not the only valuable recommendation in the review. Raxone, a drug that could help older boys with Duchenne muscular dystrophy to breathe well for longer, was approved for use earlier this year through the Early Access to Medicines Scheme (EAMS) after vigorous lobbying by Muscular Dystrophy UK. The scheme lets people access the drug while regulators and NICE complete their assessments, but many drug companies are not using this route, which can be costly. The review recommended up to £30m to help companies apply and get treatments to people faster. There are just three drugs currently being considered for EAMS; that number could rise rapidly with support, and this would be welcomed by many patients with life-limiting conditions who are desperate for faster access.

The recent ministerial acknowledgement to reassure patients and industry on the post-Brexit plans for drug approval is positive but, in truth, this is about much more than just Brexit. The Accelerated Access Review has already set out the agenda for change; the government now needs to get on with putting it into action to ensure that people who need life-saving drugs are not at a disadvantage.

Robert Meadowcroft

Chief executive

Muscular Dystrophy UK

Citation: Clinical Pharmacist DOI: 10.1211/CP.2017.20203122

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