Tough UK line on rare-disease drugs stirs concern after Brexit

Bloomberg

A tougher stance in the UK on funding expensive medicines for rare diseases is raising questions about the government’s pledges to support a life-sciences industry that’s already rattled by Brexit.
A proposal to introduce a cap on coverage of treatments within the state-run health system would have a significant impact on access to ultra-rare disease drugs, said Heidi Wagner, senior vice president of global government affairs at Alexion Pharmaceuticals Inc. Last week, the US biotech company was denied support in England for Kanuma, a drug that’s estimated to cost 492,000 pounds ($612,000) a year, because a key authority deemed it too expensive even after unspecified discounts.
“Perhaps England is not going to be as supportive of innovation as it was in the past,” she said. There are a number of signals “that would really call that into question.”
Adding a funding threshold for new therapies that tackle very rare diseases, part of broader measures to keep a lid on costs, could make it harder for makers of those drugs to recoup the costs of developing treatments used by few patients globally. UK Prime Minister Theresa May’s government last month detailed plans to encourage innovation as she sought to alleviate concerns that the UK’s exit from the European Union may curtail investment in science and technology, and erode the nation’s attractiveness for global talent.

DEADLY DISEASE
The country’s National Health Service has said it’s committed to providing timely access to new drugs and must balance helping companies with ensuring its financial sustainability.
With Alexion’s Kanuma, costs were too high even with the offered discount, according to the National Institute for Health and Care Excellence, which advises the NHS on coverage of new drugs. The drug treats a rare and deadly disease called lysosomal acid lipase deficiency, which mostly afflicts children. Babies diagnosed with an aggressive form of the disease have a median life expectancy of 3.7 months.
Alexion is still seeking to reach an agreement with the health system after the “sizable” discount it proposed was rejected, Wagner said. The company said last week that UK health authorities were disregarding expert medical opinion. Earlier this month, the firm suffered another blow from a separate UK judgment that’s likely to restrict access of its drug Strensiq to some patients.