Prepping for yet more pricey rare disease drugs, UK launches £340M 'Innovative Medicines Fund'
The UK is expanding its controversial Cancer Drugs Fund into rare disease.
On Wednesday, the country’s National Health Service announced a new £340 million effort called the Innovative Medicines Fund to cover the cost of new rare disease drugs that don’t yet have enough data to satisfy the government’s pricing watchdog but might still offer some benefit for patients. Like the similar, decade-old cancer effort, regulators would then collect data to “ensure their pricing and long term use represents good value for the NHS overall.”
The new fund comes as the UK, like many countries, grapples with the flurry of rare disease drugs pharma and biotech companies are now bringing to market, and the high price tags they’ve consistently attached to those medicines. The country spent years locked in an acrimonious battle with Vertex on their cystic fibrosis drugs and only in the last few months secured access to Novartis’ SMA gene therapy Zolgensma, often known simply as “the most expensive drug in the world.”
And that’s just in the UK, a country that’s historically been more willing than its brethren across the channel to pay for pricey drugs. Earlier this year, bluebird pulled its gene therapy for a rare blood disorder from Germany after the country refused to pay $900,000, a potential harbinger for how countries — and companies — will handle negotiations over one-and-done gene therapies.
The new £340 million adds to £340 million already allotted to the Cancer Drugs Fund. Although it is technically for all patients, NHS singled out the potential in rare and genetic diseases.
The fund, it said, could cover “cutting-edge gene therapies,” particularly in especially rare diseases where there may be too few patients to collect efficacy data in a timely manner. In addition to Zolgensma, gene therapies have reached or are nearing approval for sickle cell disease, inherited forms of blindness, hemophilia and a couple of ultra-rare immunological and neurological disorders, among others.
The fund “will significantly reduce the time it takes for the most promising new medicines to reach patients,” health secretary Sajid Javid said in a statement, “including children and those with rare diseases, saving lives and giving many people hope for a healthier future.”
The approach answers a long-running criticism that has dogged the Cancer Drugs Fund since it was conceived in 2010 as a way to cover drugs that regulators deemed not cost-effective: Why have a fund for cancer patients, but not for patients with other serious disorders?
Now, Javid said, the UK will be “bringing equal access to the best treatments for all, regardless of the condition.”
Other criticisms, though, still linger. In 2017, amid a torrent of pushback that the fund had grown into a more than £1 billion boondoggle for pharma companies with ineffective or marginally effective drugs, the government restructured the process to more resemble the accelerated approval pathway in the US, where drugs are approved on limited data and then in theory can be approved or pulled after more data on survival or other clinical outcomes emerge.
That pathway, though, remains controversial in the US, particularly after it was used to approve an unproven Alzheimer’s treatment last month. And it has received pushback in the UK, albeit far milder than when the fund was under the original model.