A lack of dedicated orphan drug fund in the UK is cause for concern, according to the All Party Parliamentary Group (APPG) for Muscular Dystrophy.
While Scotland has a fund in place for orphan drugs, the special rare disease budget in England has been merged into the overall NHS budget. This means that orphan drugs may have to compete for funding against drugs for much more common conditions, and decisions therefore based on inappropriate cost-benefit analyses.
The APPG for Muscular Dystrophy have called for the UK Government to establish a ring-fenced fund for rare disease drugs, and for the National Institute for Health and Care Excellence (NICE) to assess treatments for rare conditions differently from less rare conditions.
The UK’s Department of Health has said that it working towards developing a UK strategy for rare diseases. “NHS patients with rare diseases already receive some of the best levels of care in the world, but we are committed to improving this further. That is why we are working closely with other health departments and stakeholders to develop a UK strategy for rare diseases. This will be published by the end of the year,” said a Department of Health spokesperson.
The APPG also called for the Medicines and Healthcare Products Regulatory Agency (MHRA), NICE and NHS England to speed up access to life-changing drugs following clinical trials.
“We have seen that successfully developing an effective treatment is far from the end, with agonising waits for some families through licensing and funding issues,” said Labour MP Dave Anderson, who led the inquiry. “We must focus on ensuring that if treatments are proven to be safe and effective, the UK is in a position to license and deliver them swiftly. We urge regulators and commissioners to overcome these obstacles and find a feasible, sustainable way of delivering pioneering rare disease treatments. Otherwise the effective treatments may remain entirely out of patients’ reach.”
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