Rare disease drugs ‘need separate budget’

pharmafile | September 3, 2013 | News story | Research and Development, Sales and Marketing |  NHS, NICE, rare diseases 

A group of MPs in the UK are calling for a ring-fenced fund to pay for drugs for rare and ultra-rare conditions.

The MPs from the Parliamentary Group for Muscular Dystrophy, said this week that they are concerned about funding for high-cost drugs.

These fears have been prompted after Advisory Group for National Specialised Services (AGNSS) body which assessed low volume, high cost ‘ultra-orphan’ drugs, was abolished in April.

Its £100 million budget and remit has now been taken up by the NHS England, and there are now plans to have drug pricing watchdog NICE to take over the cost assessment of these medicines.

The MPs say they are concerned that these funds previously ear-marked for drugs for rare diseases have now been merged, meaning that the 3.5 million people in the UK with a rare disease – with most of these children – may miss out on new treatments.

And many in the rare disease community have qualms about NICE taking over this role as it is known for rejecting high-cost medicines.

Typically rare and ultra-rare drugs can only cater for hundreds and sometimes even tens of people, meaning their cost can be top £300,000 per patient given the low volume and high cost of research and development.

Rare Disease UK has expressed concern that NICE’s QALY will not be adequate to fully judge the merits of these types of medicines. The Institute has said previously that it may change the look of QALY when assessing these drugs, with new plans set to be introduced by next year.

The MPs warn that using NICE’s QALY formula as it is, could lead to hundreds of children with life-shortening conditions being denied rapid access to new therapies.

They want a drugs fund for rare diseases, similar to the one currently set up in Scotland, and a rapid system for regulators to review cutting-edge drugs.

The chairman of the all-party group, Dave Anderson Labour MP, said: “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.”

A UK-wide strategy for rare diseases will be published later this year. A Department of Health spokesman said: “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.”

Ben Adams