Scotland Creates Fund for Rare Diseases

A new $33 million (£21 million) fund has been made available to pay for rare orphan drugs that are not recommended by the Scotland’s drug pricing watchdog.

The fund will begin from March this year through to April 2014 and will help cover successful new individual patient treatment requests for drugs that target rare diseases that affect one in 2,000 people.

It builds on England’s Cancer Drugs Fund, which since 2011 has injected £200 million of additional funding into England’s National Health Service (NHS) each year to fund new cancer drugs not recommended by National Institute for Health and Clinical Excellence (NICE) or are under consideration by the watchdog.

Scottish Health Secretary Alex Neil said it was “only right” that Scottish patients with rare conditions had access to medicines which were clinically justified, and that they were not disadvantaged due to the very high cost of these treatments.

He added, “I am therefore pleased to confirm today that the Scottish government will establish a fund which will ensure that the cost of successful new individual patient treatment requests for orphan medicines are met.”

But this Fund has been controversial, and last year Eric Low, Chief Executive of Myeloma UK, said that he believed such funding allowed pharmaceuticals to “get away” with having highly priced drugs, and questioned whether this should be repeated across the UK.

And just as the Cancer Drugs Fund bypasses NICE, so too will this new fund bypass the Scottish Medicines Consortium, which could throw up political objections.

But the Chief Executive of the Rarer Cancers Foundation (RCF), Andrew Wilson, has no such qualms about this type of funding. He said, “We welcome the creation of a new fund for those treatments not routinely funded in Scotland, and we call on doctors to make full use of it to ensure that people with rarer cancers get the treatments they need.

“The RCF has campaigned for the existing system to be reformed to prevent thousands of Scottish patients being denied life-extending treatment in cancer and forced to go through inconsistent, bureaucratic and upsetting processes.”

The ABPI was, naturally, also positive about the news. Andrew Powrie-Smith, ABPI’s Scotland director, said, “This new fund is good news for access to medicines in Scotland, and the ABPI welcomes it. As with some disease areas, for example neurological diseases, there are particular challenges around rare and orphan medicines which make it difficult to fit them into the system for assessing more mainstream medicines, and we are pleased that the Scottish government is addressing them.

“We look forward to further announcements coming from the Scottish government’s new medicines review around how the criteria for individual patient treatment requests will be altered so that appropriate patients are able to access innovative medicines through the new fund.”

Reposted from PharmaTimes. Written by Ben Adams.