Scotland Unveils £21m Fund for Rare Diseases

A £21 million fund will been made available to pay for rare orphan drugs that are not currently recommended by the Scotland’s regulatory watchdog, the Scottish Medicines Consortium (SMC).

The fund will launch in March, and run until April 2014.  It will help cover successful new Individual Patient Treatment Requests for drugs that target rare diseases that affect one in 2,000 people.

The fund builds on England’s Cancer Drugs Fund, which since 2011 has injected £200 million of further funding into England’s NHS annually to pay for new cancer drugs that have not been recommended by NICE, or are currently under consideration by the regulatory agency.

Alex Neil, Scotland’s Health Secretary commented that it is “only right” that Scottish patients with rare conditions have access to medicines which are clinically justified, and that they are not disadvantaged due to the large cost of the treatments.

“This fund bridges the period to the establishment of next year’s value based pricing for medicines and any changes that are made following the completion of the on-going access to new medicines review,” Neil noted.

However, England’s Cancer Drugs Fund has been divisive, and last year the chief executive of Myeloma UK, Eric Low, claimed that such funding allowed pharma to ‘get away’ with having costly drugs, and questioned whether this should be reiterated across the UK.

In the same way that the Cancer Drugs Fund side-steps NICE, the new Scottish fund will also by-pass the Scottish Medicines Consortium, which could throw up political opposition.

But the chief executive of the Rarer Cancers Foundation, Andrew Wilson, has no such reservations about the funding.  Wilson commented: “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,” he added.

The fund will also please companies like Vertex, who had their genetic treatment for cystic fibrosis, Kalydeco, rejected by the Scottish regulators earlier this week.

Ed Owen, chief executive of the charity Cystic Fibrosis Trust, commented that “We now appeal to Vertex, the manufacturers of Kalydeco and the NHS in Scotland to work together to reach a fair and affordable solution as soon as possible.”

“The Trust will be looking for further clarification about the process going forward, including how soon a consultant can make an individual treatment request so that patients have access to Kalydeco as soon as possible after March 1, 2013,” Owen added.

The ABPI were, unsurprisingly, also positive about the news.  Andrew Powrie-Smith, ABPI’s Scotland director, noted that “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 are 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,” he concluded.


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