Rare Diseases Strategy

Stuart Blair Donaldson Excerpts

Tuesday 28th March 2017 Westminster Hall

[Martyn Day (Linlithgow and East Falkirk) (SNP)]

It is a pleasure to serve under your chairmanship today, Mr Pritchard, and I am grateful to the hon. Member for Bath (Ben Howlett) for securing the debate, for his informative speech and indeed for the work that he has undertaken with the all-party group.

As we have heard, rare disease affects considerably more people than we would at first imagine, with over 3 million people across the UK likely to suffer from a rare disease at some point in their lives. The Scottish Government’s implementation plan for rare diseases in Scotland recognises this fact in its title: “It’s Not Rare to Have a Rare Disease”.

My own constituency has had, as I am sure every other constituency has had, several fairly high-profile rare disease cases. These cases attract media attention and affect entire communities, not just the family directly affected; often, communities have to raise funds to help with treatments and raise awareness. Such a case was that of Kirsty Reid from Whitburn. In 2015, after being diagnosed with pseudomyxoma peritonei—it is one of those conditions where the acronym, PMP, is slightly easier to say—Kirsty raised over £7,000 to help others diagnosed with the rare condition, which affects only two people per million.

We also have a charity called Shavon’s Journey, which was set up in 2012 following the death of Shavon Morton from Grangemouth after a long fight with aplastic anaemia, a condition in which bone marrow does not produce sufficient new cells to replenish blood cells. I could go on with other examples, but I think we all get the point— rare diseases touch the lives of many, and therefore the importance of raising awareness, and of improving diagnosis and ultimately the services and treatments to sufferers, cannot be overstated.

[Stuart Blair Donaldson (West Aberdeenshire and Kincardine) (SNP)]

I thank my hon. Friend for giving way and I congratulate the hon. Member for Bath (Ben Howlett) on securing this debate. The Teddington Trust, which is jointly run by one of my constituents, supports people living with xeroderma pigmentosum, who lack the DNA repair mechanism necessary to repair damage caused to the skin by exposure to ultraviolet sunlight. Does my hon. Friend join me in commending the work of the Teddington Trust and the work of many other rare disease charities in supporting those living with rare diseases and keeping rare diseases on the political agenda?

[Martyn Day]

I do indeed join my hon. Friend in commending the work of so many good charities and organisations in this field.

The Scottish Government’s implementation plan, which I have mentioned, sets out the Scottish approach to delivering the UK strategy commitments. Key to the plan is recognising the importance of timely and accurate diagnosis, and allowing treatments to start as soon as possible. The Scottish Government have substantially increased access to new medicines, particularly for cancer, due to reforms and investment in recent years, which has led to a marked increase in uptake of orphan, ultra-orphan and end-of-life medicines.

Last year, the Scottish Government launched a review of the way drugs are assessed for NHS use, which was led by the former NHS Fife medical director, Dr Brian Montgomery. His review looked at how changes made to the Scottish Medicines Consortium process in 2014 had affected access to medicines for rare and end-of-life conditions. The review’s recommendations set out how the process for appraising medicines can be made more open, transparent and robust, and the Scottish Government have committed to implement all 28 of its recommendations, such as a new approval pathway, outwith the standard SMC process, for these high-cost medicines for very rare conditions.

The Scottish Government’s peer-approved clinical system, or PACS, has helped to give patients and clinicians a better say in which new medicines are approved by the SMC for use in NHS Scotland, particularly those used for rare or life-limiting conditions. PACS was first piloted in Glasgow in 2015 and has been successfully rolled out across Scotland, and a second tier of PACS will now be introduced to replace and build on the existing individual patient treatment request system. A new national appeals process will be introduced through this new tier of PACS, which will include consideration of equity of access with other parts of the UK as a material part of the decision-making process.

It is also perhaps worth saying at this point that the Scottish Government are happy to work with the National Institute for Health and Care Excellence, and indeed with any other countries, to improve access to medicines in Scotland and to obtain a fair price from the pharmaceutical industry. I emphasise that last point, because we now need the pharmaceutical companies to do their bit by bringing forward fairer prices for new medicines, so that access can be as wide as possible.

Finally, we know that the Health Secretary has said that he does not expect the UK to remain within the European Medicines Agency, which raises a number of concerns about potential delays in new drugs reaching patients in the UK. Sir Alasdair Breckenridge, who was the chairman of the UK’s drug regulator—the Medicines and Healthcare Products Regulatory Agency—for almost a decade, said last month:

“The UK market compared to the European market of course is small and they may decide not to come to the United Kingdom. So therefore there will be delay in getting new drugs—important new drugs, anti-cancer drugs, anti-infective drugs—for patients in the UK.”

He is not alone in saying that; David Jefferys, vice-president of Japanese drugs firm Eisai, has also warned that UK patients could face delays of up to two years. I would be grateful if the Minister could advise in his response to the debate how these dire warnings can be prevented from becoming a reality.