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Ayrshire & Arran NHS Board and Greater Glasgow & Clyde NHS Board have reviewed Individual Patient Treatment Requests since the publication of CEL 17 and have rejected them, although treatment was recommended by a UK specialist. The UK Guidelines have been written by specialists and the patients rejected by their NHS Boards in Scotland do fit within these Guidelines.


An appeal has been made to the Medicines Appeal Panel of Ayrshire & Arran NHS Board which has also been rejected on the grounds of cost efficacy, despite representation by a UK specialist.

A patient has written two letters to John Scott, her MSP, at the suggestion of Jackie Baillie, MSP.

This Parliamentary Question has been asked -
S3W-40287   -   Jackie Baillie:
To ask the Scottish Executive what review it has undertaken on Recommendation 11 of the Mackie Report: Access to specialist neuromuscular care and social care in Scotland by the Cross Party Group on Muscular Dystrophy.
Nicola Sturgeon (Wednesday, March 16, 2011):
“The enzyme replacement therapy for Pompe disease, Myozyme, has been appraised by the Scottish Medicines Consortium, but was not recommended for routine use within NHS Scotland. In such circumstances, NHS boards have arrangements in place to consider treatment requests from individual patients. Such decisions are made on a case by case basis, exercising professional judgement on clinical need.”

The Cross Party Group on Muscular Dystrophy in the last Scottish Parliament recommended that “The Scottish Government reviews the situation regarding the unequal treatment of the small number of patients with Pompe disease living in Scotland. While some patients are currently receiving enzyme replacement therapy, others are being refused this treatment. In England all patients are able to access this treatment.” This means that approximately 90 adult patients residing in England receive this treatment.


Robbie Warner, Chair of the Muscular Dystrophy Campaign’s Scottish Council is quoted in the Mackie report hoping for service improvements:

“I would want advice available for people living with muscular dystrophy, and I would want the planning to be done and for people to acknowledge it. I would want the whole system streamlined in such a way that you don’t have to fight to get everything, because if you get it at the end of the fight, you wonder whether all that energy was wasted in terms of them resisting it. I would like to see a change of attitude that would say that you need it and you plan for it, and get into a working relationship.”

First Minister Alex Salmond has previously intervened to support a case for one of his constituents to receive treatment for Pompe disease.


From this same article, published in The Herald on 25 March 2011:

• Allan Muir, development director of the Association for Glycogen Storage Diseases – which supports families affected, said: “Access to therapy for Pompe disease patients in Scotland currently depends on which health board a patient belongs to, and the vast majority of patients are being denied treatment. This is hugely frustrating for the families affected, especially given that patients with other related rare diseases are being treated with similar medications.”
• The Muscular Dystrophy Campaign is supporting the drive by Scotland’s sufferers to obtain access to the drug. Director of care for the charity Nic Bungay said: “We know of only 11 Scottish Pompe disease patients, yet the vast majority of them are being denied access to a ground-breaking treatment automatically available to people living in England, on the grounds that the therapy is not cost-effective.”
• A spokesman for the Scottish Medicines Consortium, which approves new drugs for use by the NHS, said: “We evaluated Myozyme in 2007 and were disappointed to find that it wasn’t a value for money medicine. This decision was based on analysis of the manufacturer’s own information. All NHS boards have mechanisms in place in order to consider drugs that are not deemed to be cost-effective.”

The Daily Record have also publicised the plight of a patient:


An article was published in the Scotsman on 1 May 2011:

“Scottish Labour's health spokeswoman Jackie Baillie said: "It is vital that those suffering from rare conditions get access to the medicines they require and we must ensure that patients in one part of Scotland are not disadvantaged compared with patients living in England and Wales."  An SNP spokesperson said: "Everyone recognises the importance of decisions on medication and treatment being made by health professionals and not politicians. When consultants recommend access to medicines we expect health boards to respond flexibly and favourably to requests."


In addition: “First Minister Alex Salmond has met the anti-cuts protesters who ambushed Labour leader Iain Gray and Scottish Conservatives leader Annabel Goldie…. issues include the reversal of a decision to move a Glasgow day-care service and the potential funding of treatment for people with Pompe disease, a genetic muscle disorder.” Following the meeting, Mr Salmond said: "Pompe disease has to be dealt with sensitively but there might be a way forward there as well."


Finally, “Scottish patients have demanded Health Secretary Nicola Sturgeon end the “postcode lottery” in drug provision… However a Scottish Government spokeswoman said: “Health boards have arrangements in place to provide this drug for individual patients in certain circumstances. This is a clinical decision based on a robust national framework – not a postcode lottery.”


But the latest Chief Executive’s letter (CEL) from the CMO states that:

“The responsibility for an application for an IPTR rests with the clinician who supports prescribing the requested medicine. It is the clinician who is expected to demonstrate the clinical case for the patient to be prescribed a medicine within its licensed indication(s) where the following criteria apply:

The patient’s clinical circumstances (condition and characteristics) are significantly different from either:

(i) the general population of patients covered by the medicine’s licence; or
(ii) the population of patients included in the clinical trials for the medicine’s licensed indication as appraised.

These circumstances imply that the patient is likely to gain significantly more benefit from the medicine than would normally be expected. Such considerations should be taken on a “case by case” basis reflecting clinical opinion and, as such, should not be generalised.”


Due to the fact that there are so few patients suffering from Pompe disease, these criteria will not be met.

Therefore, although applications have been made to access therapy via IPTRs as submitted by UK specialists, the applications have been rejected – not because clinical need has not been justified – but because the patient’s clinical circumstances cannot be significantly different from the general or trial population of Pompe patients.

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