THE head of one of the largest health boards in Europe has admitted Scottish Government criteria in the provision of certain life-saving and life-changing drugs has created a north-south divide in access to medication.
Robert Calderwood, chief executive of NHS Greater Glasgow and Clyde, made the admission in a report which says the health board was only following Government guidance in refusing pensioner Joyce Juszczak the "miracle" drug, eculizumab, for a life-threatening blood condition known as paroxysmal nocturnal haemoglobinuria (PNH), a rare disease in which red blood cells break down earlier than normal, before having to make a U-turn when she suffered a potentially fatal blood clot.
Patient groups say Calderwood's statement supports their demands for a rethink on how patients can get access to hundreds of effective drugs, particularly for rare diseases, that have not been recommended for use in NHS Scotland by the Scottish Medicines Consortium because of their cost.
The Scottish Government insists clinical decisions are "based on a robust national framework – not a postcode lottery".
Clinicians making an "exceptionality" case have to demonstrate that the patient is significantly different from the general population of patients with the condition in question and that they are likely to gain significantly more benefit than might normally be expected.
This criteria has provided a headache for NHS boards. One NHS Borders policy document the Sunday Herald has seen admits exceptionality is "difficult to define" and that "pragmatism and flexibility are necessary".
Responding to a formal complaint made by Mrs Juszczak's family about the constant refusal to provide the drug and the unclear reasons given, Calderwood said she was treated in a "fair and consistent way" and that the health board was merely carrying out "national advice" on how to manage individual patient treatment requests and "exceptionality" cases.
But he admitted: "There is variance in access between England and Scotland in accessing eculizumab. In England, the only criterion to be met before a specialist can prescribe it is that the patient must have received a minimum of four blood transfusions within one year.
"The absence of any specific threshold for NHS Scotland, therefore, creates cross-border variations in prescribing practice. This may be difficult for Scottish patients to understand and accept.''
While Calderwood's comments referred to eculizumab, experts and health board sources say it covers all drugs not recommended for use in the NHS.
In his report, which carefully refrains from being overtly critical, he said "exceptional" case processes would "remain unchanged, unless the Scottish Government reviews the definition".
But he added: "These criteria are intended to be generic, in an attempt to bring equity of response to a wide range of clinical situations and, therefore, lack definition in relation to eculizumab or any other medicine.
"The challenge for prescribers and NHS boards is that each case is unique and judgment is required against the predetermined criteria for a wide range of medicines. There is no explicit 'threshold' set by the SG for approval of eculizumab."
Patient groups, which have used Mrs Juszczak's case to highlight how Scotland is failing more than 350,000 people affected by a rare disease, want lessons to be learned from the problems she faced, and are campaigning to ensure other patients are not similarly affected.
Rare Disease UK says medication is failing to reach patients because it is considered too expensive for use in Scotland – even though the same drug is accepted in England and Wales. An analysis shows that, of 59 rare disease medicines appraised by the SMC, nearly half were not recommended and just 15% were accepted, with 42% allowed for restricted use.
The Rare Cancers Foundation says more than 20 cancer medicines are not routinely available in Scotland, but are funded by the Cancer Drugs Fund in England.
Professor Peter Hillmen, a British expert on PNH and chief investigator on the clinical trials of eculizumab, said Calderwood's admission trivialised the situation and did not recognise that it is time for the Scottish health system to develop a system of prescribing drugs which takes into account the expense of some medicines.
"In England we have a policy on the management of rare diseases which recognises that patients with rare diseases are often left without adequate care and that they need particular attention to support expert services and treatments. It is accepted that their care will be more expensive and this should be incorporated in the commissioning process," he said."The Scottish Government needs to consider a plan for commissioning services for rare diseases that are not being generally supported by the current system.
"In particular, the decisions on the treatment of patients with PNH across Scotland are inconsistent, leaving some at considerable risk – just look at the cases of William Devine and Joyce. [Mr Devine, of Newton Mearns, who had PNH, died after being refused eculizumab.]
A Scottish Government spokesperson said: "Appraisal of the clinical and cost-effectiveness of all newly licensed medicines is carried out by the Scottish Medicines Consortium (SMC) which operates independently from the Scottish Government. Until such time as the SMC publishes advice to confirm its acceptance of a medicine, NHS boards are not expected to make it routinely available.
"However, they have arrangements for the clinical consideration of SMC 'not-recommended' medicines for individual patients in certain circumstances. Such decisions are taken on a 'case by case' basis."
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