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Background Info

There are four distinct areas which are brought to attention of the Committee:

1. A National Plan for Rare Diseases

The Council of the European Union’s Recommendation on an action in the field of rare diseases was adopted unanimously by each Member State of the EU in June 2009. The Recommendation calls on Member States to adopt plans or strategies for rare disease by 2013. 

• RDUK calls upon the Committee to ask the Scottish Government what plans it has in place for the implementation of a National Plan for Rare Diseases in Scotland.
• RDUK calls upon the Committee to ask the Scottish Government what plans it has to allow a budget so that all patients can access therapy, if available, for rare diseases.

2. The current IPTR process is too onerous for orphan medicines

RDUK would like to commend the extensive work undertaken by the previous Public Petitions Committee in consideration of petition PE1108 which led, directly, to revised guidelines being issued by the Scottish Government on the ‘end to end’ process from licensing of medicines through to individual patient treatment requests (what was known as ‘exceptional prescribing’). However, this has not improved access to orphan medicines for patients with a rare disease. www.scottishmedicines.org.uk/files/CEL2010_17.pdf

Although the Scottish Government may no longer be referring to the  terminology of ‘exceptionality’  within the IPTR process, the clinical case made by the requesting physician still relies on the principle that the patient is in some way ‘exceptional’ from the general population where the drug is used. The criteria that must be met as part of this process are proving to be a particular challenge for patients with rare diseases. The CMO letter to the NHS Boards on 18 March 2011 states:

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

• the general population of patients covered by the medicine’s license; or
the population of patients included in the clinical trials for the medicine’s licensed indication as appraised.

There is also evidence that the terminology “Exceptionality” is still applied within the IPTR process at a Health Board level.  The Non-Formulary process from GG&C HB is very clear that the requesting clinician is responsible for “demonstrating exceptionality” for their patient. http://www.ggcformulary.scot.nhs.uk/Medicines%20Policies/Medicines%20Policies%20Home.htm

In rare diseases it is extremely difficult to demonstrate the above criteria. The small patient numbers who make up the clinical trial populations are those patients with the greatest clinical need for the drug and therefore the license will be based on this group of patients. It is therefore extremely difficult to show that a patient with genuine clinical need will be “More likely to benefit from the medicine than might be expected for other patients with the condition”. The patients who are likely to have the greatest need for the treatment will be the same as those patients within the clinical trials upon whom the license is based. Unlike in some of the more common conditions and even certain cancers where there is often more than one licensed treatment available, in the majority of rare diseases there is likely to be only one licensed treatment available, apart from just supportive care. In orphan diseases the above criteria are therefore more likely to lead to those patients with the greatest clinical need being refused access to therapies, which may be life changing and / or life saving.

This has been observed in several recent cases since the publication of CEL 17, whereby GG&C NHS Board and Ayrshire & Arran NHS Board have refused to fund therapy in patients with rare diseases. RDUK are aware of five cases in Scotland where patients have failed to meet the clinical circumstances highlighted in the CMO letter, despite very strong endorsements for treatment by UK opinion leaders.

• RDUK calls upon the Committee to ask the Scottish Government to review the CMO letter to the 14 NHS Boards dated 18 March 2011 which aims to provide guidance to the NHS Boards in relation to the implementation of their Individual Patient Treatment Request (IPTR) process regarding orphan medicines.

3. Assessment of Orphan Medicines

A recommendation from the report; ‘Improving Lives, Optimising Resources: A Vision for the UK Rare Disease Strategy’ is that ‘Evaluation [for orphan medicines] should be based on an appraisal of the technology against multiple criteria and not simply a cost utility analysis. A recent positive example of this approach is the decision making framework developed by the AGNSS in England. This framework involves a consideration of ‘value’ in four domains: health gain, societal value, reasonable costs and good practice. This mechanism will be reserved for medicines that are used to treat 500 or fewer patients in England.  By contrast, the SMC appraises all new medicines coming to market in Scotland. As of May 2010 the SMC had appraised 46 orphan medicines, recommending 18, rejecting 17 and recommending the restricted use only of a further 11. This situation has come about despite the addition of modifiers to the SMC process designed to give special consideration to treatments for rare disease and terminal illness.’

The SMC has stated that a medicine has to be very special to be recommended if the cost per QALY > £30,000, which is the case with the majority of innovative products treating very few patients.

• RDUK calls upon the Committee to ask the Scottish Government to use expertise developed by AGNSS to explore how drugs for very rare diseases can be more appropriately assessed.

4. Orphan Drugs Risk Share Scheme

The ODRSA in Scotland is operated by the NSD. From their 2009 /10 Annual report  (www.nssannualreport.scot.nhs.uk/nsd/orphandrugs10.html) it shows that the spend for the financial year for the risk share was £5.21 million against a budget of £5.2M. The risk share budget for the last three financial years has remained static with no increase on £5.2M despite having been slightly over spent in 2009/10. As this pays for therapy for rare disorders, and patients who have chronic long term conditions which require therapy for the duration of their life, this budget requires to be increased to allow new patients to access the therapies in the risk share. NHS Scotland announced that NHS bodies collectively underspent by £37 million on a revenue budget of £9.5 billion in 2009/10. Also Ms Sturgeon has said; “When we add this saving to the other non-clinical efficiency savings that health boards will make, it will release more than £100 million a year to be spent on vital frontline services.”  (www.scotland.gov.uk/News/Releases/2010/12/15135337)

• RDUK calls upon the Committee to ask the Scottish Government to review whether the budget for the Orphan Drugs Risk Share Scheme is sufficient to meet the needs of patients in Scotland who would benefit from these treatments.