Recent Press Releases
Friday 25 January 2013 - Genetic Alliance UK calls for transparency and fairness in the commissioning of orphan drugs
The Advisory Group for National Specialised Services (AGNSS), an expert committee that advises health Ministers on which services should be nationally commissioned, concluded that the drug Eculizumab would help save lives and improve the quality of life for children (among whom the condition is particularly prevalent) and adults with atypical haemolytic uraemic syndrome (aHUS). However, Ministers at the Department of Health have said that whilst they recognise the evidence for the clinical effectiveness of Eculizumab, they require further advice on the affordability of the drug.
Genetic Alliance UK was disappointed by the decision by Ministers at the Department of Health not to ratify the recommendation by AGNSS to make Eculizumab available nationally. We believe that it is unfair to withhold vital treatment for patients with aHUS, especially since the appraisal process by which AGNSS came to its recommendation was one that was comprehensive and robust; a process that has been widely lauded by academia, patients and industry alike. That it is not simply a rubber stamp is evident in respect of its decision not to recommend approval of Tafamidis for the treatment of Familial Amyloid Polyneuropathy.
Furthermore, Ministers have now referred Eculizumab to be considered by NICE. Genetic Alliance UK is confounded by this decision as there is no process for appraising ultra-orphan medicines within NICE. Referring the decision to NICE will therefore result in an unacceptable further delay for patients, resulting in avoidable harm to those affected.
Eculizumab is currently available to patients with Paroxysmal Nocturnal Hemoglobinuria (PNH); it should therefore be available to those with aHUS. We believe this discrimination towards those with aHUS to be wholly unjust and against the very principles on which the NHS was founded. The NHS is based on the principle of responding to need and if a drug is affordable for one (small) group of patients, then it is affordable for all who have an identifiable clinical need. To do otherwise is to create a system of arbitrary rationing that will result in institutionalisation of inequitably between patients.
Alastair Kent, Director of Genetic Alliance UK said ‘Patients with rare diseases will look at this decision and be dismayed at what they will perceive as the undermining of a trusted and fair process on purely financial grounds. It also has the potential to undermine patient confidence in the replacement process for AGNSS currently being developed by NICE if this is thought to be driven by financial expediency rather than clinical need.’
We urge Ministers at the Department of Health to reconsider their decision in delaying and preventing the effective treatment for those with aHUS.
For more information please contact:
Farhana Ali, Public Affairs Officer, email: phone: 020 7704 3141
Notes to editors
Genetic Alliance UK is the national charity of over 150 patient organisations supporting all those affected by genetic conditions. Our aim is to improve the lives of people affected by genetic conditions by ensuring that high quality services and information are available to all who need them.
We strive to do this in three ways:
Supporting: We seek to raise awareness of genetic conditions and improve the quality of services and information available to patients and families.
Campaigning: We actively campaign on behalf of those with genetic conditions on issues of policy and practice to influence governments, policy makers, industry and care providers such as the National Health Service.
Uniting: We provide a united voice for all those affected by genetic conditions, enabling us to work together towards a common goal of making life better for patients and families at risk.
As an organisation focusing on issues of policy and practice Genetic Alliance UK keeps an active watch on developments in the UK and Europe that will influence the effective transfer of knowledge and understanding into products and services for families that are supported by our member groups.
In a potentially controversial field like genetics, Genetic Alliance UK provides a strong voice advocating the benefits of scientific progress in understanding the contribution being made now and that will be made in the years to come for improving health and combating many currently incurable diseases.
For more information please go to www.geneticalliance.org.uk
Registered Office: Genetic Alliance UK Unit 4D, Leroy House 436 Essex Road, London N1 3QP
A Registered Charity in England and Wales (number 1114195) and in Scotland (number SC039299). A company Limited by Guarantee (number 05772999)