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An open letter to the Rt Hon David Cameron, Prime Minister and the Rt Hon Andrew Lansley, Secretary of State for Health.
We are writing to you as a group of clinicians* treating patients with so-called ‘orphan’ diseases to express our concern at an unintended effect of the Orphan drug legislation. The original purpose of this legislation was to encourage drug companies to research into rare diseases and to develop novel treatments. However, as the rules are currently enacted, many drug companies merely address their efforts to licensing drugs that are already available rather than developing new treatments. Having obtained a licence, the legislation then gives the company sole rights to supply the drug. This in turn allows them to set an exorbitant price for this supply, and to effectively bar previous suppliers of the unlicensed preparation from further production and distribution.
We believe this behaviour is neither in the best interests of patients nor the NHS, but is undoubtedly significantly advantageous to pharmaceutical companies. We have made representations to the Department of Health and the MHRA. In return, we have simply been quoted the rules and no-one seems willing to investigate the issues that we are raising or to consider whether the system should be changed. We are asking you to identify the appropriate individual(s) who can act upon this unacceptable situation.
One example of the effect of these rules is the drug 3,4-DAP. We have been using 3,4-DAP for over 20 years to treat two rare diseases, Lambert Eaton Myasthenic Syndrome (LEMS) and congenital myasthenic syndromes - both cause disabling muscle weakness of the limbs, body, eyes and face, together with swallowing and breathing problems, which can be fatal. 3,4-DAP improves muscle strength, and is used either because other treatments haven’t worked sufficiently or to avoid using drugs which can have serious side effects. Expert clinicians take the responsibility for informing patients about the drug and prescribing it. It has an excellent safety record. At present 3,4-DAP has been produced by a small pharmaceutical company on an unlicensed basis and costs between £800-1000 per patient year.
Biomarin has now been issued with a licence to supply 3,4DAP (named Firdapse) throughout Europe and has priced their product at £40,000-70,000 per patient per year – a 50- to 70-fold increase. Biomarin have merely had to demonstrate that their drug works, using data generated from the unlicensed version. They have simply produced a slightly modified version that meets regulatory standards and have been allowed to set the price at an exorbitant level with no clinically relevant advantage.
This high cost means that
1) Some funders (i.e. PCTs) have refused to pay for the drug because it doesn’t fulfil cost effectiveness criteria.
2) Where it is funded no additional funding source has been identified which must mean that patients in other areas are being deprived of NHS funding. The cost to the NHS is likely to be in excess of £10,000,000 per annum.
We urge you to instruct urgent review not only for the sake of our particular group of patients but also for the many more who are likely to be affected in the near future as other pharmaceutical companies take advantage of this loop hole. Extraordinarily, there is a website which lists other drugs that can be similarly exploited (http://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/HowtoapplyforOrphanProductDesignation/ucm216147.htm).
In the present economic situation it seems vital to ensure systems are set in place to prevent excessive commercial profits being made at the expense of patients and public spending. Although the pricing of 3,4-DAP is the most recent example of this trend1, massive price rises have been noted in other orphan diseases for years- e.g. from N-carbamylglutamate (for N-acetylglutamate synthetase deficiency), sodium phenylbutyrate (for ornithine carbomyl transferase deficiency), ibuprofen and indometacin (for the treatment of patent ductus arteriosus), caffeine citrate (for apnea in preterm infants) and even nitric oxide (for the treatment of pulmonary hypertension)2,3.
We feel that there are sufficient grounds for the Health Select Committee to look at the pricing of orphan drugs as this is directly affecting patients of all ages with a diverse range of conditions across the country. If an investigation was to find that there was evidence of artificially high pricing, then this is something that the Office of Fair Trading should consider pursuing.
Orphan drug legislation, far from encouraging the development of new treatments for orphan diseases, is severely limiting the availability of existing treatments. We feel the MHRA and DOH should not only simply state the rules but should take the initiative in cascading the issues of unfairness upwards, in order to instigate change.
*MM is not a clinician, but represents the Myasthenia Gravis Association which includes patients with LEMS and congenital myasthenia.
References
1. Hospitals forced to use unlicensed drugs to save millions. The Daily Mail. 27th September, 2010.
http://www.dailymail.co.uk/health/article-1315441/Hospitals-forced-use-unlicensed-medicines-save-millions.html (accessed 9th November 2010)
2. EU loophole sends drug prices soaring. The Guardian. 24th June 2002.
http://www.guardian.co.uk/society/2002/jun/24/health.medicineandhealth1 (accessed 9th November 2010)
3. Leonard JV and Richmond S. Pricing of Orphan Drugs. The Lancet 2009;373:462
Dr David J Nicholl, Consultant Neurologist, City Hospital, Birmingham
Dr David Hilton-Jones, Consultant Neurologist, John Radcliffe Infirmary, Oxford
Dr Jacqueline Palace, Consultant Neurologist, John Radcliffe Infirmary, Oxford
Dr Sam Richmond, Consultant Paediatrician, Sunderland Royal Hospital, Sunderland.
Dr Sarah Finlayson, Congenital Myasthenia Clinical Fellow, John Radcliffe Infirmary, Oxford
Dr John Winer, Consultant Neurologist, University Hospital Birmingham.
Dr Andrew Weir, Consultant Neurologist, Royal Berkshire NHS Trust, London Road, Reading
Dr Paul Maddison, Consultant Neurologist, Nottingham University Hospitals NHS Trust, Nottingham
Dr Nick Fletcher, Consultant Neurologist, Walton Centre for Neurology & Neurosurgery, Liverpool
Dr Jon Sussman, Consultant Neurologist, Greater Manchester Neuroscience Centre, Hope Hospital, Salford
Dr Nick Silver, Consultant Neurologist, Walton Centre for Neurology & Neurosurgery, Liverpool
Dr John Nixon, Consultant Neurologist, Royal Preston Hospital, Preston
Prof Dimitri Kullmann, Consultant Neurologist, Institute of Neurology, London
Dr Nick Embleton, Consultant Paediatrician, Royal Victoria Infirmary, Newcastle-upon-Tyne.
Prof David Beeson, Professor in Neuroscience, Weatherall Institute of Molecular Medicine, Oxford
Dr Maria Elena Farrugia, Consultant Neurologist, Institute of Neurological Sciences, Southern General Hospital, Glasgow
Dr Marguerite Hill, Consultant Neurologist and Senior Clinical Tutor, ABMU Local Health Board, Swansea
Dr Chris McDermott, Consultant Neurologist, Royal Hallamshire Hospital, Sheffield
Dr Gareth Llewelyn, Consultant Neurologist, Royal Gwent Hospital, Newport, Wales
Prof James Leonard, Professor Emeritus, UCL Institute of Child Health, London
*Michael Morris, Chairman of the Myasthenia Gravis Association, The College Business Centre, Uttoxeter New Road, Derby, DE22 3WZ.
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