Intended for healthcare professionals

Observations Orphan Drugs

Open letter to prime minister David Cameron and health secretary Andrew Lansley

BMJ 2010; 341 doi: (Published 17 November 2010) Cite this as: BMJ 2010;341:c6466
  1. David J Nicholl, consultant neurologist, City Hospital, Birmingham,
  2. David Hilton-Jones, consultant neurologist, John Radcliffe Infirmary, Oxford,
  3. Jacqueline Palace,, consultant neurologist, John Radcliffe Infirmary,
  4. Sam Richmond, consultant paediatrician, Sunderland Royal Hospital, Sunderland,
  5. Sarah Finlayson, congenital myasthenia clinical fellow, John Radcliffe Infirmary,
  6. John Winer, consultant neurologist, University Hospital Birmingham,
  7. Andrew Weir, consultant neurologist, Royal Berkshire NHS Trust, Reading,
  8. Paul Maddison, consultant neurologist, Nottingham University Hospitals NHS Trust, Nottingham,
  9. Nick Fletcher, consultant neurologist, Walton Centre for Neurology & Neurosurgery, Liverpool,
  10. Jon Sussman, consultant neurologist, Greater Manchester Neuroscience Centre, Hope Hospital, Salford,
  11. Nick Silver, consultant neurologist, Walton Centre for Neurology & Neurosurgery,
  12. John Nixon, consultant neurologist, Royal Preston Hospital, Preston,
  13. Dimitri Kullmann, consultant neurologist, Institute of Neurology, London,
  14. Nick Embleton, consultant paediatrician, Royal Victoria Infirmary, Newcastle upon Tyne,
  15. David Beeson, professor in neuroscience, Weatherall Institute of Molecular Medicine, Oxford,
  16. Maria Elena Farrugia, consultant neurologist, Institute of Neurological Sciences, Southern General Hospital, Glasgow,
  17. Marguerite Hill, consultant neurologist and senior clinical tutor, Abertawe Bro Morgannwg University Local Health Board, Swansea,
  18. Chris McDermott, consultant neurologist, Royal Hallamshire Hospital, Sheffield,
  19. Gareth Llewelyn, consultant neurologist, Royal Gwent Hospital, Newport,
  20. James Leonard, professor emeritus, Institute of Child Health, University College London,
  21. Michael Morris, chairman of the Myasthenia Gravis Association, Derby
  1. Correspondence to: D J Nicholl david.nicholl{at}

Neurologists and paediatricians call for action on “massive” rises in the prices of orphan drugs

We are writing to you as a group of clinicians treating patients with so called “orphan” diseases (and one representative of a patients’ group) to express our concern at an unintended effect of the European Union’s regulations on orphan drugs. The original purpose of this legislation, passed in 1999, was to encourage drug companies to conduct research into rare diseases and 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. Once a company has obtained a licence, the legislation then gives the company sole rights to supply the drug. This in turn allows the company to set an exorbitant price for this supply and effectively to bar previous suppliers of the unlicensed preparation from further production and distribution.

We believe that this behaviour is not in the best interests of patients or the NHS but is undoubtedly significantly advantageous to drug companies. We have made representations to …

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