Letters Orphan drugs

Authors’ reply to BioMarin Europe

BMJ 2011; 342 doi: https://doi.org/10.1136/bmj.d135 (Published 11 January 2011) Cite this as: BMJ 2011;342:d135
  1. David J Nicholl, consultant neurologist1,
  2. David Hilton-Jones, consultant neurologist2,
  3. Jacqueline Palace, consultant neurologist2,
  4. John Winer, consultant neurologist3
  1. 1City Hospital, Birmingham, UK
  2. 2John Radcliffe Infirmary, Oxford, UK
  3. 3University Hospital, Birmingham, UK
  1. david.nicholl{at}nhs.net

BioMarin Europe feels that our letter on the costs of orphan drugs such as amifampridine (Firdapse) for Lambert Eaton myasthenic syndrome (LEMS) and the safety of existing unlicensed treatments, such as 3,4-diaminopyridine base (3,4-DAP), contained inaccuracies.1 2 The Department of Health has confirmed that the additional cost of amifampridine in England is £9 768 668 (€11 400 035; $15 294 803) (excluding …

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