EU ORPHAN DRUG LEGISLATION AND ITS EFFECTS ON PEOPLE WITH RARE NEUROMUSCULAR CONDITIONS

That this House welcomes the letter published in the British Medical Journal by leading doctors from the Oxford Muscle and Nerve Centre and across the country, supported by the Muscular Dystrophy Campaign and Myasthenia Gravis Association, working with patients with two rare neuromuscular conditions, congenital myasthenic syndrome and Lambert-Eaton myasthenic syndrome, regarding the high cost of the one available treatment for these conditions; notes that, prior to its licensing, the drug was cheaply available to clinicians for 20 years; is concerned that the cost has now risen by 70 to 100 fold since the licence was granted, taking the cost per patient from between 800 and 1,250 per year to between 40,000 and 70,000 per year; further notes that, while the drug company is legally entitled to act in this way under the current EU orphan drugs legislation, the legislation was originally intended to encourage drug companies to conduct research into rare diseases and develop original treatments; shares the concern of clinicians that some patients are now being denied this treatment due to its high cost; and calls on the Government to review the regulations to ensure access to treatments for patients with these rare neuromuscular conditions, and best value for the taxpayer.