Survey
* Your assessment is very important for improving the workof artificial intelligence, which forms the content of this project
* Your assessment is very important for improving the workof artificial intelligence, which forms the content of this project
East Midlands draft policy: 3, 4 Diaminopyridine (phosphate form) (Firdapse®) for treatment of Lambert-Eaton myasthenic syndrome and Congenital Myasthenia Syndromes. Comments from (please insert organisational/interested party name): ……………………………………………………………………………………… Introduction: Lambert-Eaton myasthenic syndrome (LEMS) is a long term progressive debilitating condition. The symptoms of LEMS vary in severity but are characterised by muscle weakness and excessive fatigue. LEMS is strongly associated with cancer, especially small-cell lung cancer (SCLC). It is estimated that 40 to 60% of patients with LEMS have SCLC and 5% have other cancers. Congenital Myasthenic Syndromes (CMS) are a group of disorders all characterised by abnormal neuromuscular transmission but with various underlying genetic defects. Symptoms can include generalised fatigable muscle weakness. The syndromes are hereditary and usually patients have symptoms from childhood, if not birth. Both LEMS and CMS are very rare, and 3, 4, Diaminopyridine (base form) has been used to help treat these conditions for 20 years. 3, 4 diaminopyridine (base form) is an unlicensed drug. There is now a licensed form of the drug (in phosphate form) called Firdapse®. It is licensed for use in LEMS patients only and to a maximum daily dose of 60mg. The two forms of the drug are considered to be (bio)equivalent. There is no evidence to suggest that 3, 4 Diaminopyridine (phosphate form) works better or is safer that the base form of the drug. The base form of the drug costs, on average, £1200 per patient per annum. In comparison Firdapse® costs, on average, £44,000 per patient per annum. This draft policy says that Firdapse® will not be routinely paid for (commissioned). This is because similar clinical benefits will be provided for LEMS patients by 3, 4 DAP (base form) and by 3,4 DAP(phosphate form) (Firdapse®), but the additional costs of prescribing the licensed drug, 3,4 DAP(phosphate form) (Firdapse®) cannot be justified given the opportunity costs of investing those sums of money in other areas to deliver healthcare benefits for the local population. Feedback: We value your feedback upon the policy document in general and any recommendations or comments that you may have on it. We would be very grateful if you could answer the following questions: 1. Does this policy either directly or indirectly discriminate against any minority groups? 1 2. Is this policy operational? (i.e. will it work, can you see any problems with putting it into practice?) 3 Is the policy fair and appropriate? 4 Please write here any other comments/recommendations you wish to make: How to send your views to us: Your views on this policy are important to us, so we’d be grateful if you could send your feedback by e mail to [email protected] or by post to EMSCG, Public Health Team. Fosse House, 6 Smith Way Grove Park Enderby Leicester LE19 1SX. Please make sure your response reaches us by 31st March 2011. Please share our draft policy with colleagues and key network groups in your area as appropriate. 2