Download Responses to draft EMSCG BAHA policy

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?
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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.
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