Taking it a step further

 

Responding to the PCT
 

PCT arguments against funding
Counter arguments and reasons for funding
There is no national recommendation for this treatment
  • NICE has not reviewed Xyrem. However, this does not mean that it cannot be prescribed or funded
  • There is ‘no ban on prescribing licensed drugs that have not yet been appraised by NICE’
  • There are two Department of Health documents that provide guidance to PCTs / Local Health Authorities on managing the introduction of new drugs that have or perhaps will not be appraised by NICE (see resources and links)
The Scottish Medicines Consortium (SMC) has not recommended the drug for use in Scotland
  • This decision was based on a lack of evidence for cost effectiveness when used solely for cataplexy. Xyrem is licensed for all the core symptoms of narcolepsy not just cataplexy
  • Many recommendations for allocating money from health budgets in Scotland have not been adopted in England
  • The EFNS guidelines recognise Xyrem as the first line treatment for narcolepsy with cataplexy
 
Cataplexy was not the primary symptom and therefore funding Xyrem would allow the drug to be used outside its licensed indication
  • It is relatively rare for cataplexy to be the dominant symptom although it can be very disabling in some patients. The anti-depressant Clomipramine is licensed for cataplexy but is almost certainly less effective than Xyrem and more likely to produce side-effects
  • Xyrem is licensed for all the core symptoms of narcolepsy
 
Clinical data on Xyrem is not robust
  • The ENFS guidelines recommend Xyrem as a ‘viable treatment option across multiple symptoms’.
  • Data highlighting the benefit of Xyrem can be found in a variety of published clinical studies (see resources and links)
  • In order for Xyrem to gain a license from the EMEA (European Medicines Evaluation Authority) and the US FDA (Food and Drug Administration) it had to go through robust clinical trials to prove its efficacy and tolerability. Xyrem is currently used as a first line treatment by many doctors in the United States

 

Getting the support of your MP

Many local politicians take a keen interest in the services provided by their local national health service (NHS) so it is worth contacting your local MP to ask for their support. Your MP is in a position to directly influence government ministers who in turn can influence PCTs and strengthen your case for appeal. You can find out who your MP is by visiting www.upmystreet.com/commons/l/ and entering your postcode. The best way to approach your MP is by letter explaining your situation and experience in seeking funding for treatment. Ask your MP if they would be willing to write to the PCT in support of your funding request.
 

Generating local publicity

Generating media coverage in your local area to raise awareness of your situation may also add weight to your appeal. We can help you do this by developing press materials and liaising with the media in your area to encourage publicity as well as offering ongoing support. If this something you would like our assistance with then please do not hesitate to contact us.