Applying to Get Xyrem

While there is no specific cure for narcolepsy a number of drugs are available that can help manage the symptoms of the condition. The introduction of a drug called Xyrem (which is also known as Sodium Oxybate) has shown some significant advantages over other drug regimes currently available. In a very short space of time Xyrem has become the drug of choice in America for those suffering from narcolepsy with cataplexy.

Narcolepsy is diagnosed in a very small proportion of the population leading to ‘orphan disease’ status and high treatment costs for some medications. Sodium Oxybate (Xyrem) the only approved drug for the treatment of narcolepsy with cataplexy in adult patients can cost your Primary Care Trust up to £13,000 for a year’s supply.

Xyrem is believed to offer improved symptom control for patients who have not done well on conventional treatments. It reduces and in some cases totally prevents, cataplexy, hallucinations, disturbed sleep and excessive sleepiness.

It is not always routinely funded by Primary Care Trusts (PCTs). Different PCTs have different policies in place to manage funding applications for drugs like Xyrem that are not routinely funded. Some PCTs may only agree to funding treatment on the grounds of ‘exceptional circumstances’. Your specialist will be aware of the policy taken by your local PCT.

It is unlikely that you will be provided with Xyrem before you have tried other drug regimes. In most instances your PCT will want to see that you have tried at least two different drugs, and neither made a significant impact on your narcolepsy, before they will consider Xyrem. Whilst it is always helpful to have a supportive GP it is unlikely that they alone will be able (at the moment) to prescribe you with Xyrem.

Should your specialist suggest treatment with Xyrem, an application will need to be made to your local PCT to request funding. It is important to let your clinician make an official approach to the PCT on your behalf, but you are perfectly at liberty to support statements they make with your own personal statement, which should be submitted through your consultant.

Your consultant will provide the PCT with an outline of your case, highlighting how the condition effects your current day to day life. Previous treatments will be described and a clear statement of the treatment plan proposed. Your consultant will explain the benefit you will receive from the treatment and give evidence of any exceptional circumstances. It is in the evidence of exceptional circumstances that your personal statement could provide help.

Your consultant does not have to prove you are unique, just exceptional. If that can be proved you should be allowed the drug. The PCT might refuse the application for a number of reasons, the most common being that Xyrem is not approved by NICE. We provide answers to their statements in the Taking it Further section. They might say your QALY is not high enough; we explain that in the QALY section. You can appeal any decision your PCT makes and you should be considering that throughout the process of application.