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Author Topic: We need your help to ensure access to life-changing treatment.  (Read 3495 times)

Oliver Gardiner

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As some of you may be aware, burosumab (Crysvita) is being reviewed by the UK’s medicine guidelines organisation that we refer to as The National Institute for Health and Care Excellence (NICE).

They have published their first stage recommendation and unless something happens to change their mind, NICE will not recommend burosumab for use by the National Health Service (NHS) in the UK. The committee acknowledges that XLH is a serious condition, that childhood is the best time for treatment, and that burosumab is more effective than standard treatment, but NICE still concluded, in essence, that the benefits of treatment are not worth the cost. Much of their consideration, however, is based on the assumption that treatment would stop at age 18 (since burosumab is only approved for use by children in Europe), and then, as with current treatment, some symptoms would recur because there is no permanent fix to the metabolic system. Their decision also doesn't fully acknowledge the extent of both short-term and long-term benefits from the best possible treatment during childhood.

We still have time to act to change this outcome for children and families living with XLH across the UK.

NICE has given the public an extremely short window, just until July 6, to comment on their recommendation. Comments may be submitted from anywhere in the world, although UK residents' opinions are particularly important. Therefore we ask that you follow these simple steps to submit comments that will be reviewed in the next evaluation committee hearing in the hope that we can turn this decision around.

1. To comment, you must register at NICE using this link https://goo.gl/RYd2Cz.
2. To be effective in submitting your comment, it's important to focus on specific issues that might change their evaluation. We've broken their feedback down to make it easy for you. The evaluation committee is interested in receiving comments on the following:
a) How do symptoms (physical and psychological) and treatments (including any surgery) from childhood affect you or your child in adult life?
b) For children on standard treatment (phosphate and calcitriol), what are the side effects of taking current treatment? How is that a burden for the child and your family?
c) If you have a child who is 1-12 years old and on burosumab, please explain how this treatment is currently helping in the short-term and how you expect that it will help in the long-term.
d) If you have a child who is 13-17 years old and on burosumab, please explain how this treatment is currently helping in the short-term and how you expect that it will help in the long-term.

Remember that the closing date for comments is July 6, 2018, no excuses, no extensions. If you want to be heard on access to burosumab in the UK, you must speak up now.

We have been assured that every comment from the public is taken seriously and has the power to turn this decision around so that all children with XLH can benefit from this life-changing treatment in the UK.

Finally, if you have any questions, feel free to comment below, message me privately or email at oliver.gardiner@xlhnetwork.org.

We will update you on the progress as soon as we are able, and we want to thank you each of you in advance for submitting a response. #powerinnumbers #XLHStrong #SetTheRecordStraight


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Re: We need your help to ensure access to life-changing treatment.
« Reply #1 on: September 06, 2018, 05:04:46 PM »
We are absolutely thrilled to announce this morning that burosumab has been APPROVED for routine use on the UK’s NHS for children from 1 years old up to young adults aged 16/17 years.

This news completely changes the landscape for the estimated 250 children living with XLH in the UK. Burosumab will be the first and only treatment that targets the cause of the hypophosphatemia in the body. The treatment helps reduce symptoms during childhood and by reducing bone damage during childhood growth it could mean the potential for a better quality of life for children later in their lives too.

We want to say a MASSIVE thank you to all those individuals who shared their experiences with us and with NICE so that decision-makers could understand what this treatment will really mean to patients and their families. Without all of your input this decision to approve burosumab may not have been made.

For those that can’t quite believe their eyes this morning, please visit NICE for the full guidance on children with XLH. If you have further questions about the treatment and whether it is right for your child then please do speak to their paediatrician that manages their XLH.

Since this news is so important, we at XLH UK have put together a press release. See link.

Whilst access to burosumab on the NHS will just be for children for now, let me assure you that the fight for the adult license has begun.

Meanwhile please please do stay tuned and encourage other XLH’ers in the UK & their families to join our XLH-UK facebook page because we’re so much stronger together.