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How you can help make things happen

Rachel and her son Milo

One of the most exciting projects CLDF has been involved with over the past two years is helping to make the new drug Odevixibat (Bylvay) available on the NHS as a possible treatment for the rare liver disease, progressive familiar intrahepatic cholestasis (PFIC).

This involved gaining approval from NICE (the National Institute for Health and Care Excellence) a process in which we were able to play an active part thanks to those parents of children who have PFIC, who took on the role of patient expert.

What is a patient expert?

The role of the patient expert is to participate in the discussions and answer the NICE Appraisal Committee’s questions, relating to their experience of the therapies under appraisal and the condition that they treat. They are required to provide a personal statement about the impact of the condition and give an insight into the lived experience and impact of the condition – on all aspects of life –  not just physical issues.  They also receive the briefing documents which include the evidence from all the stakeholders in preparation for the meeting and attend the appraisal meeting which can be a long session.

The patient expert can be involved in any subsequent appeals process and obviously has to abide by strict confidentiality agreements.

So it represents a real commitment, but one which both Rachel and Claire were happy to take on.

“I find the process of drug licensing fairly interesting and having been through those early years with two children diagnosed with PFIC-2, the idea of helping the children and parents of the future was appealing,” says Rachel.

“One of the things that I remember when Eleanor was first diagnosed was desperately wanting a miracle drug to cure her or, at least something that might have helped with the horrible side effects of her type of liver disease,” adds Claire. “I have always felt passionately about the need for research and alternative treatments for Eleanor’s condition. The thought of a transplant as our only option hung over me throughout her childhood and was hugely difficult for us as a family. There was very little out there in terms of research into alternatives. After everything we have been through, this felt like a way to channel my feelings and experiences into something really positive.”

“It is a time commitment,” admits Rachel. “The meetings were long and there is a lot of paperwork to complete but the support from NICE staff was exceptional and getting a positive result was wonderful.”

“I found it really interesting to see how complex the process is for getting a drug approved, especially in the case of one for a rare disease,” says Claire .”It taught me a lot and opened my eyes. It was also really empowering to feel I might have had a role in helping other families like ours. I think that if Eleanor hadn’t gone through transplant and I felt this drug might have still been an option for her, I would have felt even more passionate about getting it approved. It would have given me something very empowering and positive to channel my negative energy and anxiety into. I would love to have had this opportunity when Eleanor was little.

“I also learned not to be disappointed if the drug gets rejected at the initial consultation. This is not unusual and NICE support staff prepared us for that. If they hadn’t, I think I would have felt very sad and let down. Many drugs get accepted once an appeal is made and all the initial issues raised refusing the drug’s approval have then been addressed second time around.”

Rachel and Claire agree that parents who are offered the opportunity to be a patient advocate in a drug approval process should seize it.

“Even though the patient rep role is tiny, every little helps in getting drug approval over the line,” says Rachel.

“And don’t be intimidated, “adds Claire. “You are there to represent the patients – no one expects you to be an expert on the technical stuff. Don’t underestimate what a huge impact your small contribution can make!”

Alison Taylor, CLDF’s former CEO who co-ordinated the process feels that the Bylvay result shows what can be achieved by working together. “Huge thanks are due to both to Rachel and Claire and to all those who helped us appeal NICE’s original decision not to recommend the drug for the treatment of PFIC. This drug will transform lives. We now know that CLDF is to get the opportunity to play a similar role with treatments for other liver conditions and it is so exciting for parents to know they can play such a valuable role in the future treatment of their child.”

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