Further to the recent announcement that the first drug treatment for PFIC has been approved by Medicines and Healthcare products Regulatory Agency (MHRA), CLDF are disappointed to hear that the National Institute for Health and Care Excellence (NICE) has decided to not recommend odevixibat for the treatment of progressive familial intrahepatic cholestasis (PFIC) in patients aged 6 months or older. Please note, this is not NICE’s final decision and the committee will meet again on the 10th November 2021 following review of the consultation responses.
*This decision is for England only.
Reasons for this decision
NICE state that this draft decision has been made due it being a high-cost treatment. They also state uncertainties about clinical effectiveness in rarer types of PFIC and in turn value for money. You can read the full detailed response about the decision in the Evaluation consultation document via the link below.
How you can help
It’s not over yet! While we fully respect the decisions that NICE make through their in-depth assessments of new treatments, as a patient community we shall continue to provide insight about the impact new treatments could have on children and families diagnosed with PFIC.
This is where you can help. NICE are calling on all interested parties to provide comments on this decision which will be reviewed in a further meeting to make a final decision. Anyone affected by a childhood liver condition, as well as family, friends and medical professionals can submit their comments.
Consultation | Odevixibat for treating progressive familial intrahepatic cholestasis [ID1570] | Guidance | NICE – deadline 7th October 2021. (Please note, you will need to sign in/register as indicated on the page to submit your comments).
Given your personal experience/expertise, there are several areas where we feel the consultation would greatly benefit from your insights. Therefore, we request that you consider highlighting the following in your ECD response:
- The unmet need with the current treatment options for PFIC patients
- The need to treat all PFIC patients regardless of subtype
- Any other areas where odevixibat could lessen the overall burden on patients, caregivers, NHS resources (e.g. quality of life, school, work and life, number of hospital visits needed, monitoring)
- If possible, to highlight how rare the subtypes are in UK clinical practice, especially PFIC 4, 5 and 6 (medical professionals)
Alternatively, you can simply respond by submitting a few lines about how you feel about this draft decision. Your response can be as short or as detailed as you choose.
Make a difference by providing your comments via the link above and asking your family, friends and colleagues to submit a response on behalf of those affected by a childhood liver condition. While this treatment is currently only for those with PFIC we are calling on families and young people affected by all childhood liver conditions to take part.
My nephew currently receives this treatment and it has given him the opportunity to lead a better life, whilst we as a family are unsure of what the future holds for our boy we believe that this drug will give him the opportunity to live the best life he can, he is only 4yrs old and deserves to have the opportunity to live with the help of any treatment available, cost should not be a factor in whether a child suffers, lives or dies. Every child/adult/person should have the right to treatment that is available.
My daughter is 4 years old and was diagnosed with PFIC4 at 6 months old. These trials are saving lives and the more options for these children the better. Please reconsider because as a mother of a child with one of the rarer PFICs, these drugs ARE WORKING! This means no transplant which equals a normal quality of life. We are so grateful for the doctors who create these drugs to change our children’s lives.
I looked after my grandson on a few occasions in the early days after his diagnosis with this disease. It was heart breaking to watch him always crying and covered in blood from scratching his skin off. He could not participate in any normal childhood activities and was either crying or sleeping. The medication trial was then offered to him and within months the improvement in his health and quality of life was incredible he became a different child and to any who did not know his story he was a normal healthy child. I am devastated and cannot believe that any government or other organisation would want to take away my grandsons opportunity to live as normally as possible and end his life earlier than expected
I have looked after my grandson on numerous occasions before his diagnosed with this genetic disorder and it was heart breaking to watch him constantly crying and scratching his skin off and unable to carry out the activities of a normal child. After he was eventually diagnosed properly and put on the trial medication the change has been dramatic. He is a happy cheeky lad growing up living a better quality of life, so I have seen the changes first hand. I am devastated that the government or any other organisation would take away any child’s opportunity of living a pain free and relatively normal childhood because of political fallout or cost as we give billions to other countries in foreign aid and if there was a failing in the European Union then this medical trial is not one them
My daughter is pfic1.
We take odevixibat from February this year.
It helps a lot on itches.She can play and sleep well.
We once run out of it for some weeks, itches come back and she scratches with blood.Can not sleep at night.It is horrible.She cry and scratch.
Back on odevixibat she happy girl again.
I am sure she can’t live without odevixibat.