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.