While NICE acknowledges that the clinical trial evidence for fosdenopterin suggests that it increases the time before people are unable to eat and how long they live compared with standard care; they have expressed that these results Have a high level of uncertainty due to the small number of people in the trial and the trial not using comparator arm (a group of participants receiving the currently most often treatment) for comparison. 

Due to these and other uncertainties, as well as the cost-effectiveness estimates being higher than what NICE normally considers an acceptable use of NHS resources for highly specialised technologies such as these, NICE does not recommend fosdenopterin. To address these uncertainties, NICE has opened a three-week consultation process on their draft recommendation.  

This consultation allows us, and others, to gather and submit further data from the MoCD Type A community around the world to address NICE’s concerns.  

Sharing your experiences and encouraging others to do the same is incredibly important in helping NICE understand life with MOCD Type A. You can do this by following the steps in our three part action plan.

Our new MoCD Type A community survey aims to capture information from those living with and affected by MoCD Type A around the areas of uncertainty identified by NICE. Your experiences and insight into the impacts of MoCD Type A on daily life, including seizures, medication, ability to feed orally, as well as any prior experience with fosdenopterin, will help us address NICE’s concerns.

This survey is open to all countries. We estimate this survey will take 10 minutes to complete. It will close on Sunday 6th of October to allow us to respond to NICE.

You can access the MoCD Type A community survey by clicking the button below:

NICE want to hear directly from you during this consultation phase. Let them know about the following: 

Daily Life Impact:  
How does MoCD Type A impact you as a carer and that of the person(s) living with MoCD Type A? Please describe any aspects of daily life that you feel are impacted. This can include, for example, seizures, medication, ability to feed orally, and caregiver impact.

Seizures:
Does the person affected by MoCD Type A experience seizures? Include information about how frequent the seizures are, the impact of these seizures including on the carer and overall wellbeing, and any medications used. 

Feeding: 
How does the person affected by MoCD Type A feed? Please share if they feed orally, or if they are tube-fed. If they are tube-fed. Include any details around difficulty feeding, and if relevant what age they began being tube-fed. 

Experience of Fosdenopterin:  
Do you have any experience of fosdenopterin? Please include any impact treatment has had on life with MoCD Type A, including any specific impact on seizures and seizure medication use, ability to feed orally, and carer impact. Be sure to detail any relevant timelines, such as how long fosdenopterin was taken for, if improvements stabilised or plateaued, when did this happen. 

This public consultation phase will close on Thursday 10th October. Make sure you get your responses in before then!  

The NICE portal can be accessed by clicking the button below. If you need any help with this process or would like to speak to someone, you can contact us by emailing contact@metabolicsupport.org or by calling 0800 652 3181.

Please encourage others to get involved and share their experiences by sharing this webpage with them. This is our opportunity to help NICE understand MoCD Type A and make a difference to the lives of people living with and caring for someone with MoCD Type A. 

To address NICE’s concerns in the current draft guidance of fosdenopterin for the treatment of people living with molybdenum cofactor deficiency (MoCD) type A, we are gathering additional perspectives from clinical experts with experience of treating MoCD type A patients.

You can find the draft guidance here:
https://www.nice.org.uk/consultations/2733/1/recommendations

The questions in this survey are best answered by treating physicians. As such, please answer all questions from the perspective of “under your care” (in case some or all MoCD type A patients under your care have passed away, please also include them in your responses) and please share this survey with any colleagues also treating MoCD type A patients. If you are unable to answer a question, please feel free to skip.

GDPR: This data will be analysed by Metabolic Support UK (MSUK) for the purpose of providing information to health technology assessment bodies to inform their decision on fosdenopterin for people with MoCD type A. By responding to this survey, you agree to MSUK processing your responses.We expect this survey to take approx. 20 minutes to complete. Please respond by Sunday, 6th of October.

If you have any questions, please do not hesitate to reach out to Laura Smith van Carroll from Metabolic Support UK via laura@metabolicsupport.org

We would like to thank you on behalf of the MoCD type A community for your time in completing this survey.