On 6 September 2024, NICE published an update on their evaluation of pegzilarginase for people living with Arginase 1 deficiency (ARG1d). Unfortunately, NICE does not recommend pegzilarginase for treating ARG1d.  

While NICE acknowledges that pegzilarginase reduces the arginine levels in the blood compared to the use of a placebo with usual treatment; they have expressed uncertainty around the impact of pegzilarginase in other areas, including mobility and cognitive impacts. 

Due to these uncertainties, NICE has concerns about the cost-effectiveness of pegzilarginase using NHS resources. 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 ARG1d 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 ARG1d. You can do this by following the steps in our three part action plan.

Our new ARG1d community survey aims to capture information from those living with and affected by ARG1d around the areas of uncertainty identified by NICE. Your experiences and insight into the impacts of ARG1d on daily life, including mobility and cognitive ability, as well as any prior experience with pegzilarginase, will help us address NICE’s concerns.

This survey is open to all countries and we estimate it will take 15 minutes to complete. It will close on Sunday 22nd September to allow us to respond to NICE. 

Click here to take part in the ARG1d community survey. 

NICE want to hear directly from you during this consultation phase. Sharing about any of the below topics would be especially helpful: 

• Daily Life Impact:  
  How does ARG1d impact you or the person(s) living with ARG1d? Please describe any aspects of daily life that you feel are impacted. This can include, for example, hyperammonaemia, social life, education, employment, and caregiver impact.
• Mobility:
  In your experience, how does ARG1d impact mobility? Around what age(s) did you notice changes to mobility, if any? If you use any mobility aids and/or therapies, you could also include information about this.
• Cognitive Abilities:
  How does ARG1d impact the cognitive abilities of yourself or the person(s) living with ARG1d? Please describe whether developmental milestones were met alongside examples of daily life including school and/or work. Where possible also include at what age(s) cognitive impact was observed.
• Experience of Pegzilarginase:  
  Do you have any experience of pegzilarginase? Please include any impact treatment has had on life with ARG1d, including any specific impact on mobility and cognitive ability. Be sure to detail any relevant timelines, such as how long pegzilarginase was taken for, if improvements stabilised or plateaued, when did this happen.

This public consultation phase will close on Thursday, 27 September. Make sure you get your responses in before then!  

The NICE portal can be accessed online by clicking here. 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 ARG1d and make a difference to the lives of people living with and caring for someone with ARG1d. 

In collaboration with clinical experts, we have developed a clinical survey to gather your insights. To complete the survey, please follow this link: https://forms.gle/2fdSZjQqrbeyfUzR7 (we estimate it will take 20-30 mins to complete).

The questions in this survey are best answered by treating physicians. As such, please answer all questions from the perspective of “under your care” and please share this survey with any colleagues at the same centre who are also treating ARG1d patients. As we are looking to collect as many responses as possible, please do forward this survey to any other UK-based or international clinicians who you believe can provide input.