Reflecting on the previous year

I was astounded to see from our 2019 stats that although we received 25% more requests for help than in the previous year, it took three times the number of telephone calls, emails, letters, training and meetings to resolve the issues.

Is this a sign that the difficulties families are experiencing are more complicated? Harder to resolve? Is it harder for families to access the support they have the right to? I think that the answer to all of these is a resounding yes. 

This is why we need to have the right information available for families and the expertise of our team to navigate the myriad of systems our community has to deal with. So in 2020 our team will still be working to help resolve the wide range of issues that PWS brings – and these days, we are not just a phone call, but also an email, WhatsApp, Insta, Facebook post, Facebook message, Facebook Live away! 

2019 was also the year of our first UK drugs trials! We worked with both Millendo Therapeutics and Soleno Therapeutics to get these trials through Ethics and bring them to sites in the UK. The results are not available yet, but we are very hopeful that they will want to bring a drug to market. 

Of course, we all know that in the UK, this is not straightforward. Any new drugs treatment must be approved by NICE and much depends on the price. Drugs for small markets tend to be more expensive and it will be hard to get anything approved. However, when was anything PWS easy?

So what are my hopes for 2020/2021? I hope that we need to organise a campaign to NICE! I hope that I need to mobilise our whole community to stand with us and demand a better future for our children and adults with PWS. I hope that 2020/2021 will be life changing!