Case Study: Rett Disorders Alliance

Rett syndrome is a rare, inherited, neurological syndrome consisting of a spectrum of disorders characterised by the development of severe mental and physical impairments at six to 18 months of age.

The Rett Disorders Alliance is a collaboration first formed between two charities: Rett UK and Reverse Rett. The collaboration has since expanded to include FOXG1 UK. The collaboration was set up to help the charities work together to address the unmet need of Rett syndrome and provide a unified voice for the Rett syndrome community.

The Rett Disorders Alliance is a great example of charities working in the same disease area coming together. Reverse Rett and Rett UK work in subtly different spaces within Rett syndrome but with some overlap (Reverse Rett focuses on research and Rett UK on patient support) but came together to successfully achieve common goals. FOXG1 UK became involved because they are a small charity supporting families affected by FOXG1 syndrome, a variant of Rett syndrome.

Setting Up the Rett Disorders Alliance

The first steps of the collaboration involved assessing the services of each charity and agreeing on a shared mission statement: to improve the lives of people with Rett syndrome and their families. Subsequently, the Alliance set up a terms of reference agreement (non-legally binding), which the boards of the charities agreed would be the basis for how they would work together.

Getting Started

At the first meeting for the collaboration, the Rett Disorders Alliance were determined to set up a Prescribed Specialised Service for Rett syndrome treatments. However, due to the small patient population and lack of funding surrounding Rett syndrome, it was soon realised that there would not be enough resources to be able to set up this service. The goals of the Alliance were therefore adapted and modified, to set up the Rett Disorders Working Group (RDWG).

The RDWG was successfully formed, consisting of over 20 clinicians, parents and patient groups, all with extensive Rett syndrome experience. The RDWG was extremely important in utilising everyone’s strengths, expertise and resources. This enabled the Alliance to have increased legitimacy and authority, so that it could speak as one voice for the whole Rett community. The RDWG became a credible and specialist group which professionals wanted to be involved with.

The RDWG set up several meetings in order to develop a Rett Syndrome Health Checklist. The Rett Syndrome Health Checklist is a document which helps with identifying symptoms of the disease and provides suggestions for disease management. After a lot of time spent accumulating extensive information, writing and reviewing, the Health Checklist was finally published into a booklet and online resource. The Health Checklist has been extremely valuable to Rett syndrome patients and has had a huge positive impact on the community. The Checklist is constantly reviewed and updated to improve accessibility and to account for new prescribing information.

Continuing the Success of the Rett Disorders Alliance

The Rett Disorders Alliance was essential in providing a unified voice for the patients and their families. It is important to emphasise that families should be at the forefront of the collaboration, rather than it being solely for each organisation’s benefit.

The Alliance are extremely proud of their success, and the Health Checklist would not have been so successful if the charities had not collaborated. The Alliance are now focusing their efforts on developing joint presentations at conferences and liaising with pharmaceutical companies to help share recruitment information for clinical trials.