AGSD, Association for Glycogen Storage Disease; LSD, Lysosomal Storage Disorder; MLD, Metachromatic Leukodystrophy; MPS, Mucopolysaccharide Disease.

LSDs are a group of more than 40 rare, inherited, disorders. LSDs can be progressive, life-threatening and severely debilitating.

The LSD Collaborative is a collaboration of nine patient organisations in the UK each focusing on a different type of LSD. This case study is a great example of patient organisations focusing on different diseases with similar underlying mechanisms coming together. The LSD Collaborative formed naturally because these diseases are treated under similar healthcare services.

Figure 2. Formation of the LSD Collaborative

A flowchart to illustrate the formation of the LSD Collaborative. Nine patient organisations focusing on different LSDs came together to form the LSD Collaborative. The similarities and differences between the groups were identified so that a ‘similar, but different’ approach could reduce conflicts and enhance knowledge sharing to work towards common goals. The LSD Collaborative has a clear vision and mission statement, while all the groups involved complement the work of each other and are given an equal voice.

Setting Up the LSD Collaborative

The LSD Collaborative was formed in 2005 initially from five patient organisations who met informally to share information in order to influence service provision, identify gaps, and address specific needs of patients affected with LSD. In 2008, with six patient organisations, the LSD Collaborative developed a terms of reference agreement. This consisted of a non-legally binding formal agreement, with a clear vision and mission statement: to work together to advance standards of care and enhance the wellbeing of all those affected by LSD.

The terms of reference stated that all member organisations should have an equal voice regardless of their size or financial status. It was also agreed that the Collaborative should be in regular contact but would meet formally three times a year.

Getting Started

The main areas of work that the Collaborative focused on were campaigning for better access to healthcare, supporting patients with LSD, consulting on the issues impacting the community, and developing strategies to utilise the Collaborative’s strengths. A key priority was to complement the work of respective organisations in order to achieve more than an organisation would on their own.

The Collaborative initially encountered a couple of challenges. Firstly, with no legally binding formal agreement in place, it was harder to access funding. Secondly, time and capacity were often an issue, particularly as there were many volunteers and changes of personnel.

Since overcoming these challenges, the LSD has successfully worked with key stakeholders including clinical centres, pharmaceutical companies, the National Institute for Health and Care Excellence (NICE), National Health Service (NHS) England and most importantly, patient communities. Recent successful outcomes have included patient webinars, patient surveys and a patient professional partnership network.

Continuing the Success of the LSD Collaborative

At the Collaborative’s scoping meeting held in August 2020, the benefits and challenges of the collaboration were discussed. The meeting covered possibilities for closer working and a more formal, legally binding agreement in the future. The Collaborative are also starting to organise LSD-specific events and plan to become a global collaborative in the future.