Role of Patients’ Advocacy in Rare Diseases

Headed by patients, parents or patient advocates, rare disease advocacy groups are often run by volunteers that have experienced what it is like to be given a diagnosis of something no one has ever heard of, that there is either no treatment or a treatment that is unaffordable, where there is little or no information, data or research, and that you may not meet another patient or parent for months or years. Historically patient advocacy was run from bedrooms and kitchen tables, in many countries and for many conditions it still is, however the voice of Patient advocacy is now integral in all aspects of the patient journey and their care from bench to beside and beyond.

1. Each patient has their own Journey, no two will be the same although there are essential elements that are similar and can be used to learn lessons, support others, make changes and strive to ensure that despite where a patient lives in the world the essential elements are met.

2. Advocacy can mean different things to different patients, it is about walking alongside the patient, guided by them to achieve their goals.

3. The Rare Disease community is just that, a community, all the different stakeholders have the patient at the centre. Patient advocacy, as an independent body should seek to drive the agenda to ensure that the community work together to achieve the best outcomes for the patient.