Rare Diseases in Latin America: Challenges and opportunities for the equitable attention and proposal of Patients Organizations 

The emerging concept of Rare Disease (RD) is used to define diseases with a low prevalence and a highly severe and disabling chronic course. Such diseases have certain characteristics besides the small number of affected people: patients frequently are diagnosed with a Rare Diseases in early childhood, Rare Diseases affect multiple organ systems, produce disability, reduce both life expectancy and quality of life as well as harming physical and mental capacities. People with Rare Diseases, generally, require support to develop basic and advanced activities of daily living, mainly in their domestic life (44.06%), displacements (42.10%), or mobility (39.58%). Only one in ten of affected people do not require any type of support. 

Rare Diseases impose common problems and strong equity challenges to Health Systems and social protection, in the mark of distributive justice. Latin America does not escape from these challenges, which intensify due to our demographical, geographical, economic, cultural, and social characteristics. 

In all domains and due to the minority characteristics of RD population and its number of vulnerabilities, the measures taken require two fundamental principles: differential focus and integral recognition from the population. Amidst this focus, patient organizations play a crucial role in the recognition and provision of social solutions in Latin America. 

The generation and exchange of knowledge around the social and health phenomenon of RD is urgent, in order to promote the debate relating to themes of equity and social protection for the Latin American community who suffer these diseases; as well as to determine better practices for the development of coherent and systematic public policies. 

 The solutions should be practical, applicable, and should transcend not only the geographic borders, but the association borders, the borders of particular interest for certain pathologies and, of course, the borders of knowledge in all of the domains: social, political, economic, and scientific. 

The proposal of creation of a CoP for RD is profiled as a viable solution to facilitate the interaction, collaboration, and cooperation between different agents, looking for the innovation in solutions of formulation of national, regional, and global public policy following effective patterns for this formulation. 

Source: ISPOR  website  http://press.ispor.org/LatinAmerica/2017/09/rare-diseases-in-latin-america-challenges-and-opportunities-for-the-equitable-attention-and-proposal-of-patients-organizations/