SOCIAL PARTICIPATION IN HTA FOR RD
in Latin America

Rare diseases (RDs) are low prevalence conditions, with a precise threshold that varies nationally and regionally. However, although each of the nearly seven thousand recognized RDs presents in a small number of individuals, these illnesses together impact around 50 million patients in Latin America (LA). Patients living with RDs often require constant monitoring, care, and attention. Caregivers, usually family members, provide critical support.

Drugs for RDs offer essential health benefits but challenge traditional health technology assessment (HTA), reimbursement, and pricing processes. RD research cannot meet the sample group and control versus experimental group sizes that generalized statistical analysis practices require because, by definition, a small number of people live with each RD.

Public and patient involvement (PPI) in the health HTA process is increasingly encouraged. The selection of topics for assessment, which includes identifying and prioritizing HTA questions, is a constant challenge for HTA agencies, especially in the case of RDs. PPI in these early stages of HTAs regarding RDs could make assessments more relevant and acceptable. Their participation in developing the assessment plan is crucial to optimize their influence and impact on HTA research.

To examine this challenge and create recommendations, the Americas Health Foundation conducted a virtual meeting with five LA experts in the fields of RD, HTA and economics to develop a manuscript to assess the gaps and provide actionable steps to closing them. The resulting manuscript, “Their voices matter: Challenges & opportunities for rare disease health technology assessment public and patient involvement in Latin America,” is under review for publication.