The management of rare disease patients from a grassroot perspective: the role of patients' organizations in the global recognition of rare diseases in Cameroon.

INTRODUCTION: rare diseases (RD) are extremely complex health conditions. Persons affected by these conditions in Cameroon are often neglected in society and health systems through the inexistence of policies and programs. In Cameroon, there exists no program or policy conceived to address their needs in terms of access to quality health care, timely and reliable diagnosis, treatments, education, etc. The consequence is that persons living with a RD (PLWRD) and their families do not participate in social life. The unique fate of PLWRD reveals that the principle of social justice and equity is flawed in Cameroon. However, patients, in order to survive in society, rely on patients' organizations (PO) to improve their quality of life (QoL) and advocate for a better consideration in the society. The aim of this paper is to highlight how initiatives from a grassroot perspective like POs can inform decision-makers to address the needs of PLWRD and their families.

METHODS: the study associated a systematic literature review and semi-structured interviews with parents of children suffering from a RD and who are members of a PO. Through the systematic literature review we highlighted the impact POs have in the development of research on RDs, patient literacy, patient empowerment and advocacy while semi-structured interviews brought out the needs of patients and their families.

RESULTS: findings, on the one hand show that, in Cameroon PLWRD face a number of challenges like the incurability of their condition, catastrophic medical expenses, stigmatization and marginalization, etc. and though in POs their QoL still remains poor. On the other hand, where POs are empowered they are key actors in research on RDs and help decision-makers on having a better insight into the type of RD that exists across a geographical area, the sociodemographic profile of patients, etc. for a better management of PLWRD.

CONCLUSION: the study suggests that the ministry of public health should create a network with existing RD POs to adequately meet the needs of PLWRD.