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and regulatory programs. Follow-up conversations to the Protea Declaration call for action focused on
identifying regions of the world in need of additional emphasis on RD research, diagnosis, and access to
orphan drugs beyond Africa; one of these regions is Latin America and the Caribbean.

The ERCAL initiative was therefore created in partnership with ICORD in February of 2020 to increase
awareness and with an emphasis on developing an agenda for rare diseases in LAC. In 2021, ERCAL became
an independent initiative with the mission to improve the lives of people living with RDs and their families
in the LAC region through collaboration, outreach, advocacy, and education, improving the access to
diagnosis, research, care, treatments, and services.

Between 2020 and 2021, a template was developed for investigators, clinicians, and leaders of patient
advocacy groups in LAC to elaborate on and present through video conference the specific issues in their
countries, in order to identify the themes that would guide the ERCAL initiative. The specific issues
discussed during these country-specific presentations included: (1) status of existing legislation, regulations,
and policies in each country; (2) national research and regulatory activities and areas of emphasis; (3)
challenges, barriers, or obstacles to needs of the rare diseases community; (4) legislative or policy needs in
research, regulatory, or healthcare services; (5) opportunities for future research collaboration; and (6)
suggested future activities for the country, the region, and a sustainable ERCAL initiative. Thus, a
comprehensive understanding of the landscape of RDs in the region was accomplished. In this way,
patients, patient representatives, organizations, researchers, clinicians, regulators, and all interested
stakeholders in the RD ecosystem in LAC were called upon to come together in a common collaborative
platform to sum efforts and work on the challenges faced by patients and families living with RDs in the
region.

Current activities and efforts
Over the last four years, ERCAL has brought together patients, organizations, clinicians, researchers,
advocates, industry representatives, and other stakeholders in the RD ecosystem in LAC through video
conferences and the co-organization of a very successful hybrid 1st Congress on RareDiseases in Latin
[9]
America and the Caribbean (#CongresoERCAL) in March of 2023 in Bogota, Colombia .

Including the voice and representation of patients and patient organizations as part of ERCAL has been a
priority since the establishment of the initiative to hear directly from them about the major challenges the
RD community in LAC faces and promote their involvement and empowerment to develop proposals and
solutions to address such issues. ERCAL considers individual and organization membership, which can be
requested by filling in an interest questionnaire on the website. Currently, the membership includes more
than 50 affiliated individuals representing 20 of the LAC countries plus US members [Figure 1].
Additionally, 20 allied patient or advocacy organizations across LAC are uniting efforts as part of ERCAL
and working towards solutions to the challenges faced by patients and families living with RDs in the
region. In following ERCAL’s principles of inclusivity and collaboration, ERCAL welcomes individual or
organization members who want to collaborate and pool efforts to advance the agenda of rare and low-
prevalence diseases in LAC. Current allied organizations range from disease-specific patient organizations,
larger umbrella organizations and federations, and other advocacy organizations that have voluntarily
requested membership as part of the ERCAL regional initiative.

Since 2020, the ERCAL leadership and membership have worked on identifying the major topics of
relevance and need to advance the rare diseases agenda in LAC and pinpoint the priority projects to work
on. The work of ERCAL members and projects are organized around Working Groups focused on eight