P3.110: The role of medical students in advocacy for rare diseases – Experience from a Low- and Middle-Income Country (LMIC)

Background: Over 300 million people worldwide live with rare diseases. The annual celebration of rare disease day allows communities to advocate for visibility, inclusion and better healthcare for those with lesser-known conditions. High rates of consanguineous marriages in Pakistan increase the prevalence of rare diseases. Having a rare disease and living in a developing country like Pakistan predisposes those with rare diseases to many challenges. In this light, learning from patient journeys and advocating for rare diseases is a step in the right direction to better care. We share experiences from Pakistan of medical students spearheading one such effort as stakeholders in the care for rare. We also discuss the importance of teaching health advocacy as part of the medical education framework, helping nurture empathy in medical students, who we hope to be leaders of better care in the future.

Project Description: WHO recommends communicating early, at the right time and then building on the conversation to effectively communicate messages to the public; hence, the Pediatrics For Life (PFL) interest group at the Aga Khan University (AKU), engaged in activities over the span of 3 weeks. The first phase was improving social media visibility; infographics were shared and viewed by over 800 young adults and medical students. The second phase was the ‘Launch of Newborn Screening Program for Selected Inherited Metabolic Disorders’, where we encouraged medical students to wear zebra-stripes, and dip their hands in the blue, green, pink and purple paint onto a giant canvas. For the final phase, we invited four individuals with rare diseases to share their experiences with an audience of 26 medical students. The conversation shed light on exhausting diagnostic journeys, the psychological impact of receiving a diagnosis, inequity in available opportunities becoming apparent as they age, and the support that rare-disease community groups offered, giving the audience a chance to walk in their shoes for a moment and empathize with them.

Discussion: Medical students must be equipped with skills to advocate for patients and communities, such as understanding multidisciplinary factors that impede early disease recognition, accumulate systemic biases, and accentuate health disparities. Evidence-based advocacy training is essential for future policymakers to effect meaningful change. Medical students often encounter rare diseases only in textbooks, but hearing personal stories and engaging in the activities helped shift their perspectives, and will make them more empathetic health care physicians.

Conclusion: Individuals with rare diseases are experts in their diseases and there is much that we can learn from them to tailor the care we provide according to their needs. Evidence based advocacy by medical students will mean that they understand the dynamics of policy making and are able to successfully maneuver their way to a favorable position for their patients and the community.