Executive Summary - Solidarity Amidst Rarity: Reforms for Equitable and Sustainable Rare Disease Management

Due to a combination of a lack of public awareness, a subpar standard of knowledge among professionals, and a dearth of diagnostic facilities, rare disease patients face significant delays in healthcare access. [...] With the majority of healthcare facilities and expertise concentrated in the Klang Valley, location has become a prohibitive physical barrier for those residing outside the vicinity. [...] This, coupled with the lack of entitlement to the usual forms of social healthcare protection, result in rare disease patients being left to fend for themselves at the expense of their physical, financial, and emotional well-being. [...] In order to identify and measure healthcare access for rare diseases throughout the patient pathway, this study employed Levesque’s framework to conceptualise healthcare access from the perspectives of the healthcare system and patients. [...] Lastly, in the long-term, the country’s social protection systems (Pillar 4) need to be enhanced to empower and facilitate the resilience of individuals with rare diseases along with their caregivers.