Rare diseases (RDs) are complex conditions and a worldwide healthcare challenge. The healthcare policymakers in developing countries lack templates from countries at the same level of development. This article introduced and discussed the combination of top‐down strategies and bottom‐up interventions in addressing RDs in a developing country, China, as an example. The government leads the formulation of laws, policies, and guidance to coordinate national resources, while local authorities and nongovernment organisations (NGOs) are responsible for policy localisation and complement policy gaps. This article may inspire other developing countries of improving RD healthcare.