Guidelines for Rare Diseases Now Available!

The PQMD Rare Diseases Guidelines offer a practical framework for organizations working to improve the lives of people affected by rare diseases worldwide. First developed through the GARDaccess initiative, the Guidelines have been further refined through collaboration among PQMD members in the Rare Diseases Working Group, including leaders from the private sector, global health, humanitarian response, and the rare disease community and patient advocacy.

The Guidelines provide clear, actionable recommendations to expand access to medicines for patients in low-resource settings by strengthening diagnosis, treatment, policy, and patient support. They are also intended to support the systematic development of tools that enable access initiatives. For nonprofits, healthcare providers, government agencies, and private-sector partners, the Guidelines and accompanying tools help turn commitment into meaningful impact, build sustainable access, and ensure that no person living with a rare disease is left behind or denied equitable access to quality healthcare.