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Statement for Rare Disease Day

Statement by Dr Tedros Adhanom Ghebreyesus, WHO Director-General

The vision of the Sustainable Development Goals is a world in which no one is left behind, including people who suffer from rare diseases. Just because a disease affects a small number of people does not make it irrelevant or less important than diseases that affect millions.

Rare diseases present fundamentally different challenges from those of more common diseases, especially for diagnosis. The small number of patients, the logistics involved in reaching widely dispersed patients, the lack of validated biomarkers and surrogate end-points, and the lack clinical expertise and expert centres all present significant barriers.

Medical expertise for each of these diseases is a scarce resource. Fragmented disease knowledge makes it critical that investments in research go hand-in-hand with investments in dedicated infrastructure and international networks such as biobanks, registries and networks of expertise. Where needed, these networks can also provide opportunities to train health professionals on rare diseases.

WHO’s top priority is to support countries on the path towards universal health coverage, with the aim of ensuring that all people can access the health services they need, when and where they need them, without facing financial hardship. This includes access to diagnosis and treatment for people who suffer from rare diseases. Through the Fair Pricing Forum, WHO is seeking to foster dialogue between regulators, insurers, pharmaceutical companies and patient groups to ensure sustainable access to medicines, including orphan drugs. We welcome further discussions with the rare diseases community about how we can strengthen cooperation to ensure people with rare diseases can access the health services they need.

Thank you for your commitment to raising the awareness of rare diseases and contributing to a world in which no one is left behind.