Rare diseases are not uncommon, and may be the mother lode for the world’s pharmaceutical producers and researchers
- Up to 30 million people live with as many as 7,000 types of rare diseases in the United States, the world’s largest and most advanced pharmaceutical market
- US market for rare-disease drugs is estimated at US$43 billion, just under 10 per cent of the nation’s total drug spending
Rare diseases may individually affect a relatively small number of patients compared to common ailments but the overall burden on society is heavy and drug developers should not ignore its market potential, says policy advocate.
“Rare diseases are not that rare when one considers the fact that each patient’s disease also has a big impact on each of their family members and carers,” said Li Linkang, executive director of China Alliance for Rare Disease. “Medical experts have made a conservative estimate that China has some 20 million rare disease patients, with around 200,000 new cases per year.
“The real numbers should be far higher as misdiagnoses and missed diagnoses are common … most doctors have no experience with rare diseases and it takes three to five years for most patients to be diagnosed, which is too late.”
The non-profit body is collecting nationwide data on rare disease prevalence to help government officials come up with policies to make drugs more affordable and accessible, and incentivise companies to invest in drug discovery programmes.
In the United States, the world’s largest and most advanced pharmaceutical market, between 25 million to 30 million people live with as many as 7,000 types of rare diseases, according to the Genetic and Rare Disease Information Centre funded by the Department of Health and Human Services.