More than 560 meds in pipeline for rare diseases, US report states

There are more than 560 novel treatments in development to treat the 30 million Americans currently living with a rare disease that has few or no treatment options, according to a new report.

Pharmaceutical Research and Manufacturers of America (PhRMA), the trade group representing pharma companies in the USA, published the findings in its report entitled Medicines in Development for Rare Diseases.

The report analyses 7,000 known rare diseases – which are classified as those affecting fewer than 200,000 people in the USA – and finds that, due to advances in science, there is now promise for brighter futures for American patients and families.