With new legislative initiatives from regulatory bodies in a number of healthcare markets, coupled with a high-unmet medical need, pharmaceutical companies are continuing to invest in the development of orphan drugs. This article provides an analysis of key 2019 orphan drug approvals, using IQVIA Pipeline Intelligence data.
Rare diseases, for which orphan drugs are indicated to treat, are characterized by a low frequency of occurrence in the general population of a geographical region. There are over 8000 described rare diseases, of which only 340 conditions currently have treatment options1. Some of these diseases, such as progeria, occur in 1 in every 4 million births, while others may be comparatively more common, such as multiple sclerosis, cystic fibrosis and Duchenne muscular dystrophy. The prevalence of these diseases varies between populations, making an indication rare for one region but common for another. The effects of these rare diseases range from premature mortality to severe disability due to a lack of efficacious treatment options. Growing understanding of numerous aspects of these diseases, along with improving government policies, are expanding the framework for research, development and market penetration through the introduction of novel and renovated therapies.
2019 Orphan Drug Approval Overview
The improvement in government policies over the years has been mirrored by an increase in the development activities for candidates addressing rare diseases; with less than 10 approved rare disease therapies on the market in the 1970s increasing to more than 830 US FDA approved orphan therapies as of 20192. New treatment options have been brought to market through the launch of either new molecular entities or through the renovation of older, established therapies.