Patient and Stakeholder Engagement

PM360 – Patient Advocates Assume a Large Role as Orphan Drug Development Expands

“Rare disease” may be the biggest misnomer in the pharmaceutical industry. Around the world, 350 million people live with conditions classified as rare because each afflicts a relatively small segment of the population. In the U.S., 30 million—nearly one in 10 Americans—suffer from one of about 7,000 known rare diseases.

As it applies to this population, “rare” most aptly describes the 400 or so drugs approved to treat these thousands of disorders. That most of these diseases lack a single approved therapy reflects the enormous cost associated with their development among other considerations, and the extremely poor odds of success: More than 90% of the compounds that reach clinical testing fail—and many more never get that far.

But with legislation encouraging innovation in this space, companies keep trying and 2015 was a landmark year for orphan drug development, with 21 compounds approved in the U.S. and 18 in the European Union. The pharma industry forecasts a 10.5% compound annual growth rate for rare disease products through 2020, and worldwide orphan drug sales are projected to reach almost 15% of the overall prescription market in 2016 versus less than 5% in 2000.

The government strongly encourages the trend. The FDA now receives orphan drug designation requests at a rate of more than one a day and has granted orphan status more than 3,600 times since 1983. However, with the number of approved compounds still numbering just dozens annually, there’s a long way to go—and patient advocates are stepping in and playing an expanding role in drug development and regulatory review.

Read more at PM360.