The acceleration of clinical trials studying rare diseases over the past 3 decades has occurred largely because of the Orphan Drug Act, which was enacted in 1983 as a result of patient advocacy groups working with congressional sponsors.1 This act facilitated the development and commercialization of products (drugs, vaccines, and diagnostic agents) to treat populations with rare diseases, defined as those affecting fewer than 200 000 US residents, or those without reasonable expectation that drug development costs will be recoverable by US sales. The act has resulted in more than 3900 orphan product designation requests to the US Food and Drug Administration (FDA) with the approval of more than 450 products to treat approximately 250 rare disorders, of which approximately one-quarter in the past decade have been for pediatric diseases.2,3
Some tools below are only available to our subscribers or users with an online account.
Download citation file:
Web of Science® Times Cited: 2
Customize your page view by dragging & repositioning the boxes below.
More Listings atJAMACareerCenter.com >
Enter your username and email address. We'll send you a link to reset your password.
Enter your username and email address. We'll send instructions on how to reset your password to the email address we have on record.
Athens and Shibboleth are access management services that provide single sign-on to protected resources. They replace the multiple user names and passwords necessary to access subscription-based content with a single user name and password that can be entered once per session. It operates independently of a user's location or IP address. If your institution uses Athens or Shibboleth authentication, please contact your site administrator to receive your user name and password.