What Is an Orphan Drug?
The Orphan Drug Act defines an orphan disease as a condition that affects fewer than 200,000 persons in the United States*. More than 5,000 of these rare conditions exist in about 20 million Americans, according to the National Organization for Rare Disorders (NORD). Because no one would "adopt" the products to treat these diseases in the days before the law, they became known as "orphans."
An orphan disease may affect only a few thousand people--some, such as infant botulism, have patient populations of less than a hundred--so the potential for a company to profit from developing an orphan treatment is small.
This means that few firms, including large pharmaceutical companies, have been interested in investing the time and money in orphan products.
So for years, patients suffering from orphan diseases such as Gaucher's disease, rare cancers, hemophilia, multiple sclerosis, and Parkinson's disease simply were out of luck. With no financial incentives available, companies couldn't risk the investment. Other possible development outlets, such as universities or research hospitals, often lacked the capital or business savvy to develop treatments for small patient groups1.
How does CHR fit in?
CHR incorporated has worked closely with physicians, patients, patent holders, and pharmaceutical companies to develop orphan drug products. We have been very pleased to see these products ultimately approved by the Food and Drug Administration. As part of a team working on Orphan Drug Development, we have provided the following services:
- Medical Chart Review
- Data Extraction
- Database Generation and Data Analysis
- Pharmacokinetic Study Design and Analysis
- Clinical Report Writing
- Participation in Meetings with FDA
- NDA Review
- Labeling Review
- Investigational New Drug Application Preparation
Why Does CHR handle this process so well?
We are an efficient, focused, and experienced Orphan Drug research group, that does not have the layers of bureaucracy found at other Contract Research Organizations. We understand the limited scope of Orphan Drug Projects, the difficulties associated in proving safety and efficacy in a limited number of patients, and the challenge of providing drug and clinical trial supplies for these projects. By taking a common-sense approach to solving these problems, while maintaining high scientific standards, we have worked with groups to overcome these barriers.
References:
*FDA Consumer magazine May-June 1999 at http://www.fda.gov/fbac/features/1999/399_orph.html
