| Sample User Question |
 |
|
What orphan drug is available for sickle cell anemia and in what country is this drug available? In what languages is information for the public available on sickle cell anemia?
|
| Analysis/Comments |
|
|
Orphan drugs are used to treat rare diseases, and their development may not offer the pharmaceutical industry a return on the research investment. The Orphanet database provides clinicians with the ability to search for Orphan drugs that may not be actively marketed to U.S. healthcare practitioners. Orphanet also provides a resource to locate orphan drugs that are being used in non-U.S. countries, but have not yet been approved by the FDA.
|
| Step By Step Guide |
|
OrphaNet
- From the Orphanet homepage click on orphan drugs
- From the sidebar, click under the Services for:
- Click on Professionals
- Now click on Search by disease
- Enter sickle cell anemia in the disease search box and enter on search
- Click on the sickle cell anaemia link
- Note that British spellings are used. If you don't initially locate a disease by the search box, try using the alphabetic listing
- Click on the drugs link
- Click on the links for Droxia, Exjade, and Hemoxin
- These are all American orphan drugs
- Click on Nicosan
- Note that this orphan drug is not listed as a U.S. orphan drug.
- Click on Siklos
- Note that in August 2007 this was only listed as a European orphan drug
- Use the back arrow until you are back at the listing page for sickle cell anemia
- A description of the disease is also provided in French
|
|
