What is a Clinical Trial?
The FDA watches over clinical trials and creates some of the rules for how they’re run.
The FDA decides yes or no to any new medicine, and researchers must get approval from both the FDA and the designated institutional review board (also known as IRB in researcher-speak) before starting a clinical trial.
The FDA makes the final decision as to whether a new medicine can be sold in the U.S. They look at the results from all the studies and decide if the medication is safe, and if it works.
There are many ethical rules designed to protect people that are participating in research. These rules make sure that:
- Consent is voluntary, which means each participant chose and agreed on their own to be a part of the study.
- Any possible harm to participants is made as unlikely as possible.
- Researchers use the right study design to answer the questions being asked by the study.
- Participants can choose to stop participating in the research at any time.
Orphan Drug Designation
A drug is considered an “orphan” if it’s meant to treat medical conditions that are so rare (affecting fewer than 200,000 people) there is little chance of profitability for the drug developer. The term “orphan disease” refers to these rare conditions.
The FDA supports the development of new treatments for rare diseases and has the authority to grant orphan-drug designation.
What is Orphan Drug Designation? The FDA grants orphan-drug designation to a drug or biological product. This designation qualifies the sponsor for various incentives, including tax credits, exemptions from fees, and market exclusivity for seven years post-approval of the product. This is a regulatory mechanism that helps expedite drug development in rare diseases, but does not mean the product is close to clinical trials or approved by the FDA.
Frequently Asked Questions