Clinical Trial / Drug Approval Terms
You may see these terms in articles, research studies or social media posts. Here's what they mean:
Clinical Trial
A research study in humans to test how safe and effective a drug is before it can be approved for use.
Fast Track
A program to speed up the review of drugs that treat serious conditions and fill an unmet medical need. Helps patients get access sooner.
Confirmatory Trial
A required study after a drug receives Accelerated Approval to confirm that it provides real clinical benefits. If the trial fails, the drug’s approval may be withdrawn.
Expanded Access (Compassionate Use)
A way for patients with serious conditions to access experimental drugs outside of clinical trials when no other options exist.
Inclusion Criteria
The specific requirements a person must meet to join a clinical trial, such as age, disease stage, or medical history.
Priority Review
Cuts the FDA’s review time from 10 months to 6 months for drugs that could offer significant treatment benefits.
Orphan Drug Designation
Special status for drugs treating rare diseases (affecting fewer than 200,000 people in the U.S.), providing incentives to encourage drug development.
Drug Label (Prescribing Information)
The official information about a drug’s approved uses, risks, dosage, and other important details.
Insurance Reimbursement
Exclusion Criteria
Factors that prevent a person from participating in a clinical trial, like certain health conditions or prior treatments.
Accelerated Approval
Allows earlier approval based on signs that the drug may work, with additional studies required after approval to confirm the benefits.
Pediatric Rare Disease Priority Review Voucher (PRV)
A reward given to companies developing drugs for rare childhood diseases, allowing them to speed up the review of another drug.
Off-Label Use
When a doctor prescribes a drug for a condition, dosage, or patient group that is not officially approved by the FDA. This is legal but may not be covered by insurance.
Coverage and payment for a drug by health insurance companies. Some drugs, especially new or off-label treatments, may require special approval or appeals to get covered.