While it may come as a surprise to majority of people, it holds true. It’s imperative to note, that not every unapproved drug out there can be used in patient treatment, and FDA itself has clear guidelines of when it’s acceptable.
The exception is granted under one of the below conditions:
- The drug is involved in an open drug efficacy study implementation program (DESI)- a program aimed at evaluating the efficacy of drugs that have been on the market prior to 1962
- Healthcare professionals rely on the drug in treatment of serious medical conditions, where there are no drugs available for treatment of this conditions
- There is an insufficient supply of a certain FDA approved drug
- The use is authorized by the U.S. Secretary in diagnosis, monitoring, and treatment of serious conditions in an emergency situation
An individual patient can also request a drug, that is currently unapproved, through a licensed physician. Certain criteria must be met in order to accomplish that:
- It must be determined that there is no alternative or comparable treatment currently on the market, and the risk of using the drug/device outweighs the risk from an untreated condition/disease
- A sufficient evidence of safety and effectiveness of the drug through clinical trials
- Use of the drug does not interfere with ongoing clinical investigations to support marketing approval
- The sponsor, or the investigator, submits to the Secretary a description of use of the drug by a single individual, or a group of patients
When it comes to a more widespread use of unapproved drugs in a group of eligible patients, the sponsor, or physician, must prove to the Secretary, that the drug is intended for use in a life-threatening disease/condition, that there is no alternative therapy, that all the necessary clinical trials have been completed, and the sponsor is currently pursuing marketing authorization. Furthermore, a sufficient evidence of safety and efficacy of the drug must be provided.
While it may seem counterintuitive, the use of unapproved drugs is under constant control and very strict regulations from both, the FDA, and the U.S government itself. Each case of use of such drug is thoroughly studied, and the decisions are made on a case-by-case basis by the U.S. Secretary.
Such method of regulation ensures both, a tight enough control over unapproved medications, while still allowing for the special patient needs to be met, in case of an emerging necessity.