FDA Approval Process

The FDA Drug and Biologic Approval Process: A hot topic

The high cost of prescription drugs is a hotly debated issue and poses a significant burden to individuals, families, and the federal government. Drug costs have led to Medicare Part D, a federally subsidized program meant to defray the costs of prescription drugs, particularly to the elderly. Though there are many contributing factors drug prices, their expense is due in part to the cost involved in completing the FDA approval process.
In order for pharmaceutical and biotech companies to market their drugs and biologics, companies must receive FDA approval, a rigorous, expensive, and time consuming process that can take over a decade to complete. Of 5000 compounds discovered in the pre-clinical stage, only about 5 will make it through the entire FDA approval process. Therefore, companies have to cover not only the cost of successful development of a single drug, but of many drugs that never make it to market.

The Phases in the FDA Approval Process

Pre-Clinical Phase

In the pre-clinical or drug discovery phase of the approval process, researchers look for potential new compounds to treat targeted diseases. Once a compound has been identified and refined to a formula that can be tolerated by humans, its toxicology is tested in animals and living tissue. The process takes roughly three and a half years. During this phase researchers look for:

  • correct dosage level
  • how frequently it should be administered
  • the best delivery system (oral, topical, intravenous, etc.)
  • short- and long-term survival of the animals

After pre-clinical testing is completed, the company then files an Investigational New Drug Application (IND) with the FDA. Fast Track Designation is an expedited review of a drug that is given to a company whose drug or biologic makes both a product and a marketing claim that addresses an unmet medical need. It can be granted at any point after the FDA approves an IND.

Phase I

If the FDA approves the IND, the experimental drug then moves into Phase I human testing. In this phase, the drug is tested in a small number (under 100) of healthy participants. Researchers look to see how well the drug is tolerated, how it is processed by the human body, and the correct dosing. This process takes a year.

Phase II

Once a compound is found to be well tolerated in healthy individuals, it is then tested for effectiveness for a targeted disease in a small number of patients.  In this phase 100-300 people are administered the investigational drug to see if it actually works, and to determine its short-term effects. This process takes about two years.

Phase III

Phase III is a large-scale study of the effectiveness and side effects of the drug in a larger population, usually ranging from 1000-3000 patients. If the drug is submitted to the FDA for approval, the FDA will look at the Phase III data to determine if the drug is safe and effective. Aside from testing the drug’s viability, the company producing the drug also determines the logistics involved in creating a large supply of the treatment.  Phase III of the FDA approval process takes about three years.

New Drug Application (NDA)/ Biologics License Application (BLA)

If the drug proves to be safe and effective, the company then files an NDA or BLA with the FDA. NDAs and BLAs are typically 100,000 pages long and include results of human and animal trials as well as information on how the drug is manufactured. It usually takes the FDA 1-2 years to complete the review process and approve a drug. However, there are cases when approval can be accelerated.

  • At the time of application Priority Review can be granted to drugs that treat an unmet medical need.
  • Orphan Drug Status is granted to drugs that treat rare diseases, or diseases that have no other available treatments.

Phase IV

Once a drug has received FDA approval it is then marketed to the general population. Short- and long-term side effects continue to be monitored and results are submitted to the FDA. Companies will also look for additional indication for the drug. In order for the drug to be approved for a new indication, it must receive approval from the FDA.

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