Among the various products that the U.S. Food and Drug Administration (FDA) regulates are brand-name and generic prescription medication and non-prescription medication sold over the counter. Under the FDA, the Center for Drug Evaluation and Research (CDER) is the consumer watchdog responsible for ensuring the quality, safety, and effectiveness of all medication marketed in the country.
Medication Development and Approval
Companies who intend to introduce a new medication to the U.S. market bear the responsibility of having it laboratory-tested to check whether it is safe and performs as intended. The medication is then tested on people to determine its effectiveness in treating disease and to observe any side effects. The company submits evidence from these tests with an application to CDER.
The pharmacologists, chemists, physicians, statisticians, and other scientists of CDER do an independent and unbiased review of the data and the proposed labeling for the medication. The FDA approves a medication for sale if the CDER review shows that its health benefits outweigh the known and possible risks for the intended users.
CDER usually requires results from two separate and well-designed clinical trials to eliminate bias or chance. If the medication is for a rare disease, though, and it is not feasible to do multiple trials, CDER can accept compelling evidence from one trial that shows the benefits compensating for the risks.
The assessment of risks and benefits also depends on the analysis of the medication against the illness it is for and other available treatments. For instance, medication for an illness with other existing treatments will undergo more stringent standards than medication for a life-threatening condition with no existing medication.
Because all medications have risks, one of the risk management strategies required by the FDA is an approved label that clearly describes the medicine’s benefits and risks and how to detect and manage such risks. If the risk is high, the FDA can require the company to execute a Risk Management and Mitigation Strategy (REMS).
For instance, if there is a risk of a serious adverse reaction to medicine within three hours from intake, the company can require its administration in a certified health care facility where the patient can stay for observation for three hours afterward.
Prioritizing Medications Based on Need
The FDA can expedite the release of a new medication for a life-threatening illness through Accelerated Approval if the medicine is for long-term use. A clinical trial will take a prolonged period to show effectiveness. In such cases, the FDA requires the company to carry out post-marketing clinical trials to prove the medication’s value. If the trials do not substantiate the company’s claims, the FDA can withdraw its approval.
The FDA can also do a Priority Review for a medication, meaning the review will be within six months instead of the standard 10 months. This is for medications that radically improve the prevention, diagnosis, or treatment of serious illnesses.
A company can request a Breakthrough Therapy designation for medication that is a significant improvement over available treatments for a serious illness. This will expedite its review. If granted, the company can likewise request a Fast Track process for medication that fills an unmet need.
Medication Performance Tests
Performance tests are a requirement for medicines from the development stage to the rest of their market life. Dissolution testing checks how much of the active ingredients of the medicine are being released over a certain period in its dosage form under standard conditions and how this is being absorbed through the patient’s gastrointestinal tract.
During drug development, the manufacturer can see how absorbable the active pharmaceutical ingredient in the formulation is and whether there is not enough or too much of it. Too little will mean the medicine will be ineffective, while too much can cause unwanted side effects. The manufacturer will also see how the medication’s release mechanism performs. Formulations are then adjusted accordingly. This process is also followed for new formulations or changes in dosage form of the same medication, for instance, from tablet to capsule form.
Even after the medication is out on the market, tests continue to ensure that all lots are always of the same quality. Tests also check product stability, especially when scaling up manufacturing or transferring manufacturing sites.
Industry standards for testing come from the United States Pharmacopeia (USP), including four apparatus setups. The USP also has its National Formulary (USP–NF), a collection of official standards on dissolution, disintegration, and drug release requirements.
It is reassuring to know that experts and the authorities thoroughly vet the medications available to the public for quality and safety before their release to the market. It is, therefore, crucial to check that medication has FDA approval in its label before intake.