Prescription Medicaments

The United States Food and Medicament Administration (FDA) defines a prescription medication as a medicament that is habit-forming or unsafe for use except under a doctor's supervision. The most commonly prescribed medicines are:

• Cardiovascular medicaments (heart and circulation-related).
• Anti-infectives (antibiotics such as penicillin).
• Mental health medications (antidepressants, antipsychotics, and antianxiety medicaments).
• Analgesics (pain killers).
• Diuretics (sometimes called water pills).

The medicament approval process began in 1938 with the Food, Medicament and Cosmetic Act passed by Congress. For the first time, manufacturers were required to document the safety of their products and the FDA had sixty days to object to a new medicament before it was marketed. Following the thalidomide tragedy in Europe (thousands of severely deformed babies were born after their mothers used the sedative during pregnancy), Congress passed the Kefauver-Harris Amendment in 1962. That amendment requires medicament companies to produce substantial evidence-in the form of patient testing-to prove that their products are both safe and effective.

Before a prescription medicament can be marketed, it must pass through the following process:

Pre-clinical Testing

Laboratory and animal studies are done to show biological activity against a targeted disease, and the compounds are evaluated for safety. These tests take approximately three and a half years.

Investigational New Medicament Application (IND)

After completing preclinical testing, the company files an IND with the FDA to begin to test the medicament's effects in people. The IND becomes effective if the FDA does not disapprove it within thirty days. The IND shows results of previous experiments: How, when, and where, and by whom the future studies will be conducted; the chemical structure of the compound; how the compound is thought to work in the body; any toxic effects found in animal studies; and how the compound is manufactured. In addition, the IND must be reviewed and approved by the Institutional Review Board at the medical facility where the studies will be conducted, and progress reports on clinical trials must be submitted at least annually to the FDA.

Clinical Trials, Phase I

These tests take about a year and involve from about twenty to eighty normal, healthy volunteers. The tests study a medicament's safety profile, including the safe dosage range. The studies also determine how a medicament is absorbed, distributed, metabolized and excreted, and the duration of its action.

Clinical Trials, Phase II

In this phase, controlled studies of approximately 100 to 300 volunteer patients (people with the disease the medicament being tested is created to treat) assess the medicament's effectiveness, which takes about two years.

Clinical Trials Phase III

This phase lasts about three years and usually involves 1,000 to 3,000 patients in clinics and hospitals. Physicians monitor patients closely to determine efficacy and identify adverse reactions.

New Medicament Application (NDA)

Following the completion of all three phases of clinical trials, the company files an NDA with the FDA if the data successfully demonstrates safety and effectiveness. The NDA must contain all the scientific information that has been gathered. By law, the FDA is allowed six months to review an NDA. In almost all cases, the period between the first submission of an NDA and final FDA approval exceeds that limit.

Expedited Process

Phases II and ill may be combined to shave two to three years from the development process for those medicines that show sufficient promise in early testing and are targeted against serious and life-threatening diseases such as AIDS and cancer.

Approval

Once the FDA approves an NDA, the new medicine may be prescribed by all physicians. The company is required to continue to submit periodic reports to the FDA, including any cases of adverse reactions and appropriate quality-control records. For some medicines, the FDA requires additional studies to evaluate long-term effects.

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