Today, the process of bringing a drug to a patient's bedside takes an average of 8.5 years, costs about $500 million, and includes a rigorous review by the FDA. With drug development starting in laboratories, where scientists probe the effects of chemical compounds on enzymes, cell cultures or other substances involved in the disease whose treatment they seek. The potentially effective chemicals are then tested in two or more species of animals to determine whether they can be safely used in humans. No more than 5 in 5,000 tested compounds pass these preclinical trials and are proposed for clinical studies. If the FDA finds the approach promising and an institutional review board of scientists, ethicists, and health-care specialists approves the sponsor's study protocol, the drug enters a progression of tests in humans. Each new trial phase is predicated on a successful outcome of the previous one:
Phase III studies- seek to determine the drug's safety, effectiveness and dosage.
The results of Phase III trials are submitted to the FDA for review by a team of chemists, physicians, epidemiologists and other specialists. This group's task is to judge whether the trials have demonstrated that the product's health benefits outweigh its risks. Only products that pass this test may be approved for marketing. For more information, visit www.fda.gov/cder/handbook/.
The FDA frequently uses timesaving processes for speeding important new drugs to patients who need them. Accelerated approval may be granted to priority drugs that show promise in the treatment of serious and life-threatening diseases for which there is no adequate therapy. Treatment Investigational New Drug (IND) designations enable patients not enrolled in the clinical trials to use promising life-saving drugs while they are still in the testing stage.
The nation's more than $40 billion annual investment in biomolecular research is fueling a deluge of promising new medications that will eventually land at the FDA's door, seeking approval to enter the market. For many of the most exotic treatments-from cellular replacement therapies to animal organ transplants-the gate-keeper is the Center for Biologics Evaluation and Research (CBER). CBER regulates biological products for disease prevention and treatment that are inherently more complex than chemically synthesized pharmaceuticals. For more information, visit www.fda.gov/cber.
Accomplishments
