New Drug Approval Process and Testing

New Drug Approval

  • All new drugs need FDA approval before marketing.
  • A new drug is any drug proposed for marketing after 1938 that was not already recognized as safe and effective.
  • Drug manufacturer provides proof of safety and efficacy to FDA's Center for Drug Evaluation and Research (CDER).
  • Testing includes lab analysis, animal testing, and clinical trials with people.
  • The FDA estimates that it takes 8.5 years for the testing process.

Clinical Trials

  • Clinical trials test drugs on people, starting with small groups and expanding to larger groups over longer periods.
  • Participants must give informed consent, understanding risks and treatment options, and can leave the trial at any time.

Key Definitions

  • Pediatric: Having to do with the treatment of children.
  • Placebo: An inactive substance given in place of a medication.

Testing Phases

  • Animal Testing: Drugs are tested on animals after lab testing; multiple species are tested.
  • Placebos: Inactive substances used for comparison in trials; patients must agree to possible placebo and be informed of effective treatments.
  • Human Testing Phases:
    • Phase 1: Small number of participants, short time; mainly safety.
    • Phase 2: Up to several hundred patients, several months to 2 years; short-term safety but mainly effectiveness.
    • Phase 3: Several hundred to several thousand patients, 1 to 4 years; safety, dosage, and effectiveness.
    • Only about 25% of drugs tested in phase 1 successfully complete phase 3 and proved for marking.

Investigational New Drug (IND)

  • During trials, a proposed new drug is called an "investigational new drug (IND)".
  • It is available only within trial groups unless granted special "treatment" status for critically ill patients.

Control Groups

  • Tests compare drug effects on one group of patients with the effect of a different treatment on other patients.
  • Control groups may receive no drug, a placebo, an effective drug, or a different dose of the same drug.
  • Blind and Double-Blind Tests:
    • Patients are always blind to the treatment.
    • In a double-blind test, neither patients nor physicians know who receives the drug or placebo.

Testing Children

  • Children are not included in trials until drugs are fully tested on adults.
  • Pediatric Labeling Rule mandates pediatric dosing and safety info if the drug has potential pediatric use.
  • The Pediatric Research Equity Act (PREA) requires testing on children for new drugs likely to be used in children.

Other FDA Responsibilities

  • Medical devices and biological products also meet FDA testing requirements.
  • Center for Devices and Radiological Health (CDRH) is responsible for devices.
  • Center for Biologics Evaluation and Research (CBER) is responsible for biological products.