FDAAA Pediatric Studies Characteristics

From October 29, 2007 through March 19, 2009 N= 56 (BPCA = 27; PREA = 29)

The table above contains pediatric assessments (or studies) made under sections 505B and 505A of the Federal Food, Drug, and Cosmetic Act (the Act), as amended by the FDA Amendments Act of 2007 (FDAAA). In accordance with sections 505B(f)(6) and 505A(f)(6) of the Act, as amended by FDAAA, the list identifies those products approved on or after September 27, 2007, that fell within the scope of the Pediatric Research Equity Act (PREA) and/or the Best Pharmaceuticals for Children Act, and contained new pediatric information.

This list specifically highlights the number of assessments (or studies) conducted under the sections referenced above, the specific drugs and their uses, and the types of assessments conducted, including trial design, number of pediatric patients studied, and the number of centers and countries involved.

Definitions of the specific study types and designs used in the table above

* = Patient number includes adults

Study Types

1. Efficacy: Efficacy studies are done to provide evidence of a drug’s ability to diagnosis, cure, mitigation, treatment, or prevention of a disease. Efficacy information may be found in the drug labeling Indications & Usage, Use in Specific Populations, and Clinical Studies sections.

2. Safety: Safety studies provide evidence of a drug’s medical risk to humans. Safety is usually determined by collecting information on laboratory and/or other tests (for example, electrocardiogram and vision), vital signs, and drug side effects. Safety information may be found in the drug labeling Warnings & Precautions, Adverse Reactions, and Use in Specific Populations sections.

3. Pharmacokinetic (PK): Pharmacokinetic studies provide information on what the body does to a drug. More specifically, it covers how a drug is absorbed, distributed, metabolized and eliminated by the body. Pediatric PK studies are generally performed in patients, and focus on the measurement of drug in blood, urine, or in other body fluids or tissues.
Pharmacokinetic studies are often conducted during initial drug development in order to:

• evaluate a new formulation of an existing drug;
• study how a drug may interact with other drugs;
• study what the body does to a drug in a special population of patients (for example, children and the elderly).

Pharmacokinetic information may be found in the drug labeling Clinical Pharmacology section.

4. Pharmacodynamic (PD): Pharmacodynamic studies provide information on what a drug does to the body. PD examines how a drug works in the body and the amount of drug needed to provide an effect. Pharmacodynamic information may be found in the drug labeling Clinical Pharmacology section.h

5. Pharmacokinetic/Pharmacodynamic (PK/PD): A drug’s pharmacokinetic effect is usually expressed along with its pharmacodynamic effect. (The relationship between what a drug does to the body and what the body does to a drug.) PK/PD information may be found in the drug labeling Clinical Pharmacology section.

6. Tolerability: Tolerability of a drug is the degree to which drug-related side effects can be accepted by a patient. Tolerability is usually associated with the safety of a drug. Tolerability information may be found in the drug labeling Warnings & Precautions and Adverse Reactions sections.

7. Nonclinical: Nonclinical studies are usually conducted in a laboratory setting, and may involve in vitro (test tube) and in vivo (animal) studies. There is no testing of human subjects. Nonclinical information may be found in the drug labeling Nonclinical Toxicology section.

8. Neonatal: Neonatal studies involve pediatric patients 0 to 1 month of age. Neonatal study information may be found in the drug labeling Use in Specific Populations/Pediatric Use section.

9. Actual Use: Actual Use studies provide information about whether subjects can self-diagnose a medical condition, use a drug product safely, and follow labeling instructions without the assistance of a healthcare professional (for example, doctor, nurse, or pharmacist). These studies are conducted for products intended to be marketed over-the counter (OTC). Actual Use study information may be found in the drug labeling Clinical Studies section.

10. Oncology: Oncology studies focus on the diagnosis, cure, mitigation, treatment, or prevention of cancer in patients. Oncology study information may be found in the drug labeling Clinical Studies section.

11. Immunogenicity: These studies test efficacy by measuring the antibody response of the patient to the vaccine.

12. Other: Any study that does not involve the types described above.

Study Designs 

1. Traditional Pharmacokinetic (PK): Traditional PK studies involve administration of the study drug to a subject followed by intensive sampling of blood, urine or other body fluid, which is used to measure the drug in those fluids. These studies usually enroll small groups of patients (less than 20).

2. Population Pharmacokinetic: Sometimes called "Pop PK studies", they involve administration of the study drug to a larger group of patients, followed by less frequent sampling of body fluids, Computer models are then used to identify certain demographic characteristics, such as weight or age, that influence how the drug passes through the body, without having to conduct a traditional pharmacokinetic study.

3. Active Control: Studies that compare a known and effective drug to the study drug.

4. Placebo Control: Studies that compare the study drug to an inactive substance or treatment that does not have any therapeutic activity.

5. Historical Control: Studies that use past information from studies or drug experience to compare with the effect of the study drug.

6. Blinded: During a blinded trial, the participant and/or investigators do not know what treatment the patient is receiving. “Double blinding” means that neither the investigator nor participant knows what treatment is being given. During a “single blinded” trial, the participant is unaware of the treatment, although the investigators know. Blinded studies help to minimize study bias regarding the efficacy and safety of the drug being studied.

7. Open Label: In such a study, participants and/or investigators may be aware of which treatment the participant is receiving.

8. Parallel Group: During a parallel group study, each treatment group is studied simultaneously. For example, in a controlled study, one group may receive the study drug while at the same time a second, separate group receives a placebo or alternative treatment.

9. Crossover: In such a study each patient serves as his/her own control and each treatment is examined sequentially. For example, in a single treatment, controlled study, Group 1 receives the study drug first while at the same time the other Group 2 receives a placebo or alternative treatment first. Then, Group 1 receives a placebo or alternative treatment while at the same time Group 2 receives the study drug.

10. Dose Response: A measure of the body's response to escalating drug doses. A dose response pattern usually shows little or no drug effect at very low dosages and increasing levels of effect or toxicity at higher doses until the effect plateaus. Usually, several doses of a drug are tested to find out which one is the safest and most effective.

11. Animal: Such studies are used to measure the potential toxicity of a drug. Animal studies are also performed when it would be unethical to test the drug in humans due to the nature of the disease or condition being studied (for example, bioterrorism agents such as anthrax).

12. Other: Any study that does not involve the study designs described above.

For more information regarding clinical trials, see:
http://www.fda.gov/oashi/clinicaltrials/clintrialdoc.html.