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Pediatric assessments (or studies) made under Section 505B of the Federal Food, Drug, and Cosmetic Act (the Act), as amended by the FDA Amendments Act of 2007 (FDAAA).
In accordance with section 505B(f)(6)(A)-(C) of the Act, as amended by FDAAA, the following list identifies those products approved on or after September 27, 2007, that fell within the scope of the Pediatric Research Equity Act (PREA), contained new pediatric information, and were not related to studies conducted under 505A of the Federal Food, Drug and Cosmetic Act (i.e., pediatric exclusivity incentive program).
This list specifically highlights the number of assessments (or studies) conducted under the section 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 below
Approval Date |
Product |
Indication(s) |
Study # |
Study Type |
Study Design |
# Patients |
# Centers |
# Country |
---|---|---|---|---|---|---|---|---|
1/10/2008 |
Alvesco (ciclesonide) Inhalation Aerosol, 80 mcg & 160 mcg |
Treatment of asthma in patients 12 years of age and older |
1 |
Efficacy/Safety |
Blind/Placebo/Parallel Group//Dose Response |
514 |
67 |
2 |
2 |
Efficacy/Safety/Dose Response |
Blind/Placebo/Parallel Group |
517 |
64 |
2 |
|||
3 |
Safety |
Open Label |
193 |
26 |
1 |
|||
4 |
Safety |
Open Label |
190 |
26 |
1 |
|||
5 |
Safety |
Open Label |
232 |
29 |
1 |
|||
6 |
Safety/Pharmacodynamic |
Placebo |
24 |
1 |
1 |
|||
7 |
Efficacy/Safety/Tolerability |
Blind |
728 |
51 |
1 |
|||
2/1/2008 |
Asmanex Twisthaler 110mcg (mometasone furoate inhalation powder) |
Maintenance treatment of asthma as prophylactic therapy in children 4 years of age and older |
1 |
Efficacy/Safety |
Blind/Placebo/Parallel Group |
296 |
39 |
6 |
2 |
Efficacy/Safety |
Blind/Placebo/Parallel Group |
349 |
25 |
1 |
|||
3 |
Efficacy/Safety |
Blind/Placebo/Parallel Group |
290 |
20 |
6 |
|||
4 |
Safety |
Blind/Placebo/Parallel Group |
50 |
1 |
1 |
|||
5 |
Safety |
Blind/Placebo/Parallel Group |
187 |
29 |
1 |
|||
6 |
Safety |
Open Label/Active Control |
233 |
21 |
1 |
|||
7 |
Safety |
Blind/Placebo/Crossover |
23 |
1 |
1 |
|||
8 |
Safety |
Blind/Placebo/Active Control/Crossover |
51 |
1 |
1 |
|||
12/12/2007 |
Derma-Smoothe (fluocinolone) Topical Oil, 0.01% (Body Oil) |
Treatment of atopic dermatitis in pediatric patients 3 months and older for up to 4 weeks |
1 |
Safety |
Open Label |
32 |
2 |
1 |
5/8/2008 |
Desmopressin acetate tablets, 0.1 mg and 0.2 mg |
Determine the capacity of the kidney to concentrate urine in pediatric patients (Renal Concentration Capacity Test or RCCT) and management of primary nocturnal enuresis (PNE) |
1 |
Efficacy/Safety |
Blind/Placebo |
145 |
6 |
1 |
2 |
Efficacy/Safety |
Open Label |
58 |
2 |
1 |
|||
3 |
Efficacy |
Blind/Parallel Group |
31 |
14 |
1 |
|||
4 |
Efficacy |
Blind/Crossover |
23 |
1 |
1 |
|||
5 |
Efficacy |
Open Label |
30 |
1 |
1 |
|||
6 |
Efficacy |
Open Label |
38 |
11 |
1 |
|||
7 |
Efficacy |
Open Label |
249 |
9 |
1 |
|||
1/23/2008
|
Moxatag (amoxicillin extended-release) Tablet, 775 mg |
Use for tonsillitis and/or pharyngitis secondary to Streptococcus pyogenes patients 12 years of age and older |
1 |
Efficacy/Safety |
Blind/Placebo/Parallel Group |
142 |
64 |
1 |
2 |
Efficacy/Safety |
Blind/Placebo/Parallel Group |
139 |
50 |
2 |
|||
9/19/2008
|
Nasacort AQ (triamcinolone acetonide) Nasal Spray
|
Treatment of the nasal symptoms of seasonal and perennial allergic rhinitis in adults and children 2 years of age and older |
1 |
Safety/PK |
Open Label/Traditional Pharmacokinetic |
15 |
6 |
1 |
2 |
Safety |
Placebo/Blind/Crossover |
59 |
1 |
1 |
|||
3 |
Efficacy/Safety |
Blind/Placebo/Parallel Group |
474 |
64 |
1 |
|||
3/14/2008 |
NovoLog (insulin aspart [rDNA origin] injection) |
Insulin analog indicated to improve glycemic control |
1 |
Efficacy/Safety |
Open Label/Active Control/Parallel Group |
298 |
45 |
1 |
2 |
Efficacy/Safety |
Open Label/Active Control/Parallel Group |
61 |
5 |
1 |
|||
|
||||||||
11/21/2007 |
Omnaris (ciclesonide) Nasal Spray |
Treatment of seasonal allergic rhinitis (SAR) in patients 6 through less than 12 years of age |
1 |
Safety/Tolerability |
Blind/Placebo/Parallel Group |
125 |
3 |
1 |
2 |
Efficacy/Safety |
Blind/Placebo/Parallel Group |
618 |
69 |
1 |
|||
|
||||||||
5/9/2008 |
OraVerse (phentolamine mesylate) Injection 0.4 mg (0.235 mg/mL) |
Reversal of soft-tissue anesthesia, i.e., anesthesia of the lip and tongue, and the associated functional deficits resulting from an intraoral submucosal injection of a local anesthetic containing a vasoconstrictor |
1 |
Efficacy/Safety/Pharmacodynamic |
Blind/Placebo |
31 |
18 |
1 |
2 |
Efficacy/Safety/Pharmacodynamic |
Blind/Placebo |
24 |
16 |
1 |
|||
3 |
Efficacy/Safety |
Blind/Placebo |
152 |
11 |
1 |
|||
4 |
Efficacy/Safety |
Blind/Placebo |
26 |
7 |
1 |
|||
5 |
Safety/PK |
Open Label/Traditional Pharmacokinetic |
19 |
3 |
1 |
|||
|
|
|
|
|
|
|||
4/15/2008 |
Patanase (olopatadine hydrochloride) Nasal Spray |
Relief of symptoms of seasonal allergic rhinitis (SAR) in patients 12 years of age and older |
1 |
Efficacy/Safety |
Blind/Placebo/Parallel Group |
56 |
33 |
1 |
2 |
Efficacy/Safety |
Blind/Placebo/Parallel Group |
50 |
7 |
1 |
|||
3 |
Safety |
Placebo/Parallel Group |
(924) |
43 |
1 |
|||
4 |
Efficacy/Safety |
Blind/Placebo/Parallel Group |
(192) |
7 |
1 |
|||
5 |
Efficacy/Safety |
Blind/Active Control/Placebo/Parallel Group |
(166) |
13 |
1 |
|||
6 |
Efficacy/Safety |
Blind/Active Control/Placebo |
(397) |
17 |
1 |
|||
7 |
Efficacy |
Blind/Active Control/Placebo/Parallel Group |
(90) |
1 |
1 |
|||
|
||||||||
3/20/2008 |
Prilosec (omeprazole) for Delayed-Release Oral Suspension |
Maintenance healing of erosive esophagitis |
1 |
Pharmacokinetic |
Open Label/Traditional Pharmacokinetic |
25 |
11 |
3 |
2 |
Efficacy |
Other |
43 |
8 |
3 |
|||
9/16/2008 |
Proair HFA (albuterol sulfate) Inhalation Aerosol |
Treatment or prevention of bronchospasm with reversible obstructive airway disease & prevention of exercise-induced bronchospasm (EIB) in patients 4 years of age and older |
1 |
Pharmacokinetic |
Open Label/Active Control/Trad Pharmacokinetic/Crossover |
16 |
1 |
1 |
2 |
Efficacy/Pharmacodynamic |
Blind/Placebo/Crossover |
55 |
10 |
1 |
|||
3 |
Efficacy/Safety |
Blind/Placebo/Parallel Group |
95 |
14 |
1 |
|||
6/24/2008 |
Viramune (nevirapine) Tablets (200 mg) & Oral Suspension (10 mg/ml) |
Use in combination with other antiretroviral agents for the treatment of HIV-1 infection |
1 |
Safety/Tolerability/Pharmacokinetic |
Open Label/Traditional Pharmacokinetic |
123 |
4 |
1 |
(#) Patient number includes adults
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:
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.
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. Other: Any study that does not involve the types described above.
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.