FDA Advisory Committees - FDA Office of the Commissioner (OC) - Pediatric Advisory Committee (PEDAC)
The committees will meet to discuss the pediatric-focused safety review for OxyContin (oxycodone hydrochloride) extended-release tablets, as mandated by the Food and Drug Administration Safety and Innovation Act (Pub. L. 112-144), and to discuss pediatric data considerations for opioid analgesics labeling and Pediatric Research Equity Act studies for opioids generally, using Opana IR as an example.
The committees will meet to discuss a pediatric-focused safety review of neuropsychiatric events with use of Singulair (montelukast).
The committee gave in put on drug development for testosterone replacement therapy in male adolescents for conditions associated with a deficiency or absence of endogenous testosterone resulting from structural or genetic etiologies (“classic hypogonadism”).
The committee heard pediatric-focused safety reviews for Intuniv (guanfacine) and Lexapro (escitalopram). For each of these drugs, the committee voted in favor, by a vote of 11-Yes and 1No, of the monitoring proposed by the FDA’s Division of Pharmacovigilance (DPV). For Intuniv, the DPV proposed to continue monitoring for adverse events (AEs), including suicidal ideation and behavior, pancreatitis, and medication error involving name confusion. For Lexapro, the DPV proposed to continue routine safety monitoring
On Friday May 11, 2018 the Pediatric Advisory Committee (PEDAC) and the Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) met jointly to discuss drug development for the treatment of children with achondroplasia. The following topics were discussed: evidence required to establish dose-response, study design, study duration, intended population, and endpoints.
From 8:00 am to 11:00 am, the meeting was closed to permit discussion and review of trade secret and/or confidential commercial information (5 U.S.C. 552b(c)(4)). During this session, the Committees discussed the premarketing drug development program of an investigational product.
Originally, this meeting was scheduled for March 22, 2018, but it was postponed due to inclement weather.
The Committee discussed a new drug application (NDA for stannsoporfin injection, for intramuscular use, submitted by InfaCare Pharmaceutical Corporation, proposed for the treatment of neonates grdeater than or equal to 35 weeks of gestational age with indicators of hemolysis who are at risk of developing severe hyperbilirubinemia. 21 of 24 members, voted that the overall risk-benefit profile of stannsoporfin does not support approval. Three of the 24 members did support approval with a REMS.
The Committee will meet to discuss pediatric-focused safety reviews, as mandated by the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA).
The Committee will meet to discuss the following products (listed by FDA Center):
(1) Center for Drug Evaluation and Research
(2) Center for Devices and Radiological Health
a. FLOURISH (Humanitarian Device Exemption (HDE))
b. ACTIVA (HDE)
c. LIPOSORBER (HDE)
d. IMPELLA RP SYSTEM
The PEDAC and EMDAC will meet to discuss the major objectives of a phase 3 drug development program indicated for the treatment of children with achondroplasia (ACH). The following elements of a phase 3 program should be considered for discussion: evidence required to establish dose-response, study design, e.g., placebo control, study duration, intended population, e.g., infants and toddlers and/or older children and adolescents, and endpoints that have a clinically meaningful impact on the patient’s functional or psychological well-being.
On Monday, September 11, 2017, the Pediatric Advisory Committee (PEDAC) discussed and voted on the use of prescription opioid products for the treatment of cough in pediatric patients.
On Tuesday, September 12, 2017, the Committee discussed and voted on pediatric-focused safety reviews for certain products that are mandated by the Best Pharmaceuticals for Children Act (BPCA), the Pediatric Research Equity Act (PREA), and the Pediatric Medical Device Safety and Improvement Act (PMDSIA).
The Committees jointly supported, by a vote of 14-Yes to 0-No, with no abstentions, the use of an indwelling central venous access devices, in certain circumstances, in the clinical trial “A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients with Duchenne Muscular Dystrophy (ESSENCE).”
The Committee will discuss pediatric-focused safety reviews, as mandated by the Best Pharmaceuticals for Children Act, the Pediatric Research Equity Act and the Pediatric Medical Device Safety and Improvement Act .
On March 6, 2017, the Committee will discuss the following products (listed by FDA Center):
Center for Drug Evaluation and Research (CDER)
a. NITROPRESS (sodium nitroprusside)
b. KUVAN (sapropterin dihydrochloride)
FDA will provide the analysis of a possible safety signal regarding the use of the drug product EXJADE (deferasirox) in children with fever and dehydration, which was discussed at the September 2015 meeting.
The Committee will also discuss the role of pharmacogenomics in pediatric product development.
On March 7, 2017, the Committtee will discuss the following products (listed by FDA Center):
Center for Biologics Evaluation and Research (CBER)
a. EPICEL (cultured epidermal autografts) (humanitarian device exemption (HDE))
b. NOVOEIGHT (Antihemophilic Factor (Recombinant))
c. RIXUBIS Coagulation Factor IX (Recombinant)
Center for Devices and Radiological Health (CDRH)
a. IMPELLA RP SYSTEM (HDE)
b. LIPOSORBER LA-15 SYSTEM (HDE)
c. MEDTRONIC ACTIVA DYSTONIA THERAPY (HDE)
The Committee discussed pediatric-focused safety reviews, as mandated by the Best Pharmaceuticals for Children Act, the Pediatric Research Equity Act, and the Pediatric Medical Device Safety and Improvement Act.
For 12 of the products that were reviewed, the Committee unanimously concurred with FDA’s proposal to continue routine surveillance. For the other products, the Committee did not have a unanimous vote or, in some cases, added or modified FDA’s recommendations.
The Committee discussed 18 pediatric-focused safety reviews, as mandated by the Best Pharmaceuticals for Children Act, the Pediatric Research Equity Act, and the Pediatric Medical Device Safety and Improvement Act of 2007. For all of the products that were reviewed, the a majority of the Committee members concurred with FDA’s proposal to continue routine surveillance.
On Monday, March 23, 2015, the Pediatric Ethics Subcommittee to the Pediatric Advisory Committee (PEDAC) discussed the general topic of how procedural sedation for nontherapeutic (research) interventions or procedures in the pediatric population should be considered under the Additional Safeguards for Children in Clinical Investigations, at 21 CFR 50 subpart D. A slight majority of the Subcommittee, 9 of 16 members, voted that there are no approaches to procedural sedation that would present no more than a minor increase over minimal risk.
On Tuesday, March 24, 2015, the PEDAC discussed pediatric-focused safety reviews, as mandated by the Best Pharmaceuticals for Children Act (BPCA, Pub. L. 107-109), the Pediatric Research Equity Act (PREA, Pub. L. 108-155), and the Pediatric Medical Device Safety and Improvement Act of 2007 (PMDSIA). The PEDAC unanimously agreed with the FDA’s proposal to continue routine pharmacovigilance for the following products: QUILLIVANT XR (methylphenidate hydrochloride), LUNESTA (eszopiclone), RISPERDAL (risperidone), OXTELLAR XR (oxcarbazepine), REVATIO (sildenafil), ADVAIR HFA (fluticasone propionate/salmeterol xinafoate), DYMISTA (azelastine hydrochloride/fluticasone propionate), QNASL (beclomethasone dipropionate), VENOFER (iron sucrose), INVIRASE (saquinavir), ALTABAX Ointment (retapamulin), FluMist QUADRIVALENT (influenza vaccine live, intranasal), FLUARIX QUADRIVALENT (influenza virus vaccine), and LIPOSORBER LA-15 System. All but one of the Committee members agreed with the FDA’s proposal to continue routine pharmacovigilance for CYMBALTA (duloxetine hydrochloride). All but two of the Committee members with the FDA’s proposal to continue routine pharmacovigilance for the Medtronic ACTIVA DYSTONIA THERAPY; those two members abstained.
Pediatric Advisory Committee (PEDAC)
PEDAC advises and makes recommendations to the FDA Commissioner regarding:
- Pediatric research conducted under the Public Health Service Act (sections 351, 409I, & 499) and the Federal Food, Drug, & Cosmetic Act (sections 501, 502, 505, 505A, and 505B).
- Identification of research priorities related to pediatric therapeutics and the need for additional treatments of specific pediatric diseases or conditions
- The ethics, design, and analysis of clinical trials related to pediatric therapeutics.
- Pediatric labeling disputes as specified in sections 3 and 5 of the Best Pharmaceuticals for Children Act (BPCA) (Public Law 107-109),
- Adverse event reports for drugs granted-pediatric exclusivity and any safety issues that may occur as specified in BPCA (section 17).
- Any other pediatric issue or pediatric labeling dispute involving FDA-regulated products
- Research involving children as subjects as specified in 21 CFR 50.54, and
- Any other matter involving pediatrics for which FDA has regulatory responsibility.
- Pediatric Ethics Subcommittee
- Neonatology Subcommittee of the Pediatric Advisory Committee