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Background Analysis: US FDA Advisory Committee to Review Drug Products and Devices Under PREA/BPCA – SEP 20, 2018


The US FDA has scheduled a meeting of the Pediatric Advisory Committee (PEDAC) for Thursday, September 20, 2018. The Committee will discuss pediatric-focused safety reviews, as mandated by the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA). In addition, the FDA will give updates on general safety topics that pertain to the FDA Adverse Event Reporting System (FAERS), the generic drug approval process and labeling updates for EXJADE (deferasirox) , inhaled corticosteroid long-acting b-2 agonists, and safety labeling for gadolinium.

This meeting is the rescheduling of the meeting that was planned for March 23, 2018, but was postponed.

Product Background

Products for review under BPCA/PREA

The PEDAC will discuss pediatric-focused safety reviews for the listed below with their indications (as of August 20, 2018). Typically, the FDA reviews the pediatric safety of these products, proposes how to continue surveillance and asks the Committee to vote on whether they are in agreement with the FDA’s proposals.


INTUNIV is indicated for the treatment of Attention Deficit Hyperactivity Disorder (ADHD) as monotherapy and as adjunctive therapy to stimulant medications.


1.1  Major Depressive Disorder

Lexapro (escitalopram) is indicated for the acute and maintenance treatment of major depressive disorder in adults and in adolescents 12 to 17 years of age [see Clinical Studies (14.1)].

A major depressive episode (DSM-IV) implies a prominent and relatively persistent (nearly every day for at least 2 weeks) depressed or dysphoric mood that usually interferes with daily functioning, and includes at least five of the following nine symptoms: depressed mood, loss of interest in usual activities, significant change in weight and/or appetite, insomnia or hypersomnia, psychomotor agitation or retardation, increased fatigue, feelings of guilt or worthlessness, slowed thinking or impaired concentration, a suicide attempt or suicidal ideation.

1.2 Generalized Anxiety Disorder

Lexapro is indicated for the acute treatment of Generalized Anxiety Disorder (GAD) in adults [see Clinical Studies (14.2)].

Generalized Anxiety Disorder (DSM-IV) is characterized by excessive anxiety and worry (apprehensive expectation) that is persistent for at least 6 months and which the person finds difficult to control. It must be associated with at least 3 of the following symptoms: restlessness or feeling keyed up or on edge, being easily fatigued, difficulty concentrating or mind going blank, irritability, muscle tension, and sleep disturbance.

Additional Meeting Topics

The FDA will provide general safety updates on the following topics:

·      Overview of the FDA Adverse Event Reporting System and lack of efficacy;

·      Generic drug approval process; and discussion on trade versus generic drugs; exceptions;

·      Summary of the FDA’s completed review of pediatric safety issues and updated labeling changes for EXJADE (deferasirox);  

·      Update labeling change for inhaled corticosteroid long-acting b-2 agonists; and

·      Safety labeling for gadolinium.

Regulatory Background - BPCA/PREA

The reports on these products are mandated through the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA).


BPCA and PREA were made permanent by the Food and Drug Administration Safety and Innovation Act (FDASIA) in 2012.

In 2003, PREA was first signed into law. It was reauthorized by the Food and Drug Administration Amendments Act (FDAAA) in 2007 and later made permanent with FDASIA in 2012. The act requires pediatric studies and covers drugs, biologics and medical products derived from living sources, such as vaccines, blood, and blood derivatives. Under the PREA, the FDA can require pediatric studies of a drug submitted in a new drug application if the FDA determines the product is likely to be used in a substantial number of pediatric patients, if the product would provide a meaningful benefit in the pediatric population over existing treatments, and absence of adequate labeling could pose a significant risk. At the same time, PREA does not delay the availability of drugs for adults. PREA does not apply to drugs that qualify for orphan drug designation or to generics.

In addition, the voluntary pediatric exclusivity provision of the Food and Drug Administration Modernization Act of 1997 (FDAMA) was continued through BPCA since 2002.The Pediatric Exclusivity Provision of the BPCA allows companies to qualify for an additional six months of marketing exclusivity if they do the studies in children as requested by the FDA. Since the incentive under FDAMA did not apply to old antibiotics and other drugs that lack marketing exclusivity or patent protection, some categories of drugs have remained inadequately studied. For these products, BPCA provides a contract mechanism through the National Institutes of Health (NIH) to fund pediatric studies. In addition, if a company that has a drug with existing exclusivity or patent protection chooses not to conduct the requested pediatric studies, the FDA can refer the Written Request to the Foundation for the NIH to award grants so that third parties can conduct the needed studies.

What’s Next? (With Subscription)

Tarius will send a Briefing Summary after briefing materials are posted to FDA’s website (typically within 2 days of the meeting). This report will provide a summary of the FDA and the Sponsor’s briefing materials.

Tarius will send a Results Wire soon after the meeting. This report will include the voting outcomes, if applicable, and key outcomes of the discussion.

METADATA: Sponsor: none Drug Name: several Drug Class: several Indication: pediatric uses

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DISCLAIMER: The information in this document is for informational purposes only. The SAC Tracker Background Analysis contains information from publicly available sources, including FDA, sponsor, scientific, and clinical websites. Tarius A/S assumes no liability for any inaccurate or incomplete information, or for any actions taken in reliance thereon. © Tarius A/S. All rights reserved.