November 10-15

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ACR Convergence 2023

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FDA leaders will address challenges and opportunities in pediatric study designs


4 minutes

Applying the laws and regulations that govern drug development in pediatric patients, employing new approaches to study design, and leveraging adult data in similar pediatric rheumatology conditions is at the forefront of the session Pediatric Rheumatology Diseases Drug Development: Challenges and Opportunities on Monday, Nov. 9, from 12 – 1 p.m.

The first live viewing will feature a live question-and-answer period, and registered attendees have on-demand access to a replay through Wednesday, March 11.

This session is particularly important and timely because the U.S. Food and Drug Administration (FDA) is actively employing extrapolation of adult efficacy data in drug development programs in rheumatology conditions that demonstrate a similar disease pathophysiology and clinical presentation, similar co-morbidities, and similar response to therapy in pediatric rheumatology patients to achieve the goal of shortening timelines for pediatric drug development, according to the session presenters and FDA leaders Nikolay Nikolov, MD, medical office team leader at the FDA; Rebecca Rothwell, PhD, lead mathematical statistician at the FDA; and Carolyn Yancey, MD, senior medical officer at the FDA, who answered questions for ACR Convergence Today ahead of the session.

What are some of the scientific and operational challenges clinical trials in pediatric rheumatic diseases present?

Pediatric rheumatology clinical trials are challenging due to the small numbers of patients, creating difficulties in recruitment. Product development in smaller pediatric rheumatology patient populations such as systemic lupus erythematosus, psoriatic arthritis, and spondyloarthropathies are particularly challenging. 

Furthermore, pediatric clinical trial designs need to be acceptable for parents/caretakers and patients and meet ethical standards for evaluation in pediatric patients. This may raise challenges with the use of traditional study designs used in adult indications, e.g., parallel group designs using a placebo control arm, and require alternative design approaches.

Diagnostic criteria present another challenge for clinical trials; older children and adolescents may present with diverse signs and symptoms that may not clearly meet adult criteria on presentation. The nuances of disease presentation need to be considered in the context of disease progression through childhood and into adulthood. These differences in presentation based on age must also be considered in selecting the appropriate trial design because efficacy endpoints that capture a clinically meaningful benefit applicable for a pediatric rheumatology patient population may differ from those applicable for an adult rheumatology patient population with established disease.

How will this session be helpful and beneficial in facilitating an open discussion among regulators and academic researchers?

We seek an open dialogue to understand the challenges from academia and clinical trial investigators committed to pediatric rheumatology drug development and to discuss novel, efficient approaches to pediatric drug development. Our session will provide time for questions from colleagues and the collective pediatric rheumatology ACR community as well as adult rheumatologists who care for pediatric patients.

What are some of the regulatory and clinical trial design considerations in pediatric rheumatology that you’ll be addressing?

The Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research and Equity Act (PREA) are the regulatory bases for pediatric drug development at FDA. We consistently employ BPCA or PREA, as it applies to a clinical development program, to further pediatric drug development. In this session, we will discuss the potential use and considerations of alternative trial designs and statistical methods to address these regulations, such as Bayesian approaches for borrowing adult data. We will also consider the appropriateness of enrolling adolescent patients into adult clinical trials to address the need for pediatric safety data.    

What are a few points you’d like to get across in the session?

This session will present FDA pediatric rheumatology drug development milestones and recent progress on use of the extrapolation paradigm in the context of drug development for pediatric rheumatology conditions. We aim to summarize the key considerations for approaches to pediatric drug development to foster further discussion in the rheumatology community.

Why would you encourage people to attend this session?

We are excited to discuss the constantly evolving approaches to drug development for pediatric rheumatology diseases. Clinicians and researchers who want a better understanding of how FDA evaluates safety and efficacy of drugs for rheumatology diseases in children should not miss this session.