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Pediatric Clinical Trials and Tribulations

Last month, Certara sponsored the 11th Pediatric Clinical Trials conference in Philadelphia. First, I have to say that the content of this conference was among the best I have ever heard. Each speaker added to the dialogue, and the group was able to share and collaborate on how to make meaningful improvements to the field of pediatric drug approvals.

The subject is complex, often heartbreaking, and in many ways can be defined as a “Catch 22.” It is extremely challenging (both logistically and ethically) to enroll children in clinical trials, yet without a proper and approved clinical process, physicians are left with inaccurate dosing and therapeutic approaches for children. The result is a continuation of the off-label, experiential ‘wild wild west’ of prescribing. Here’s some interesting factoids that were revealed at the conference:

  • 42% of recently pediatric drug trials failed for reasons of safety or efficacy
  • Historically, there are about 9 years between the approval of a given new drug for adult and pediatric populations
  • While children represent about 40% of the world’s population, only 10% of the drugs on the market have been approved for pediatrics

Fortunately, the carrot and stick of the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA), combined with the FDA’s Pediatric Study Plan (PSP) and the EMA’s Pediatric Investigation Plan (PIP) regulatory requirements are starting to move the pendulum. Between 2007 and 2013, 469 pediatric studies were completed in the US, and 526 labeling changes were made by August 2014. In Europe, more than 300 products have had changes approved for safety, efficacy or dosing for pediatrics.

A key theme of the meeting was to engage with the regulatory agency early (and often). Dr. Dionna Green, from the FDA’s Office of Clinical Pharmacology reminded us that between Food and Drug Administration Safety and Innovation Act (FDASIA) and PSP requirements, the agency’s emphasis has been on early study planning and establishing a timeline of communications with the agency to assure efficient completion of pediatric trials. Dr. Green also echoed GSK Director of Clinical Pharmacology, Dr. Mohammad Hossain’s comments on the benefits of modeling and simulation tools in drug development as well as lessons learned from failed pediatric drug development programs in refining future approaches. In her summary remarks, Dr. Green added that an increased understanding of the natural history of diseases/conditions, identification of biomarkers, and the use of quantitative tools to inform dose selection can all help to advance pediatric and neonatal drug development.

Kelley Kendle, CEO of Synchrogenix (a Certara company) gave first-hand experience of how a well thought out regulatory writing strategy can give a sponsor a clear advantage, not only in agency interactions but in terms of business development, including pediatric exclusivity. She made a clear case for the many benefits of early planning, developing coordinated and global strategies to maximize results, leveraging early phase data, and using innovative approaches including bridging analyses between non-clinical and clinical studies.

To learn more about how to integrate regulatory writing and modeling & simulation (M&S) into the drug development process, read our whitepaper.

About the author

Ellen Leinfuss
By: Ellen Leinfuss

Ellen Leinfuss serves as SVP and Chief Commercial Officer for Certara’s Simcyp Division. Simcyp is the global leader in mechanistic modeling, which includes the renowned Simcyp Simulator for physiologically based pharmacokinetics (PBPK) and its Quantitative Systems Pharmacology (QSP) immunogenicity, immune-oncology, vaccine and gene therapy modeling & simulation platforms. Ellen has been at Certara for the past seven years, serving for many years as Chief Marketing Officer for the corporation. With a degree in chemistry and MBA in marketing, Ellen has provided management, business development and marketing leadership to organizations in the biopharmaceutical, environmental and energy fields. She is a published author and speaks regularly on topics related to model-informed drug development.

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