What are the current pediatric requirements and incentives for new products in four major markets: US, Europe, Japan, and China?

• In the US, PREA requires the submission of a Pediatric Study Plan (PSP) and BPCA provides for certain incentives for pediatric drug development. Under FDARA 2017, oncology products with orphan drug designation are no longer exempt from PREA. How will FDA implement the Molecular Targets List?
• In EU, Regulation No. 1901/2006 requires the submission of a Pediatric Investigation Plan (PIP) and provides for certain incentives for pediatric drug development. EMA has recently re-assessed the impact of the pediatric requirements, following 10 years of implementation and a stakeholder workshop in March 2018. What are the findings and expected actions?
• In Japan, there is no requirement to study a new drug in pediatric patients; however, there are incentives for conducting pediatric studies. Are changes likely from PMDA?
• In China, NMPA is dramatically changing many previous regulations and requirements but pediatric studies are not required for new drugs. What new flexibilities can provide incentives for the registration of new drugs for pediatrics in China?

In addition, ICH is preparing a new guideline, ICH E11A: Pediatric Extrapolation. How is extrapolation different in pediatrics vs adults?

Participants are invited to share their recent learnings in pediatric drug development along with the speaker.

Susan Watts has more than 25 years of experience in global regulatory affairs and clinical development. She recently spent 14 years as director of regulatory affairs and global regulatory team leader at GlaxoSmithKline in product development for phases I-IV. She also has 13 years of regulatory experience in local CROs and one year of experience at a small biotechnology company.

Her regulatory focus is agency meetings, scientific advice, and major submissions for US, Europe, and Canada from pre-approval through post-approval. Her therapeutic area regulatory experience includes cardiovascular/metabolic, anti-inflammatory, immunotherapeutic, antiviral, oncology, respiratory, women's health, and neurosciences. She has developed regulatory strategies for expedited development, rare diseases, pediatrics, monoclonal antibodies, gene therapies, and combination products. Susan has a PhD in microbiology and immunology and has been RAPS US Regulatory Affairs Certified (RAC) since 1998.

The pharmaceutical industry is at a tipping point.  Clinical trials in the United States have become increasingly more expensive and complex with 70% of clinical trials missing their timelines and 11% of research sites failing to enroll a single patient.  Data are abundant and the recent FDA guidances around the use of RWD in clinical research has made the investment in vast clinical data sets even more compelling.  By harnessing clinical data, specifically electronic medical record data, and leveraging technological advances, the current clinical trial paradigm can be re-engineered to execute clinical trials that are faster, that utilize fewer resources, and that are more grounded in real world practice.

Kristen K. Buck MD is Senior Vice President, Chief Clinical Development for the Life Sciences business at Optum.  She delivers expert therapeutic advice and strategic support to sponsors for the design and implementation of their clinical trials using the Digital Research Network (DRN). Prior to joining Optum in 2018, Dr. Buck served as Vice President Strategic Drug Development in a Global CRO where she delivered strategic support to pharma and biotech companies worldwide in the development of TPPs, CDPs, trial designs, due diligence, portfolio prioritization, project valuation, and risk-sharing opportunities. Prior, Dr. Buck served as a Chief Medical Officer, SVP of Scientific Affairs and Board Member for a global medical device company and held multiple leaderships roles in Pharma including designing and delivering several global studies for an NME up through registration and FDA Advisory Committee. Dr. Buck also served as a Medical Officer at the United States Food and Drug Administration (FDA) where she was responsible for sponsor guidance on drug development strategies (Phases I-IV) and review of new drug applications and investigational new drug applications. Dr. Buck holds a Bachelors in Biochemistry from the University of Virginia, an MD from Penn State University College of Medicine, and board certification from the American Board of Internal Medicine (2002/2012).