Pediatric Drug Development – Developing a Successful Strategy Through Case Studies

12.0
RAC Credits
Thursday, 18 February 2021 (9:00 AM) - Friday, 19 February 2021 (2:00 PM) Eastern Time (US & Canada)

Please note that this session requires attending the live workshop due to the interactive breakout rooms and is not recommended for people who are unable to attend the live workshop. 

Regulations in the US and EU require and incentivize development of pediatric medicines to ensure every new drug is evaluated for pediatric use and studied in this population when appropriate. A key requirement is the submission of detailed plans outlining studies to be conducted as part of the clinical development program and/or waivers or deferrals for such studies. Successful pediatric regulatory strategies can be complex. Differences in regulatory requirements for drugs for pediatric use, including timing, extrapolation of adult data, and use of precedent, present challenges to the development of a robust pediatric global strategy. Knowing these differences is critical to success and avoiding delays in approval that can adversely impact patients. This workshop will provide an overview of the US and EU pediatric drug development regulatory requirements. The key focus will be application of knowledge by engaging participants through the development of mock of pediatric strategies, pressure-testing those strategies with a panel of experts, and identifying regulatory risks and approaches to mitigate those risks.


Pricing Amounts & Deadlines

19 November 2020 – 17 January 2021: Early Bird $800.00 Member | $900.00 Nonmember
18 January 2021 – 17 February: Regular $900.00 Member | $1000.00 Nonmember


Objectives

After this program, participants will be able to:

  •  Describe the pediatric requirements for US, and EU.
  •  Understand the similarities and difference.
  •  Explain the use of extrapolation of adult data to pediatrics.
  •  Apply learning to pediatric regulatory strategies and identify mitigation strategies to support a successful submission/approval.


Agenda

Day 1

9:00 a.m. - 9:15 a.m. –  Welcome and Review of Workshop Goals and Learning Objectives

9:15 a.m. - 9:30 a.m. – Interactive Ice Breaker

9:30 a.m. -10:45 a.m. – Pediatric Drug Development Requirements and Strategic Challenges

   • Introduction to Pediatric Drug Development 

   • Highlights of the US Requirements

   • Highlights of the EU Requirements

10:45 a.m. - 11:00 am – Break

11:00 a.m. - 11:15 a.m. – Interactive Activity / Knowledge Check

11:15 a.m. - 12:15 p.m. – Considerations on Study Design and Conduct of Pediatric Trials – a Pediatrician’s Perspective

12:15 p.m. - 12:45 p.m. – Break

12:45 p.m. - 1:15 p.m. – Selection of the Control Group – Case Studies

1:15 p.m. - 1:45 p.m. – Introduction to Case Studies for Day 2 & Team Assignments

1:45 p.m. – 2:00 p.m. – Interactive Activity / Knowledge Check, Q&A


Day 2

9:00 a.m. - 9:15 a.m. – Welcome to Day 2 and Q&A from Day 1

9:15 a.m. - 9:30 a.m. – Interactive Activity / Knowledge Check

9:30 a.m. - 10:00 a.m. – Adapting Development Programs for Success

10:00 a.m. - 10:15 a.m. – Case Study Readiness Q&A (logistics, Ask the Expert)

10:15 a.m. - 10:30 a.m. – Break

10:30 a.m. - 11:00 a.m. – Team breakouts to work on Case Studies

11:00 a.m. - 12:30 p.m. – Breakout Group Case Study Presentations

12:30 p.m. -1:00 p.m. – Pediatric Drug Development Recap and Q&A

1:00 p.m.  – Adjournment


Speakers


Kimberly Belsky, Executive Director, Regulatory Policy and Intelligence and Labeling Operations, Mallinckrodt Pharmaceuticals

Kimberly (Kim) Belsky is an experienced leader skilled in innovative and strategic thinking as it relates to a broad range medical products and functional areas. In her current role as Executive Director of Regulatory Policy & Intelligence at Mallinckrodt Pharmaceuticals, she is responsible for assessing changes in the regulatory environment relevant to the company portfolio to identify opportunities and challenges in rare diseases and unmet medical needs. Kim has more than 29 years in the pharmaceutical industry including 19 years in regulatory affairs, that covers a diversity of medical products including Rx drug/biologics, OTCs/Nutritionals/Cosmetics, and medical devices,. Kim holds a master’s degree in chemistry from SUNY Stony Brook and has several scientific publications including a 2019 poster entitled, “The Regulatory Environment for Tissue-Engineered Products for the Treatment of Burns in the United States”. Additionally, Kim is an active member of RAPS, the vice-chair of RAPS Convergence 2020, on the RAPS Convergence Planning Committee, recipient of the 2017 RAPS volunteer award.


Karl-Heinz Huemer, Medical Assessor AGES (Austrian Agency for Health & Food Safety)

Karl-Heinz Huemer, PhD MD, medical assessor at the Austrian Medicines & Medical Devices Agency, Vienna / Austria delegate to SAWP (Scientific Advice Working Party) and PDCO (Paediatric Committee) at EMA (European Medicines Agency), London / UK for a more detailed CV please refer to the European Medicines Agency homepage https://www.ema.europa.eu/docs/en_GB/document_library/contacts/huemerk_CV.pdf


Linda McBride, Senior Director Regulatory Professionals Inc

Linda McBride is the Senior Director, Reglatory Affairs at Regulatory Professionals, Inc. Previously she was the head of regulatory affairs and clinical compliance at Revance Therapeutics. She has over 30 years of biopharmaceutical industry experience, including more than 20 years in regulatory affairs. Her experience includes head of the regulatory department along with global regulatory lead during development of investigational products through marketing application submission/approval including post-approval lifecycle management, including advertising and promotional activities, addition of new indications including pediatrics. Her therapeutic area expertise consists of analgesia, anti-infectives, cardiorenal, dermatology, neurology, oncology and pulmonology and the products supported include small molecules, biologics, combination products and over-the-counter products. She began her career in the pharmaceutical industry as a research pharmacist with Novartis Consumer Health. She has a B.S. in Pharmacy and earned a RAC for the US. She is a member of the Regulatory Affairs Professionals Society and sits on the Editorial Advisory Committee


Susanne Schmidt, Senior Medical Director, Covance


Cancellation Policy

RAPS reserves the right to cancel this program at its sole discretion. RAPS will not be responsible for travel or other costs incurred due to cancellation. All cancellation requests must be submitted in writing via our online cancellation form. RAPS is unable to accept cancellations by phone. Please specify the name of the person registered and the event title. Substitutions may be accepted with written approval from RAPS and must be submitted to raps@raps.org.


Proof of Attendance

RAPS will send an electronic letter or a certificate of attendance upon request.


Questions

Call RAPS Solutions Center at +1 301.770.2920, Ext 200 or emailraps@raps.org.