Regulatory bodies have demonstrated flexibility in applying the standards of safety and efficacy to rare disease trials given the unique challenges that drug sponsors face. Developing a sound strategy to navigate the regulatory landscape for rare diseases can literally save (or cost) a sponsor months, or even years, of development. That translates into lives saved and quality of life for those suffering with the disease, and financial gains or losses as well as a competitive advantage for the drug sponsor. In this free webinar, Medpace’s global regulatory experts will discuss some of the key considerations for accelerating development.
Topics will include:
- Which way should we go? A deep dive into the available designations and regulatory pathways that can be used to expedite and accelerate treatments for rare diseases in the US and EU including: orphan drug designation, fast track designation, breakthrough designation, rare pediatric disease vouchers, PRIME and accelerated and conditional approvals.
- What is required and how do we prepare? An in-depth assessment of the requirements for these designations and accelerated or expedited pathways, how to prepare your application and what to expect when you’re granted a designation or an expedited or accelerated pathway, as well as considerations around best-timing for submission will be provided from a global perspective. The presenters will also describe how to prepare for meetings with the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA), and what to expect when engaging with regulatory authorities, particularly in a face-to-face meeting.
- With or without pediatric populations? The pros and cons of including pediatric patients in adult trials for rare diseases will be presented, including suggestions for when it is time to engage regulators in discussions about including pediatrics patients earlier in the clinical development program. The webinar will wrap up with an overview of requirements for pediatric plans in the US and EU, and how these plans fit into the overall development program for rare diseases.
To register for the other parts in this webinar series, please click below:
Christina Vonderhaar, MBA, Sr. Director, Regulatory Affairs, Medpace
Christina Vonderhaar is a senior director in regulatory affairs with over 13 years of drug development experience. Christina’s work in drug development includes experience developing and implementing successful regulatory strategies to support global applications for rare and orphan diseases that impact the pediatric population. She has led and attended multiple successful FDA meetings, including Pre-IND and End-of-Phase meetings across several divisions at FDA. These experiences give her an in-depth understanding of global regulatory processes and allow her to lead and provide experienced insight into potential expedited and accelerated regulatory pathways.
Sargon Daniel, Associate Director, Regulatory Affairs, Medpace
Sargon Daniel is an associate director of regulatory affairs with over 19 years in global regulatory affairs including acquisitions, divestment and product transfers. Sargon has worked in both pharmaceutical companies and contract research organizations (CROs) which has given him excellent rapport with a number of European agencies. Mainly focused on the EU and international markets, Sargon’s work includes several successful applications for orphan drug designation and pediatric investigation plans, with an integrated strategy include PRIME applications. He has attended several EU and EMA agency meetings related to scientific advice and has developed several drug development programs for sponsors.
Who Should Attend?
This webinar will be suitable for VPs, directors, managers and department heads working within:
- Clinical Affairs
- Clinical Research
- Clinical Pharmacology
- Clinical Outsourcing
- Project Management
- Regulatory Affairs
- Medical Affairs
What You Will Learn
This webinar will cover:
- A deep dive into the available designations and regulatory pathways that can be used to expedite and accelerate treatments for rare diseases in the US and EU
- Requirements for these designations and accelerated or expedited pathways and considerations for best-timing for submission
- What to expect in face-to-face meetings with the FDA and EMA
- Pros and cons of including pediatric patients in rare disease trials
- Requirements for pediatric plans in the US and EU
Medpace is a scientifically-driven, global, full-service clinical contract research organization (CRO) providing Phase I-IV clinical development services to the biotechnology, pharmaceutical and medical device industries. Medpace’s mission is to accelerate the global development of safe and effective medical therapeutics through its high-science and disciplined operating approach that leverages local regulatory and deep therapeutic expertise across all major areas including oncology, cardiology, metabolic disease, endocrinology, central nervous system and anti-viral and anti-infective. Headquartered in Cincinnati, Ohio, Medpace employs approximately 3,400 people across 37 countries.