David Berner, PhD
Associate Director, SFL, a Veristat Company
2 min read
Did you know approximately 1 in 10 Americans are affected by a rare disease or condition according to the National Institutes of Health (NIH)? That’s 30 million people in the US, and similarly in the EU affected.
Before 1983 in the US and 2000 in the EU, development of treatments for these diseases were largely ignored by industry due to the research challenges.
Sponsors developing drugs/biologics intended to prevent, diagnose, or treat a rare disease or condition should consider applying for Orphan Drug Designation (ODD) with the FDA, EMA and other local health authorities. However, due to varying regional and local regulations, ODD applications should be considered from different angles.
Listen to a RAPS and Veristat on-demand webinar on ‘Orphan Drug Designation - the Considerations, the Rewards, and How They Differ Between the US and Europe’.
Regulatory experts working with FDA, EMA, MHRA and Swissmedic – Mara Holinger, Ellen Truitt, David Berner and Julien Gaudias discuss:
- Their experiences in navigating the requirements, benefits, and nuances of ODD applications
- Specific examples of ODD applications in various regions
Learning points:
- Why Orphan Drug Designation came about
- The criteria and determining prevalence
- The incentives and how they differ between the EU vs. US, with specific mention of Swissmedic
- Logistics of the procedure and how to apply for Orphan Drug Designation
Meet the Speakers:
Mara Holinger, PhD, RAC
Senior Vice President, Regulatory Affairs, Veristat
Mara earned her PhD in Molecular Microbiology from Tufts University School of Medicine and began her career as a lead scientist in anti-cancer research. She now provides pharmaceutical and biotech firms with strategic consulting to prepare their regulatory pathway; to assist in the design of clinical programs; and to assess program gaps and design regulatory road maps.
Additionally, she is an expert in regulatory agency meeting preparation, moderation and logistical support, and serves as an Authorized Regulatory Representative and US Agent. Mara has written, reviewed, or provided oversight for more than 20 Investigational New Drug Applications (INDs), multiple Orphan Drug Designation (ODD) applications, breakthrough designation applications, special protocol assessments, fast track applications and several New Drug Applications.
Ellen Truitt, RAC
Regulatory Affairs Strategist, Veristat
Ellen is a Regulatory Affairs Strategist who supports biopharmaceutical firms looking to advance new drugs intended to treat rare diseases across multiple areas of medicine, as well as numerous products in the oncology space. She began her career in biotech and pharma at CBR International Corp. in Boulder, Colorado. Ellen came to Veristat in August of 2019 as a Regulatory Specialist and has since moved to the role of Associate Regulatory Strategist.
Ellen holds a degree in Chemical & Biological Engineering from Colorado State University. In December 2020, Ellen received the RAC – Drugs certification from RAPS.
David leads regulatory projects including defining strategy, preparation and review of regulatory documentation to support SFL clients in the development, MAA filing and maintenance of healthcare products. He leads regulatory submissions to Swissmedic and other international Authorities.
David holds a PhD in Neurobiology from the University of Basel, Switzerland, and an MSc in Molecular Biology from the Friedrich Miescher Institute/University of Basel.
David holds a PhD in Neurobiology from the University of Basel, Switzerland, and an MSc in Molecular Biology from the Friedrich Miescher Institute/University of Basel.
Julien Gaudias, PhD
Senior Director, SFL, a Veristat Company
Senior Director, SFL, a Veristat Company
Julien is team leader and leads projects from early development to MAA filing and launch of healthcare products. He advices on regulatory strategy, oversees preparation and submission of MAA and interacts with Authorities globally for clients’ products.
Julien has extensive experience in pre-submission activities such as CTA, ODD, PIP, scientific advice meetings for medicinal product across broad range of therapeutic areas. He also provides strategic and operational support for the development and life-cycle management of medical devices, IVDs and combination products, including product classification, preparation of Clinical Evaluation Reports and Technical Documentation, and interactions with Notified Bodies. He also advices on regulatory intelligence projects, preparing high quality documents and customized newsletters for clients.
Julien holds a PhD in Neurobiology from the Center of Molecular Life Sciences of the University of Basel, Switzerland, and obtained an MSc in Neuroscience from the University of Strasbourg, France.