Accelerating the development of therapeutic products intended to treat serious conditions has been an interest of the public, legislators and the scientific community for many years. Responding to this interest, the US government has passed a number of laws intended to encourage the development of products for these indications, including the Prescription Drug User Fee Act of 1992 (PDUFA), the Food and Drug Administration Modernization Act of 1997 (FDAMA), and most recently, the 21st Century Cures Act of 2016. Responding to these directives from Congress, the US Food and Drug Administration (FDA) has issued regulations and guidance documents to establish development programs designed to speed the availability of new therapies to patients with serious conditions, especially when there are no satisfactory alternative therapies.
This free webinar will highlight the requirements, benefits and procedures for Fast Track Designation, Breakthrough Therapy Designation, Priority Review Designation and Accelerated Approval. The speakers will also discuss two newer programs, Regenerative Medicine Advanced Therapy (FDA guidance, Feb 2019) and Qualified Infectious Disease Program (FDA guidance, Jan 2018).
Participants will also learn about the importance of establishing a therapeutic area as a serious condition with an unmet medical need for each of these programs and hear examples for this.
John P. Balser, PhD, President and Co-Founder, Veristat
John Balser co-founded Veristat in 1994 and has grown the company to be a scientifically focused clinical research partner that leads emerging and growing biotech clients through the clinical development and regulatory approval process. John is actively involved with clinical projects in his role as one of Veristat’s principal statistical consultants. He assists clients with clinical study design and program development based on his many years of experience in the statistical aspects of clinical research. He regularly assists clients as they prepare for and meet with the regulatory agencies, particularly the FDA.
John received his Master of Science and PhD in Biometrics from Cornell University and has been actively engaged in clinical biostatistics for over 30 years.
Barbara Balser, VMD, Executive Vice President and Co-Founder, Chief Scientific Officer, Veristat
Barbara Balser co-founded Veristat in 1994 and continues to provide biopharmaceutical clients with medical writing and particularly, regulatory submission expertise. With more than 30 years of clinical research experience, her passion lies in providing strategic planning and consulting, medical oversight and production of regulatory submission documents. Her experience includes oversight and production of Investigational New Drug (IND) applications, regulatory agency briefing documents, preparation for and representation at advisory panel meetings and consultation across Phase 1 to 4 trials in multiple therapeutic areas with a specific focus in oncology, rare diseases and anti-infectives. Barbara has participated in the writing, oversight and submission of countless marketing applications to US, Canadian, Japanese and European Union regulatory authorities.
Barbara holds a Bachelor of Science in Chemical Engineering from the University of Pennsylvania, School of Engineering and Applied Science, and a VMD from the University of Pennsylvania, School of Veterinary Medicine.
Mara Holinger, PhD, RAC, Vice President, Regulatory Affairs, Veristat
Mara Holinger provides pharmaceutical, biotech and medical device firms with strategic consulting to prepare their regulatory pathway, design their clinical program and trials, perform gap analysis for CMC and preclinical reporting and write regulatory applications. Additionally, she is an expert in regulatory agency meeting preparation, moderation and logistical support, and services 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, including 505(b)(2).
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.
Kevin Hennegan, Regulatory Consultant, Veristat
Kevin Hennegan has provided strategic clinical and regulatory guidance to the pharmaceutical, biotech and medical device industry for over 15 years. He has led and participated in the preparation of many successful FDA filings, including Investigational New Drug Applications (INDs), New Drug Applications (NDAs), Biologics License Applications (BLAs), Breakthrough Therapy Designations, Orphan Drug Designations and Fast Track Applications. His experience covers a wide array of therapeutic indications (oncology, neurology, rheumatology, infectious disease, etc.) and product classes (therapeutic proteins, small molecule drugs, cell therapies, gene therapies, vaccines and more). He is an advocate for patient-focused drug development and excels in engaging the FDA and other regulatory authorities as development partners.
Kevin earned a Bachelor’s degree in Microbiology from Colorado State University and a Master’s degree in Molecular, Cellular and Developmental Biology from the University of Colorado at Boulder.
Who Should Attend?
This webinar will benefit regulatory professionals and pharmaceutical/biotech companies that are developing products intended to treat serious conditions with an unmet medical need.
What You Will Learn
- The requirements that must be met to qualify for each expedited regulatory pathway
- The benefits of the various pathways and the information needed to receive these designations or approvals
- Strategic elements to consider when choosing expedited pathway(s) to pursue at a given time
Veristat is a scientifically oriented and impactful full-service clinical research organization (CRO) that is committed to partnering with emerging and small to mid-sized biotechnology firms to advance their therapies throughout the entire clinical development and regulatory submission process. Veristat helps growing clients solve the unique and complex challenges that arise when trying to accelerate therapies along the development pathway to successful regulatory approval, beginning with regulatory planning, study design, protocol development, site selection and start-up through to patient recruitment, clinical monitoring, data collection, analysis and reporting. Ultimately, we guide our clients to market success so that their therapies become available to improve and save people’s lives. Our impact is proven by our regulatory track record of success — in 2018, Veristat supported 12% of all NMEs approval by the FDA.