expert insights webinars
Award-winning experts share their detailed and actionable insights to inform the future of pharma, biotech, and medical devices
View our complimentary webinar offerings below and register for one or more webinars.
Expert Insights webinars, presented by MMS
The Expert Insights webinar series offers free learning opportunities on current trends, high-value topics, and complex initiatives in the evolving pharmaceutical, biotechnology, and medical device industries. Current and archived webinars are available as follows:
Rare Disease Research in the United States: Understanding FDA Guidance and Flexibility in the Application of Regulatory Standards (August 17 part 1 & part 2 August 31, 2021 at 2 PM EST)
The Orphan Drug Act (ODA) defines a rare disease as a disease or condition that affects less than 200,000 people in the United States. Within this range we see conditions like sickle cell disease with a prevalence of over 100,000, down to rare genetic diseases that may occur in a handful of patients. As approaches to clinical development are constrained by issues of enrollment and statistical powering, the categories defined by prevalence distribution (rare, ultra-rare and uber-rare) require differing approaches within the framework of identical statutory standards for safety and efficacy. At the level of the ultra and uber-rare conditions that make up the majority of rare disease, there is an unmet need not only for treatments but also for the tools to evaluate whether a given treatment works.
This webinar will focus on challenges and areas of flexibility within the application of the statutory standards as they apply to rare diseases. Particular attention will be paid not only to the laws and guidelines that specifically relate to orphan drugs but also how the general regulatory standards that govern safety and efficacy are uniquely applied to rare diseases at different levels of prevalence.
This webinar will cover rare disease research:
- Core challenges – knowledge of the disease, clinical trial design and enrollment and others
- The legal framework and guidance relevant to rare disease research with case studies focusing on the role of innovative clinical trial design, Expanded Access data, and the natural history studies in recent approvals
- Strategies specific to ultra- and uber-rare diseases
- Amanda Beaster, Senior Global Regulatory Affairs Manager
- Ben Kaspar, Senior Global Regulatory Affairs Manager
- Uma Sharma, Chief Scientific Officer
Orphan Drug, Rare Pediatric Disease, and Expedited Program Designations: FDA Incentives to Promote Rare Disease Drug Development (Oct 6th 11-12 EST)
Substantial progress continues in the development of treatments for rare diseases or orphan products. In 2020, 32 novel drugs and biologics were FDA approved with orphan drug designation status. Notably, 1 of 5 (20%) novel biologic approvals in the Center for Biologics Evaluation and Research (CBER)—although not orphan designated—was used in rare diseases. In the Center for Drug Evaluation and Research (CDER), 31 of 53 (58%) novel drug approvals were orphan designated products. Unfortunately, 1 in 10 (25 to 30 million) Americans is affected by rare diseases (more than half of which are children), and approximately 95% of the more than 7,000 rare diseases have no treatments available.
To advance the development of rare disease treatments, the FDA Office of Orphan Products Development grants orphan drug and rare pediatric disease designations (ODD and RPD) to eligible products for the prevention, diagnosis, and treatment of rare diseases. Additionally, CBER and CDER provide four expedited program and approval pathways—Fast Track Designation (FTD), Breakthrough Therapy Designation (BTD), Accelerated Approval, and Priority Review Voucher Designation (PRV)—to facilitate and expedite the development and review of drugs to address the unmet medical need in the treatment of serious and life-threatening conditions. Because most rare diseases are serious or life-threatening, with a high unmet medical need, most qualify for at least one expedited program.
This rare disease webinar provides an overview of the FDA’s compendium of rare disease and expedited programs and provides sponsors with recommendations to leverage these opportunities and speed the development timeline. In this webinar, attendees will understand:
- Features and benefits of ODD, PRD, FTD, BTD, Accelerated Approval, and PRV
- Qualifying criteria for each designation or approval pathway
- When to submit a designation request
- Real world examples highlighting expedited drug development timeline with designations
- Key differences between FDA and European Medicines Agency (EMA) rare disease and expedited programs, including EMA orphan drug and PRIority MEdicines (PRIME) designations.
- Christine Clarke, PhD, Senior Medical Writer, Regulatory and Medical Writing
- Ben Kaspar, Senior Global Regulatory Affairs Manager, Regulatory Affairs