Beyond one indication: Unlocking the full potential of orphan drugs

Orphan drugs are essential for addressing unmet medical needs in rare diseases, but their development faces significant challenges, including small patient populations, high development costs, limited disease understanding, and complex regulatory pathways. Despite these hurdles, orphan drugs hold untapped potential to address multiple conditions beyond their initial indication. Expanding indications can help overcome market constraints, increase therapeutic impact, and foster sustainable innovation. This webinar will explore strategies for expanding indications for orphan drugs to maximize their therapeutic impact, unlock opportunities for innovation and collaboration, and address unmet needs across multiple conditions. Industry leaders will discuss how indication expansion can optimize R&D investments, balance ROI with patient access, and enable sustainable growth. Attendees will gain a comprehensive understanding of how pharmaceutical and biotech companies can leverage partnerships with Contract Development and Manufacturing Organizations (CDMOs). Experts will highlight how CDMOs can accelerate orphan drug development, provide scientific expertise to support and scale production to meet growing demands.

Paul T. Kim

JD MPP, Kendall Square Policy Strategies, LLC

With 30+ years of experience in Washington DC, Paul Kim advises patient advocates, global life science leaders, and innovative emerging companies on FDA and public health policy- developing legislative and policy strategies for regulatory reforms, drafting and enacting Federal legislation, and securing funding or creating effective P3s. Paul's experience extends across drug and device law; rare disease innovation; public health and immunization policy; biodefense policy; and congressional oversight & investigations. Paul has represented leading biotechnology, pharmaceutical and medical device companies, trade associations, and patient advocacy groups before the US Food and Drug Administration (FDA), the Centers for Medicare and Medicaid Services (CMS), HHS ASPR/BARDA, and Congress.

Cornell Stamoran

Vice President, Strategy and Government Affairs, Catalent

Cornell Stamoran is a life sciences industry strategist and has spent nearly 25 years engaged with the health care industry, including 20 years in advanced drug delivery. Cornell has directly participated in the development of drug design-enabled adherence and outcomes enhancement strategies for more than 150 branded drugs over the last 10 years. He holds several professional certifications and is a graduate of the University of Michigan.

Matthew Mollan

Vice President, Operations, Early Phase Development, Catalent

Dr. Mollan, Ph.D., joined Catalent in 2012 as General Manager, Kansas City following the acquisition of the site from Aptuit, and during his tenure, he successfully grew the business from less than $30M in revenue to over $100M last year while overseeing the launch of 16 new oncology and rare disease products over that timeframe. Prior to joining Catalent, Dr. Mollan worked at Aptuit from 2007, following a number of years with Pfizer and Warner-Lambert in roles of increasing responsibility. He holds a bachelor’s degree in pharmacy and a doctorate in pharmaceutical sciences from Rutgers University.

moderator

Surani Fernando

Healthcare journalist, writer & podcaster

Surani Fernando is a seasoned healthcare journalist and editor with over 13 years experience covering the biopharma industry. A Sydney native, she started her investigative journalism career in London covering clinical trials, M&A and financing deals for BioPharm Insight, later moving to New York to continue her work as an enterprise journalist and editorial leader for GlobalData and Reorg. She is now based in Madrid working as a freelance journalist, consultant writer and podcast producer. In November 2023, she launched the Raising Biotech podcast.