Small molecule orphan drugs: Status quo, challenges, and perspectives

The development of orphan drugs in the past year has continued to trend upwards in the US, the European Union, and Japan. This is due to the financial incentives offered through regulation, with orphan designation appearing to be successful at generating opportunities and positive value for drug sponsors and patients. Despite that, many rare diseases remain without effective treatments despite the rising number of approved orphan drug products. In addition, the decision to invest in the orphan drug space is more complex than simply responding to regulatory incentives.

In this webinar, experts will discuss the key drivers that motivate drug sponsors in developing orphan drugs. In addition, experts will explore the challenges of orphan drug development and manufacturing and will discuss how the industry is leveraging strategic partnerships to help mitigate these challenges to create a viable orphan drug business model.

Key learning objectives:
  • Understand the drivers of growth in orphan drug investment and development
  • Learn how to navigate the complex development and manufacturing space of orphan drugs
  • Discuss practical considerations for supply chain when bringing an orphan drug to the clinic and market
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Cornell Stamoran

Cornell Stamoran

Vice President, Strategy and Government Affairs, Catalent

Dr. Stamoran, Ph.D., leads Catalent’s strategic planning, market intelligence, and government affairs efforts. He has spent nearly 30 years engaged with the healthcare industry, including more than 25 years in advanced drug and biologic delivery and outsourcing. In 2012, Dr. Stamoran founded the Catalent Applied Drug Delivery Institute, whose mission is to educate and advocate for the enhanced use of drug delivery technologies to improve patient outcomes.

Evelyn Kelly

Evelyn Kelly

CEO and Founder, Orphan Drug Consulting

Evelyn has over 20 years of experience in the pharmaceutical industry in both distribution and orphan drug companies, working across Supply Chain and Quality Assurance. Evelyn brings years of experience in manufacturing and distributing orphan and speciality drugs to over 50 countries globally. Evelyn specializes in the management of supply via virtual, outsourced models and has been named as both a QP and RP on multiple licenses. She has also held various supply chain leadership positions in Shire, NPS Pharma and PTC Therapeutics.

Evelyn is experienced in facilitating cross-functional strategic entity decisions, organizational redesign, selection of vendors and corporate activities. Evelyn founded Orphan Drug Consulting in 2017, with the vision of supporting companies across key areas such as Supply Chain, Quality Assurance, Launch and Finance/Tax.

Evelyn holds a Master of Pharmacy from Robert Gordon University and a Master of Business from the Irish Management Institute, as well as being an Adjunct Professor for the Trinity College Dublin School of Pharmacy.

Matt Mollan

Matt Mollan

Vice President, Operations, 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.




Arsalan Arif is a news media entrepreneur who set out in 2015 to build his vision of an independent biotech news company at Endpoints News.