According to the National Institutes of Health (NIH), there are approximately 7,000 rare diseases and approximately one in ten Americans will suffer from one.1 The Orphan Drug Act (ODA) of 1983 incentivized drug manufacturers to enter these difficult niche markets. Over the years, development activity in this segment has shown no sign of slowing down. In 2018 alone, the FDA approved 35 novel products with an orphan drug designation, the most since the enactment of the ODA.2 This area is estimated to continue growing and become a $242 billion global market by 2024.3
Drugs to treat rare diseases were once immune to patient access management strategies due to the high unmet need, few, if any, treatment options, and small patient populations. However, as the cost of these treatments continues to spiral upwards due to the high cost of drug development, patient access is no longer a given. Due to the changing environment and economic pressures, price can no longer be assessed using traditional methods, and new paradigms must be considered.
Pricing pressures on payers to afford and manage costs of treating rare diseases and drug companies to demonstrate the value of their products are mounting. Securing a price sufficient to make a reasonable return on investment (ROI) is key to a drug program’s commercial success. But, how exactly do we determine the “value-based price” of a drug? And, who is the arbiter of what is or is not justifiable (health systems, formulary committees, patient associations)?
Join this webinar to learn the following takeaways:
- How pricing has evolved from the 1990s to today for the rare disease drug market
- What new pricing concepts are gaining a foothold in the US and elsewhere
- Why you should prepare to assess price and value through emerging lenses
References
- https://rarediseases.info.nih.gov/diseases/pages/31/faqs-about-rare-diseases
- https://www.fda.gov/news-events/fda-voices-perspectives-fda-leadership-and-experts/fda-working-bridge-gaps-and-meet-needs-rare-disease-product-development
- https://www.evaluate.com/sites/default/files/media/download-files/EvaluatePharma_Orphan_Drug_Report_2019.pdf
About Our Speakers

Roman Casciano is the General Manager and SVP in Analytica Laser, Certara’s Evidence and Access Group. Based in New York City, Roman’s role comprises the executive management of all strategic and scientific teams across the company’s eight offices and three divisions. Roman continues to actively participate in client engagements as a market access strategist, ensuring that customers can leverage all of the benefits of our multidisciplinary team and integrated offer.
Roman was one of the founding members of the Analytica Group in 1997 and remained with the company for over 14 years, serving in various capacities during his tenure ultimately serving as President from 2008 onwards. Prior to joining Analytica, Roman worked for 4 years as an environmental engineering consultant with HDR Engineering Inc. in White Plains, NY. Roman went on to participate in the founding of the Analytica Group in 1997, initially serving as Director of Operations and subsequently serving as General Manager for the German subsidiary from 2004-2006. After returning to the US from his tenure as General Manager in the German subsidiary and prior to taking on his role as President of Analytica, Roman worked for 2 years in Analytica’s previous parent company, Accentia Biopharmaceuticals as the Sr Vice President and head of the NY-based product development team with full responsibility for the conduct of its two on-going phase III clinical programs.
As an applied health economist and market access strategist, Roman has personally led hundreds of engagements in the international market access and HEOR context related to product value demonstration and has deep experience in the management of multidisciplinary teams of scientific and strategic consultants across continents. Roman’s experience ranges from analytical activities such as economic modelling and database studies, to strategic activities such as developing product pricing and market access plans and communicating evidence internally and externally for our customers. Roman has experience across the product lifecycle, including assessment of early phase assets, licensing due diligence, and go/no-go decision-making support, as well as launch planning and commercialization activities.
Roman received both a Bachelor of Science degree in Mechanical and Aerospace Engineering and a Master of Engineering Degree in Mechanical Engineering and Engineering Decision Making from Cornell University.