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The Rare Disease Drug Dilemma: Great Value, High Price, Limited Access?

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.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)?

Watch 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

  1. https://rarediseases.info.nih.gov/diseases/pages/31/faqs-about-rare-diseases
  2. https://www.fda.gov/news-events/fda-voices-perspectives-fda-leadership-and-experts/fda-working-bridge-gaps-and-meet-needs-rare-disease-product-development
  3. 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.

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