World Orphan Drug Congress LIVE: Workshop Day

In Orphan Drug Congress, Orphan Drugs Live by Freya Smale

Today’s pre-conference workshop was an informative success.

To recap a bit on the day, we sat with David Lapidus from Lapidus Data Consulting and George Milstein from Cowen Group this morning to learn how to forecast for an orphan drug and how to factor in market conditions such as epidemiology, natural history, treatable population, diagnosis, uptake and price.

Meanwhile, in the marketing orphan drugs workshop, we learned about some of the challenges in patient compliance and how to manage patients for the long haul. Moderated by Florian Brey, Medical Director, Juice Pharma, we learn that it’s not about good science, but much to do as well with reaching out and maintaining patient relationships. This 3-hour workshop also discussed how successful orphan drug launches, such as that of Cinryze and Translarna. Great presentations were given by Kim Gariepy, Associate Director, Product Marketing – Rare Diseases, Shire, Greg Gorgas, SVP, Commercial, Mast Therapeutics, Paul Stickler, VP, Commercial Operations, Recordati, and Malcolm McKenzie, SVP, Strategic Planning & Customer Insights, Juice Pharma

Following the morning sessions, Mapi’s Ron Christensen, Charles Makin, Patricia Anderson, and Kelly Franchetti cover the topic of building rare disease and orphan drug programs with all the stakeholders in mind during a 4 hour classroom styled workshop. Their discussion included

  • Forecasting and best thoughts at an early stage rather than solely prior to commercialization.
  • Areas around health economics and market access as well as optimized operational pre and post approval programs to support all stakeholders

After the workshop we were able to take away an understanding of patient insights and regulatory support. It was a very interactive session where at one point, the audience broke into small teams to have more intimate discussions building ideas with each other.

The patient access workshop discussed how accessing rare disease patients continues to be the most critical in the orphan drug industry. Robert Hawes of Marshfield Clinic, Kerry Goetz of NIH, and Nick Sireau of AKU Society gives a great presentation on the various innovative ways to reach out to patients; even when they seem to be nowhere to be found.

Overall a successful pre-conference workshop. Looking forward to Day One of World Orphan Drug Congress USA 2015! Don’t forget the hashtag #WODC

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