World Orphan Drug Congress USA 2017
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[[whitepaper]] The New Reality: Maximizing Value in Rare Disease Launches

The landscape is changing. Clinical development plans no longer depend solely on the outcomes of a phase 3 trial, as companies are adapting their approach to strategy development for rare disease assets. This means that companies must plan earlier, with less data, to identify key components for their phase 2 trials that will address the breadth of future competitive levers. There are more rare disease candidates entering the funnel than ever—2015 and 2016 saw an all-time high with 354 and 333 orphan designations, respectively (source:, accessed 3/9/2017).


With rare disease assets being granted expedited review, launch is no longer a fixed point in time, and research and development (R&D), medical affairs, and commercial teams need to collaborate early in the process to reach critical consensus on where to focus, when to engage key health care provider (HCP) stakeholders, how to reach patients and caregivers, and what to say about the relative value and impact the asset will have in the market.


Download whitepaper for more on:

  • Strategize Earlier, Prepare to Flex
  • Patients: Engage Them Early and Often
  • Map the Journey, Uncover Clues to Establish a Sound Tactical Plan
  • It’s All About Education 
  • The Cost Conundrum

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