[[whitepaper]] The New Reality: Maximizing Value in Rare Disease Launches

In Clinical Development, Featured on App, Orphan Drug Congress, Uncategorized, Whitepapers by Karina Kusova


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: https://www.accessdata.fda.gov/scripts/opdlisting/oopd/index.cfm, accessed 3/9/2017).

Download whitepaper >>

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 >>

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

Download whitepaper >>

Whitepaper has been provided by Cello Health.

Cello Health creates customized solutions for rare disease brands and assets.  We combine the expertise from our three core capabilities – Cello Health BioConsulting, Cello Health Insight, and Cello Health Communications – to help clients gain deeper insights into the patient journey, market realities  and advocacy landscape, and create focused clinical, regulatory and commercial strategies that optimize limited data sets, engage and educate key stakeholders, and effectively communicate the core value story.  With offices throughout the US and UK, Cello Health offers global resource and local support to help bring orphan drugs to market.

Learn more about Cello Health’s offerings and capabilities here >>

Cello Health are sponsors of World Orphan Drug Congress USA 2017 being held April 19-21, 2017 at the Washington Wardman Park, Washington D.C. Register now to hear more from Cello Health. Make sure to visit their booth (209).