With over 1,200 attendees and 350 speakers, this year’s WODC in Oxon Hill, MD did not disappoint. It was our pleasure to, once again, be a part of the world’s largest gathering of rare disease focused pharma execs, researchers, governing bodies, and patient groups to network with and learn from during the week.

If you were unable to addend the Congress, here are a few of the key subjects we heard throughout the event:

  • With more biotechs launching each year than ever before, investors will continue to place more emphasis on early commercialization projections, many times prior to entering the clinic. Companies will need to put their marketing caps on earlier as the years progress.
  • A focus carried forward from last year was access and affordability, an ongoing battle between pricing and the need to remain innovative in rare disease. The industry is becoming more cautious of Payer push-back, with hopes that it doesn’t affect its current concentration on addressing the 7,000+ known rare diseases.
  • Last year marked two milestones in gene therapy, with RNAi approved and CRISPR gene editing technology beginning its first human trial, the future holds promise of cured disease and new treatments, including advancing RNAi therapy as an entire new treatment class
  • As expected, there was a concentration on patient data, digital health, and how the role of machine learning and AI in drug discovery, patient mapping, precision clinical trial recruitment, and drug development risk mitigation will greatly increase in 2019
  • There is a rising trend of going beyond a “one disease at a time” mentality and moving to platform approaches to rare disease drug development, which will lead to more efficient advancements and approvals of new therapies
  • A new level of patient engagement is needed when preparing for rare disease clinical trials. Not only to identify the patient story, but to deliver education that facilitates patients/caregivers and their families’ understanding of the process (potential benefits, requirements, etc.,). Earlier patient engagement will lead to more successful trial design and outcomes.

Please contact us if you have any questions about last week’s WODC. If you missed us at our booth and would like to learn more about our outlook on rare disease, click here.

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