Expert Insights

Expert Insights

World Orphan Drug Congress Recap

World Orphan Drug Congress Recap
30 April 2019

There are several challenges facing rare and orphan disease clinical trials, including low disease prevalence, lack of validated outcome measures, and burden of disease. Earlier this month, our experts attended the World Orphan Drug Congress (WODC USA) in Oxon Hill, Maryland, where these and other aspects of trials were discussed.

The two-day event, held April 10 – 12, was well-attended and brought together representatives across all stakeholders, including patients, advocates, caregivers, researchers, trial sites, regulators and biotech and pharma professionals.

On April 11, our Vice President of Rare and Orphan Diseases, Lisa Dilworth, participated in a roundtable discussion “Meaningful endpoints – navigating the differences in what it means to patients, caregivers, physicians, and regulators during advanced therapies clinical development.” The roundtable was standing room only. There was a wide range of participants in the roundtable, including regulators, small biotechs, big pharma, founders of advocacy groups, and family members of patients with a rare disease. The roundtable was a lively discussion and covered several timely topics and challenges impacting rare and orphan disease clinical trials.

A few trends discussed included:

  • At-home video – Using video at the homes of patients, specifically children, so caregivers/parents can capture their patient achieving milestones to support clinical trial data.
  • Innovation Gaps- New technologies such as wearables, that can passively record endpoint data while reducing the burden of trial participation
  • Novel endpoints – Pushing the industry to move forward by designing and implementing new endpoints that are sensitive to meaningful change for caregivers, clinicians, patients and other stakeholders. Pharma and biotech companies typically use endpoints that were used previously, regardless of whether or not they have been successful or not. Creating new endpoints will enable the industry to provide meaningful change and results.
  • Meaningful cognitive data – There is a need for more frequent cognitive testing to capture important achievements and data that might be missed between clinical trial visits

We enjoyed interacting with and learning from the broad spectrum of participants at WODC and came away even better prepared to address the challenges we know are part of the rare disease space.  WODC is always an innovative event that pushes the envelope and addresses new changes in the industry. We look forward to follow-up discussions and interactions stemming from this year’s event and are eager to see what 2020 WODC US has in store for us.  

Contact us today if you want to learn how we can bring your rare and orphan study to life.

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