The New England Rare Disease Statistics (NERDS) Workshop, the first of its kind in the nation, attracted more than 160 attendees from all over the country on October 11, 2019. This sold-out event filled a void for statisticians working to bring cures for rare diseases.
The last 10–15 years have seen an emergence of drug development efforts in the rare disease space. Contributing factors include increased public awareness, encouraging drug regulation changes, scientific advancements in cellular/molecular biology and genetics, development of innovative trial designs, an influx of capital investment, and availability of scientific talent through decades of cultivation. As a result, a number of regulators, academicians, and industry statisticians now work to bring these orphan drugs to patients, facing unique technical issues and challenges.
Recognizing the need for a conference like NERDS, Ouhong Wang, vice president and head of biostatistics at Vertex, proposed the conference so statisticians across the rare disease drug development spectrum would have a forum to exchange ideas, share experiences, and network.
Yang Song, Vertex (Co-Chair)
Sammi Tang, Servier (Co-Chair)
Kun Chen, University of Connecticut
Charlie Cao, Biogen
Roee Gutman, Brown University
Mike Hale, Takeda
Daniel Meyer, Pfizer
Jeffrey Palmer, Pfizer
John Zhong, REGENXBIO
The one-day workshop included detailed presentations and discussions. Vertex’s incoming CEO, Reshma Kewalramani, kicked off the event by delivering the keynote speech, “Tyranny of Numbers.” The scientific program featured speakers including L.J. Wei of Harvard University, Robert Beckman of Georgetown University, Jingjing Ye of FDA, Ziliang Li of Vertex, Chenkun Wang of Vertex, Rima Izem of Children’s National, Ming-Hui Chen of the University of Connecticut, Qing Liu of Quantitative and Regulatory Medical Science, LLC, Balram Gundapaneni of Pfizer, Feng Tai of Agios, and Peng Sun of Biogen. In addition to several actual case studies, topics discussed included treatment effects in rare diseases, trial designs, pediatric trials, historical control, comparative effectiveness, and matching methods.
Attendees’ feedback was overwhelmingly positive, and there are plans to continue the workshop.
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