Rare Disease Report

John Scott Explains the Difficulties in Working with Small Patient Populations

NOVEMBER 09, 2017
John Scott, PhD
John Scott, PhD is the Chairman of Pharmacology at the University of Washington.

Rare Disease Report caught up with him at the Fibrolamellar Cancer Foundation Summit in Stamford, Connecticut where he presented data on behalf of a multidisciplinary research group who have dedicated recent studies to fibrolamellar hepatocellular carcinoma.

In the video below, he discusses why conducting scientific research can be compared to participating in a highly competitive environment like professional sports, the importance of the public holding his work in high regard, and the challenges of working in small patient populations.

Scott: In some respects, I see research like professional sports because it’s a highly competitive environment and you’re always striving to be successful and coming up with new ideas. You’re only ever as good as your last paper. That’s a very challenging thing, but I think a very important element of that is that we’re funding by the public to do work that the public generally considers to be of high value. That’s the driving factor in terms of being able to maintain a level of precision and alertness and the integrity and the rigor of the work that we do.

The real issue, though, in getting back to the rigor and reproducibility, is being able to get the numbers of samples that are necessary to be able to make a fairly definitive mechanistic statement about something when you can only evaluate a few patients. In a general situation, you may have 100 or 200 samples that will allow you to come with a then fairly significant finding; it’s much, much more difficult when you have 5 or 6 or 7 different samples.

We’re all on a different genetic background, and that has a huge effect on the outcomes. That’s how disease works, and I think that actually an important and compelling factor that drives me to my work in human disease is that we’re not all genetically-inbred mice; we are all different. That’s one of the issues with rare diseases that makes for a real challenge. If there are 15-20,000 samples of cases in this country every year, how are you going to be able to get the numbers in your region that are statistically significant?


For more from the Fibrolamellar Cancer Foundation, visit the organization’s website: fibrofoundation.org.

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