Original trial design and sham control ethics

uniQure is a business that develops gene therapies. uniQure's AMT-130 is currently in the headlines. This podcast episode attempts to present the Huntington's Disease community's arguments for why the FDA should work collaboratively to design a palatable drug trial.

Last week the FDA told uniQure that AMT-130 would be subject to a double blind, Phase 3, randomized controlled study. While the scientific basis for that request is sound in most cases, the Huntington's Disease community argues that path is overly burdensome; perhaps impossible.

This episode cites:

Dr. Sung - https://www.youtube.com/watch?si=VhMQaFAZRvQA5NRj&t=62&v=mwEEJ91LeJY&feature=youtu.be

Sr. Sarah Tabrizi - https://www.youtube.com/watch?v=wKj1JRfJBRE&t=356s

Dr. Ed Wild - https://www.spreaker.com/episode/uniqure-update-with-dr-ed-wild--67900959

FDA papers - External Control Guidance https://www.fda.gov/media/122425/downloadUse of Bayesian Statistics - https://www.fda.gov/regulatory-information/search-fda-guidance-documents/use-bayesian-methodology-clinical-trials-drug-and-biological-products

Senate Committee on Aging - https://www.youtube.com/watch?v=yLm1SltbRxw&t=2702s

Sponsorship Information

Thank you to ⁠⁠⁠⁠⁠⁠Trata⁠⁠⁠⁠⁠⁠ for sponsoring the show.

If you're listening to this podcast, you'll like Trata. Trata is buyside to buyside conversations on individual stocks. Trata makes finding a bull or bear on any stock as easy as clicking two buttons. Over 125 funds globally contribute that collectively cover 2000+ tickers. Trata raised over $3mm coming out of Y Combinator. Before you would track 13Fs, now you can understand what funds are actually thinking. You can join as a lurker or you can join as a contributor and Trata will pay you hundreds of dollars per call. For a free trial, go to ⁠⁠⁠⁠⁠⁠trytrata.com/brew⁠⁠⁠⁠⁠⁠ 

OG Sponsor Shoutout!

Thank you to ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Fiscal.ai⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠ for sponsoring the show.

DISCOUNT INFO: If you use the affiliate link ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠fiscal.ai/brew⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠, you will automatically get 2 weeks of Fiscal Pro for Free and if you find that you want to upgrade, my link will get you 15% off any paid plans. About ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Fiscal.ai⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠

⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Fiscal.ai⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠ is the complete modern data terminal for global equities.

The ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Fiscal.ai⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠ platform combines a powerful user experience with all the financial data capabilities that professional investors need. 

Users get up to 20 years of historical financials for all stocks globally that they can easily chart, compare, or export into their own models. 

And unlike legacy data terminals where it can take hours or even days, ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Fiscal.ai⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠’s data is updated within minutes of earnings reports. 

⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Fiscal.ai⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠ also tracks all the company-specific Segment & KPI data so you don’t have to. 

• Like to track Amazon’s Cloud Revenue? They’ve got it.

• How about Spotify’s premium subscribers? 

• Or Google’s quarterly paid clicks?

They’ve got all of it.