1 / 10

Overview

Development and Approval of Drugs and Devices EPI260 Lecture 12: Lessons from Drug Development June 3, 2010 Richard Chin, M.D. Overview. How regulations evolve Risks of drug development CAST FIAU University of Pennsylvania Vioxx Tysabri TeGenero. Regulations.

yitro
Download Presentation

Overview

An Image/Link below is provided (as is) to download presentation Download Policy: Content on the Website is provided to you AS IS for your information and personal use and may not be sold / licensed / shared on other websites without getting consent from its author. Content is provided to you AS IS for your information and personal use only. Download presentation by click this link. While downloading, if for some reason you are not able to download a presentation, the publisher may have deleted the file from their server. During download, if you can't get a presentation, the file might be deleted by the publisher.

E N D

Presentation Transcript

  1. Development and Approval ofDrugs and DevicesEPI260 Lecture 12: Lessons from Drug DevelopmentJune 3, 2010Richard Chin, M.D.

  2. Overview • How regulations evolve • Risks of drug development • CAST • FIAU • University of Pennsylvania • Vioxx • Tysabri • TeGenero

  3. Regulations • Regulations tend to be reactive • Elixir of Sulfanilamide • Thalidomide • CAST • Risk management • New tools for FDA • Practice of medicine • Difference in other countries

  4. Risks of Drugs and Drug Development • Drugs are poisonous • Clinical trials are dangerous by design • Testing limits • Testing new drugs and indications • Informed consent and IRBs • No direct relationship with patients • Social benefits outweigh risks

  5. CAST • Post-MI patients are at risk for sudden death • Ectopy predictive of death • Antiarrhythmic drugs reduce ectopy

  6. CAST

  7. FIAU • Fialuridine was a Hep B drug candidate • 15 patients exposed • 5 died • 2 required liver transplantation • Study conducted at NIH – NIDDK • Investigations by IOM, FDA, NIH • Conclusion was that studies were justified, consents were obtained properly, the studies were adequately monitored for the most part

  8. University of Pennsylvania Gene Therapy • Gene therapy study of ornithine transcarbamylase deficiency, an X-linked genetic disease of the liver • Jesse Gelsinger volunteered for the study, and died • Multiple failures in conduct of the study • Improper informed consent (key information missing) • Protocol violation in enrollment (Jesse has high ammonia levels) • Other failures • Significant financial conflict of interest

  9. Tysabri • Alpha 4/Beta 7 inhibitor • Highly effective for MS • Approved on basis of reduction in relapses • PML found in several patients • Drug pulled from market • Now back on with closed distribution channel

  10. TeGenero • TGN1412 – agonist antibody to CD28 (BAD idea!) • 6 patients enrolled rapidly into Phase I • All developed DIC and cytokine storm • All admitted into ICU, all survived • TeGenero out of business • New rules for Phase I studies

More Related