Changing the FDA's culture

The U.S. Food and Drug Administration

Irvine, CA – An FDA chemist tests the melting point of chemical compounds in drug samples.

Changing the FDA's culture

Download PDF

When an experimental enzyme-replacement drug for Hunter syndrome came along a decade ago, parents of children with the disorder were understandably desperate to get their kids the new medicine, called Elaprase.

Many families traveled hundreds of miles so that their children could take part in the drug’s key clinical trial. They may have expected that the trial would be an example of effective regulation hastening the timely arrival of a safe, new treatment. Instead, what these families experienced exemplified a broken and dysfunctional approach to drug trials, driven by an FDA culture poorly suited to serving the needs of the sickest patients.

The story of the Elaprase trial is important not because it stands out as an exception, but rather because it is increasingly characteristic of the FDA’s drug-review culture. That culture is the product of a poorly understood, but now well-established, attitude within the agency: an excessive desire for certainty.

This desire is primarily driven not by fear of unforeseen dangerous side eff ects caused by drugs under review, but rather by a deepening mistrust of the doctors who eventually prescribe such medicines and the companies that market them. And that mistrust, in turn, is impeding the availability of safe, effective drugs that could today be helping real patients.

Fortunately, however, this harmful culture can be readily improved — by implementing a few straightforward reforms of the FDA’s responsibilities and structure.

To read more, download the pdf.

 

Also Visit
AEIdeas Blog The American Magazine
About the Author

 

Scott
Gottlieb
  • Scott Gottlieb, M.D., a practicing physician, has served in various capacities at the Food and Drug Administration, including senior adviser for medical technology; director of medical policy development; and, most recently, deputy commissioner for medical and scientific affairs. Dr. Gottlieb has also served as a senior policy adviser at the Centers for Medicare & Medicaid Services. 

    Click here to read Scott’s Medical Innovation blog.


    Follow Scott Gottlieb on Twitter.

  • Phone: 202.862.5885
    Email: scott.gottlieb@aei.org
  • Assistant Info

    Name: Kelly Funderburk
    Phone: 202.862.5920
    Email: Kelly.Funderburk@AEI.org

What's new on AEI

image Recovering from tax time blues
image 10 welfare reform lessons
image Let HHS nominee Sylvia Burwell explain Obamacare lie
image Why bold ideas backfire in politics
AEI on Facebook
Events Calendar
  • 14
    MON
  • 15
    TUE
  • 16
    WED
  • 17
    THU
  • 18
    FRI
Wednesday, April 16, 2014 | 10:00 a.m. – 11:00 a.m.
Calling treason by its name: A conversation with Liam Fox

Join us at AEI as the Right Honorable Liam Fox sits down with Marc Thiessen to discuss and debate whether America’s intelligence agencies have infringed on the personal privacy of US citizens.

Event Registration is Closed
Thursday, April 17, 2014 | 4:00 p.m. – 5:00 p.m.
The curmudgeon's guide to getting ahead

How can young people succeed in workplaces dominated by curmudgeons who are judging their every move? At this AEI book event, bestselling author and social scientist Charles Murray will offer indispensable advice for navigating the workplace, getting ahead, and living a fulfilling life.

No events scheduled this day.
No events scheduled this day.
No events scheduled this day.
No events scheduled this day.
No events scheduled this day.
No events scheduled this day.