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Compliance In Focus
Posted by Brandy Smith on Wed, Sep 5, 2012

FDA Helping or Hurting? What’s the Right Balance?

FDA helping or hurtingIn the United States, all new drugs and/or medical devices are subject to various levels of scrutiny by FDA.  The Food, Drug, and Cosmetic act of 1938 set into play the requirement for new drugs to be determined safe before they can be marketed.  Sometimes the regulatory process can be time consuming, but the whole reason regulations exist is for the protection of the U.S. population at large.    History is full of examples of treatments with good intentions causing more harm than good.  Think of it as a necessary evil if you will. What is the perfect balance between fastest path to market and proper safety evaluation?

Recently, a poignant example of this system at work was covered by Susan Donaldson James for Good Morning America.  She writes of the story of two brothers from Vermont suffering from Duchenne Muscular Dystrophy.  One of the brothers qualified to be in a clinical trial for a new drug from Sarepta Therapeutics called Eteprlirsen, which is aimed at treating the degenerative symptoms of the disease (loss of muscle strength and motor movement).  The other did not.  The brother who received the experimental treatment improved significantly, while the other was left on the outside looking as his condition worsens.  It’s a heartbreaking story, but a perfect example of the ethical dilemma faced by FDA everyday.  As Serepta Therapeutics works diligently to complete the trial and secure regulatory approval so the drug can be marketed, there are many other kids out there who may benefit from it now (only 12 children total are enrolled in the trial and are therefore able to receive the drug as a treatment).

With a story like this, it’s easy to take the side of the debate that would urge for faster approval.

What if we think about this example further in a hypothetical sense? 

  • What if FDA threw caution to the wind, changed its rules, and approved the drug tomorrow (after only 12 people receiving it for a limited amount of time)?
  • What it if 6 months from now everyone taking the drug suddenly develops a dramatic recurrence of symptoms, or they spontaneously combust? 

Of course these examples are over the top, but the point is that something could happen.  And, if it were to happen FDA would be lauded for not taking the proper amount of time to fully evaluate the drug.

Where do you stand on this issue?  Do you think FDA does a good job of balancing the need to allow access to helpful medical treatments with scrutiny to protect society at large?  Comment below and tell us what you think the right balance is for investigational products.

Photo Credit: DigitalnativeALCOA Checklista989a7b7-a2d4-43fc-9f0f-c5265e00587f



Topics: Right Balance, FDA


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