Mindy Leffler…What I Said to Congress

Posted by: Christine McSherry on February 08, 2014

My name is Mindy Leffler, and I am from Seattle, Washington. I am the mother of Aidan, who is ten years old. Aidan wants more than anything to dedicate his life to the preservation of jaguars. For the past two years, he has been flying from Seattle to Vancouver, BC every Wednesday in order to participate in a clinical trial that was eventually halted. He is part of the generation that is being asked to sacrifice his childhood to advance science on behalf of the boys who are younger than him and boys who are yet to be born.

When Aidan was diagnosed, I dug into the science because no matter what happened, I wanted to be able to tell him that I had done everything I could. As parents, not only are we educating ourselves on the science, we are funding research to the tune of millions of dollars, and in some cases, we are actually purchasing drugs and taking them through the development process ourselves.

We are here today because Congress has a role to play as well. You all have spent years funding research and passing policy that’s designed to get treatments to the patients that need them in a timely way. And we are now in a time where all of those efforts have come to fruition, and the FDA is standing in the way.

Two years ago, Congress passed the Food and Drug Administration Safety and Innovation Act (FDASIA) as a special tool designed to expedite drug development past the normally decade-long process it normally takes to get a drug to market. FDASIA establishes the following criteria:

  • That the disease is rare and deadly

  • That is acknowledges a surrogate marker that is reasonably likely to predict clinical benefit for patients

  • That it look at the balance of risk versus benefit and take patient opinion into account

Eteplirsen meets all of these criteria.

A year ago, we as parents starting meeting with the FDA to explain the need to get Eteplirsen to patients as quickly as possible. Last summer, we had hope for the first time; FDA told the company that they were open to a new drug application. But in November, FDA abruptly reversed it’s decision with no scientifically-valid explanation. And after five meetings, we moms realized that we needed help. And so we enlisted the world’s most renowned doctors in the treatment and research of Duchenne. We met with the FDA yesterday with four additional experts, but in front of you today are Dr. Kunkel, the man who led the team that originally discovered the dystrophin gene, Dr. Wilton, the man who first accomplished exon skipping in the lab, and Dr. Mendell, the primary investigator of the Eteplirsen study and the man who has been a primary investigator on more clinical trials for Duchenne than any doctor in the world.

The FDA is ignoring something that is both obvious and extraordinary. And I will let these world renowned experts in Duchenne tell you why.

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