Changing the Climate at the FDA

Posted by: Christine McSherry on October 15, 2013



Thank you and Congratulations to our Friend Janet Woodcock


Last week, Jenn and I were honored to attend the RareVoice Award Gala.  We were invited after I was nominated for an advocacy award, truly an amazing honor for a mom who has been advocating for accelerated approval for a compound that is in trials for Duchenne.  The event was hosted by the EveryLife Foundation.  A warm thank you to Dr. Emil Kakkis, President and Founder of the Foundation,  and Julia Jenkins, Director of Public and Government Relations, who brought together people from all different areas of the rare disease community – all working for the same goal, getting treatment to those affected by rare disease.  Each and every person nominated was unique in their approach, yet the same, by acting as a catalyst for change.  Their backgrounds included; legislators, the nonprofit sector, parents, government officials, regulators and others. The event brought us all together – under one roof, but clearly, perhaps unknowingly, made us recognize that while we are all working in different areas, we are working for a united goal, all trying to save the lives of children, faster.

Most notably, Dr. Janet Woodcock was recognized as a hero and advocate.  Dr. Woodcock was celebrated for her  work in advocating for faster approval pathways, advocating for innovative approaches in clinical trials for rare disease, and trying to ensure that every patient, every child has access to potential therapeutics. She was the winner of the award for Government Agencies.

Dr. Woodcock was extraordinarily candid and endearing in her conversation with Jenn and me.  She talked to us about her commitment to the Duchenne community and all rare disease and her dedication to staying at the agency to continue the work she started. Further, we discussed how our efforts are helping to support her and the agency in making positive changes internally.  She told us how the climate at the FDA was changing, and that we (Jenn, Mindy and I) are part of that change.  I was thrilled to get to have a conversation with Dr. Woodcock and to get her input. She shared how FDASIA is allowing parents and patients to have meaningful conversations with the agency about risk/benefit. She spoke honestly about how every parent of every child with a rare disease – has a voice and that used, in the same way we are using ours, through thoughtful dialogue and good science, these conversations are working. She explained that our tactic of fervently supporting our mission and with the backing of letters, congressional briefings, and the media, we are effectively creating the right pressure to achieve accelerated approval. I was impressed that she is so dedicated to our cause.

The true highlight of the evening was that Dr. Woodcock was able to meet our boys (picture pending). Her communication with the boys was enthused and spirited, as if she had been looking forward to this moment for quite some time. She thanked us for the opportunity to meet the boys – noting that opportunities like this were rare, how nice it was to meet the patients and families that it represents.

Time, we just can not afford to lose another minute.

If accelerated approval is lost for eteplirsen, the cascade of losses will be insurmountable, immeasurable.  It will not just be an added year, several years or even a decade…it will be the loss of other, future and potential therapeutics as well.   While exon skipping appears to be an effective technology that can help boys now, its limits are unknown, therefore, we must continue the momentum to bring accelerated approval for eteplirsen, all the while creating a pathway for all other complementary therapies that could be used alone or in conjunction with exon skipping. We don’t want to be back in this position years from now…we have the ability to influence and educate the regulators now.  We must keep “Duchenne” on the lips of the FDA, they need to hear the community, now for eteplirsen – and consistently for other potential therapeutics: laying the groundwork for the cocktail(s) that will treat this generation of affected individuals.


  • All the dystrophin data that is needed to consider this compound under accelerated approval is there.  Given all the information that we have today, eteplirsen is “reasonably likely to predict a clinical benefit”.




  • Dystrophin is the most reasonable surrogate marker, it is the lack of dystrophin that is the causes Duchenne.




  • Given all the information that we have today, It would be unethical to suggest a placebo randomized control trial for the eteplirsen trials and/or other exons.




  • The accelerated approval for eteplirsen, the Duchenne community – is a victory for all rare disease, as it changes the landscape for patients, regulators and industry.




The time is now to make this happen or we risk losing momentum  - for not just exon skipping – but also the future of other compounds.

Today, I heard from over a dozen organizations, from around the Globe – all members of the Duchenne Alliance.  An international effort to join all Duchenne specific organizations in a way that increases our communication, collaboration and streamlining of funding promising research.  All of them agreed, it’s time – time to speak up and be heard.  Together, we endorsed the next step in attaining our goal of accelerated approval for eteplirsen.

The RareVoice Awards were an amazing evening for Duchenne, celebrating the progress we have made. It was incredible to gather so many legislators, parents, government employees, all under one category “advocates”…working towards one goal, saving children’s lives, faster. And I’m so pleased Janet Woodcock was recognized for the hero she is and the friend she is to the Duchenne community. It is through her inspiration that we will take our next step…and for that, the highest award will be in finding the path that will  deliver treatments to our children and patients, especially those in rare disease, faster.




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