What Next?

By March 24, 2014 November 27th, 2018 Tracy's Blog

As we approach the White House petition deadline  on Friday of this week, we are hyper-focused on two things:

1) reaching the 100k goal

2) planning what happens next

First, a word on #1.

The word on #1 is “wow.”  Wow describes the emergence of families who – some for the first time since their son’s diagnosis — feel they have something to fight for, even if their own son is not in the group who can benefit from eteplirsen.  Wow describes the collaboration of organizations that have come together to urge the FDA to approve the first Duchenne treatment ever for use by all eligible children.  Wow describes the courage of the 100+ families across the United States and beyond who have told their stories on the local news, bringing a surge of awareness and support to this crucial effort.  Wow describes the ever-growing list of celebrities and the thousands of non-famous citizens who have joined The Race to Yes by signing our petition and encouraging their followers to do the same.

Wow also describes the anxiety brought on by the realization that we still need 3,051 signatures each day for the rest of this week (including today!) to reach our goal.  I know we can do it.  But I also know it will be close.  Please continue to hit up every store clerk, waitress, neighbor, and person-next-to-you-on-the-bus we come across this week.  Please share the petition link across all social media platforms.  Please check in daily on the Race to Yes Team 100 Facebook page to share signature-getting ideas with others who have joined the cause.  We are so close. We can bring this home.

And when we do, then what?  This is a crucial question.  It is easy to lose ourselves in the drive to reach 100,000 signatures.  But remember, that milestone is just one mile-marker to winning the Race to Yes.  It is a step toward getting the FDA to say YES to eteplirsen and to be more urgent and responsive when evaluating all new therapies for Duchenne.  Our message to the White House is clear:  The citizens of this country have spoken.  They support the use of the Accelerated Approval pathway for safe and effective therapies for Duchenne Muscular Dystrophy.  Now the ball is in the Obama Administration’s court to examine why the FDA is not using the tools it has been granted by Congress to expedite therapies for rare and fatal diseases.  We eagerly await their response.

In the meantime, we will continue to raise awareness and repeat again and again that the FDA has to act today, not tomorrow or next month or next year, to help our kids.  This includes continued media attention — so keep your eyes peeled for more news and continue to speak to your local media outlets.  We also need to remember that approval of eteplirsen is a first step toward ending Duchenne.  Eteplirsen helps a subset of children with the disease.  And while it slows the disease progression, it is not expected to reverse muscle damage that has already set in.  This first approval must pave the way for a speedy and ultra-efficient regulatory process that can be applied to follow-on exon skipping drugs and other new medicines.

Eteplirsen is a modern speed train, based on technology that was unimaginable ten years ago.  But tragically our tracks have not changed with the times, and the train is being held back.  Two years ago, Congress recognized the need to update the tracks that lead to approval and passed the FDA Safety and Innovation Act.  FDASIA was designed to allow exciting breakthrough drugs to quickly make their way to the patients who desperately need them.  Eteplirsen meets and even exceeds the criteria for this expedited pathway.  The FDA should be standing at the express track waving the flags for this drug to speed right through.