Why I'm Fighting for Access to Off-Label Medicine


My story with #CURESNow and OPEN ACT started as many do – with a rare, serious disease, and the timid words “there is no FDA approved treatment.”

I was uncompromising and filled with the indefatigable hope that only a wide-eyed 13-year-old could have. Unwilling to accept the fact that my future was dense with uncertainty, I did my research and found an off-label infusion therapy that maybe, just maybe, could help.

It did, and here I stand — an activist who has lived an incredible 17 years and has become a little too obsessed with making history.

This is exactly what the 21st Century Cures initiative will achieve. It will give this generation of youth the chance to see treatment developed in their lifetime. By incorporating patient perspective into drug development, identifying disease earlier, modernizing clinical trials, incentivizing rare disease drugs, accelerating the cycle of discovering, developing and delivering new cures and treatments, and providing an extra 1.75 billion dollars for the NIH and 110 million dollars for the FDA for five whole years, this may just be one of the most transformative bills to pass Congress in decades.

Youth are often grossly underestimated. It is beyond important to highlight the fact that ill young people are still intellectuals capable of playing a vital role in the passing of legislation that will directly impact their lives and the lives of many others.

It is time to turn these silent hurts into screams of survival and pleas for change.

We are not the vulnerable, sick youth the media so often portrays. We are change-makers. We are movers and shakers. We are the next generation of leaders. In a world that so frequently silences based on age or circumstance, we must be bold, brave and loud.

The conversation surrounding Cures has been gaining momentum and it is crucial we don’t allow it to peek here. With both Speaker Ryan and Senate HELP Chairman Alexander naming the 21st Century Cures initiative a top priority, we know that Washington is listening. Patient voices are important in securing a vote and gaining bipartisan support now more than ever. Cures truly impacts every American, regardless of age, gender, religion class, race, or creed.

Illness affects everyone, and therefor 21st Century Cures does too.

Want to get involved? There are a few ways you can help:

1. Contact your representatives. Ask your Senators to support the 21st Century Cures initiative, and your Congressman to co-sponsor OPEN ACT (HR 971/S 1421).   Bi-partisan support is crucial.

2. Tweet “I need #CuresNow because” with your own story. I’ll be retweeting.

3. Change your Facebook and Twitter profile pictures in support of #CURESNow by using this link.

Never doubt the power of a single voice…your voice. You never know who is listening and what change you can bring.


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