Dear Sen. Warren,

When you killed Orphan Product Extensions Now Accelerating Cures and Treatments Act (OPEN ACT) you effectively killed me. I know that writing this will not bring back OPEN ACT, but I also know that I cannot sit idly by when my future is at stake.

At age 17, I am living on borrowed time because of rare disease. I have become a strong advocate for health policy, knowing it is the only way to ensure a future, but your strong opposition to Cures Now feels like the trigger going off in an already loaded gun. 95% of rare diseases do not have treatments, so patients  rely on off label medications.  As I am sure you are familiar with, OPEN ACT intended to take medication already approved by the FDA and prove these safe and effective for rare disease patients. Yes, increased research for rare disease patients is necessary, but the patient populations are so small, there is little to no incentive for drug development and research. What we need are treatments that have already gone through the approval process to be repurposed.

Personally, I have to take over twenty medications a day, none of which are used for their approved purpose. I am fortunate because my family can afford it, but insurance will not cover a majority of these medications and I will be put in a position of choosing to pay for college or life saving/changing medication. Again, I am in the privileged few, but there are many families who have the choice between putting food on the table and paying for these necessary medications.

Without the ability to use off label medication I would not be alive. There will come a time in the future where a doctor will refuse to prescribe a life saving medication because it is off label, or I will not be able to afford it. You claim to not be bought off, to be a woman for the people, but this decision does not seem to be for the people - it seems to be for bio-pharma.

I am thankful that the 21st Century Cures package, which I have been advocating passionately for, for over a year, looks like it will be passed despite your opposition. There are still many important sections/sub-sections, but OPEN ACT was vital to my, along with many others’, future.

I know that because I am not a constituent you will most likely disregard this. You have already taken so much from me, I will not allow you to take away my voice or my hope.

Be ready for a fight with 21st Century Cures because rare and common disease advocates aren't backing down. I urge you to join us instead of going against us.


Shira Strongin

Founder of Sick Chicks, Patient Activist