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A quick and dirty guide to healthcare in America

The healthcare briefing at the the National Health Institute was packed with enough information to make ones’ head explode.  Seriously.  Blow right off.

I made you an easy-to-follow guide of the information from the day’s events so your body can stay intact.  Here we go in no particular order:

rare disease day #rddnih

  1. There are over 300 million people in the United States.  30 million of them, or 10 percent have a rare disease.
  2. Of the 7000 rare diseases, only 350 or 5 percent of them have approved drugs. 
  3. At the current rate, it would take 2000 years to find a treatment for the rest to have treatment because of slow methodology, lack of communication, no funding, and bureaucracy.
  4. Biologics, biosimilars, and precision medicine is the wave of the future in medicine
  5. The FDA doubled rare drug approvals last year.
  6. Bi-partisan collaboration in both House and Senate to change the research and funding paradigm of rare diseases is unprecedented.
  7. Non-Pharma support organizations for rare diseases donated over $50 million for research last year and are putting together many programs to bring people and trials together (more on that later).
  8. International research data sharing is being streamlined for quicker availability to doctors.
  9. There is a lot of BIG legislation on the floor right now that could be a game changer for rare disease patients (and every other patient).  The biggest is the bi-partisan 21st Century Cures Act.  It sailed through the House with a vote of 344-77 but has stalled in the Senate. In a nutshell HR 6 accelerates the discovery, development and delivery of life saving and life improving therapies.  The other is the OPEN ACT.  This one could bring hundreds of safe, effective, and affordable medicines to rare disease patients within the next several years by incentivizing drug makers to “repurpose” therapies for the treatment of life-threatening rare diseases and pediatric cancers.  Together they could have to potential to speed up treatments by decades. 
  10. Your voice matters.  It’s an election year.  That means things don’t pass just because it’s an election year.  If you don’t think you can make a huge difference in your own life, you are fooling yourself.  Anyone with an illness should really take a look at these bills.  If you think they are a good idea, let your Congressperson know.  The more pressure we put on them, the faster they will take action.  The faster they take action, the faster we can get the drug protocols we desperately need at a fraction of the time and cost it takes now.  Sending a quick letter that could save your life is really easy to do too.  For the OPEN ACT, click HERE and a pre-filled letter will be sent to your Senator with your name on it.  For the 21st Century Cures Act, click HERE to send a letter.  The Senators count the letters received on a daily basis.  The more they get, the more they pay attention.  Get their attention now.

I think the most encouraging part was the smart (really smart) medical team from the National Institute of Heath.  They are thinking outside the box to shift the way disease is viewed and researched so medicine can move into the 21st Century.  I just hope it doesn’t get bogged down by politics.

About the Author

#rddnih Rare Disease Day A Quick and Dirty GuideLisa Douthit is the author of Amazon’s #1 bestselling book, Wellness Warrior – Fighting for Life in Fabulous Shoes and an Integrative Health Consultant who is passionate about healing from all perspectives.  After struggling with multiple bouts of cancer and autoimmune disease, no one understands the physical, spiritual, and emotional rollercoaster better than she does. As one of the 40 million American women currently suffering with an autoimmune disease, she made it her mission to have a voice for all those with an invisible illness, and prove you can be well without perfect health. She has a private Facebook Group called Wellness Warrior Tribe for all those with chronic illness looking for support and encouragement.  For more out of the box thinking that is guaranteed to bring you joy, Like her FaceBook Page Here or join the tribe Here.

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  • Deborah Collyar

    Hi Lisa,
    Thanks for your post. As you described there are lots of things going on, and pharma is definitely becoming more interested in rare diseases. There are also many valid concerns with the 21st Century Cures Act that could actually take away rights, and could cause a raft of new antibiotic drugs that could actually hurt people. As usual, the devil is in the details which I hope the Senate is working through. It is an important Act – need to keep the good and shore up the problems.

    Also wanted to mention a great group Called Cures Within Reach. Disclosure: I’m on their advisory board! They focus on re-purposing existing drugs for other diseases, so it might be worth looking into. They have a grant that helped them create the Cure Accelerator, and they just got 100 projects submitted in 100 days! Please tell the researchers you work with. I’d be happy to help connect them.

    Thanks for all of your efforts. I also write about health and medical research, and how we as patients can get involved to create more effective results at

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