FDA Drug Approval Based on Real World Evidence

I just got back from the 2019 Global Genes Rare Patient Advocacy Summit, which I attended last weekend in beautiful San Diego. It was f*cking awesome! as always.

I have gone to the last six Summits. I will go to many more.

I come home refreshed, relieved, informed, invigorated, catharted, and ready to take on more Ehlers-Danlos!

This year I learned that as part of the 21st Century Cures Act, the FDA will consider real-world evidence, such as data from electronic health records, in approval of drugs. Wow!!!! Yay!!!! 🤗🤗🤗

It’s no secret. The FDA said it here!

“The 21st Century Cures Act, passed in 2016, places additional focus on the use of these types of data to support regulatory decision making, including approval of new indications for approved drugs. Congress defined RWE as data regarding the usage, or the potential benefits or risks, of a drug derived from sources other than traditional clinical trials.”

Dress by Vince Camuto. Shoes from Discount Dance Supply. Get competition dance shoes, hypermobile ladies, so you can party safely. They are built for moving in. Sparkly bag from the vintage shop next to Dr. Plance’s office, Pattye’s Closet, where over the years I have had so much fun spending way too much money on pretty things I don’t need, trying to gather myself for the long drive home after another wild afternoon with Dr. Plance.

This news from the FDA is verrrrrrrrry exciting.

Are you using Ascor to treat Ehlers-Danlos Syndrome?

Many people have reached out to me over time. I say, “Good luck!”

Many have asked me to organize a study, but I am not a medical professional and am not qualified. I say, “Why wait for a study, when Ascor is so cheap and safe?”

Many have told me they are doing much better with their shots. I should have kept track of them! Darn!

I didn’t because I didn’t see the point.

I do not want to name a protocol or theory after myself.

I am not selling anything.

I do not want a following.

I do not want to convince anyone this works. EDSers are confused enough!

This may only work on people with my genetic cause, but who knows. I am still cause not found.

I want you to get better any way that you can.

For myself, I got what I wanted:

My life is about something other than Ehlers-Danlos Syndrome.

Ah! Relief! Joy! Happiness!

But now, it would be possible to get FDA approval for the treatment of Ascor for EDS based on your electronic health records and mine. You can read more at Use of Electronic Health Record Data in Clinical Investigations Guidance for Industry.

Would you kindly email me the name of your prescribing physician?

I will keep a list in hopes of accomplishing this at some point in the future.

less flexible at gmail dot com

I am qualified to blog and pay Squarespace to host the story of my life.

Which is fine with me.

Writing my blog has been tremendously therapeutic. Putting your experiences and pain into words (talking or writing) is healing. This is my private journal, posted on-line for anyone to read. I don’t like to discuss my disability in person.

The hero of my story is Dr. Plance. He was the one who made my tremendous improvements possible, even if the daily injecting of Vitamin C to treat EDS was not his idea, just a happy accident. It would not have happened without him being him.

I am firmly committed to my life’s purpose. That is to look good and have fun. Getting mired down in studies or running a foundation may not exactly fit my mission.

These days, the world is a scary new adventure. How lucky I am to be in it.

Adjusting to life beyond surviving unlivable pain and fatigue was so strange and difficult. It requires different skills. It is a different rhythm. It is not dramatic. It is about simple things and small enjoyments. It is the opposite of high-tension, high-stress living, my old life of hopelessness and no relief. It is strange not to struggle. I am glad to say I have succeeded at making the transition.

This is what I did to get better:

I imagined life without debilitating Ehlers-Danlos. What might that be like?

I injected Vitamin C (now the drug Ascor) every day, starting in December of 2013. I have done this every day since.

I worked hard in physical therapy and exercise as if I were training for the Olympics, or better yet as if my life depended on it, because it does! Yes, it hurt, as it would for anyone weakened by illness or injury.

We have a complex musculoskeletal disorder.

I do not believe EDS can be treated without exercise. Weakness and being sedentary makes us worse.

The disaster of our proprioceptive-poor and intrioceptive over-loaded nervous system can be improved through exercise. Mine has, especially through balance and coordination training. Those modalities make me feel alive.

We need circulation to reduce symptoms of POTS.

We need exercise to strengthen the fascia to stabilize joints and organs.

I got off all brain-draining drugs. I refuse drugs that are anticholinergic (unless very short term) because I already have enough problems. Those drugs ultimately make us worse.

I avoid all extreme joint positions and stretching altogether.

I got a job, with help from the California Department of Rehabilitation. Thank you, taxpayers.

I powered through this huge change. It took courage. And time. I integrated with the collagen-tycpials. I got on with my life. Don’t fail to appreciate how hard change is, how it is human nature to keep complaining about the problems we have rather than upgrade to new ones. And if you go from disabled to functioning, it will change every relationship in your life.

I continue to grieve my disabled years, and all that I missed out on. Oh well.

I continue to embrace healing modalities, expanding on exercise and taming my unstable nervous system. Right now I am focusing on kettlebells, specifically the swing, snatch and get-up, perhaps the greatest exercises ever.

Healing took time. At my worst I was hardly able to get out of bed and unable to walk for more than a short distance. That is a lot to come back from.

I must be still healing, because I have not yet stopped improving.

“You can do it!!! You are f*cking awesome!!”