The fight for 21st century cures: A plea for help from a 16 year old girl.

Hello all,

This morning I woke up with a migraine. I was unable to get out of bed and I slept until 3:00 in the afternoon. Recently I have been very sick and my health has gotten beyond tolerable. When I was able to look at my phone, I was overwhelmed by the supportive comments that I have received from everyone. People reaching out to me and thanking me for standing up for the rare disease community. As I was very touched by this I was also heartbroken. I was heartbroken because I realized that so many voices are unheard. I believe in many things, but I believe more than anything that God wants us to stand up for ourselves and stand up for others. I am standing up not only for myself in this letter, but I am standing up for all of you zebras out there. The patients with diseases so rare that your doctors don’t know what to do. We all need to fight. We need to get everyone who is on our side to support us. We need to go far and wide and scream at the top of our lungs. One person cannot change the world, but together we can accomplish anything. Please everyone call your senators even if you do not live in Massachusetts. Contact everyone you can and bug the hell out of them. It is our time to stand up and fight. It is our time to make our voices heard. We can no longer sit by and watch the world forget us. We are rare, but we are not less. At the bottom of the page is my letter to Elizabeth Warren. Along with a link to “The Sick Chick’s” page. I have also included links to Elizabeth Warrens speech, the senate contact list, the Mighty to read stories about people with rare disease and non profit organizations websites. Please share this with everyone you know! No matter if this bill passes or fails, this movement has shown us that we can work together and together we can make a ripple in the pond no matter how small that ripple may be. Whatever you do please never loose hope in yourself and your ability to make a difference in this world. 21st Century cures is so important to rare disease patients for the reasons I list in the letter. Please do not let this bill die!

Dear Elizabeth Warren,
Today I am embarrassed and ashamed to live in Massachusetts. The state I love, the state I go to school in, has been hijacked not by “big Pharma and republicans”, but by you. I want to first say that I am a democrat. You see, I am also a disabled American. I have many rare diseases and have undergone 20 surgeries in my 16 years of life. I have had more surgeries than years on this Earth; think about that for a second. When I heard you are against the 21st century cures act, my stomach went into my throat and tears welled up in my eyes. Now I feel as though my home state is against me. Against helping people like me. I feel as though you have forgotten me and left me out in the cold to die. I read your arguments and I truly hope that you take the time to read mine…
1.) “Legalizes Fraud”: You tell us about how the 21st century cures act is allowing patients to get medications they do not need. “You can’t sell a headache pill as a cure for cancer.” Yet you forget that there is still a medical doctors prescription required to obtain these medications. In short, you are saying that you, someone who is well educated in law, knows more about someone who is well educated in Medicine. Let doctors do their jobs as doctors should. If someone has brain cancer, they might need a headache pill as well, also there is no “cure” for cancer as of yet, but treatments, which may help them. As a rare disease patient I have suffered because of the “off label medications” rule. Medications change the molecular biological processes that go on in our body. Especially for when dealing with neurological diseases, some of the exact effects that these medications have are unclear until later. For example the drug Doxipin, is thought of as an anti-psychotic, and yet, some studies have found that it is helpful for allergic reactions. This drug saved my life when I had anaphylaxis. Benadryl is alike Doxipin in the respect that it makes people seem sedated, yet people don’t take it to calm them, they take it for their allergies.
2.) Covers up bribery: “Once again, drug companies cozied up to Congress to gut that disclosure rule – allowing any gift related to “medical education” to be exempt.” When I first became ill, my family was clearly devastated as I was only six years old. By the time I was eleven, my mom had started up her own non-profit organization with a few friends. Since then, they have grown in size and are doing amazing things. If my Mom were a pharmaceutical representative, this means she couldn’t ask doctors to do studies for her non-profit due to conflict of interest issues. I understand what you are saying, but many doctors are not frauds. They go into their profession for the same reason, you became a politician, to help others. This means that a pharmacy company cannot put on a medical conference. The regulations on drug companies are so strict that drug companies are having to spend an extreme amount of money on legal help. One drug company I know of has 20 lawyers on staff. When companies have to pay this much for legal help, they run up the prices of their drugs. What you are suggesting, contributes to the already high drug prices.
3.) Hands out special deals to GOP donors: “The Cures bill creates a special deal so people can sell these treatments without meeting the FDA gold standards for protecting patient safety, undermining the integrity of the FDA and the safety of untold numbers of desperate patients.” To get a treatment approved by the FDA is very difficult. Frankly, it is unrealistic for rare diseases where only 10 people in the entire country are affected. How can you collect proof of a treatment when there are not enough resources to collect that? When a person is dying of a rare disease it should be their choice what to do with their body. This choice should be made alongside a medical professional who took the Hippocratic Oath and is not going to do wrong by the patient. You support abortion, why wouldn’t you support this? A patient should have a choice in this country which treatments they try and do not try. Once I had a brain surgery that may or may not help. It was proven, but my doctor was unsure if it would make a difference. My doctor told me going back into the operating room: “this can kill you.” My family and I knew that I could die. We knew the risks of the surgery. Still I also understood that my life would BE MISERABLE without the surgery to help me. Thankfully the surgery did help, and I am glad that I live in a free country where I was able to decide my own fate. If I had not had that surgery I would be dead.
I truly hope that you change your mind about your position on the 21st century cures act or at least take the time to meet with me. I truly admired you before this and I am hurt by this betrayal. You once said, “Donald Trump chose racism as his weapon.” Now you have chosen negligence as your weapon. You are turning your back to the rare disease community and forgetting that we exist. In my book, this is immoral. Please prove that you are not neglectful towards the rare disease community. We are rare, but please don’t make us less.

Karen Richards (A resident of Medfield Massachusetts)

This is a picture of me in my wheelchair hugging my little brother. We both suffer from rare disease. He has had one major spinal surgery already at the age of 11.
This is a picture of me in my wheelchair hugging my little brother. We both suffer from rare disease. He has had one major spinal surgery already at the age of 11.

 

Here is a picture of me on life support trying to smile despite a difficult situation. I was fifteen in this picture.
Here is a picture of me on life support trying to smile despite a difficult situation. I was fifteen in this picture.
Here is a picture of me and Sue Pinkham. Sue is also a board member of TCAPP and a resident of Massachusetts.
Here is a picture of me and Sue Pinkham. Sue is also a board member of TCAPP (The Coalition Against Pediatric Pain) and a resident of Massachusetts.
This is a picture of my boyfriend and I. I am including this picture not only because I love to talk about him, but because he also has a rare disease. We both have Ehlers Danlos Syndrome and Complex Regional Pain Syndrome. We have both suffered a great deal in our life because of people misunderstanding rare disease.
This is a picture of my boyfriend and I. I am including this picture not only because I love to talk about him, but because he also has a rare disease. We both have Ehlers Danlos Syndrome and Complex Regional Pain Syndrome. We have both suffered a great deal in our life because of people misunderstanding rare disease.

This is a photo taken when I met with Representative Kennedy in August of 2015 about the 21st century care act. I told him "you don't see people with rare disease because we are rare and sick. Just because you don't see patients with rare disease every day, doesn't mean they don't exist. Please don't forget us and our families." There was also a man there dying from a rare form of cancer. Mr. Kennedy was extremely kind and listened to all of our concerns. He even said he would vote for the bill please talk to him about this meeting and let him tell you the hell rare disease patients go through. This is a photo taken when I met with Representative Kennedy in August of 2015 about the 21st century care act. I told him “you don’t see people with rare disease because we are rare and sick. Just because you don’t see patients with rare disease every day, doesn’t mean they don’t exist. Please don’t forget us and our families.” There was also a man there dying from a rare form of cancer. Mr. Kennedy was extremely kind and listened to all of our concerns. He even said he would vote for the bill please talk to him about this meeting and let him tell you the hell rare disease patients go through.

 

This is a picture of my mother and I accepting a check for TCAPP (the non-profit organization she runs). To the left of us are our friends. The little girl in the front, also suffers from rare disease and will suffer if this bill is not passed.
This is a picture of my mother and I accepting a check for TCAPP (The Coalition Against Pediatric Pain). To the left of us are our friends. The little girl in the front, also suffers from rare disease and will suffer if this bill is not passed.

 

"I would say that the most obvious reason I need 21st century cures is because I use quite a few "off label medications". They've been life-changing and if i weren't allowed to have them due to that I would be bedridden again. Which would stink because I'm finally becoming more functional." Emily is a 27 year old trying to live her life.
“I would say that the most obvious reason I need 21st century cures is because I use quite a few “off label medications”. They’ve been life-changing and if i weren’t allowed to have them due to that I would be bedridden again. Which would stink because I’m finally becoming more functional.” Emily is a 27 year old trying to live her life.

 

My life for the past 7 years has been doctor after doctor, treatment after treatment, a constant effort to find something that works. The act would let me try more things, I wouldn't be held back by regulations. I could have a helpful treatment, I could have a chance to get better because I could have treatment NOW. I should have the right to try a treatment when it could help me, not wait years for it to get approved. Until you have walked in our shoes, don't dismiss a quicker approval, you don't know what it is like to sit idle when you know that there is something just beyond your reach that could make you feel slightly better.
My life for the past 7 years has been doctor after doctor, treatment after treatment, a constant effort to find something that works. The act would let me try more things, I wouldn’t be held back by regulations. I could have a helpful treatment, I could have a chance to get better because I could have treatment NOW. I should have the right to try a treatment when it could help me, not wait years for it to get approved. Until you have walked in our shoes, don’t dismiss a quicker approval, you don’t know what it is like to sit idle when you know that there is something just beyond your reach that could make you feel slightly better.

 

This is a picture of Hana Belanger, a high school student with Ehlers Danlos Syndrome; a rare disease.
The rare disease community is left dying Because Senator Elizabeth Warren is against the 21st Century Cure Act Leaving my family and I destined for the ground Senator. Why are you letting my people die? Your ignorance can not be blamed on “Big Pharma” or “Republicans” when it is you. You are the leader of this genocide, preventing us treatment options. When most of us rely on “off label” medications to survive. We manage symptom by symptom instead of illness as a whole. 95% of rare diseases do not have 1 single FDA approved treatment Yet as you say, “I know the difference between compromise and extortion.” Meaning our lives are nothing but booby traps set to catch and torture the country. In reality, you are the most dangerous contraption in the way of medical research. Also: here’s a quick FYI This bill does not “legalize fraud” The use of “off label” medications does not make us criminals. The problem is not that I take an off label medications for my wide spread pain, the problem is that without these medications, I can’t move from my bed. You say “you can’t cure cancer with a headache pill” But let me tell you this, my whole herd needs Excedrin Migraine to cure the aches you give us. Besides, there is know cure to cancer yet, but passing this bill could find one with the new council. The Council of 21st Century Cures is A non-profit established for acceleratiing the discovery, development, and delivery of innovative cures, treatments, and preventive measures. America runs on innovation, we are fueled by the dreams of tomorrow just to make it to today. You don’t want to be responsible for letting this country run out of gas… Do you senator?”

 

 

This is a picture of Natalia Rijos who suffers from Complex Regional Pain Syndrome and many other rare diseases.

 

Karen Cogswell, Age 32, Disabled, Ehlers Danlos Syndrome, Celiac Disease, Fibromyalgia, Dysautonomia, Traumatic Brain Injury (from a car accident where i wss hit by a woman texting and driving), Depression, Anxiety, OCD, PTSD. Filed medical bankruptcy before turning 30.

 

I'm 24 and diagnoses include EDS, CRPS, POTS, PCOS, myokymia, spinal cord injury, spontaneous spinal fluid leak and gastroparisis. I recently had a DVT and have a possible blood clotting disorder as well but that hasn't been fully worked up yet.
I’m 24 and diagnoses include EDS, CRPS, POTS, PCOS, myokymia, spinal cord injury, spontaneous spinal fluid leak and Gastroparisis. I recently had a DVT and have a possible blood clotting disorder as well but that hasn’t been fully worked up yet.

 

Emily Lambert age 19, her symptoms started September 2011 and by Dec 31 her Complex Regional Pain Syndrome had spread. Since she has also been diagnosed with POTS (Postural Orthostatic Tachycardia Syndrome).
Emily Lambert age 19, her symptoms started September 2011 and by Dec 31 her Complex Regional Pain Syndrome had spread. Since she has also been diagnosed with POTS (Postural Orthostatic Tachycardia Syndrome).

 

Sammie Barret
I’m Sammie and I’m 23. I have CRPS, EDS, POTS, MCAD, and both panic and anxiety disorders. More than 75% of my medications are “off label,” not to fill someone else’s pockets, but to help me get through every day. I don’t want to lose any progress I’ve made because I can no longer take the medications that I need. I’m getting married in 2017. This wouldn’t just effect me, it would effect my husband to be. This doesn’t just effect people with rare diseases; it would effect our families, spouses, and friends as well. We need to move forward, not backward.

 

I am a mom of a child with multiple rare diseases . Emma Parcells age 14 . She has Ehlers Danlos , Complex Regional Pain Syndrome, Mast Cell Activation Syndrome and Cyclic Vomiting . What was said about off label uses for drugs is WRONG . As a patient you should have the right to choose with your doctor which medications will help you . Medicine is not exact at times and patients suffer everyday without cures or treatments for rare diseases . Pharmaceutical companies will only sponsor clinical trials for drugs they can make large profits on . Therefore many drugs are used in what’s considered an off label way to help patients . This means that the drugs do work and can and in some cases are lifesaving , but they aren’t FDA approved for that condition . This is only because the clinical trusts needed are not funded . My daughter has CRPS , a severe case . She is bedridden many days and uses a wheelchair . She lives in constant pain excruciating at times . The only thing that help her is infusions with a medication called ketamine that is not FDA approved for CRPS .Yet this drug is considered safe for use and is approved by the FDA . It’s just not approved for her condition but without it she remains bedridden in pain . Who are you as our representatives to make choices that should only be made between a patient and a doctor ? What choices would you make if the only drug that cured you , saved your life or helped relieve you of daily constant excruciating pain was considered off label for your condition ? Would you want the ability to make that choice with your doctor ? Yes you would . Yes you would choose a drug even if it was off label of it saved your life , or stopped you pain levels that are unbearable and leave you without the ability to function . Yes all patients with rare diseases deserve these choices . Without them you are leaving suffering children to live in pain and possibly die without the drugs they need to treat them and keep them alive .

 

My name is Marissa Piatelli, age 26. My diagnoses include Bipolar disorder, depression, generalized anxiety disorder and hypothyroidism. The 21st Century Cures Act would provide mental healthcare research and funding which is so important for people like myself who are diagnosed with mental health disorders. This research and funding would be beneficial to have to help myself and others living with mental health disorders in our day to day life.

 

Savannah Orth, Age 17. Suffers from rare disease

If you have a rare disease send me your picture with a description and I will include it in this post!

To learn more about TCAPP click here

To learn more about what complex Regional pain syndrome is click here.

To read a letter to senator Warren from ‘The Sick Chick’ click here.

To read stories of those with rare disease and their every day struggles, click here.

Click here to read exactly what Elizabeth Warren said.

To contact your local senator click here.

3 Comments on The fight for 21st century cures: A plea for help from a 16 year old girl.

  1. I truly appreciate your well thought out response to Senator Warren & your concern. What I don’t easily understand is how this bill actually helps you. I am admittedly skeptical of big pharma & insurance companies because at their heart I don’t know if they are governed by a desire for better health or for more money but I lean to the latter. So your perspective as a self described democrat but also someone with a rare disease who supports this bill is immensely important to me. I want to know how specifically it helps you & people like you, & how that outweighs the concerns Warren expresses in her speech. (By the way I found this post through a comment your mom left under Warren’a speech.)

  2. Hi Christie, thank you for the thoughtful response. The rare disease community is largely unheard of and unspoken for in washington. The fact that there was a bill proposing to help us was exciting in itself. The actual reasons though are that the FDA gold standards Warren talks about in her speech are ridiculous to be frank. There aren’t enough rare disease patients to put in these studies to fit the requirements. That is why I believe that there should be an exception for rare disease patients for these rules. Also, I truly believe that Senator Warren is misunderstanding the off label medication use. Last year I started a drug off label for fainting. I was having to get IV infusions for the problem every day after school at my home. This was not the best because the PICC line I had went straight to my heart (leaving a direct highway for infection into my bloodstream). The medication was not covered by insurance, but my mother (who is truly a saint) looked and found Walmart had the cheapest price for the medication and then she found a coupon for the medicine as well to where my family could afford it. The medication helped me and I was able to get my PICC line out, which was awesome. So that is why when she said “a headache pill can’t be sold as the cure for cancer” it really bothered me. This bill would have made my families financial situation a lot easier last year. If my mom had not found the discount, I might still have an IV in my arm today. Thank you so much for reading the post and commenting. If you have any more questions I am willing to respond to those as well. 🙂

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