Since the end of last year when the OPEN Act was removed from the 21st Century Cures bill, I have spent many sleepless nights trying to understand why Washington was not comprehending the critical need for repurposing drugs. We are now working to convince Democrats that the OPEN Act is not a handout for pharmaceutical companies, as I am living proof that repurposing an FDA approved drug works wonders. Continue reading
This morning, the Food and Drug Administration announced that it granted accelerated approval to Sarepta Therapeutics’ drug Eteplirsen. This comes after months of waiting for a decision after an extremely intense Adcomm meeting. Continue reading
Mr. Trump, I’m writing this in the wake of the Republican National Convention. I was so excited to see your son Eric acknowledge the special needs community. See, before I ever became “Rare Candace”, I was the sister to an incredible young man with autism and cerebral palsy. Growing up, I was his sister, his protector and his best friend. One day, I will be his caretaker all while managing my rare disease and three additional chronic conditions.
I am not looking for sympathy or money, I believe I have been blessed with a second chance that few others in my position will ever have. I am about six weeks away from my 30th birthday, a day I am not suppose to see because my rare disease almost stole it from me.
In the two years I have been sick, I have become a voice for the members of society hidden away from plain view. I am a voice for the caretakers, the siblings. The people in hospitals and at home fighting for a “new normal” with illness. I have been working with members of Congress in both parties to pass legislation that will help the largest minority in America- people with disabilities.
My rare disease does not define me, just as my brother’s conditions do not define him. He graduated with a degree from Florida Atlantic University, despite my parents being told he would never walk or talk. I was hospitalized with a platelet count so low, when I hit my head on a 26 foot box truck at work the day before, I should have bled to death. We both have defied the odds. Your children spoke to us, as did you. So I am reaching out.
I know we can do better for America. I know our orphan drug approval process is overcomplicated and leaves people to die from awful diseases while we approve other drugs that carry little benefit to the American public. I am the victim of Obamacare, which I have to pay through my law school loans, yet covers little while I manage my health. We can fix this, and I know you can help.
Mr. Trump, over 30 million Americans have a rare disease. Factor in caregivers and families members, that number grows to well above 100 million. We need your help to change the way this country assists people like me and people like my brother. I see the things that need to be changed, things that can be improved, and in your speech you touched on many of them.
I would like to talk to you about how we can work towards Making America Great Again by increasing funding for medical research, reforming the FDA and encouraging biotechnology and pharmaceutical companies to create new treatments and potential cures for the over 7,000 known rare diseases. Currently, only 5% of those disease have an FDA approved treatment. America can do better, and with you we can reach new heights.
Candace H. Lerman
Discrimination and bullying of disabled students is rampant in colleges and universities. Now that I have a full year of law school under my belt since being diagnosed with a rare disease and three other chronic conditions, I connect with those who have had similar experiences. I refuse to be silent about “administrators” who torment students with invisible disabilities because they are ignorant. It is obvious that they disregard the Americans With Disabilities Act and university policies with their outrageous and targeted discriminatory behavior. It is an epidemic for the largest minority in the United States. Continue reading
There has been a great deal of criticism of pharmaceutical and biotechnology companies in recent months. Most of the anger surrounds drug pricing, and many in the rare disease community have participated. While affordable healthcare is a priority and we need access, an even more alarming need is actually treatments for orphan disorders. Many rare disease patients are struggling day to day, without anything to help them manage their symptoms and conditions. Drugs for small populations cost money, the FDA is particularly critical of rare disease drugs making investments risky and the cost of treatments sky rocket. Continue reading
I’m sorry for the extended absence. I have been suffering from Celiac Disease! Who would have thought that I was poisoning myself with gluten?
It is no secret that since I became sick and was stuck on prednisone for months that I began to hate my body. After all, I’m up about 30 pounds from where I was nearly two years ago and I lost a lot of muscle that I had worked so hard to gain. I never acknowledged to myself how much I hate my body until I was out for a walk tonight. My hatred isn’t just for the weight gain or loss of muscle. It comes from a feeling of betrayal: my body tried to kill me. My immune system malfunctioned and for some miraculous reason, I survived months of physically challenging work in heels without internally bleeding to death.
I haven’t abandoned this blog, and if you follow me on Twitter or on the Facebook page you have seen my involvement in many projects. This month I have law school exams so I am extremely busy. I will update everyone soon!
I will be spending the day at the National Institutes of Health, checking out the Rare Disease United Foundation’s Beyond the Diagnosis art exhibit, touring the facilities and speaking with researchers. In the evening I’m set to attend a cocktail reception and documentary screening.
Today is my second rare disease day as a patient and completely different from my last one. Now I feel a sense of authority over my disease, the new normal I have created and my spot in working toward change for our community. The game is totally different now, I am not looking to play along, I am working on my own projects.
Wednesday is when I will be on the hill, but at the moment my schedule is uncertain as I have been asked to participate in a few exciting projects. Follow my Instagram and Twitter pages for up-to-the-minute updates on what’s shaking in DC. And if you’re a VC reading this, let’s chat.
If you see me out and about, please come and say hi. My hair will stand out, I promise you can’t miss me (and I don’t bite).
I am headed up to Washington, DC on Sunday for Rare Disease Week. I am excited to be able to meet with a lot of different groups to work on legislation for the 30 million of us fighting these disorders.
Last year was about me, I was focused on what I went through. This year is about YOU, the patient, the caregiver, the healthcare provider, the researcher. Now we have to work on unity between everyone involved in the rare disease space. We need to see to it that the 21st Century Cures bill is signed into law as soon as possible.
I expect this trip to be quite different, as I have found myself at peace with what life has thrown at me.
If you’re going to be in DC and at the events, I would love to meet you. Tweet me @RareCandace and let me know what you’re up to.