Who are we, and how can we help you if you have a rare disease?
There are 350 million patients with a rare disease globally. In the US alone there are 30 million people with rare diseases. On the continent of Europe, there are 30 million people with a rare disease. We seek to have a global outreach so that we can help as many rare disease patients as possible.
The Putting Rare Diseases Patients First!® organization is a 501 (c) (3) non-profit, and a registered charity. It has tax exempt status with the US tax authorities (IRS), and in California. Donations will be tax deductible.
What do we do? We provide information that can help you find new treatments and diagnostics for your rare disease. Why do we do this? We believe that Knowledge is Power. Patients with rare diseases need effective diagnostics and treatments. For this to happen, they must be an active part of the process of developing new diagnostics and treatments. This organization seeks to provide empowering information to rare disease patients, and the parents of children with rare diseases, about the clinical trial and drug development process.
Many rare disease patients suffer for many years, and some even die without a conclusive diagnosis. Even after they receive a diagnosis, many patients with rare diseases do not have access to a treatment that is effective. For most of them, the search for effective new treatments continues for a very long time. Do you have a rare disease? What if we could give you information that would lead you to a conclusive diagnosis, or even a treatment or a better treatment? We believe that would make a difference to your life.
The Putting Rare Diseases Patients First!® organization provides information on the clinical trial process to patients and parents using several different methods, including Webinars (online seminars in real time), newsletters, Emails, using social media (Facebook and Twitter), and using a Blog.
Meet the Members of the Board of Directors
Putting Rare Diseases Patients First!® is run by a Board of Directors. The Board of Directors governs the non profit charity using accepted governance principles. We meet approximately once per month to discuss the goals of the organization.
- I was wondering if we could set up a call with my colleague, EG to gather some insights on what you have been able to accomplish, and how you have been able to successfully put the patients voice first. Let me know your interest and availability and I can go ahead and coordinate this call. Thanks so much Dr. Speid, I hope all is well with you!
- “Please reflect on sharing and consolidating between National History Studies. How many National History Studies allow patients to review their own data and add patients generated data, like quality of life statements?”
- “Do Natural History studies usually run concurrently with clinical trials?”
- “What a touching story Tim shared with us last Friday! The story of his mother so deeply resonated with me and the hardship so many people suffering from rare diseases have.I think it is a wonderful idea to repeat this webinar if we can.”
- “Excellent Webinar today! That had significant impact.“
PRDPF! Research Survey
Putting Rare Diseases Patients First® is conducting a research survey. We are seeking responses from 1000 patients and parents impacted by rare diseases. We will analyze the results and publish them and/or present them to policy makers to help to address the needs identified by the research survey. The research survey will take you about 10 minutes to complete, and does not require you to divulge any personal or confidential information about your rare diseases. The research results will also not divulge your name or individual situation.
Take the research survey here:https://www.surveymonkey.com/r/XP6P5C6