Who are we?
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.
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.
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 serve the global rare diseases patient community by providing empowering information to rare disease patients, and the parents of children with rare diseases, about the clinical trial and drug development process.
Many of these 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 despite the growing interest in rare diseases.
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.
Founder and President
Dr. Speid is registered as a Pharmacist with the Royal Pharmaceutical Society of Great Britain.View profile
- “Do Natural History studies usually run concurrently with clinical trials?”
- “Excellent Webinar today! That had significant impact.“
- “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.”
- “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?”
- 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!