1. Part 1 - Information about Putting Rare Diseases Patients First!®

There are 7000 rare diseases impacting 350 million people around the world. Many of these patients have no effective treatment. Indeed, many patients with a rare disease have not received a diagnosis as to the cause of their suffering, or they endured a great deal of discomfort before they were diagnosed. To make matters worse, the number of treatments for rare diseases is very limited (200 to 400 reported). Most patients with a rare disease, cannot understand why there are no effective treatments for their disease.

The PRDPF! organization is a non-profit charity organization that is seeks to provide unbiased information about the process by which new treatments are developed for patients with rare diseases.   The educational information is delivered online via Webinars, Blogs, Newsletters, Emails, etc.

Putting Rare Diseases Patients First! is overseen by a Board of Directors. Dr. Lorna Speid is the Founder and President. Leah Rihela is the Treasurer, and John Gilbert is in charge of Marketing and Social Media. We are always looking for others who want to volunteer their time to help us to serve patients with rare diseases. Send enquiries to LSPEID@PRDPF.ORG.

Why has the organization created this Survey? We want to hear from you. What are your needs as a patient with a rare disease, or a parent of a child with a rare disease?  Do you find it easy to find information on new treatments and the process by which new treatments are developed? Do you have views about the regulatory process? What challenges have you faced in finding information about new treatments or in taking part in clinical trials?

This short survey will give you the opportunity to provide your views. When all results have been received and collated, we will publish the results on our Website and Blog, and in other arenas. We will also determine if we can use the results to bring about changes in various spheres.  Please tell other patients with a rare disease to take part in the survey. Our goal is to survey a minimum of 1000 rare disease patients/parents.

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