Pharmaceutical companies, academia, and governmental agencies are frantically engaged in the search for new treatments for rare diseases. However, without patient involvement in clinical trials these efforts will be futile. Patients with rare diseases, and parents of children with rare diseases, are aware that they are in the driving seat. By definition, patients with rare diseases are a scarce resource. Because of this, their involvement and retention in clinical trials is crucial for success.
Patients with rare diseases want treatments to be developed quickly and effectively because their very lives often depend upon the rapid delivery of new effective treatments. Desperation creates vulnerability, and for this reason they must be protected by an effective, but non-impeding regulatory process. How can they contribute to this process and make it more effective? Patients have never had as much information at their fingertips as they have now via the internet. Knowledge is power. However, information does not necessarily equal knowledge. The Rare Diseases Patients First! organization seeks to provide education about the clinical trials and drug development process in an unbiased and independent way.
The next Webinar “The Rare Diseases Patient at the Center of it All” will take place on the 23rd of April at 8 am PST (11 am EST, 4 pm GMT, 5 pm Europe). Dr. John Whyte Director of Professional Affairs and Stakeholder Engagement at the Center for Drugs Evaluation and Research at the US FDA will be the Keynote Speaker.
Dr. Speid said “It is not often that patients with rare diseases are able to ask someone from the FDA about the clinical trial process, and how they can better engage with it.”
Dr. Whyte said “The FDA is committed to engaging with individuals with rare diseases and their families to find new and better ways to include their voices in the development and evaluation of drugs. This is a valuable opportunity for the Agency to place the focus on patients.”
Webinar 003 will take place on the 23rd of April 2015 at 8 am PST.
To register, participants should send an email to Dr. Speid at lspeid@sndtm.com with RDPF WEBINAR 003 in the subject line.
About Rare Diseases Patients First!
The RDPF! organization is a non-profit charity that provides unbiased education about rare disease clinical trials and drug development to patients with rare diseases, and parents of children
For more information visit
RDPF! Website: The website will be activated after the trademark (in process) is approved.
RDPF! Facebook page:https://www.facebook.com/rarediseasesPF
RDPF! Twitter:https://twitter.com/rarediseasespf
RDPF! Linkedin page: https://www.linkedin.com/company/rare-diseases-patients-first
