The Webinars run by Putting Rare Diseases Patients First! are designed for patients with rare diseases, and the parents of children with rare diseases. We are often asked why we have designed content for this audience and not those who represent them.
We do receive many requests from advocates and professionals who work with rare disease patients. They have often read the descriptions of the planned Webinars, and want to attend because they recognize the value of what will be presented. So why does PRDPF! design these Webinars for patients and the parents?
One important reason is that there are other organizations that develop information for professional advocates, and those who represent patients. We presume these organizations have taken the time to design the content appropriately for this professional audience. We have no desire to duplicate their efforts.
We have chosen to develop the content for patients and parents because there is very little available to them that addresses their needs, especially in relation to issues of drug development and clinical trials, in an informed way. There are many professional conferences that seek to address the topic of patient engagement. The only problem with many of these conferences, is that they are very expensive, and therefore “the patient” is generally not present to voice his or her concerns. Additionally, a lot of the content presented at these conferences is policy based, philosophical, or theoretical. In other words, it is not very useful to the real patient living in the trenches with their rare disease.
Who speaks for “the patient”? We feel that the patient should speak for the patient. Who knows better the drug development needs, than the patient? Why filter the information by first providing it to a professional who will then determine how much of what was gleaned should be provided to the patient?
Patients and parents often voice concerns, urgency and ideas that would be missed if they were in a professional setting. By allowing them to speak, we have found that they are very creative in relation to the regulatory and drug development approaches that we often take for granted. From running the Webinars we have also discovered that patients/parents can decide for themselves what they want to follow up on and what is not of importance to them. They are clearly best placed to speak about the issues that concern them.
Lorna Speid, Ph.D.
Lorna is the author of Clinical Trials: What Patients and Healthy Volunteers Need to Know, published by Oxford University Press.
Putting Rare Diseases Patients First! Board of Directors
Lorna Speid, Ph.D., is the Founder and President of Putting Rare Diseases Patients First! She is joined on the Board of Directors by Patricia Ortiz, and John Gilbert.
Patricia Ortiz – VP of Patient Liaison
Patricia is a Biomedical Engineer with a strong background in Medical Device Development, Diagnostic Instrument Development, and Clinical Site Management. Patricia is an important addition to the Board because she has a rare disease, and is active in LAM, the patient organization for her disease. Patricia is the VP of Patient Liaison.
John Gilbert – VP of Public Relations, Marketing and Social Media
John is a commercial business executive for a pharmaceutical company. He has a deep background in life science commercialization. He is also an expert in public relations and social media. He is The VP of Public Relations, Marketing and Social Media.
Lorna Speid, Ph.D., President and Founder
Lorna is a Clinical Pharmacist, and is registered in the Royal Pharmaceutical Society of Great Britain. She has worked in the pharmaceutical industry, developing and obtaining approvals of new medicines since the early 1990s. in 2014 she set up a 501(c) (3) non–profit, Putting Rare Diseases Patients First! in January 2014 to educate patients with rare diseases about the clinical trial and drug development processes. Lorna has a special interest in developing new treatments for rare diseases, and in empowering patients with rare diseases, and parents of children with rare diseases.