On December 10th, Putting Rare Diseases Patients First! hosted its fifth Webinar. The subject was Natural History Studies. Dr. Nuria Carrillo from the National Institutes of Health spoke to an audience of patients with rare diseases, and parents of children with rare diseases.
She explained that Natural History Studies are the foundation that allow the building of a drug development program. Without Natural History Studies it will not be possible to know how a disease will progress in a patient. For instance, how quickly will it progress or worsen? As it worsens, does the patient experience pain? Do they lose their cognitive abilities? How can these changes be measured? Without this type of information, new medicines cannot be developed.
It was very interesting that many of the patients and parents that took part in the Webinar were unaware of Natural History Studies. Many had never been asked to take part in a Natural History Study. These are patients and parents who are earnestly seeking to find solutions to their diseases, and yet the one area in which they could make a contribution had not been brought to their attention.
On the 25th of February, we will be holding Part 2, a follow-up to Part 1. This time we will be discussing the role of Natural History Studies in children. If you would like to take part in the Webinar, please email firstname.lastname@example.org with PRDPF! in the subject line. You will then be registered. We hope you will be able to join us. Patients with rare diseases, and parents of children with rare diseases, from anywhere in the world, are welcome to register. If your support group would like to sponsor the Webinar, please email Dr. Speid.
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 pharmaceutical sales executive for a pharma company. He has a deep background in pharmaceutical sales. He is also an expert in 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 Patient 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.