This is a Blog written by Putting Rare Diseases Patients First! The goal of this Blog is to provide useful information to patients with rare diseases and parents of children with rare diseases. Our goal is to communicate with the real patient that is grappling with rare diseases. The information that we provide will relate specifically to clinical trials and drug development of new medicines for rare diseases.
We have been asked about our funding. We are a formal charity based in the US (California), but the rare diseases patient population that we seek to serve is global. We have set up a GoFundMe page to raise funds to support our operations. We may from time to time take charitable donations from various sources, including pharmaceutical companies, patient foundations, etc, but only as long as there are no strings attached. At the moment we are not funded. By ensuring that Putting Rare Diseases Patients First! remains independent of any organization or group, we believe we can best serve the needs of the rare diseases patient. We are open to funding from organizations that have anti corruption policies and donations.
Who We Are
Lorna Speid, Ph.D., is the Founder and President of Rare Diseases Patients First! She is joined on the Board of Directors by Patricia Ortiz, 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 the patient organization for her disease. Patricia is the VP of Patient Liaison. John Gilbert is a pharmaceutical executive for a pharmaceutical company. He has a deep background in pharmaceutical commercial operations. He is also an expert in social media. He is The VP of Public Relations and Marketing. Any notices that you have seen on Facebook were as a result of John’s efforts.
The Putting Rare Diseases Patients First! organization is based in San Diego, but plans to provide educational content to patients around the world. Forming collaborations with the major patient foundations and organizations around the world is an important goal to facilitate a global reach.
Lorna Speid, B.Pharm.(Hons)., M.R.Pharm.S., Ph.D., RAC works in the area of global and strategic regulatory affairs, and is considered by her peers to be an expert. She works with small and large pharmaceutical companies, assisting them at various stages of the drug development process. Dr. Speid has an excellent track record of success in regulatory affairs, having secured registrations with all the major regulatory authorities, including after conducting appeals to overturn rejections.
Lorna has experience with many therapeutic areas, including oncology (hematological and solid tumors), diabetes, obesity, anti-infectives (anti-bacterial and anti-viral), pulmonary (asthma, COPD), influenza, women’s health, bone (Paget’s disease and osteoporosis), lupus, arthritis, transplantation, autoimmune diseases, Anti-malarials, and CNS (psychiatry, Alzheimer’s disease). She has experience working with small and large molecules, including gene therapy and Biosimilars.
Dr. Speid began her career as a clinical pharmacist in the UK, after which she completed a Ph.D. at the Centre for Medicines Research International into the Safety Assessment of Medicines: Pre and Post Marketing. She has worked for large pharma as well as small biotech companies. These companies include Sanofi Winthrop in the UK (now Sanofi-Aventis), Ciba Geigy and Novartis in Switzerland (at Headquarters). Small companies that she has worked for include GeneMedicine/Valentis, Inc. (Director of Regulatory Affairs), NewBiotics (Vice President Regulatory Affairs and Project Management), and Avera, Inc. (Vice President of Regulatory Affairs). Dr. Speid was an officer at the last two companies. She founded and incorporated Speid & Associates in 2004. Since that time she has been able to use her expertise to make a difference for many other companies and organizations. She has a Bachelor of Pharmacy degree from the University of London, UK (King’s College), and a Ph.D. from the University of Wales, College Cardiff (now University of Cardiff), UK.
Dr. Speid founded, and Co-chairs the Drug Development Boot Camp with Harvard University OTD in November of each year. The Boot Camp provides intensive training in drug development to pharma and biotech companies, academia, NIH/NCI, and other institutions interested in honing these skills. Dr. Speid’s book, Clinical Trials: What Patients and Healthy Volunteers Need to Know was published by Oxford University Press in 2010 and is being promoted to patients in the US through major bookstores and online, as well as in other countries.
Lorna has a special practice in rare and neglected diseases. She has a strong background in drug safety, and its interface with drug information functions. 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.
To find out more about the Putting Rare Diseases Patients First! organization, or to volunteer on a committee, please send an email to Dr. Lorna Speid at firstname.lastname@example.org with PRDPF Board Positions, or About PRDPF in the subject line. We will respond promptly.