YourEncore Insights - Life Sciences

Patient Engagement: Putting Rare Disease Patients First (Ep. 10 on The Pharmcast)

Posted by Jim Combs on 1/30/17 12:00 PM


This week on The Pharmcast, we turn our attention to the topic of rare disease, and more specifically: how to engage and educate rare disease patients to advance development. It's estimated that nearly 30 million Americans, or nearly 1 in 10 people,  are living with a rare disease - and almost 95% of rare diseases don't have any FDA-approved treatment. 

One of the unique challenges and perhaps keys to rare disease development is patient engagement. Patient populations are small by definition. Roughly 50% of patients are children, and benefit-risk equations are completely different from those of more traditional diseases. To learn more about engaging rare disease patients, we sit down with Dr. Lorna Speid, to discuss her stories of rare disease drug development, outline the unique challenges of engaging the rare disease population, and her advice to developers.

Dr. Lorna Speid is the author of Clinical Trials: What Patients and Healthy Volunteers Need to  Know, and founder of the nonprofit organization, Putting Rare Disease Patients First. With an extensive background in R&D, Dr. Speid has dedicated her career to working within the rare disease space - particularly in drug development, clinical trials and patient engagement. Dr. Speid earned her Ph.D. in Safety Assessment of Medicines, Pre and Post Marketing, and has served on the NIH Committee for Therapeutics for Rare and Neglected Diseases. 

Dr. Speid began her nonprofit in 2014, after spending a year writing her book, aiming to empower and inform patients and professionals alike. With hard-to-diagnose diseases, Dr. Speid felt especially passionate about helping patients understand their disease and the pharmaceutical industry - informing them on everything from the drug development process, to genomics, to natural history, and clinical trials - through easily accessible information such as webinars and blogs. The nonprofit also uses social media to share information and offer support to patients - which is especially important in a time when inaccurate information can spread like wildfire. 

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Topics: Rare Disease, R&D, Pharmcast