Drug Safety and Rare Diseases
Small Patient Populations Pose Unique Challenges for Safety Scientists
“These are diseases that are not often taught in medical school,” said Peter Hawkins, Senior Director, Head of Rare Genetic Disease Risk Management at Agios Pharmaceuticals, about the types of diseases he and his team are working to cure.
Mr. Hawkins joined the Safety Signals Podcast for a discussion on the specific safety challenges faced by sponsors developing medicines for very small patient populations. The episode covered a wide variety of topics, including:
– The importance of real-world data in understanding rare disease patients
– The need to look at quality of life metrics
– Reasons why safety scientists should actively participate in clinical trial design
With limited data available, Hawkins acknowledged that he and his team look at any information they can get to understand what patients are going through, and whether or not new safety signals are from a drug they’re studying, from the disease, or something else, such as comorbidities. “Understanding the whole world this patient lives in is essential to making sure that the patient makes the right decision,” he said.
* The views of the hosts and guests featured on Safety Signals are their own and do not necessarily reflect the views of Saama or the individual companies for which the guests may work.
About Saama Safety Signals
Introducing Safety Signals, the show for pharmacovigilance professionals, benefit risk managers, epidemiologists, and other patient safety stakeholders. From pharma leaders to academics and scientists, we provide expert perspectives on the challenges, opportunities, and technologies that are shaping the future of the safety sciences. Get ready to be inspired and energized as you work to reduce the risk of life-changing–and life SAVING–medical treatments.
Meet Your Hosts & Guests
Vice President & Partner
Senior Director, Head of Rare Genetic Disease Risk Management