Monday, April 27, 2015

The Ethics and Implications of Compassionate Use (And People Still Don’t Understand the Drug Development Process)

By Kristina Lopienski, Product Marketing Manager, Forte Research Systems, Inc.

The general session, “Fast Tracking Clinical Development in a Moment's Notice: Patient Advocacy, Social Media and the Implications of Compassionate Use” featured Kenneth Moch, former CEO of Chimerix, giving his firsthand experience in dealing with expanded access to investigational drugs.

He recounted the story of Josh Hardy, a young boy who was refused treatment to his company’s experimental drug. Outraged by this, the story was taken to social media and picked up by angry reporters from around the world. As the story blew up, Ken received death threats, and it became clear that people don’t understand the drug development process. He cited an example of someone who thought all patients have a fundamental right to experimental medicines after Phase 1 is complete, because that’s when the sponsor knows all of the effects of the drug (and phases 2-3 are just to make the FDA happy). There are right to try laws which vary by state and give terminally ill patients the right to have access to investigational drugs, but this became a case of compassionate use. The outcome was the FDA helped craft a novel solution: a phase 3 trial that enrolled the boy in this story to the trial within days.

Because the outcry from the social media campaign was so loud, was this a case of special treatment? The ethics are complex, but making exceptions is not ethical. Once the door is opened, you can’t say ‘yes’ to this patient and ‘no’ to everyone else. What would you have done if this boy was your son? Of course, we all want to save the child, but we also want to save people in the future. There are no simple answers to moral dilemmas. The good news is that the boy in the story is alive today, but what would have happened if he died or had a rapidly negative response to the drug? Because there was so much attention on this story, if that was the case, it’s very possible that potential future patients wouldn’t want to enroll in trials.

Ken believes that we as an industry continue to fail to inform the public on how complicated drug development is, and we need to do a better job educating the public now – not as an aftereffect. His experience in dealing with the “mob” of ignorant reporters and ill-informed citizens was done reactively, which he stressed is too late. Rather, we must take a proactive approach to explaining the complexities of the clinical trials process, requirements, regulations, etc. so those who are not in the industry can get the fundamentals right.  

About the Author: Kristina Lopienski is the Product Marketing Manager for Overture EDC at Forte Research Systems​. In her role, she works to bring educational resources to clinical research professionals. She writes on a variety of topics affecting clinical trials on the Forte Clinical Research Blog. Kristina is currently the guest blogger covering Partnerships in Clinical Trials 2015. 

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