Advocates Discuss Barriers and Strategies to Including Children in Clinical Trials

Samantha Swamy

Ella Murray is a happy, inquisitive elementary school student. However, because she was born without collagen factor VII, she suffers from a disease called epidermolysis bullosa (EB). This causes her to develop blisters all over her body, and she has to endure 3 hours of bandage changes multiple times a week. There are currently no therapies for EB, in part due to low prevalence of the disease and low enrollment in clinical trials. Joe Murray, Ella’s dad, told her story and called on researchers to inform communities about existing clinical trials during the Coalition for Clinical Trials Awareness panel discussion on child inclusion in clinical trials held in Washington, D.C. on May 1.

This event marked the beginning of Clinical Trials Awareness Week, which occurred between April 30 and May 4 this year, during which researchers, industry partners and patient advocates came together to discuss barriers to clinical trial enrollment and how to include underserved populations like children in these trials. It has been 20 years since a new medication has been approved for newborns, and 90% of medications used for children have never been tested in children. Rather, they are tested in adults and adjusted for children based on body size.

David Charles, moderator of the clinical trial briefing and chairman of the Alliance for Patient Access, noted that there was indeed a push from researchers to include children in clinical trials, however such efforts are often blocked by the Institutional Review Board, which oversees clinical trial design. Charles emphasized the necessity of more clinical trials for children due to a current lack of data to inform how to effectively treat them.

However, there is hope on the horizon. Andrew Rosenberg, director of the Newborn Health Initiative, spoke about how the initiative provides incentives for research companies to develop medications for neonate babies in rare diseases. This resolution offers companies extended patents on drugs that are already in the market providing that they work on developing neonate specific drugs as well. There is still much work to be done to include children in clinical trials, the success of which hinges on the year-round efforts of parent and doctor advocates like those who came together this past week.

For more information on Clinical Trials Awareness Week, visit http://cctawareness.org/ctaw-2018/.

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Without research, there is no hope.
The Honorable Paul G. Rogers