The Child Participant’s Burden

Researchers should see this slide deck. At the end of Patricia Furlong’s keynote address at the 2016 Advancing Ethical Research Conference, I realized that this was the only note I had written during the session (though for the purposes of this post, I have gone back to the proceedings to quote other relevant thoughts).

Ms. Furlong delivered a powerful message about the burdens that families endure and the risks they are willing to take for the chance—and it’s only a chance—that a new treatment will be effective. Embedded in the story she told about her own family’s experiences was a clear message about the inequitable allocation of research funding and the power of strong advocates to make a positive change.

Ms. Furlong is the mother of four children, two of whom succumbed to the progression of Duchenne Muscular Dystrophy. She spoke pointedly about the disruptions to family life and childhood that research participation may cause: “We were medicalizing his life at a time where he would ordinarily only be going to the hospital once or twice per year. He was a much sadder kid during that year.” She also discussed the differences between how research participants and patients are treated, saying, “when you are a patient, you are treated with urgency,” but in research, the procedures are done to collect data; the person may be secondary.

In sharing her perspective as a parent of children who were the participant of research, Ms. Furlong’s talk also highlighted the insights researchers can gain from hearing more from different stakeholders. At one point, she even made the statement that parents expect benefit from research participation, no matter what was said in the consent process or written in a consent form—a statement that can inform discussions between IRBs and researchers, and could lead to more effective consent processes. As Ms. Furlong presented results of research involving parents of children with Duchenne, it seemed clear that researchers would benefit from knowing these results (e.g., that parents of children with Duchenne prioritize the slowing of muscle weakness even more than the potential for a longer life).

Ms. Furlong described herself, following her sons’ diagnoses, as “a desperate mom” who borrowed $100,000 without her husband’s knowledge, immersed herself in research about the disease, and developed a plan to try to save her children. NIH funding for research on this disease, which is terminal in all cases, was minimal. Ms. Furlong was instrumental in founding Parent Project Muscular Dystrophy, which advocated successfully for more awareness of the disease and its impact, studies parent perceptions of clinical trials, and worked to change the relative paucity of research funding. Today, NIH funding for Duchenne Muscular Dystrophy is 45 times greater than what it was then, and numerous drugs are in development and clinical trials.

Thinking about this session after the fact, I realize that it’s not only researchers but all who are involved in research with human subjects who should see these slides and hear this presentation. We all need to remember that the objective of all that we do is to help and protect the people who sacrifice the most to enable research to move forward—the subjects. For them, participation in research is not just an experiment; their very lives may depend on it.

Brenda L. Ruotolo, BA, CIP, is executive director of the Human Research Protection Office at Columbia University, and has been affiliated with Columbia since 2003. She has extensive experience with IRB review of both biomedical and social science/behavioral research. As executive director she is responsible for the functions of the seven IRBs and oversight of approximately 6000 active research studies. Ms. Ruotolo has been a member of PRIM&R for approximately 20 years. She presents at local and national conferences on a variety of topics related to the protection of human subjects.