Since 1989 the NIH has required that graduate students supported by its grants have training in the responsible conduct of research. However, up until recently, much of the training had been minimal and fragmented and only made use of websites or short seminars. In response, CIRGE scholars began working in collaboration with the SCBE Program in Bioethics & Film to create documentary films which can be used as dramatic and interactive teaching tools that will engage students and scientists in thinking about the ethical, philosophical and social issues involved in their research.
No medium is better suited to present research ethics than film, which has the potential to educate far more powerfully than any amount of didactic explanation. Its visual imagery can portray complex situations from multiple perspectives and can easily engage viewers in meaningful discussions of the issues. Film is one of the only media forms that compels the viewer to experience and empathize with the situation deeply enough to truly weigh ethical dilemmas.
Therefore, CIRGE and SBCE scholars have been prolifically creating bioethical, educational and interactive films, from Hold Your Breath (pictured to the right) to the current project, Rare: The Story of a Citizen Scientist. A synopsis of Rare, along with information on the accompanying facilitator guide and suggested audiences, is detailed below.
Distribution of Rare with the accompanying guide and evaluation form will begin later this year. For more information and to request the film, please email CIRGE Program Manager Maya Wolpert at firstname.lastname@example.org.
Truly in a race against time, parents of children with rare genetic diseases are joining together to change the way scientific research is conducted. Rare, a new hour-length documentary by award-winning filmmakers Maren Grainger-Monsen, M.D., and Nicole Newnham, explores this movement and major issues affecting people living with rare genetic diseases, through the intimate lens of one unforgettable mother and her quest to find a cure.
Rare follows Donna Appell, whose twenty-one year old daughter Ashley was diagnosed as a toddler with Hermansky-Pudlak Syndrome (HPS), a rare genetic disorder often characterized by albinism, bleeding diathesis and fatal pulmonary fibrosis. At the time of Ashley’s diagnosis, Donna was informed that less than thirty people had been diagnosed with HPS in the United States and that there was no way to speak with or locate these similarly affected families. Realizing that researchers were not going to focus their efforts to help cure “just one child,” Donna set about gathering HPS individuals and forming an advocacy group.
Armed with a few patients, Donna cold-called the National Institutes of Health (NIH) and begged them to research her daughter’s rare disease. It was the beginning of a long-term effort to study HPS. Today, Donna and the NIH team are involved in a Phase III clinical trial for a drug that could slow the progression of pulmonary fibrosis in HPS patients. Moreover, Donna’s database now includes over seven hundred patients.
Nevertheless, significant challenges remain. Even with a registry of hundreds, filling tens of spots in the clinical trial is extremely difficult. Additionally, although affected by pulmonary fibrosis, Ashley has been excluded from the drug trial for which her mother so desperately advocated. Will Donna be able to fill the trial and get the medication approved for her daughter in time to save her life?
Rare takes viewers into the world of a rare genetic disease, exploring Donna’s annual conference, meeting patients enrolled in a clinical trial, and attending a board meeting of scientists and advocates. The film also highlights the lobbying efforts of rare disease groups, which petition for increased funding on Capitol Hill.
Rare is designed to inspire thoughtful debate of some of the major ethical and social issues inherent in the advocacy movement: What are the lived experiences of patients and research subjects? How do leaders such as Donna balance the sometimes dueling roles of parent and advocate? What conflicts are raised for researchers who develop an emotional relationship with their subjects? How is research study design impacted by input from patients?
The facilitator guide, which accompanies the film, delves into these issues and explores a range of additional topics with extensive references. The guide has been created to enhance discussion, reflection and debate on both the full-length and the 30-minute medical version of the film. Supplementary issues covered include the history of advocacy movements and institutional review boards, cultural attitudes toward research, study enrollment and inclusion criteria, therapeutic misconception, and differing notions of progress among advocacy groups, scientists and clinicians.
Rare has been very well received at pilot screenings, including at the ASBH 2008 Annual Conference and the PRIM&R 2009 Annual Conference. Written evaluations and oral feedback from these screenings indicate that the film would be of great utility in many settings and in four areas specifically: 1) institutions funded by Clinical and Translational Science Awards; 2) Responsible Conduct of Research programs; 3) Institutional Review Boards; and 4) undergraduate medical education professionalism, humanism and introductory clinical courses.
Maren Grainger-Monsen, MD
SCBE Senior Research Scholar
Mildred Cho, PhD
CIRGE PI, SCBE Associate Director
Maren Grainger-Monsen, MD
CIRGE Program Manager, 2008-2010
CIRGE Program Manager, 2010-2012
Facilitator Guide Layout Designer: