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.