Although gene therapy has been widely publicized as an impending cure for numerous diseases, including cystic fibrosis (CF), little empirical research has been conducted to determine the impact of this publicity, and its promise of cure, on patients and their families. Such knowledge of consumer perspectives is essential for enhancing trust in the biomedical establishment, for protecting vulnerable consumer groups from potential harms, and for the design of effective consumer and clinician educational materials. The Education Development Center, Inc., in collaboration with the International Association of Cystic Fibrosis Adults (IACFA), Temple University, St. Vincent's Hospital, and Brigham and Women's Hospital propose a study to (a) assess how gene therapy research, and the publicity surrounding it, are perceived by adults with CF, parents of children with CF, and CF Center physicians; (b) determine the knowledge, beliefs and attitudes that inform consumer perceptions and felt needs; (c) discover how those needs and perceptions affect patients' and families' treatment decisions and life plans; and (d) identify the ethical and psychosocial implications of gene therapy developments on patients, families, and their physicians. The team will do a content analysis of information materials disseminated to CF patients and the public; conduct qualitative interviews with 30 adult CF patients, 30 parents of children with CF, and 30 physicians. These interviews will be used to develop a survey questionnaire to be administered to al consenting adult CF patients and parents of children with CF cared for at all 13 New England CF treatment centers (N=1595) and to IACFA members in the U.S. (N=713). Findings will be presented to an expert advisory group of CF consumers, researchers, clinicians, ethicists, and representatives of other disease perspectives, such as AIDS and cancer. This goup will assist the study team in drafting recommendations for how best to reconcile the promise with the reality of CF gene therapy and identify the possible implications for other areas of gene therapy research. Recommendations will focus on consumer and clinician education; however, they will also have relevance for a broader array of issues, such as recruitment into gene therapy trials and priorities for research and care delivery.
