ABSTRACT
INTRODUCTION: It has become increasingly important to include patient preference information in decision-making processes for drug development. As neuromuscular disorders represent multisystem, debilitating, and progressive rare diseases with few treatment options, this study aimed to explore unmet health care needs and patient treatment preferences for two neuromuscular disorders, myotonic dystrophy type 1 (DM1) and mitochondrial myopathies (MM) to inform early stages of drug development. METHODS: Fifteen semi-structured interviews and five focus group discussions (FGDs) were held with DM1 and MM adult patients and caregivers. Topics discussed included (1) reasons for study participation; (2) disease signs/symptoms and their impact on daily lives; (3) top desired benefits; and (4) acceptability of risks and tolerance levels for a hypothetical new treatment. Data were analyzed following a thematic 'code' approach. RESULTS: A total of 52 participants representing a wide range of disease severities participated. 'Muscle strength' and 'energy and endurance' were the disease-related unmet needs most often mentioned. Additionally, improved 'balance', 'cognition' and 'gut function' were the top desired treatment benefits, while 'damage to the liver, kidneys or eyes' was the most concerning risk. Factors influencing their tolerance to risks related to previously having experienced the risk and differentiation between permanent and temporary risks. A few differences were elicited between patients and caregivers. CONCLUSIONS: This qualitative study provided an open forum to elicit treatment-desired benefits and acceptable risks to be established by patients themselves. These findings can inform decisions for developing new treatments and the design of clinical trials for DM1 and MM.
Subject(s)
Myotonic Dystrophy , Patient Preference , Caregivers , Humans , Qualitative Research , Rare Diseases/drug therapyABSTRACT
This paper begins by highlighting the necessity of combining sociological and psychoanalytic approaches in the study of social movements, but acknowledges that psychobiographical studies of prominent movement leaders sit uneasily within the sociological tradition. The author attempts to illustrate that leader psychobiography can make a contribution to understanding social movements, however, provided it offers a way into understanding broader psychosocial issues within the movement. This is achieved through a psychobiographical portrait of Bruce Gagnon, a leader within the outer space protection movement. The author argues for the central importance of both paranoid-schizoid and depressive mechanisms throughout Gagnon's activist career. These eventually came to underpin Gagnon's commitment to protecting outer space as a Kleinian "good object." The paper concludes by suggesting how Gagnon's psychobiography might be instructive in attempts to understand the wider movement, utilizing Erikson's model for psychohistorical study as a framework.
