Navigating Therapeutic Ambiguity: Patient Narratives on Off-Label Drug Use in Rare Disease Management
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Abstract
This study explores the lived experiences of patients using off-label medications to treat rare diseases, focusing on the emotional and existential challenges they face. Rare diseases often present limited treatment options, prompting patients to turn to off-label drugs when conventional therapies fail. The research employs a descriptive phenomenological approach, utilizing in-depth interviews with ten patients to uncover how they navigate the uncertainty surrounding off-label use. Thematic analysis identified four core themes: off-label treatment as a last resort for hope, the ethical and emotional complexities of uncertain medical decisions, the influence of family dynamics, and the assertion of personal agency. Findings emphasize that off-label drug use is not just a clinical decision but a deeply personal journey marked by hope, fear, moral dilemmas, and a desire for autonomy. This study contributes to the growing body of literature on rare diseases and off-label drug use by highlighting the profound psychological and relational impacts on patients, offering new insights for patient-centered care, and suggesting the importance of integrating these narratives into clinical practice and policy.
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