Expectations and experiences of investigators and parents involved in a clinical trial for Duchenne/Becker muscular dystrophy.

ABSTRACT:

Background

The social context of rare disease research is changing, with increased community engagement around drug development and clinical trials. This engagement may benefit patients and families but may also lead to heightened trial expectations and therapeutic misconception. Clinical investigators are also susceptible to harboring high expectations. Little is known about parental motivations and expectations for clinical trials for rare pediatric disorders.

Purpose

We describe the experience of parents and clinical investigators involved in a phase II clinical trial for Duchenne and Becker muscular dystrophy: their expectations, hopes, motivations, and reactions to the termination of the trial.

Methods

This qualitative study was based on interviews with clinical

SUBMITTER: Peay HL 

PROVIDER: S-EPMC3935382 | biostudies-literature | 2014 Feb

REPOSITORIES: biostudies-literature