Background Charcot-Marie-Tooth disease is a common inherited neuropathy where patients may be sensitive to adverse effects of certain medicines; however, information about medication safety in this group of people is limited. Objective This study aimed to investigate the experience of Australian individuals with Charcot-Marie-Tooth disease in using medications, including perceived impact of drug-induced adverse effects. Secondarily, it aimed to determine whether individuals with Charcot-Marie-Tooth disease feel adequately supported to make decisions about medication safety. Setting Focus groups and interviews (face-to-face or telephone) of individuals with Charcot-Marie-Tooth disease in Australia. Method A mixed methods qualitative study was conducted between September 2015 and August 2016 using semi-structured interviews. Thematic analysis of interview transcripts was conducted independently by two researchers using inductive coding until concept saturation was achieved. Main outcome measure Perceptions of medicines safety in people with Charcot-Marie-Tooth disease, including barriers to making informed decisions about medication safety. Results Twenty-four adults with Charcot-Marie-Tooth disease participated. Anaesthetics (18%) and pregabalin (15%) were the medications most frequently reported as impacting on Charcot-Marie-Tooth symptoms. Participants sought medication information primarily from general practitioners or neurologists. The main barriers identified by participants were a perceived poor understanding in non-specialist health professionals about Charcot-Marie-Tooth disease and lack of attention to medication safety concerns in people with Charcot-Marie-Tooth disease; this resulted in dissatisfaction about the advice provided. Many individuals who faced uncertainty in obtaining and understanding medicines information turned to internet resources, peer groups, and use of complementary and alternative medicines to self-manage Charcot-Marie-Tooth exacerbations. Conclusion Participants reported drug-related adverse effects and a difficulty in obtaining safety information about medication. This study highlights the need for improved evidence about medication safety in people with Charcot-Marie-Tooth disease. Development of evidence-based resources, increased awareness amongst health professionals about Charcot-Marie-Tooth disease and a team-based care approach could facilitate shared decisions about medication use for people with Charcot-Marie-Tooth disease.