case study

Supporting Patient Access to Treatment

In order for the European Medicines Agency to maintain the marketing authorization


Patients with non-dystrophic myotonic disorders (NDM) experience unpredictable frequency and severity of myotonic episodes, associated with life-long symptoms and negative impact on physical functioning and quality of life. Treatment has included unlicensed products such as mexiletine.

For the European Medicines Agency to maintain the Orphan Drug Designation, data was necessary to evaluate the use and access to mexiletine across Europe. There is limited data on the impact of and disrupted availability of mexiletine in the EU.

Our Solution

  • Leading the creation of the Myotonia Observation Survey of Patient Access to Therapy avoiding Harm (MyoPath)
  • Using a well-defined mixed methodology of interviews and an online survey to gather information on the availability and use of mexiletine in the EU by patients with NDM in a short time frame
  • Gathering important data regarding patient use of mexiletine and the harm due to lack of treatment access
  • Presenting results of the survey to the Committee for Orphan Medicinal Products (COMP) along with the client. Read the COMP’s public assessment report


The COMP maintained the orphan drug status of mexiletine which will significantly support patient access across Europe. admedicum thereby helped the internal team to create great value both for patients and their company.