Alongside the clinical trial a number of other projects are being completed that aim to ensure the drug can be accessed by patients if the trial proves successful.
Biomarker development – A biomarker is a biological signal, often a chemical produced in the body, which can be used to monitor one of the body’s processes or the effect of a drug on a disease.
There are currently no biomarkers available for MCDS. This makes it more difficult to measure the effect of a treatment on MCDS, and to identify different variations of MCDS in order to tailor treatments to individual patients.
As part of the MCDS-Therapy project Sciomics will be working to identify new biomarkers for the condition. At the same time, the team at The University Medical Centre Freiburg will be working to develop a new line of human stem cells derived MCDS cells that can be used to help to test these biomarkers.
Health Economics – Health economics is the study of the value of healthcare. It primarily aims to determine the cost-effectiveness of health interventions, such as treatments, by comparing the overall cost to society of a treatment to the overall benefit to society of treating that condition. This should help governments decide which expensive treatments should be paid for and delivered to patients.
As part of MCDS-Therapy we will be working to understand the financial cost of MCDS to patients, the healthcare system, and society, and to provide evidence that treating the condition with carbamazepine (if proved to be effective in the clinical trial) is cost-effective.
Building the MCDS community – A rare disease diagnosis can be an isolating and devastating experience. Many patients struggle to find doctors who understand their condition and treatment needs. Some may never meet someone else with the same condition. As part of the MCDS-Therapy project we aim to work with patients, to make sure that both the treatment and the clinical trial works for them. At the same time we would like to help them to connect with one another, and make their voice heard beyond the project.