11. Does GSK use the DMD patient registries to find out about numbers of eligible patients in a country?
Data from patient registries and databases can be a very important tool for recruitment into clinical trials and are often used by study investigators. To date, GSK has not collaborated with DMD patient registries. To date, Prosensa has commissioned two feasibility reports via the registries, which have been important in programme design and implementation.
12. Does GSK use the patient registries when recruiting for a trial?
Responsibility for recruiting study participants lies with the investigators undertaking the clinical study. Individual investigators often work with patient registries to identify eligible patients.
13. What factors does GSK take into account when deciding which countries will be involved in the DMD trials?
GSK’s primary goal is to deliver high quality clinical data that will inform decisions regarding drug efficacy and safety. As such, there are a number of factors that are taken into consideration when deciding whether a country will be involved in the DMD trials. These include the availability of suitably qualified investigators with access to the necessary equipment and personnel to be able to conduct the study assessments, as well as the availability of patients that would meet the eligibility criteria of a trial. Other factors that impact the number of countries and sites in a trial include the regulatory timelines and local GSK expertise and experience.
14. I am prepared to travel to another country to take part in the trial. Is it up to GSK or the individual trial site to decide whether that is acceptable?
Responsibility for recruiting study participants lies with the investigators undertaking the clinical study. Any questions regarding participating in the study should be addressed to the appropriate investigators once the study sites are posted on www.clinicaltrials.gov.
15. Will GSK pay for my travel and subsistence costs if I am travelling from another country?
GSK provides funding to the investigator sites to conduct the study. Typically study sites reimburse reasonable travel expenses for patients involved in the studies. Any questions regarding participating in the study, including what travel costs are considered reasonable, should be addressed to the appropriate investigators.
These questions have been translated into Japanese and are available from the Japan’s Registries website – Remudy.