How to Ensure Endpoint Integrity?

Clinical trials for rare diseases can be very challenging because only a limited number of patients can be included in the trial.

FREMONT, CA: Rare disease clinical trials can be very challenging as far as endpoints is concerned. There are typically a limited number of patients that can be included in the trial, and often the endpoints themselves are novel or complex. A collaboration between the subject matter experts on the teams at Medpace and ATOM International has led to better training and improved data visualization, pattern detection, and problem-solving in rare disease clinical trials.

Medpace has partnered with ATOM International to optimize the clinical trials for rare diseases and also clearly define trial endpoints.

Clinical Trial Endpoint Advancements and Quality Control in Data Collection

Being involved in their trials from the outset, they can provide advice on endpoint selection. ATOM offers consultation on protocol development, target population inclusion and exclusion criteria, endpoint information, guidance on endpoint selection, and advancements of a clinical manual of operations to standardize the accumulation of data across the different clinical trial sites. 

As most of their studies are global, training all the clinical evaluators who will be gathering data on primary and secondary endpoints is vital. The ATOM team depends on Medpace's data management team to jointly develop their source documentation.

Planning a Rare Disease Study

Medpace'sdata management team is working with the ATOM International group in their clinical trials for nearly six years. They began reviewing the data periodically to find any signals in the data that could indicate issues with how the clinical evaluators were performing the tests at the trial sites. They have quarterly data review meetings where they offer subject profiles, including the demographics, adverse events, concomitant medications, and any other factors that might affect the clinical evaluator tests, as well as data visualizations so that errors can be detected as quickly as possible.

Practical CE Training for Global Clinical Trials

A global reach is usually necessary to obtain the needed sample size for a clinical trial involving rare disease, and Medpace works to ensure the site staff who perform the procedures are correctly prepared. They have worked to finalize training manuals early in the process to allow adequate time for language translations and to ensure that translators are available for investigator meetings or on-site training.