Duchenne’s MD Guide Will Allow Researchers to Compare Diverse Datasets

Two non-profits that work on standards that can facilitate drug development have created a guide to help those doing Duchenne’s muscular dystrophy research.

The two are Critical Path Institute (C-Path) and the Clinical Data Interchange Standards Consortium (CDISC). The resource they created is the Duchenne Muscular Dystrophy Therapeutic Area User Guide.

Some of the topics the guide covers are genetic facets of the disease, imaging information, and disease measures, including movement and lung function assessments. The standard format the guide uses allows datasets from different sources to be compared or combined for data collection, sharing, and analyses.

The two organizations’ researchers are also developing standards for a series of functional tests so that scientists and others can easily compare results.

Critical Path Institute’s mission is to bring scientists from the Food and Drug Administration, industry and academia together to collaborate on and improve the drug development and regulatory processes. CDISC’s mission is to establish patient data standards that can improve the research process and facilitate connections with the healthcare system.

“We are delighted to partner with C-Path’s Duchenne Regulatory Science Consortium to create a standard for Duchenne muscular dystrophy that will facilitate improved data collection, sharing, and analysis,” David R. Bobbitt, the president and chief executive officer of CDISC, said in a press release. The effort could help increase “hope that new treatments may be discovered,” he said.

C-Path formed the consortium in August of 2015 to develop tools that could accelerate DMD therapy development and approval. It partnered with Parent Project Muscular Dystrophy to establish the group.

The consortium’s first objective was to put medical data provided by partner organizations into a common database, using CDISC standards to integrate the information.

Consortium members provided information and ideas to help develop the guide.

“I avidly follow DMD drugs in the pipeline that are soon to be evaluated by the FDA,” said Christopher (Buddy) Cassidy, a patient representative on the consortium. “I am very enthusiastic about the role the CDISC data standard for Duchenne will play in the near future with particular regard to FDA approval.

“A standardized method for data collection will in turn make it easier for clinicians [doctors], researchers, drug development companies, and the FDA to share and compare data,” Cassidy said. “Only through the constant exchange of like sets of data can we truly evaluate the effectiveness of new DMD treatments.”

“We are excited to publish the new Duchenne Therapeutic Area User Guide,” said Jane Larkindale, the consortium’s executive director. “We hope the research community rapidly adopts the standard in new studies, which will allow future data to be compared, contrasted, and combined in a methodical manner, and lead to a more rapid and comprehensive understanding of disease progression and drug effects.”

Treatment developers use CDISC standards when they submit research and other materials to the FDA, Japan’s Pharmaceuticals and Medical Devices Agency, China’s FDA, and to those involved in the European Innovative Medicines Initiative.

So far, standards have been developed for more than 30 types of diseases. The use of the standards has saved time and resources.

DMD researchers have said they believe the standards will make international and cross-institutional cooperation in their field easier.