Roche and representatives of parent-driven organizations are collaborating on the first part of a study whose objective is to design better clinical trials and speed up the development of Angelman syndrome treatments.
The aim of the initial part of the study is to gain a better understanding of the condition’s impact on patients and their families. It will be based on interviews with the doctors and caregivers of 33 patients worldwide.
The company, which advocates a patient-centered approach to drug identification and development, will fund the creation of a conceptual model for Angelman syndrome trials.
“The findings of this research will be a key step towards identifying and developing the best outcome measures and biomarkers for future clinical trials,” Tom Willgoss, the principal scientist at Roche, said in a news release.
Roche expects the results of the study, expected to be released in 2017, to offer valuable insight into the way clinical trials for Angelman syndrome should be developed.
“To have such a cutting-edge biotech giant join all of us in the quest for a treatment and cure for Angelman syndrome is a very hopeful sign of significant movement for our families who struggle with the impact of this disorder,” said Allyson Berent, chief scientific officer of the Foundation for Angelman Syndrome Therapeutics, or FAST.
Alliances between pharmaceutical companies and foundations that focus on particular diseases have advanced research and development of therapies for those diseases. In 2016, FAST and the Angelman Syndrome Foundation worked with Agilis Biotherapeutics to create the Angelman Biomarkers and Outcome Measures Alliance. The initiative is aimed at encouraging doctors, patients’ families and caregivers to join the Angelman registry to help collect information about the disease.
“We need rigorous ways to measure how potential treatments may improve the quality of life for individuals with Angelman syndrome and their families,” said Terry Jo Bichell, director of the biomarkers alliance. “Roche’s conceptual model will set a standard that will help researchers determine what to measure, how to measure it, and how to interpret their findings when they are trying to identify possible treatments.”
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