New FDA Recommendations for the Clinical Development of Drugs Will Make It Easier for Patients to
Participate in Clinical Trials for Novel Alzheimer's Treatments
February 16, 2018 - UsAgainstAlzheimer's (UsA2) welcomes the U.S. Food and Drug Administration's (FDA) revised draft guidance, Early Alzheimer's Disease: Developing Drugs for Treatment, which reflects the Agency's commitment to the patient community and to addressing the Alzheimer's crisis. The guidance, which updates 2013 draft guidance for industry, was accompanied by a statement from FDA Commissioner Scott Gottlieb prioritizing the need for new medical treatments for serious neurological disease like Alzheimer's.
"UsAgainstAlzheimer's applauds Commissioner Gottlieb for recognizing the value of patient engagement in advancing our understanding of the impact of Alzheimer's on human lives and how that real-world impact informs our understanding of what is clinically meaningful," said UsAgainstAlzheimer's Chairman George Vradenburg. "Patient engagement will be critical to the development of a successful intervention in early Alzheimer's disease and this is an important step in moving us closer to this reality."
The FDA draft guidance reflects the Agency's engagement with UsA2 and is consistent with previous UsA2 advocacy. The FDA Division of Neurology Products engaged directly with UsA2's Alzheimer's Disease Patients and Caregivers Engagement (AD PACE), which played a central role in bringing the voice of patients to the guidance process. The FDA draft guidance also highlighted the value of data sharing and standardization across the Alzheimer's disease scientific community, which UsA2 has previously advocated for through its Global CEO Initiative on Alzheimer's (CEOi) Biomarkers Collaborative Initiative. Finally, UsA2 has called on FDA to clarify its position on the historical dichotomy of functional and cognitive assessments. The FDA revised draft guidance rejected this dichotomy as inappropriate due to the potentially clinically meaningful nature of changes in cognition. This responsiveness to the patient community and clarity is critical to paving the way for new treatments that millions of families desperately await.
"UsA2 looks forward to continuing to work with FDA in the areas of increased patient engagement and biomarker development and we urge the Agency to take the lead in engaging other national regulatory bodies in the international community to take similar measures," added Vradenburg. "As a community of advocates and patients, scientists, drug developers, and regulators, we all share the mission and have the collective ability to advance the development of therapies that will address this dreadful disease, particularly in its early stages."