Pharma
Angelman Biomarker and Outcome Measure Consortium (A-BOM)
It may seem obvious to caregivers, but science and regulators require metrics—outcomes that are clearly defined in advance, measurable, sensitive, replicable, and most important, measuring a change that is meaningful to patients and caregivers.
Tools used to assess change in how a patient feels or functions are called “outcome measures.” If a drug provides a benefit, it needs to be clearly determinable, to support drug approvals.
The ABOM Consortium is made up of a team of over 350 people that have interest in advancing the regulatory science for Angelman syndrome to understand, develop, test, and validate different tools that can assess meaningful clinical outcomes for individuals living with Angelman syndrome. If a drug provides a benefit, we want it to be easily determinable—because this, in turn, helps support drug approvals using specific tools that become widely available to our pharma partners. That’s why, to date, FAST has invested over $4M into the efforts of the ABOM Consortium.
There are currently 45 members of the Steering Committee (SC) of the ABOM, who meet 4 times a year to drive clear objectives and deliverables. This team is Co-Directed by FAST’s own Dr. Allyson Berent, our Chief Science Officer, and Jennifer Panagoulias, FAST’s head of Regulatory and Compliance, who have been working in industry for over 20 years. The SC is made up of 12 pharmaceutical and biotechnology companies working on Angelman syndrome, alongside the Foundation for Angelman Syndrome Therapeutics, The Angelman Syndrome Foundation, academic clinicians who are experts in Angelman syndrome neuropsychological testing, and global parent representatives.