Table 2:

Priorities to advance individualized therapies

Challenge	Solution
Length of the diagnostic odyssey	Early use of next-generation diagnostics
Day-to-day burdens of living with rare disease	Engage support networks and advocacy organizations
Fundraising and reimbursement	Define development pathway and commercial incentive
Procuring drug and satisfying regulatory requirements	Partner with experienced investigators, companies
Monitoring patient response to treatment	Perform comprehensive molecular and clinical assessments
Lack of generalizable evidence	Standardize collection of outcome data