The US Food and Drug Administration (FDA) has chosen chronic fatigue syndrome and myalgic encephalomyelitis (CFS/ME) to be first in a series of 20 patient-focused drug development meetings funded by the Prescription Drug User Fee Act re-authorization of 2012 (PDUFA V). “This initiative in PDUFA V could lead to other longer-term efforts and collaborations that could advance drug development,” says FDA spokesperson Lisa Kubaska, but “that will require further exploration with various stakeholders as we learn more about the patient perspective on specific disease areas.” CFS/ME drew public attention last fall when the FDA Arthritis Advisory Committee voted 8–5 against approval for rintatolimod (Ampligen), a Toll-like receptor 3 agonist from Hemispherx Biopharma of Philadelphia. “One reason we got on the FDA radar is because there is significant unmet medical need in the CFS community,” says Suzanne Vernon, scientific director of the CFS and immune deficiency advocacy group, CFIDS Association of America, in Washington, DC. “Now there's nothing in the pipeline.” The FDA effort is distinct from the Patient-Centered Outcomes Research Institute (PCORI) (Nat. Biotechnol. 30, 482–484, 2010). “PCORI's focus is on evaluation of therapies and services that are already available,” says executive director Joe Selby, “not in research on drugs in development.” As some research methods are of mutual interest, the group is having discussions with FDA on a common approach to developing patient-reported measures, he says.