An observational study goes where randomized clinical trials have not.

“The cancer focus, I think, is a natural,” he said. “To be able to ramp that up is timely and very appealing.” Even prior to the announcement of the Precision Medicine Initiative, the NIH launched 3 trials that are screening patients’ tumors for genetic abnormalities. Based on the screening results, patients are enrolled into a clinical trial of an investigational drug that might target the specific molecular pathology driving their cancer (http: //1.usa.gov/1EdWhDQ). The NCI MATCH Trial is enrolling adults with advanced solid tumors and lymphomas that have stopped responding to standard therapy. Pediatric MATCH is the counterpart trial for children. The third trial, the Lung Master Protocol, or Lung-MAP, is a public-private collaborative effort implementing a similar approach for patients with squamous cell lung cancer, which currently has few treatment options beyond surgery (http://bit.ly/1zIq85a). The participants in these trials could potentially become a part of the Precision Medicine Initiative cohort. “Efforts such as Lung-MAP and the NCI MATCH program are within the scope of this initiative,” said Vassiliki Papadimitrakopoulou, MD, professor of medicine at MD Anderson Cancer Center and a co–principal investigator for Lung-MAP. In September 2011, Dana-Farber also launched its Profile program, becoming the first hospital to offer tumor genotyping to all patients with cancer, Rollins said. “We see 16 000 new patients a year. We want to turn them into a giant cohort study, like the Framingham Study,” he said. “Breast cancer patients with a mutation in gene X: do they live longer? do they live shorter? That’s a very powerful scientific thing to do.” Rollins said Dana-Farber “would definitely consider contributing the Profile patient cohort to the Precision Medicine Initiative if the protections for patient confidentiality are sufficiently stringent.” While improving the treatment of cancer is one of the initiative’s shorter-term aims, it has a more ambitious longer-term goal, Collins said at last month’s NIH meeting. Eventually, he said, the plan is to “generate the knowledge base necessary to move precision medicine into virtually all areas of health and disease.”