A few things that caught Vector’s eye during the week:
FDA drug evaluation staff: An unmet need
New laws or initiatives to expedite drug approvals, especially for orphan drugs, have prompted the Food and Drug Administration to create new positions to review the applications. Unfortunately, these slots are proving hard to fill. Kaiser Health News reports that the FDA has more than 700 job vacancies in its Center for Drug Evaluation and Research (CDER), which approves new drugs. The problem? Pharma pays more — “roughly twice as much as we can,” said CDER director Janet Woodcock at a recent rare-disease summit. The 21st Century Cures Act, still awaiting Senate approval, could help by allowing the FDA to offer higher salaries and relaxing postgraduate degree requirements for some positions.
“Precision medicine” caveats
An excellent series in STAT this week provides a reminder that genetic testing is still an evolving science. First, a report out of the Mayo Clinic describes how some two dozen family members underwent genetic testing after a 13-year-old boy died from a heart syndrome. As detailed in STAT, all tested positive for variants in a gene linked to long-QT syndrome and were told they had a potentially fatal illness. Yet none had cardiac symptoms or positive EKGs — except for the boy’s brother, who seemed to have findings on one EKG that didn’t recur. A Mayo team reanalyzed the test results using a more up-to-date genetic database and concluded the mutation is harmless. Too late for the brother, who received an implanted defibrillator.
Meanwhile, a study from the University of Michigan looked at the costs of genetic testing for inherited thrombophilia, which can indicate a patient’s risk for developing blood clots. For patients in the hospital, the test results don’t change treatment — patients with blood clots receive anticoagulants even if they don’t have the gene. Yet doctors still order the test, to the tune of some $300 to $670 million a year for Medicaid patients alone, reports STAT. Another STAT writer recounts trying out five genetic fitness tests, which have infiltrated the world of sports. Costing $154 to $400 each, the tests returned sometimes contradictory results. “In one case,” she writes, “two companies cited the same journal article to support their opposing conclusions.”
Exploring the potential of sharing clinical trial data
The data gathered during clinical trials are a rich source of medical insights. But much of it doesn’t go into journal publications and thus is lost to medicine, write Jeffrey Drazen, MD, editor-in-chief of The New England Journal of Medicine, and Isaac Kohane, MD, PhD, chair of Harvard Medical School’s Department of Biomedical Informatics, this week in STAT. To help rectify the problem, NEJM has launched a challenge. Researchers are invited to analyze the dataset from a randomized trial of intensive versus standard blood pressure control published in NEJM a year ago, and to “identify a novel scientific or clinical finding to advance medical science.” The challenge will culminate in an online summit to share out the findings on April 3-4, 2017. A recent set of NEJM Perspectives sets out potential approaches and incentives for data sharing and an example of one such effort.