Executive Summary Agency staff discuss how they offer early development advice to sponsors before a breakthrough decision is made and reassure them if the status is denied. Sponsors may desire breakthrough status because of the perks that come with it, but US FDA officials said promising non-breakthrough products also could receive a lot of…
Executive Summary Siding with coalition of hospitals, judge ruled that HHS lacked authority to cut Medicare reimbursement for drugs purchased at 340B prices. That should change the dynamics of the 340B reform debate heading into 2019 – but don’t expect the Trump Administration to back down on its push to rein in what it sees…
Executive Summary Synthetic control group derived from historical clinical trial data could augment smaller randomized trials and yield better information than single-arm studies, but this approach should not be viewed as a substitute for randomized trials where feasible, US FDA officials said at a Friends of Cancer Research meeting. Use of a synthetic control…
A bipartisan, broad-based effort is needed to ensure better access to the robust pipeline of new medical technologies unleashed, in large part, by the 21st Century Cures Act, health care leaders and researchers write in a Health Affairs blog published Wednesday. The authors urge that health care stakeholders and federal agencies, particularly the Centers…
The US Food and Drug Administration (FDA) announced Monday that Dr. Amy Abernethy will replace Rachel Sherman as Principal Deputy commissioner of Food and Drugs – the highest position at the agency that is not a political appointee. Abernethy is the former chief medical officer, chief scientific officer and senior vice president, oncology, at Flatiron…
One of the leading voices in using health data to advance targeted treatments known as precision medicine has been tapped for the second-highest post at the FDA. Amy Abernethy, chief medical officer for Flatiron Health, will join the Food and Drug Administration as its principal deputy commissioner early next year, FDA Commissioner Scott Gottlieb…
Executive Summary O’Brien assumes role from his late colleague and friend Dan Best as HHS closes out administrative items in the blueprint. The Trump Administration appears to be ramping up towards some kind of action on drug pricing early in the New Year. Over the last two weeks, HHS Secretary Alex Azar has…
Executive Summary Abernethy comes to FDA from Flatiron Health, which develops oncology-specific electronic health record software and real-world evidence for cancer research. She succeeds FDA veteran Rachel Sherman, who will retire from the agency in January. Incoming US FDA Principal Deputy Commissioner Amy Abernethy will bring extensive experience in real-world evidence and health information…
Executive Summary Record-setting pace of NME approvals is a cause for celebration. But is it a sign that drug industry R&D productivity has actually improved? The US FDA’s top drug review official thinks so – but the data aren’t yet clear. The US Food & Drug Administration is already in record territory with 55…
Federal regulators put two experimental drugs targeting autism’s core symptoms on a fast track for testing and approval this year, but they’re still a long way from pharmacy shelves. Balovaptan, from drugmaker Roche, aims to improve communication and social skills for people with autism by signaling a hormone linked to behavior. In January, the…
