Congress Must Pass 21st Century Cures Act Now Posted: 07/08/2015 7:38 pm EDT This is an incredibly exciting time in biomedical research. New discoveries are announced almost every week that bring us closer to managing cancer and other life-threatening diseases. We are attacking these illnesses at the molecular and genetic levels. In cancer, for example, scientists…
The enactment of FDASIA, including the fifth reauthorization of PDUFA, really put patient engagement on the radar beyond some of the traditionally active disease communities (e.g., HIV/AIDS, cancer, neurological diseases). FDASIA’s section 1137, the provision on Patient Participation in Medical Product Discussions, led FDA to seek input on “strategies to solicit the views of patients…
Roughly half of deaths from 12 smoking-related cancers may be linked directly to cigarette use, a U.S. study estimates. While the largest proportion of deaths associated with smoking were for cancers of the lung, bronchus, trachea and larynx, about half of fatalities from tumors of the oral cavity, esophagus and bladder were also tied to…
The amount of genetic variation between cancer patients is astounding. Two people diagnosed with cancer in the same organ may, in fact, have two very different diseases. Consequently, we are now in the middle of a transition: we no longer classify cancer based solely on its tissue of origin, but also on the key mutations…
FDA codified an expedited review pathway for breakthrough-designated drugs in a manual of policies and procedures (MAPP) released this week, instructing FDA review teams to act at least one month before the Prescription Drug User Fee Act (PDUFA) goal date for these applications. FDA confirmed it had been informally putting drugs through expedited review for…
Dr. Margaret Hamburg, who as commissioner of the U.S. Food and Drug Administration (FDA) for almost six years has overseen public health initiatives ranging from tobacco control and food safety to personalized medicine and drug approvals, is stepping down, the agency said on Thursday. Hamburg, 59, is one of the longest-serving FDA commissioners in the…
Days after the White House announced a new precision-medicine initiative, an influential group of US lawmakers has released its own wish list for biomedical research. On 27 January, several members of the House of Representatives released the first draft of their long-anticipated proposalto speed the translation of research into medicine. The effort, known as 21st Century Cures,…
Representatives from the pharmaceutical and biotechnology industries said last Friday (Dec. 12) they expect work on the bipartisan 21st Century Cures initiative this spring to be a staging ground for negotiations over the sixth iteration of the Prescription Drug User Fee Act (PDUFA) in 2016 and 2017. Industry leaders also said they expected the biotechnology…
More than 60% of US prescriptions for cancer drugs call for using the medicines in ways that are not approved by the government. Often, they are the only hope against a fatal illness that has thwarted conventional treatments. But although such ‘off-label’ use is common, it is hard to know how effective it is because…
FDA’s evaluation of the first two years of the breakthrough therapies program found common threads among designation requests that were granted and denied, which may help sponsors better understand the agency’s criteria for awarding the incentive. Below are key characteristics of products that have gained and were denied breakthrough designation, as well as reasons why…
