The Trickett Wendler Right To Try (RTT) Act was introduced in May 2016 by Sen Ron Johnson (R-Wis). According to Johnson, the bill would ensure terminally ill patients, their doctors, and pharmaceutical manufacturers were allowed to administer investigational treatments where no alternative treatment exists. But for all the fanfare around RTT and the slow federal drug approval process, would the RTT legislation actually help patients?
“Rare diseases are bad enough, but the ones that affect children are even more heartbreaking,” says Mike Snape, CEO of AMO Pharma, a virtual biotech focused on rare disease. “The medical need in these areas is greater than ever. They also present challenges for drug developers, especially with clinical trials.”
Clinical news roundup for the week of May 14, 2017 with information on a new head of the FDA, inVentiv Health and INC Research merging, helping patients navigate cancer clinical trials, new standards for trial results, and more.
Samus Therapeutics is a privately-held, biopharmaceutical company focused on developing novel therapeutics and diagnostics targeting the epichaperome, a foundational protein complex emergent from multiple disease states, including oncology and neurology. “We are the ‘anti-epichaperome company,’ says Jonathan Lewis, the company’s executive chairman and CEO. “Following various forms of cellular stress, chaperome units are rewired into the epichaperome network. Targeting and disrupting the epichaperome in cancer results in cell death and in neurologic diseases, neuronal survival, with no apparent effect on normal cells.”
The Apple Watch, along with Apple’s ResearchKit, has already found a home in the clinical space. But it just got some competition. In April 2017, we saw the introduction of the Verily Study Watch. If you don’t know, Verily (formerly Google Life Sciences) is the research organization of Alphabet Inc. devoted to the study of life sciences. According to Verily, the Study Watch is the next step in the company’s targeted efforts to create new tools for unobtrusive biosensing.
For companies wanting to outsource their trials but also harbor concerns over CRO size and staffing, is there a solution? One group thinks they have come up with the answer. Four companies have come together to form a consortium designed to provide pharma with a dedicated team of trial experts focused on making studies more efficient and cost effective. Current members of the alliance include FMD K&L, inSeption Group, Pyxa Solutions, and OneSource Regulatory, with a plan to add more members in the future.
Patient recruitment remains a significant challenge for pharma companies conducting clinical trials. If all potential participants were aware of clinical trials, convincing patients to participate in them would still be a challenge. But that task becomes significantly more difficult when a large percentage of the population does not even know such trials exist.
FDA started the Breakthrough Therapy Designation program in 2012. Since then, Genentech has garnered 15 designations, more than any other company. Jeffrey Siegel has been an instrumental component of that success. Siegel is Genentech’s senior group medical director and spent 14 years with FDA prior to joining the company. He has spent most of his career in pharma developing products to address the unmet medical needs of patients.
Clinical News Roundup for the week of April 10, 2017 with information on Scott Gottlieb and adaptive approaches, preparing for epidemic clinical trials, proposed tax law changes for clinical research, and using selfies to improve medicine adherence.
Pharma companies that conduct clinical research inhouse essentially get a 100 percent tax break on the expenses incurred from that research (about 70 percent of which are normally wages). If they instead outsource that research to a CRO, they may claim only 65 percent of eligible expenses. The CRO gets no tax break, so that 35 percent difference simply disappears. If a group of congressmen get their way, that situation might soon change.