Expansion cohorts can help bring life-saving drugs to market quickly, but sponsors should be sure to update informed consent for patients and convene expert safety panels that meet regularly, the FDA says in new draft guidance.

NIH has delayed a controversial new policy that would have required basic brain and behavioral researchers to treat their work as clinical trials.

Researchers are launching the first human trial of an experimental live Zika vaccine. The vaccine, which contains a weakened version of the virus, was de­veloped by scientists at the National Institute of Allergy and Infectious Diseases (NIAID). The trial plans to enroll a total of 28 healthy, non-pregnant adults, ages 18 to 50, at Johns Hopkins School of Public Health Center for Immunization Research and at the Vaccine Testing Center at Larner College of Medicine at the University of Vermont. Trial participants will be randomly assigned to receive a single subcutaneous dose of the experimental vaccine (20 participants) or a placebo (eight participants). Neither they nor investigators will know who is receiving the real thing. Participants will keep track of their tem­perature on diary cards at home – and return to the clinic for periodic checkups for about six months. Researchers will take blood samples during visits to see if they’re developing antibodies in response to the vaccine. Anna Durbin, a professor of International health at Hopkins School of Public Health, is leading the Phase I clinical trial. Most people infected with the Zika virus — primarily transmitted by certain mosquitoes — have mild to no symptoms. But it can cause serious birth defects and developmental problems in babies born to women infected during pregnancy. That’s why the Centers for Disease Control and Prevention advises women who are pregnant or planning to become pregnant not to travel to areas with a known Zika risk. “Zika virus infection remains a significant threat to pregnant women and their developing fetuses, and we can expect to see periodic outbreaks and cases in areas where Aedes aegypti mosquitoes thrive,” said NIAID Director Anthony Fauci. “NIAID remains committed to developing safe and effective Zika vaccines, and we are pleased to begin clinical testing of a live attenuated candidate.”

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The FDA has asked Congress for $100 million to help build an advanced analytics system the agency says will help it, at long last, master real-world evidence.

Sponsors may not have to prove a proposed therapy helps addicts totally kick opioids to win FDA approval. But they will have to show it significantly cuts dependence on the potent pain meds, the agency says in new draft guidance.

The FDA says sponsors considering adaptive studies for early phase cancer treatments should focus on patients with no other options. The agency notes that so-called expansion cohort trials — which start out relatively small but expand if a potential treatment shows promise — can be a way to get a treatment to market more quickly. The thinking is, the more participants in earlier trials the more likely it is to find and fix (if possible) glitches — ensuring faster approval or scrapping the potential treatment in its infancy if it doesn’t work or if risks outweigh benefits. “A lot of time and cost of clinical development is spent waiting in between the start and end of the phases of trials,” FDA Commissioner Scott Gottlieb said in a statement. “Expansion cohort trials can bring efficiency to drug development, potentially reducing development costs and time.” In a 17-page draft guidance released last week, the FDA says it wants to “establish an infrastructure” designed to help sponsors use cohorts to speed drug approvals without compromising safety. Its top recommendation: Focus on patients who are most seriously ill and don’t have any other treatment options. The agency also encourages researchers to hire centralized data management committees and IRBs and have them meet regularly to discuss latest findings and best next steps. “It is critical that investigators, IRBs and regulators are updated with new safety information so that they can provide the necessary oversight for the protection of human subjects and so that investigators can ensure that patients can provide adequate informed consent,” the draft document says. The jury’s still out on the effect of expansion cohorts. A 2017 study by Texas researchers, published in the Journal of the American Association for Cancer Research, analyzed 533 Phase I cancer trials between 2006 and 2011. They found the ones that used expansion cohorts went on to have successful Phase II trials at nearly twice the rate (48 percent) as studies that didn’t (27 percent). But just a year earlier, a team of Cleveland researchers analyzed 252 cancer trials conducted between 2004 and 2014 and found expansion cohorts made little or no statistical difference between success or failure in Phase II trials. Read the FDA’s draft guidance here: www.fdanews.com/08-10-18-DraftGuidance.pdf.

Monthly injections of the anti-addiction drug naltrexone (brand name Vivitrol) may help HIV-positive patients cut down on heavy drinking but it doesn’t help them stick to their med schedules, Yale researchers found. Researchers recruited 51 HIV patients for a four-year trial; all were heavy drinkers and strayed from their daily treatment plans. During the trial, patients received monthly naltrexone injections and regular counseling. The findings, published the journal AIDS and Behavior: Naltrexone helped patients reduce heavy drinking days. But it didn’t improve their antiretroviral adherence. Antiretroviral drugs have converted HIV and even AIDS from deadly diseases into chronic ones. But they only work if patients take them at least 95 percent of the time, researchers said.