N-of-1 Trials: FDA Plots Path to Regulation | RAPS“At the very least, during the time needed to discover and develop an intervention, quantifiable, objective measures of the patient’s disease status should be identified and tracked, since, in an N-of-one experiment, evaluation of disease trends before and after treatment will usually be the primary method of assessing effectiveness,” Woodcock and Marks explained.
The Accuracy of Noninvasive Peripheral Pulse Oximetry After Palliative Cardiac Surgery in Patients With Cyanotic Congenital Heart Disease - Gemma E. Scrimgeour, Michael J. Griksaitis, John V. Pappachan, Andrew J. Baldock, 2017SpO2 (Masimo SET LNCS Neo pulse oximeter) overestimated oxyhemoglobin saturation in 82% of measurements (mean 4.6% ± 6.6%). There was a strong negative correlation between mean bias and SaO2 (r = −.96, P = .002, 95% confidence interval: −0.99 to −0.68).
Wrist-worn step trackers accurate in predicting patient health outcomes -- ScienceDailyIn the study, researchers conducted a 12-week, blinded, randomized, cross-over trial with 52 patients, a group that included adults with a history of respiratory problems during periods of elevated air pollution. Wrist step counters tracked patient steps for those 12 weeks; and patients also filled out respiratory symptom questionnaires. Researchers found they could effectively estimate a patient's 6MWD results by using step counters, instead of having patients come in a clinical setting to do the 6MWD test. "Instead of having one measurement every few months, you could have weekly measurements, and have information at disease progression at more frequent intervals. This is a significant improvement and enhanced convenience for our patients," said Dr. Blagev. The implications? Using wrist step counters will allow physicians to track how their patients are doing, the progression of the disease, and whether a patient requires an immediate intervention. "Being able to distill step counts into this clinically important metric is a first step in being able to think about how to use step counters in order to better manage health and detect deterioration earlier," Dr. Blagev added.
New chip poised to enable hand-held microwave imaging: Researchers shrink bulky imaging systems down to millimeter sized chip that could be used to see through walls or detect tumors -- ScienceDaily"Today's practical microwave imagers are bench-top systems that are bulky and expensive," said research team leader Firooz Aflatouni from the University of Pennsylvania, USA. "Our new near-field imager uses optical, rather than electronic, devices to process the microwave signal. This enabled us to make a chip-based imager similar to the optical camera chips in many smartphones." Hand-held near-field microwave imagers would be useful for many applications including high-resolution brain imaging and monitoring heart motion and breathing. Miniaturization of microwave imagers would also benefit applications such as tracking objects in radar systems and low-power, high-speed communication links.
Many leading universities still failing to report clinical trial results - STATSpecifically, findings were not posted for 31 percent — or 140 —of 450 studies that were to have been disclosed in public registries as a result of transparency requirements in the FDA Amendments Act, according to Universities Allied for Essential Medicines, a student-led organization concerned with access to medicines, and TranspariMED, a nonprofit research advocacy group. Moreover, only 15 of 40 universities previously found to have lax reporting are in full compliance. For instance, MD Anderson Cancer Center, which sponsored the largest number of applicable trials, disclosed only 77 percent of the findings. Similarly, the Mayo Clinic divulged 42 percent of studies. Columbia University had the worst track record, reporting 17 percent of trials, according to the analysis.
Time to say goodbye to “statistically significant” and embrace uncertainty, say statisticians – Retraction WatchThe bright-line thinking that is emblematic of declaring some results “statistically significant” (p<0.05) and others “not statistically significant” (p>0.05) obscures that uncertainty, and leads us to believe that our findings are on more solid ground than they actually are. We think that the time has come to fully acknowledge these facts and to adjust our statistical thinking accordingly.
Drug Companies and Doctors Battle Over the Future of Fecal Transplants - The New York TimesMark Smith, a microbiologist at M.I.T., was halfway through his pitch with a group of pharmaceutical executives when one of them interrupted to ask if the meeting was a prank. “I can’t believe you wasted my time with this crazy idea,” the man said, Dr. Smith later recalled. That was 2012. Later that year he helped found OpenBiome, the nonprofit stool bank that now supplies most of the fecal matter for transplants in the United States. Three years ago, he started his own drug company, Finch Therapeutics, which has raised $77 million. Over the past decade, tens of thousands of Americans with C. diff have been cured through fecal transplants, often with a single dose that can bring patients back from the brink of death. The treatment has more than an 80 percent success rate, according to several studies, and many patients feel better within hours of receiving the procedure, which is usually administered through colonoscopy or capsules containing desiccated fecal matter. The F.D.A. has not formally approved the therapy but it has suspended enforcement of its rules for patients who have failed on antibiotics while it figures out the best way to regulate a regimen that, until recently, was sometimes performed at home by desperate patients using an enema, saline and a relative’s stool. Ms. Duff, the head of the C. diff patients group, credits her own recovery from the disease to a homemade concoction her husband created with his own stool in the kitchen blender.
Medical Device Makers Report Malfunctions And Patient Injuries In FDA Database Hidden From Public ViewKaiser Health NewsAgency records provided to KHN show that more than 480,000 injuries or malfunctions were reported through the alternative summary reporting program in 2017 alone. Alison Hunt, another FDA spokeswoman, said the majority of device makers’ “exemptions” were revoked that year as a program took shape that requires a summary report to be filed publicly. More than a million reports of malfunctions or harm spanning about 15 years remain in a database accessible only to the FDA. But with the agency’s new transparency push, the public may find a public report and submit a Freedom of Information Act request to get information about incidents. A response can take up to two years. The long-standing exemption program “has allowed the FDA to more efficiently review adverse events … and take action when warranted without sacrificing the quality of our review or the information we receive,” Hunt said in an email. Madris Tomes(Courtesy of Madris Tomes) To those outside the agency, though, the exceptions to the reporting rules are troubling. They strike Madris Tomes, a former FDA manager, as the agency surrendering some of the strongest oversight and transparency powers it wields. “The FDA is basically giving away its authority over device manufacturers,” said Tomes, who now runs Device Events, a website that makes FDA device data user-friendly. “If they’ve given that up, they’ve handed over their ability to oversee the safety and effectiveness of these devices.”
Peter Thiel vs. the FDA - VoxThiel, a libertarian iconoclast, has repeatedly made the case that the FDA gets in the way of drug innovation by making it too difficult for new medicines to get to the market. Some of the FDA candidates he’s identified — including Silicon Valley’s Jim O’Neill and Balaji Srinivasan — have similarly argued that the agency should dump its requirement that drugs be proven effective before reaching the market, and that we’d be better off if the FDA operated more like a “Yelp for drugs.” In other words, bringing the same speedy and disruptive approach to medical regulation that Silicon Valley brought to the taxi and hotel industries, for example, will unlock cures — fast.
FDA Plans To Modernize 510(k) Program - Covering the specialized field of orthopedic product development and manufacturingImportantly, we’ve increased expectations for the quality and quantity of information required in 510(k) submissions, resulting in a more than doubling of the size of submissions—now, an average of 1,185 pages, compared to 475 pages in 2009. And while our reviewers have spent more time reviewing applications during this same period—an increase of about 32 percent—the average total time for the agency to reach a decision has decreased, reflecting a more robust and efficient program. These metrics reflect not only the strengthening of the medical device review program, but also the dedication of the talented CDRH career staff in carrying out our public health mission and continuing to drive forward critical program enhancements.
The F.D.A. vs. Personal Genetic Testing | The New YorkerA fifty-five-year-old who is confused and depressed and learns that he carries two copies of the risk gene and stands an eighty-per-cent chance of getting Alzheimer’s might reach for a gun, which is the kind of scenario that some genetic counsellors worry about.
Star Neuroscientist Tom Insel Leaves the Google-Spawned Verily for ... a Startup? | WIRED“I spent 13 years at NIMH really pushing on the neuroscience and genetics of mental disorders, and when I look back on that I realize that while I think I succeeded at getting lots of really cool papers published by cool scientists at fairly large costs—I think $20 billion—I don’t think we moved the needle in reducing suicide, reducing hospitalizations, improving recovery for the tens of millions of people who have mental illness,” Insel says. “I hold myself accountable for that.”
In 1977, an expert panel convened by the FDA issued urgently worded advice, saying it was “obligatory” to put a warning on the drug’s label that it could cause “severe liver damage.” After much debate, the FDA added the warning 32 years later. The panel’s recommendation was part of a broader review to set safety rules for acetaminophen, which is still not finished. Four years ago, another FDA panel backed a sweeping new set of proposals  to bolster the safety of over-the-counter acetaminophen. The agency hasn’t implemented them. Just last month, the FDA blew through another deadline.