The Maryland-based company, which has never brought a product to market before, just made the biggest deal to date with the Trump administration’s Operation Warp Speed.
Jonathan Kimmelman'many clinical trials fail when vaccines turn out to be ineffective...' Actually, those trials are a success. They protect us from unsafe/ ineffective vaccines. Its the candidate that fails. @katie_thomas
AbstractBackground. Phase 1 oncology trials are often regarded as a therapeutic option for patients. However, such claims have relied on surrogate measures of
Jonathan KimmelmanIf you participate in a #cancer phase 1 trial, what are the chances of receiving a drug, at the right dose, that will eventually be approved for your cancer? About 1 in 83. Latest from @STREAM_Research. More below: 1/
The company said its preliminary test in 8 healthy volunteers was safe. It is on an accelerated timetable to begin a larger human trial soon.
Jonathan Kimmelman"Moderna Coronavirus Vaccine Trial Shows Promising Early Results." Uh, some context here? 90-95% of Rx's/vaccines showing similarly "promising results" (i.e. they don't kill you in a phase 1) never show sufficient safety / efficacy for approval.
This Viewpoint uses publicly available data to analyze the number of deaths from seasonal influenza deaths compared with deaths from coronavirus disease 2019.
Jonathan Kimmelman...'Directly comparing data for 2 different diseases when mortality statistics are obtained by different methods is inaccurate. The repeated failure of govt' officials & others to consider these statistical distinctions threatens public health.' END/
The drug company, along with a German partner, is running tests in healthy volunteers. It’s one of several companies on an accelerated timetable to try to find a safe, effective vaccine.
Jonathan KimmelmanGiven that no RNA-based vaccine has yet to be approved, & that most novel platforms take decades of iterative research, the odds of this candidate showing substantial protection are very slim (if not 0)- no matter how compressed / staggered their approach.
Developers and funders are laying the groundwork for efficacy trials, but only a handful of vaccines are likely to make the cut.
Jonathan KimmelmanDistressing & encouraging themes in this article on vaccine development. 1st the former: WHO orchestrating platform trials that will test vaccines side by side. Important to know how vaccines compare w/ each other. And important to have >1 vaccine!
What is replication? This Perspective article proposes that the answer shifts the conception of replication from a boring, uncreative, housekeeping activity to an exciting, generative, vital contributor to research progress.
Jonathan KimmelmanGorgeous, illuminating essay from @BrianNosek & Tim Errington, on replication: 'declaring a study is a replication is a theoretical commitment. Replication provides the opportunity to test whether an existing model can predict outcomes.' @PLOSBiology '
This Viewpoint reviews notable examples of clinical trial misconduct identified by routine US Food and Drug Administration (FDA) clinical trial site inspections and argues that making inspection reports publicly available on the agency’s and trial...
Using breast cancer as an example, we evaluated the underlying evidence for the surrogate
endpoints for solid tumors listed in the FDA's Table of Surrogate Endpoints and found
weak or missing correlations of treatment effects on these surrogates...
Jonathan Kimmelman'With oncology training focused on treatment, clinical trials, and drug development, it is not surprising that palliative care is still not integrated into cancer care.' Thoughtful comment by @ramsedhom et al
Simple design aims to let even overwhelmed physicians and hospitals participate
Jonathan KimmelmanQuestion 2: ‘The WHO scientific panel had originally decided to leave chloroquine/hydroxychloroquine
out of the trial, but had a change of heart at a meeting bc the drugs received significant attention. Are you f’ing kidding me? 4/
The following review has been prepared by members of the MRC-NIHR Trials Methodology Research Partnership. The reviewers are all qualified statisticians with experience in clinical trials. Our objective is to provide a rapid review of publications,...
Jonathan KimmelmanThat nonradnomized hydroxychloroquine / azithromycin trial for COVID19 Rx? Review from @statsepi et al: 'control patients included those who refused consent for the active arm. The fact that patients who didn’t give such consent is a red flag'
This study uses data from annual financial reports to compare the profitability of large pharmaceutical companies vs other large companies in the S&P 500 Index from 2000 to 2018, measured via gross profit; earnings before interest, taxes,...
Jonathan KimmelmanUsing 3 different metrics, pharma co.'s in S&P have > median profitability than all non-pharma co's in S&P (including energy and tech- w/ a few near ties for individual measures).
Jonathan KimmelmanFrom the vaults ('13): 'none of the 9 docetaxel-based combo strategies [tested for metastatic castration resist prostate cancer] had sufficient phase II data to warrant further development;' all lead to (-) phase 3 trials.
The era of precision medicine has arrived. A large share of new pharmaceuticals are tested and approved on the basis of biomarkers. Pharmacogenetics—the tailoring of drugs to patients based on 1 or more biomarkers—is used in conditions as diverse as...
Jonathan KimmelmanHealth economists: is there evidence for the following claim from @Cutler_econ? "The more drugs there are in a particular therapeutic area, the more competitive is pricing." [my impression is that- w/ pharmaceuticals in USA it has not been the case].
Let's talk Sarepta. And FDA approval, because you can't bring up that company without immediately starting a regulatory affairs argument. I was not happy when their initial exon-skipping therapy (Exondys, eteplirsen) for Duchenne muscular dystrophy...
Jonathan Kimmelman“What the hell is going on?” with FDA’s Sarepta / muscular dystrophy approvals??
Objective To determine whether patients randomized to unapproved, disease-modifying interventions in neurodegenerative disease trials have better outcomes than patients randomized to placebo by performing a systematic review and meta-analysis of...
Is it better to be in the placebo arm when participating in trials of disease modifying Rx's for neurodegenerative diseases? Yes, says just published analysis from @stream_research@amandamacp5 & aden feustel. Why? Because... 1/