Unbiased Analysis of Today's Healthcare Issues
That is the title of my recent piece in STAT News. Earlier this month, the Food and Drug Administration announced the creation of Project Facilitate. This pilot program facilitates access to innovative treatments for cancer that have not yet been approved by individuals who aren’t able to enroll in clinical trials. This move suggests that the…
How do you design a good clinical trial? Clearly, the trial design depends on what question you are answering. For instance consider the dichotomy between an explanatory and pragmatic trial: A pragmatic randomised trial is undertaken in the “real world” and with usual care and is intended to help support a decision on whether to…
That is the title of my latest article in Pharmaceutical Market Europe. An excerpt is below. Randomised controlled trials (RCTs) are regarded as the gold standard of scientific evidence, and for good reason. By randomizsing a treatment across study arms, RCTs eliminate patient-treamtent selection bias, resulting in reliable causal inference. In contrast, in the real…
As research in new cancer treatments has grown, scientists may have run into a serious roadblock: there many not be enough patients to fill the needed clinical trials. As the New York Times reports: There are too many experimental cancer drugs in too many clinical trials, and not enough patients to test them on. The logjam…
Clinical trials for cancer treatments aim to demonstrate whether one treatment is better than another. What is of most interest to patients, providers and payers, however, is which treatment works best in the real-world, not in a randomized controlled trial. Further, clinical trials often use progression free survival to measure treatment outcomes rather than overall…
An interesting paper from some of my colleagues at Precision Health Economics: Approval of new drugs is increasingly reliant on “surrogate endpoints,” which correlate with but imperfectly predict clinical benefits. Proponents argue surrogate endpoints allow for faster approval, but critics charge they provide inadequate evidence. We develop an economic framework that addresses the value of…
Academic integrity is one of the bedrocks upon which research is founded. With that said, it is with great concern that I came across an article that stated that 80% of Chinese clinical trials data is fabricated. Science Alert reports: The review looked at data from 1,622 clinical trial programs of new pharmaceutical drugs awaiting regulator approval for mass…
Efficacy describes the technical relationship between the technology and its effects (whether it actually works), whereas effectiveness concerns the extent to which application of an efficacious technology brings about desired effects (changes in diagnoses, altered management plans, improvement in health)…Efficiency is an economic concept which relates efficacy and effectivness to resource use. Assessment of efficiency is…
Your knee-jerk reaction (and mine) is No! However, an interesting article from Mosiac makes the case that the argument is not clean cut. Because it has long been considered unethical to include expectant mothers in clinical trials, scientists simply don’t know whether many common medicines are safe for pregnant women. Of the more than 600…
An concise article from Sean Khozin and Gideon Blumenthal (2015) try to explain. “Personalized (or precision) medicine has been broadly described as the administration of the right therapy to the right patient at the right dose and intensity.” However, this is a fairly broad definition. Some more concrete examples of personalized medicine include: Modern concepts in…