Unbiased Analysis of Today's Healthcare Issues
That is the question posed in paper by Baker, Larcker and Wang (2022). I summarize their key arguments below. The validity of…[the DiD]…approach rests on the central assumption that the observed trend in control units’ outcomes mimic the trend in treatment units’ outcomes had they not received treatment. As the authors write: First, DiD estimates…
Previously, CMS implemented a Tukey outlier deletion method when calculating Medicare Advantage (MA) and Medicare Part D Prescription Drug Plans (PDP) star rating. A Final Rule implemented in 2022, however, removed the use of Tukey outlier deletion from quality measures. Based on 2020 historical data, 17% of MA plans would have lower star ratings as…
When using observational data, assignment to a treatment group is non-random and causal inference may be difficult. One common approach to addressing this is propensity score weighting where the propensity score is the probability that a person is assigned to the treatment arm given their observable characteristics. This propensity is often estimated using a logistic…
While randomized controlled trials (RCTs) are the gold standard of experimental research, there are some limitations. For instance, in some cases use of an RCT is not feasible (e.g., the sample size may be too small as in the case of rare disease), not ethical (e.g., if the previous standard of care is not effective)…
At first glance, one may think the answer is no. Randomization should insure that baseline characteristics are balanced across trial arms. In practice, however, sometimes baseline characteristics due differ somewhat by trial arm simply by chance, especially in smaller randomized controlled trials (RCTs). A JAMA Guide to Statistics and Methods by Holmberg et al. 2022…
Clinical trials have detailed study protocols and are registered on ClinicalTrials.gov. What level of details are needed for real-world data (RWD) analyses that aim to estimate treatment effects? In Europe, the European Medicines Agency (EMA) requires registration of many study protocols using a template for observational post-authorization safety studies (PASS) conducted by marketing authorization holders.…
The Yost Index is a measure of socioeconomic status (SES) across geographic areas. The index was developed by Kathleen Yost and coauthors in their 2001 paper titled “Socioeconomic status and breast cancer incidence in California for different race/ethnic groups.” There are seven key inputs to creating the Yost index were calculated at the Census block…
One would think that this question would be a relatively easy question to answer empirically: simply compare patients with delays against those without delays and see if there is a difference in health outcomes (e.g., survival). This approach, however, is problematic as due to three key methodological concerns. Measurement issues. Ideally, one would like to…
Today, the Food and Drug Administration (FDA) approved the drug Relyvrio (sodium phenylbutyrate/taurursodiol; also known as AMX0035), which is indicated for the treatment of patients with amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease. Most people with ALS die within 2-5 years of diagnosis. In justifying the approval, FDA’s press release stated: “This…
It seems like everything just gets more and more expensive. But are prices for medical care declining? Cutler et al. (1998) found in seminal paper “Are medical prices declining? Evidence for heart attack treatments” that while unadjusted prices of medical care were in fact rising, quality-adjusted health care costs actually were falling. In my own…