Goal Attainment Scaling

What outcomes should be used to measure if a medicine is a “good” medicine? Improved survival? Improved ability to function? Few side effects? Ability to get back to work? Living to attend your daughter’s wedding? All of the above? Oftentimes, health economists measure value of a treatment for the average person. Preferences for efficacy, safety,…

Should you adjust for covariates when analyzing data from randomized controlled trials?

FDA draft guidance published this month says you should. In most cases, adjusting for covariates is not necessary. Randomization generally insurers that covariates are balanced across clinical trial arms. Randomization, however, may not always result in perfectly balanced trial arms. In these cases, the FDA notes that covariate adjustment is perfectly acceptable. There are some…

How to justify your survival curve extrapolation methodology

Clinical trials are typically of (relatively) short duration, but innovative treatments may impact patient survival over multiple years. Health economists and outcomes researchers often are faced with the challenge of extrapolating clinical trial survival curves to estimate long-term survival gains for the typical patient. These estimates may be done parameterically (e.g., exponential, Weibull, Gompertz, log-logistic,…

Human Challenge Trials

Should we infect people with COVID-19? Although most people would say ‘no’, this prospect could help speed the development of a vaccine. In these trials, half the people would be treated with a test vaccine for COVID-19 and half the people would receive a placebo. Then, people in both arms would be infected with COVID-19…

The gold standard of scientific evidence

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…

Predicting Real-World Effectiveness of Cancer Therapies Using OS and PFS Clinical Trials Endpoints

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…