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…

Off-label prescribing

How frequently are pharmaceuticals used off label?  Perhaps more than you think.  Although these figures are a bit dated, Tabarrok (2000) details the extent of off-label prescribing in the U.S. as follows: According to a study by the U.S. General Accounting Office, 56 percent of cancer patients have been given non-FDA-approved prescriptions, and 33 percent…

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…

China fabricates clinical trial data?

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…

How the FDA evaluates Cancer Drugs

The Food and Drug Administration (FDA) reviews and approves all drugs. But what criteria does the FDA use to evaluate new drugs? Namely that they are effective (i.e., improve health outcomes) and safe (i.e., have limited adverse health events). In a 2007 guidance document from the FDA’s Center for Drug Evaluation and Research (CDER) and…