Daniel Michaeli: Our new article ‘Clinical trial design and treatment effects’

Daniel Michaeli, Medical Doctor at National Center for Tumor Diseases (NCT) Heidelberg, shared a post on LinkedIn:

“Excited to see our new article ‘Clinical trial design and treatment effects’ published by BMJ Evidence-Based Medicine with T. Michaeli, S. Albers, and J. Michaeli.

What is known on this topic:

• Clinical trials demonstrating a new drug’s safety and efficacy for their approval ought to provide unbiased treatment effect estimates.
• Prior meta-epidemiological studies found trial design can bias reported treatment outcome.
• This is the first study to analyse the association between sources of bias arising from clinical trial design features and treatment effects in trials supporting the FDA approval of new cancer drugs.

What the study adds:

• Larger treatment effects were reported for smaller (small study effect), shorter (short study effect) and single-centre trials.
• Greater treatment effects were observed in trials comparing a new cancer drug to an inactive comparator (placebo/no treatment) rather than an active comparator (anticancer drug), particularly for surrogate endpoints.

Practice and Policy implications:

•  Regulators, pharmaceutical companies and trialists must strive to conduct large, multicentre trials that test clinically relevant hypotheses.
• Patients and physicians should critically review clinical trial design for the interpretation of overall survival, progression-free survival and tumour response outcomes.”

Additional information.
Source: Daniel Michaeli/LinkedIn