Defining Efficacy Estimands in Clinical Trials: Examples Illustrating ICH E9(R1) Guidelines

INTERNATIONAL CONCIL FOR HARMONISATION OF TECHNICAL REQUIREMENTS FOR PHARMACEUTICALS FOR HUMAN USE ICH HARMONISED GUIDELINE Estimands and Sensitivity Analysis in Clinical Trials E9(R1)

Clinical Trials in Oncology and Defining Benefit

In the editorial “The Evolution of Clinical Trials in Oncology: Defining Who Benefits from New Drugs Using Innovative Study Designs” [1], Goldberg et al. nicely discuss many changing aspects of clinical trial design in oncology. However, I believe their title is misleading. No study will define “who benefits” but only who might benefit. For example, human epidermal growth receptor 2 overexpress...

A structured approach to choosing estimands and estimators in longitudinal clinical trials.

An important evolution in the missing data arena has been the recognition of need for clarity in objectives. The objectives of primary focus in clinical trials can often be categorized as assessing efficacy or effectiveness. The present investigation illustrated a structured framework for choosing estimands and estimators when testing investigational drugs to treat the symptoms of chronic illne...

Guidelines for clinical trials in melasma

There have been very few well-conducted trials in melasma and this makes the process of comparing treatment outcome between trials difficult. The Pigmentary Disorders Academy has examined the issues relating to clinical trials on melasma, and has proposed recommendations on how they should be conducted. This covers all aspects including correct diagnosis of the condition, evaluation of efficacy...

Predictors of high quality clinical practice guidelines: examples in oncology.

OBJECTIVE Clinical practice guidelines are widely used as effective tools for improving the management of patients with cancer. However, there is increasing concern about variation in guideline quality. In this study we identified predictors for high-quality guidelines in oncology. DESIGN The quality scores for 32 oncology guidelines from 13 countries were determined by four independent appra...