Implementing Current Regulatory Guidance: An Industry Perspective

Mouna Akacha, Statistical Methodologist, Novartis Pharma AG

There has been a long debate in the statistical literature on missing data issues and health authorities are increasingly concerned about the impact of missing data on statistical inference for clinical trials. In 2010, the Committee for Medicinal Products for Human Use (CHMP) published a guidance document on missing data in confirmatory clinical studies. At the same time, the National Research Council published a report on ‘The Prevention and Treatment of Missing Data in Clinical Studies’ which was commissioned by the U.S. Food and Drug Administration. Both documents highlight the importance of the choice of estimand and sensitivity analyses.

In this talk, we will give a review of regulatory guidance on missing data. We will discuss why the assumption of ‘missing at random’ may be of limited interest to regulators and touch upon the usefulness of ‘retrieved data’, i.e. collection of data after study treatment discontinuation.

Based on case studies, we will provide direct insight to health authority responses and discuss the proposed and accepted analysis techniques. In this context, we will also discuss potential differences in viewpoints of different health authorities.