Clinicians and FDA Decisions

Abstract

TO THE EDITOR: The last two issues of the American Journal of Gastroenterology contain articles arguing that the FDA should seek input from clinicians before limiting the availability or withdrawing a drug that affects patients that are cared for by these physicians (1, 2). Both articles fail to acknowledge explicitly that FDA is a data-driven regulatory organization that requires data to support whatever decisions it makes. Data Monitoring Committees (DMCs) for clinical trials, like FDA when considering safety data for a previously approved product, make risk-benefit assessments based on safety data that become available sequentially over time. The nature of the safety database used in these assessments is almost always robust because of the global standards that exist for safety data (3). On the other hand, there are no standard methods for collecting data to quantify the benefits of a pharmaceutical product used to treat a non-life threatening condition. Data from quality of life measures, such as the SF-36 or Sickness Impact Profile, are often unavailable or not adequate to make risk-benefit assessments. Physicians with relevant clinical expertise usually serve on DMCs and participate in the risk-benefit assessments, but the lack of methods for assessing benefit can make risk-benefit assessments quite complicated (4). I believe that without robust methods for quantifying the benefit of long-term treatment for a non-life threatening condition, FDA, like DMCs, will still be forced to emphasize safety issues. Changing the FDA decision-making process along the lines suggested in the editorials is unlikely to change the nature of the final decision.