Assuring Data Quality and Validity in Clinical Trials for Regulatory Decision Making: Workshop Report (1999)

A major finding of the workshop was the need for collaborative systematic improvements. Related themes included the need for greater openness and communication; broader involvement of stakeholders, including consumer groups; greater efforts to build quality into the process; and careful attention to maintaining public confidence and the scientific integrity of the clinical trials and regulatory process. In the course of the workshop, participants identified a number of topics about which further discussion might contribute to this effort. The Roundtable members therefore proposed that a 1-day symposium be convened to focus on the following three topics identified as important follow-up activities to the workshop: (1) clinical investigator training, (2) the role of consumers, and (3) better integration of the Food and Drug Administration (FDA) and industry with regard to monitoring.

FDA is making plans to convene a working group of its members as well as with the Institute of Medicine (IOM), National Institutes of Health, and stakeholders in clinical investigations (credentialing bodies, university representatives, clinical trials societies, pharmaceutical industries, and study personnel representatives) to discuss clinical investigator training issues. Topics expected to be covered in this first follow-up activity include training of clinical investigators for pediatric trials, clinical pharmacology, informed-consent issues, and the most effective type of training.

This workshop report highlighted the fact that; the goal of improving health through the use of new medicines cannot be achieved without public confidence in the clinical trials process. Subsequently, this objective cannot be achieved without participation of the public and without bringing into the spotlight the importance of informed consumer groups' opinions. An effective means of measuring the public's confidence in the process by which new investigational drugs are evaluated is their willingness to participate in clinical trials. Although

to date plans for addressing the third proposed topic are still in their infancy, IOM has initiated communication with FDA and industry to begin making preparations to convene a symposium on consumer involvement issues, the second follow-up activity identified by the Roundtable members. A major goal of this symposium will be to demystify the drug development and drug safety process for consumers. Messages on such issues to consumers have been developed by FDA as well as by Public Citizen and the Consumer Federation of America, consumer organizations that watch over the activities of federal agencies. Some of the proposed discussion topics may include messages to consumers on drug development and drug safety in the areas described below.

• ways to determine whether a drug is safe and effective,
• whether new drugs need to be more effective and safer than older drugs,
• knowledge of long-term safety of new drugs at time of approval,
• quick review of new drugs by FDA for serious and life-threatening diseases, and
• which patients can have access to experimental drugs that have not completed the final FDA review.

• determination of whether the benefits of a drug outweigh the risks,
• acknowledgment of the fact that risks exist in taking every drug,
• difficulty in determining all of the individual adverse reactions to a prescription drug,
• voluntary nature of the adverse drug reaction reporting system and the fact that health professionals are therefore not required by law or regulations to report any adverse drug reactions to FDA,
• the fact that manufacturers are required to report to FDA adverse drug reactions reported on any of their medicinal products, contrary to the requirements for health professionals,
• monitoring of adverse drug reactions by FDA to determine whether labeling changes are necessary or whether the drug should be removed from the market,
• encouragement of consumer reporting of an adverse reaction to a medication to one's health care provider or directly to FDA, and
• insistence that consumers obtain a drug's approved product labeling from the pharmacist before taking a new drug.

By demistifying the drug safety and development process, it is hoped that public confidence and public participation in the clinical trials process will be maximized.