Drug Approval in a Learning Health System

Abstract

The current system of FDA approval seems to make few happy. Some argue FDA approves drugs too slowly; others too quickly. Many agree that FDA — and the health system generally — should gather information after drugs are approved to learn how well they work and how safe they are. This is hard to do. FDA has its own surveillance systems, but those systems face substantial limitations in practical use. Drug companies can also conduct their own studies, but have little incentive to do so, and often fail to fulfil study commitments made to FDA. Proposals to improve this dynamic often suggest gathering more information after approval in various ways and incorporating that information into FDA’s decision-making process, making the information/access tradeoff more nuanced than a sharp binary at approval. The drug approval regime has already begun to move in this direction.

In this Essay I describe parallels between this move and the move to a learning health system more broadly, which blurs the line between health care and health research. A learning health system creates opportunities for broad postapproval studies, both observational and interventional, with lower costs and more systematic applicability than traditional mechanisms. FDA itself has recognized some of these possibilities, especially in its actions to consider uses of real-world evidence as mandated by the 21st Century Cures Act.

Unfortunately, while learning health systems blur the line between care and research, some parts of the law do not — at least not yet. In particular, the federal Common Rule requires relatively elaborate informed consent procedures for research, and the Health Insurance Portability and Accountability Act (HIPAA)’s Privacy Rule tightly limits the use of identifiable patient information in research. These unblurred lines fail to reflect the evolving nature of the health-care system, and both hamper and bias the generation of systematic, generalizable knowledge to improve the use of drugs and other medical interventions in patient care.

Keywords: FDA, Learning Health Systems, Innovation, Privacy, HIPAA, Informed Consent, Big Data, Medical Innovation

JEL Classification: K23, L52, L65, O31, O34, O38