Law and Regulation in a Systems Setting

In our last team week note we listed five categories that will be the focus of our Phase 2 work. This week, let’s take a closer look at one of those categories – law and regulation – and examine how modern drug legislation evolved.

In the early twentieth century, there were no effective laws controlling the marketing of drugs. That changed in the 1930s with the passage of the Food Drug and Cosmetic Act in 1938 (the FDCA), which Franklin Roosevelt signed into law to strengthen a statute passed in 1906. Later in the twentieth century (in 1962), Congress passed, and President Kennedy signed, a major amendment to the FDCA. These two pieces of legislation form the core of legal principles that shape the discovery, evaluation and marketing of drugs and biologics today. Both of these laws were enacted because of tragedies involving drugs. In the first case (Elixir of Sulfanilamide, 1937) a sulfa drug to combat strep was marketed largely for children without any pre-market testing. The solvent used to dissolve the sulfa drug proved poisonous, and killed over 100 people. In the second case, (thalidomide, 1950s through 1961) a morning sickness drug was not fully tested for developmental effects, and its use by pregnant women resulted in babies with birth defects. Medical devices share a similar story. Congress passed, and President Ford signed, a comprehensive medical device bill in 1976 after it became known that medical devices resulted in 10,000 injuries and more than 700 deaths.

While these are the highlights, we should not forget that the FDCA has been amended over 100 times. It has seen several important changes in the past 15 years. Provisions were added to create approval pathways and incentives for drugs that are meant to treat “orphan diseases” (a disease whose prevalence is fewer than 200,000 people), for “fast track” review (for drugs and biologics targeted to serious or life threatening illnesses for which there is an unmet need) and “breakthrough therapies” (to expedite the development and review of drugs that could be substantial improvements over available therapies.)

For a systems thinker, the nature, pace and process of amending the FDCA teach at least one important lesson. Simply put, the history of the FDCA is a history of after-the-fact actions and patchwork legislating. Congress has been reactive by responding to misfortune and perceived gaps, but it has not been proactive. Nor has it thought about the broader implications of these individual changes from a systems perspective. It has mostly left the FDA and the regulated community with the job of figuring out how these alterations can be incorporated into the overall process of drug discovery and approval. By promulgating regulations and issuing guidance documents, FDA has woven together a system that meets Congressional goals and created the drug development and approval structure that exists today.

As we examine the laws and regulations more deeply, we will be outlining the steps that products must successfully navigate to meet FDA approval and stay on the market. We anticipate developing recommendations about how to change this system so it can more successfully meet its goals of delivering drugs that meet high standards for safety, efficacy and quality as well as spark innovation and encourage research.

Paul Locke