Moreover, for many children with severe medical conditions, decisions about treatment are not limited to a one-time surgery or other episode of treatment. Rather, the balancing of potential benefits and harms is ongoing as new options for treatment are proposed or the shortcomings of past interventions are revealed.
Beyond treatment decisions, parents weigh risks in structuring their child’s daily life and determining the degree to which a tightly controlled, medically monitored environment should be moderated to allow a more normal childhood experience. For example, parents weigh the risk of damage to an implant against the opportunity for a child to play sports with his or her peers.
Often parents and clinicians must make decisions in the absence of good evidence about the relative safety and effectiveness of medical devices and procedures. Many complex medical devices used with children have not been systematically evaluated in pediatric populations. Even for adults, who are the typical subjects of clinical trials involving devices, uncommon problems may not be evident in clinical trials used to support applications for marketing approval. The clinical studies undertaken in support of a product’s approval for marketing are usually conducted for relatively short periods in carefully controlled populations that do not fully represent the population of expected users (e.g., patients with multiple health conditions). For that reason, once medical products enter the market, government health agencies, clinicians, manufacturers, and others sometimes continue to study them for longer periods and with broader populations. In certain circumstances, the U.S. Food and Drug Administration (FDA) can require such studies. Policymakers have also created requirements for manufacturers, health care facilities, and others to report problems—adverse events—that are caused by or associated with legally marketed drugs, devices, and other medical products.
Until 1976, federal officials had limited authority to regulate the safety or effectiveness of medical devices. In that year, Congress added the Medical Device Amendments (P.L. 94–295) to the Federal Food, Drug, and Cosmetic Act (P.L. 75–717). By this step, Congress acknowledged the increasing sophistication and importance of medical device technology while also recognizing that both the benefits and the risks of this advanced technology warranted more systematic attention by FDA, manufacturers, and others.
In the past decade, with enactment of the FDA Modernization Act of 1997 (P.L. 105–115) and the Medical Device User Fee and Modernization Act of 2002 (P.L. 107–250), Congress streamlined certain regulatory procedures for medical devices. The 2002 legislation also included several provisions related to pediatric uses of devices, one of which called for this