Gene editing, specifically somatic gene therapy, cures maladies with traceable genetic origins. This revolutionary science is rapidly advancing, but not without major setbacks. Regulations designed to protect patients from dangerous applications of gene editing, both real and imaginary, are increasing the cost, slowing development, and preventing life-saving research. This hurts all patients, but especially the economically disadvantaged. If unnecessary regulations are eliminated, the poor will benefit most. For a more effective way forward, the history of vaccines provides a guide.
The genetic codes for diseases that predominantly hurt those in poverty, such as HIV, diabetes, alcohol-attributed illnesses, and heart failure, are well understood and treatable. Improved treatments for diabetes, for example, would disproportionately benefit individuals with less income, less education, and less access to health care. Furthermore, a new treatment for diabetes could save approximately $327 billion for the U.S. health care system. Cures for diseases that are not associated with lower socioeconomic groups but are nevertheless gene editing candidates, such as cystic fibrosis, cancers, muscular dystrophy, and Huntington’s disease, would reduce billions of dollars in costs from the health care system.
Gene therapy is a precise and highly effective treatment for many diseases, but currently the prices are extremely high. The process of multi-party deal making, not a lack of innovation, is the primary culprit. Safety and efficacy regulations impose significant cost burdens on research and development, suffocating smaller and more innovative firms. As a consequence, smaller firms must sell their products to larger firms. Insurance companies reimbursement policies, likewise, incentivize larger firms to charge even higher prices because of drug price regulations.
During this laborious process, gene editing is overseen by both the Food and Drug Administration (FDA) and the National Institutes of Health (NIH). The duplication is an expensive and unnecessary burden. Safety is important, but overregulation can inhibit supply, especially in areas where there is great demand. This regulatory trajectory leads to either adopting a national health care system that severely limits new therapies but has broad socioeconomic access or making gene therapies available to the wealthy alone. Neither scenario is optimal.
The best way to avoid these two outcomes is to change the regulatory framework. According to senior leaders at the FDA and NIH, gene editing should no longer be treated as a special case of clinical research. Its hazards are not unique. In fact, gene editing is most comparable to vaccines, requiring diligent research, not regulation, to ensure safety. When vaccines moved away from governmental oversight and toward commercial manufacture, it resulted in lower prices from competition and superior production methods. Vaccines saved millions of lives and $68.6 billion in societal costs. In lower socioeconomic strata, the difference was the most dramatic.
If health promotes economic growth at all levels, how much more could gene editing save? Here is a technology that has the capacity to prevent a wide array of diseases, no matter how rare or common. To stall gene editing, therefore, is akin to, if not worse than, inhibiting vaccines.
Many are afraid that fewer regulations will promote genetic enhancement among the wealthy while leaving the poor behind. This is unlikely. While genetic diseases have a straightforward code, enhancement traits do not. Intelligence, a trait most significantly correlated with financial success and independent of culture, relies heavily on the environment. Furthermore, the assumption that humans can enhance without limit ignores a certain evolutionary principle: tradeoff. A larger neocortex assists cognitive function at the expense of energy, reproduction, and muscular growth. Likewise, cognitive function is preserved at the expense of physical strength and fighting performance. Genes are only modified within current evolutionary boundaries, serving little use for immediate and meaningful enhancement. The fear of enhancement for the wealthy obstructs the quest to find cures for knowable, treatable diseases. Rather, policymakers should let competition generate life-saving therapies for the economically disadvantaged.
The regulatory road is long and difficult. Removing unnecessary restrictions will decrease the cost of gene editing, making more treatments accessible to more people, especially to the poor. Let diseases be the targets, not patients.