Much of the progress in medicine during the past half-century has involved expensive, high-tech diagnostic tests and therapies. The trend in this direction worries health economists and politicians because it can potentially send already-high healthcare costs into the stratosphere.
However, in both medicine and dentistry, there is an important role for ingenious, low-tech, less expensive approaches to improving health and increasing longevity.
Several years ago, the FDA approved a high-tech gene therapy drug, Zolgensma, for a rare childhood genetic disease, spinal muscular atrophy, that costs just over $2 million for the single dose of the treatment. The illness, caused by a defect in a gene called SMN1, affects about 400 babies in the United States annually and kills those with the most common form of the disease in the first few years of life. The treatment uses non-pathogenic, genetically engineered viruses to deliver healthy copies of the SMN1 gene to patients’ cells so they can synthesize a protein needed to develop normal muscle neurons.
Gene therapy is treating more and more genetic diseases, but the cost is prohibitive. Last year, the FDA approved a product called Hemgenix to treat hemophilia B, which is caused by a mutation in hemoglobin. It is the most expensive drug in the world, at $3.5 million for a one-time infusion. Another application of gene therapy was recently reported in a Phase 3 trial of a biological called aloctocogenve roxaparvovec, which led to significantly reduced bleeding rates that persisted in men with severe hemophilia A despite standard Factor VIII prophylaxis. Pharmacokinetic modeling suggested that Factor VIII activity levels would remain in the mild hemophilia range for at least five years following gene transfer.
Another remarkable genetic engineering feat was reported in the journal Nature in 2017. An experimental gene therapy procedure used to transform and grow sheets of healthy skin saved the life of a 7-year-old boy who suffered from a genetic disease, junctional epidermolysis bullosa, that had blistered and destroyed most of his skin. He was on the verge of death, but two years after the treatment with genetically engineered cells produced by a multi-national team, he had healthy skin and was leading a normal life.
These kinds of high-tech interventions are spectacular, but there are many simpler and cheaper yet tremendously important innovations for diagnosing and preventing illness. Among the most cost-effective are checklists for personnel in operating rooms and ICUs. According to a Norwegian research group, “Safety checklists appear to be effective tools for improving patient safety in various clinical settings by strengthening compliance with guidelines, improving human factors, reducing the incidence of adverse events, and decreasing mortality and morbidity.”
Sometimes, a simple tool or device is important to clinical diagnosis. One example is the hand-held direct ophthalmoscope, which allows a medical practitioner to look into the back of the eye to ascertain the health of the retina, optic nerve, vasculature, and vitreous humor (the liquid inside the eyeball). Invented in 1851, it costs less than $200.
Another example is how a single blood test can ascertain that a patient in the emergency room is not having a heart attack and can then forego the inconvenience and expense of additional invasive tests or unnecessary hospitalization. The highly sensitive blood test measures levels of cardiac troponin, a protein involved in muscle contraction; if the level is undetectable – that is, below the limit of detection of the test – there is a greater than 99% likelihood that the patient is not experiencing a heart attack and is at very low risk of other cardiac adverse events for at least 30 days.
That innovative approach is advantageous to patients and helps to reduce the frequency of hospitalizations and, therefore, healthcare costs.
Falls are both a cause and effect of declining health in the elderly. They are the leading cause of injury-related visits to emergency rooms and the primary cause of accidental deaths in Americans over 65. To measure the potential benefits of a low-tech approach to preventing injuries from them, a research group in New Zealand compared rates of falling and injuries from falls on low-impact flooring (LIF) compared with standard vinyl flooring on an “older persons health ward.” Falls were prospectively monitored with written reports of all incidents, noting the location and consequences of each fall. The frequency of falls and injuries on LIF and those occurring on standard vinyl flooring (controls) were compared.
The investigators found that over the 31 months of the study, there were 278 falls (among 178 persons who fell). The rate of falls was indistinguishable in the two groups, but “fall-related injuries were significantly less frequent when they occurred on LIFs (22% of falls versus 34% of falls on control flooring).” And many of the injuries that were averted were serious: “Fractures occurred in 0.7% of falls in the LIF cohort versus 2.3% in the control cohort.”
Thus, the New Zealand study provides a compelling rationale for adding low-impact flooring (along with other modifications) to housing for seniors.
Dentistry has also benefited from costly high-tech innovations such as dental implants, but low-tech prophylaxis can provide much-needed dental health benefits. Tooth decay remains one of the major public health concerns for both developing and developed countries, according to the World Health Organization. It is one of the most common chronic problems in the United States, where most adults will have at least one cavity in their lifetime. The decay causes inflammation in surrounding gum tissue, abscesses, and eventually, tooth loss. In addition to taking a significant toll on the quality of life, decay, and periodontitis have been linked to an increased risk of cardiovascular events, systemic infections such as endocarditis, and complications in pregnancy.
Perhaps the ultimate in low-cost, low-tech interventions is dental flossing, which offers tremendous health benefits. It helps to remove debris and plaque from between teeth and underneath the gums that tooth brushing alone cannot reach. By regularly removing plaque from these areas, flossing can prevent gum disease, tooth decay, and bad breath. This is especially important, given the link between periodontal disease and serious cardiovascular events.
The Centers for Disease Control estimates that approximately 100 million Americans still lack access to fluoridated water. Introducing this low-tech, high-impact measure more widely would substantially decrease the need for many dental procedures.
The high-tech miracles will continue to garner headlines, but to advance public health, simpler and relatively inexpensive innovations are also essential. That has policy implications: We need to put research funding into potential big-ticket, high-tech blockbusters and ingenious, low-tech innovation.
Henry I. Miller, a physician and molecular biologist, is the Glenn Swogger Distinguished Fellow at the American Council on Science and Health. He was the founding director of the Office of Biotechnology at the FDA. Shiv Sharma is a practicing dentist and the owner of Palo Alto Oral Health.
Henry I. Miller, MS, MD
Henry I. Miller, MS, MD, is the Glenn Swogger Distinguished Fellow at the American Council on Science and Health. His research focuses on public policy toward science, technology, and medicine, encompassing a number of areas, including pharmaceutical development, genetic engineering, models for regulatory reform, precision medicine, and the emergence of new viral diseases. Dr. Miller served for fifteen years at the US Food and Drug Administration (FDA) in a number of posts, including as the founding director of the Office of Biotechnology.
