As Congress barrels toward passage of the 21st Century Cures Act, it has failed to ask a crucial question: Will it help or harm the nation's drive to derive greater value from its healthcare dollars?
No one opposes innovation in medicine. Half a million people a year are dying from terrible cancers. Millions more are suffering from rare genetic disorders where their only hope lies in more rapid advancement of medical science. Anyone who can speed the wheel of scientific progress has the right to ask for the support of taxpayers and the insurance premium-paying public.
But before we give the drug and device industries the blank check created by an easier pathway to new Food and Drug Administration approvals, shouldn't we have an honest discussion about medical innovation—where it comes from, why it is so difficult, and whether the additional investment spurred by the law represents the best use of society's resources?
Such a discussion starts by understanding why medical innovation advances in fits and starts; why it is growing more expensive; and why recent achievements are almost always incremental in nature.
Medical innovation depends on an in-depth scientific understanding of the causes of a disease, whether genetic, microbial, environmental or the inevitable process of aging. Once that is understood, it requires a molecular understanding of its biological pathways.
That knowledge is usually created by university-based scientists supported by government grants. So the 21st Century Cures Act's boost in National Institutes of Health funding is a wise investment. Only when that scientific understanding is in place can medical interventions with a good chance of success be imagined and developed.
To put it in more concrete terms: If scientists don't know what causes dementia (is beta amyloid plaque build-up a cause or an effect?), how can they discover a drug to treat it except through blind luck?
This process is getting more difficult a century into medicine's scientific revolution. The low-hanging fruit has been picked. Truly life-saving interventions such as antibiotics and insulin, which marked the beginnings of the pharmaceutical and biotechnology eras, are becoming rarer.
Today, a new cancer chemotherapy agent that extends life for six months is considered wildly successful. A drug combination that helps cystic fibrosis patients breathe easier is hailed as a miracle, even though there is still no evidence that it will extend the average life expectancy beyond 37.
This diminishing return on investment in biomedical innovation shouldn't be a surprise. The first “chemotherapy” agent—the antibiotic salvarsan to treat syphilis—was developed by German chemist Paul Ehrlich on the eve of World War I. Today, the FDA has approved more than 1,500 drugs and biologics for virtually every imaginable disease and condition—from glioblastoma to toenail fungus.
Economics teaches us that the progress from more recent therapies will be less than earlier ones since drugs and devices are but a single factor of the production process we call healthcare. The law of diminishing returns states that pouring more investment into a single factor of production over time reduces the marginal return on that investment.
For proof of that concept in healthcare, we need go no further than the longevity statistics. The average lifespan of people who take care of themselves, have a stable social and economic environment, and take advantage of existing therapies is approaching the mid-80s. As Dr. Nortin Hadler, author of a dozen books including Rethinking Aging: Growing Old and Living Well in a Over-Treated Society, told graduates of the University of Michigan Medical School last week, “if you manage to stay well to 85, you're off warranty.”
So let's return to the value proposition posed at the top of this editorial. How do we keep more people healthy into their 80s while keeping healthcare costs under control?
Will the answer come from pouring more of society's scarce resources into developing a new drug to temporarily shrink lung cancer tumors? Such a drug will cost the healthcare system billions of dollars a year at current prices when it comes on the market.
Or should society invest those same billions in the most cost-effective smoking cessation programs for the 20% of the population that still smokes? Ask the same question about better asthma inhalers and pollution control.
It's a choice—one that Congress, ignoring the value proposition, is about to make in favor of new drug development.