Precision Medicine Is the Best Medicine
There is an old cliché about how a 3-out-of-10 success rate will get you fired, unless you are a baseball player, in which case you are headed to the hall of fame. As it turns out, there are a few other areas in which a .333 average could be considered a winning statistic.
Take, for example, the world’s best-selling drug, Humira, which produced $21 billion in sales in 2021 and can cost upwards of $85,000 annually per patient. It is part of a class of newer biologic drugs called tumor necrosis factor inhibitors (TNFi) that are widely prescribed for autoimmune conditions like rheumatoid arthritis or Crohn's disease.
What is less commonly known, however, is that two-thirds of patients do not adequately respond to TNFi treatment.
The case of TNFis, a widely prescribed and therefore wildly successful class of drug, is emblematic of the imbalance between the investment in drug development and the tools that can make those drugs more effective for individual patients and the healthcare system.
Humira was approved by the Food and Drug Administration in 2002, but for most of that drug’s time on the market, physicians have had no means of determining whether a patient would respond to the therapy other than by deploying expensive, frustrating, and potentially harmful try-and-fail periods. It’s not just patients who are feeling the drawbacks either.
Setting aside for a moment the cost, frustration, and potential negative effects on an individual patient’s health, the cost to the American healthcare system of these try-and-fail protocols is astronomical. Billions of dollars have been spent on a medicine that had no chance of success in those patients whose bodies simply did not have the right makeup to respond to the drug.
Thankfully, things are changing.
In the last few years, advances in precision and personalized medicine are permitting physicians, for the first time, to determine whether an individual patient is likely to respond to a therapy before a decision is made about which therapy to prescribe. (Disclosure: the company for which I work as Chief Medical Officer has developed just such a test for patients with rheumatoid arthritis to determine whether they are likely to respond to TNFis.)
The emerging field, called pharmacogenomics, examines patients on an individualized basis to determine that specific patient’s likely response to a given therapy. The field is in its infancy, but it has the potential to dramatically change patient outcomes on an individual level as well as drastically increase the efficiency of health care spending at a systemic level.
That there is a prescription drug pricing crisis in this country is not news. Similar to the 1990s when we had an antibiotic prescribing issue related to acute illnesses that caused more harmful strains of bacteria to spread because patients were not being treated according to the best possible drug for their illness, the same issue is now happening with chronic diseases like RA. It is not newsworthy either that at least some of that crisis is driven by the explosive growth of highly innovative and effective (but extraordinarily expensive) new pharmaceutical and biologic therapies. What is often overlooked in this debate, however, is the toll this all takes on patients.
We, as a society, focus our attention on the drugs and the diseases that they manage well (or, increasingly, cure). But that gives short shrift to the individual patients in whom the drug and the disease converge. Often, that individual patient’s unique biology plays a determinative role in whether the drug has the intended effect, no different than when a patient is infected with a specific strain of bacteria that requires a targeted antibiotic to cure them.
Investment, both in terms of dollars as well as regulatory and legislative reforms that encourage the use of predictive testing by physicians as a tool for making prescribing decisions, is necessary. Given the billions of dollars that are spent each year on drug research and development (and the tax expenditures that incentivize that investment), we should direct significantly more resources to ensuring the products of the research and development are ideally suited to the patients they are intended to heal.
There is an immense opportunity to direct these investments towards pharmacogenomics and ensure that our healthcare system exceeds a 30% success rate. Oncologists have demonstrated the value of such testing in their field in recent years, and rheumatologists and those suffering from rheumatoid arthritis now could benefit as well, especially if approved by a panel for coverage by Medicare, as is being considered right now. By working together, we can help advance these types of tests and improve patient’s livelihoods nationwide and around the globe.
Dr. Sam Asgarian is the chief medical officer for Scipher Medicine, a Massachusetts-based precision immunology company. He previously held the same role with Thrive, an Exact Sciences company focused on early cancer detection.
