Doctors have always known that patients may respond differently to the same therapy. Despite the current pharmacological advances, drugs prescribed today are effective in 60% of patients, ineffective in 30%, and harm 10%. This knowledge was not actionable for most of medical history due to a limited scientific understanding of its nature. Accordingly, drugs were developed and prescribed for the “average patient,” which worked great for blockbuster economics but not always for individual patients. Even when it was massively successful, medicine remained largely imprecise.
The Cost Of Imprecise Medicine, Human And Financial
Traditionally, doctors have dealt with the unpredictability of response to medication via a trial-and-error approach, turning each such treatment into a “clinical trial,” which is often protracted and painful. In most cases, therapies are eventually optimized or abandoned at the cost of precious time, which patients can rarely afford. Take, for example, anti-inflammatory drugs known as tumor necrosis factor inhibitors (TNFi) used for the treatment of rheumatoid arthritis (RA). TNFi drugs gross around $40 billion in revenue annually, including the best-selling drug of the decade, Humira, but they work only in one-third of patients. It can take six to 12 months of trial-and-error treatment to determine whether the patient responds to the TNFi therapy. During this period, nonresponding patients are not receiving alternative medications while the disease progresses, which may lead to permanent joint damage.
Aside from medical implications, prescribing drugs to patients who do not benefit from them has enormous economic consequences. Remember, 40% of patients do not benefit from prescribed medications to the full extent. We estimate that out of more than $300 billion in annual retail prescription drug spending in the US, tens of billions is wasted on ineffective medicines. Just the mega-blockbuster Humira alone, prescribed to 2 million patients in the US and grossing $20 billion annually, has contributed tens of billions to this loss since its introduction in 2002. But this waste pales compared to the estimated annual cost of drug-related morbidity and mortality from nonoptimized medication, which was a whopping $528 billion, equivalent to 16% of US healthcare expenditures in 2016. Such is the cost of imprecise medicine.
The Promise Of Precision Medicine
Advances in genetics, molecular diagnostics, data science, information technologies, and artificial intelligence are expanding doctors’ power to personalize therapies, gradually transforming one-size-fits-all care into precision medicine (PM). At the core of PM is the notion that responsiveness to treatment can be predicted based on the patient’s biology. By leading straight to optimum therapy, PM gives a much clearer prospect for treatment, sparing nonresponding patients the unnecessary side effects and lost opportunity for alternative therapies. And for some patients, the choice between protracted trial-and-error treatment and the rationally predicted outcome may be life or death.
The recent innovative approach to TNFi therapy is the textbook example of PM in action. Based on decades of academic research, Scipher Medicine developed an AI-driven molecular diagnostic platform that identifies patients’ unique proteomic signatures predicting individual responses to TNFi drugs. In 2020, a blood-based molecular diagnostic test, PrismRA, was introduced to determine whether patients would be nonresponsive to TNFi therapy. Under the new PM-guided protocol, patients with a molecular signature of nonresponse can be directed to an alternative treatment from the onset of medical intervention without waiting to fail TNFi first. Two years after PrismRA’s introduction, more than 20% of prescribing US rheumatologists have used this reimbursable test, resulting in improved patient outcomes and lower healthcare costs.
Precision Medicine Is An Interdisciplinary Endeavor
Like most PM initiatives, the diagnostic test for TNFi therapy is a multidisciplinary project bringing together the resources of different industries and public institutions. It is reflective of PM in general, where success will come only through the collective efforts of multiple contributors. We identified nine stakeholders representing an incredibly diverse PM ecosystem: healthcare providers, life sciences, the pharmaceutical industry, diagnostic companies, informatics, regulators, patients, health insurers, and policymakers. Providers bear ultimate responsibility for PM implementation, but they can’t do it alone. To realize PM’s potential and business benefits, providers should not view their relationships with other stakeholders as purely transactional; they must proactively engage in technical and organizational collaboration and knowledge sharing throughout the entire PM ecosystem.
Hospitals, healthcare systems, and individual doctors must engage with:
- Life sciences to lay the foundation for PM practice and contribute to PM research.
- Pharmaceutical companies as providers’ natural allies in PM science and business.
- Diagnostic companies to keep physicians abreast of the overwhelming number of tests and to assure validity of test data.
- Informatics to operationalize PM by processing vast amounts of data from multiple sources.
- Regulators to influence the formation of the PM regulatory infrastructure.
- Patients to be educated on PM and encouraged to share personal data for PM development.
- Insurers to pursue PM reimbursement.
- Policymakers to continue promoting PM at the highest national legislative level.
Stay tuned for the upcoming report, Precision Medicine Is An Imperative In Healthcare, But Providers Can’t Do It Alone, in which we describe a provider-centric PM ecosystem and guide healthcare organizations in their collaboration with other PM stakeholders. If you are looking to learn more about trends in precision medicine, schedule a call with us. We would love to talk with you!