The concept of the Triple Aim—improving the experience of care, reducing the costs of care, and improving the health of populations—is increasingly becoming important to drug manufacturers, who must demonstrate how their products acheive these aims in order to ensure placement on clinical pathways.
Since Berwick et al first introduced the Triple Aim concept in 2008, its impact continues to reverberate throughout the health care system in several ways. Notwithstanding its key role in the development of the Affordable Care Act legislation, many clinical pathway developers are attempting to make progress on all three aims: to improve the experience of care, to reduce the costs of care, and to improve the health of populations.1 Certainly, the pharmaceutical industry was never outside the scope of the Triple Aim; however, as health systems and payers make significant changes to their business models to align with quality, performance, and value goals, manufacturers are compelled, more than ever, to demonstrate how they can fit into clinical pathways aimed firmly at achieving these goals.
The Evolution of Clinical Pathways for Oncology
The Place for Biosimilars in Clinical Pathways
IMPROVING THE INDIVIDUAL PATIENT EXPERIENCE
One overarching approach in achieving the Triple Aim goal of improving the patient experience has been described as getting the right treatment to the right patient at the right time, or, more succinctly, personalized medicine. While clinical assessments, approaches, and pathways have been tailored for various patient populations, pharmaceutical treatments have made great strides in personalizing treatment by identifying very specific conditions and matching them with equally specific drugs through companion diagnostics.
Companion diagnostics were first launched in the 1980s with Herceptin (trastuzumab; Genentech) and Gleevec (imatinib; Novartis), both of which required companion diagnostic testing prior to prescribing. This helped to establish the therapeutic approach in oncology, one that has recently expanded into other therapeutic areas such as cystic fibrosis, human immunodeficiency virus (HIV), and severe growth failure.2
Evidence of the effectiveness of combining diagnostics with specific medications has spurred consistent growth in this area. For example, a recent study showed that a targeted blood test could predict high-risk cancers in patients treated with chimeric antigen receptor–modified T cells (CAR-T). Researchers at the University of Pennsylvania (Philadelphia) identified two biomarkers for CAR-T therapy in patients with acute lymphoblastic leukemia that could result in cytokine-release syndrome, which can be fatal if not treated. Lead investigator David T Teachy, MD, stated: “If we check certain cytokines in the blood in the first 48 hours after infusion of CAR-T before patients develop full-blown [cytokine-release syndrome]…we have a high likelihood of predicting who will become critically ill and who won’t.”3
From a financial perspective, medications that target small and defined populations using drug–diagnostic combinations are known as “niche busters,” commanding high prices with corresponding hefty revenues for manufacturers. For example, Herceptin and Gleevec, even with reduced but targeted patient populations, had global annual sales in 2012 of $6.5 billion and $4.7 billion, respectively. Some believe that Kalydeco (ivacaftor; Vertex Pharmaceuticals) for cystic fibrosis could be a niche buster, with projected annual sales of more than $2 billion in 2018. And this is just the beginning, with ~100 drugs in development with diagnostic information being included as part of the primary and secondary outcome measures and inclusion/exclusion eligibility criteria.2
Although the outlook for companion diagnostics seems bright, challenges quickly surface when we examine the conflicting interests of stakeholders. For example, pharmaceutical companies generally are not interested in developing companion diagnostics and would rather a diagnostic company make the investment to develop tests. However, diagnostic companies may not be very interested in developing a product that is used once per patient in a small population. The manufacturer, on the other hand, can gain a patient for life as a result of one test.
As the players circle each other in this amorphous market, manufacturers will probably make the first moves, as they have the most to gain. The ways in which drug developers will be involved revolve around a few different scenarios. One is that they will develop diagnostic capabilities internally; a second is that they will partner with a diagnostic company; and a third is that they will purchase an existing diagnostic company. Versions of these scenarios are already taking shape; it will be interesting to watch as this part of the market matures to better address the patient experience.
TOTAL COST OF CARE
A second emphasis of the Triple Aim is to lower costs of care. Prescription drugs are a typical target to reduce costs, with reports in the media of extremely high profit margins and sharp price increases underlying the high costs of specialty drugs for cancer and hepatitis C.4 Although pharmaceutical manufacturers are a long-time target for reducing the costs of prescription drugs, it is important to note that retail drugs still only represent ~10% of the total health care spend.5 In addition, a long-standing argument supporting the value of high-cost prescription drugs is that pharmaceutical treatments prevent many patients from needing more expensive care with both inpatient and outpatient services.
In any case, our health care system is placing tremendous pressure on drug manufacturers to demonstrate how their products provide value to the treatment equation. Sometimes providing value while reducing cost can mean the use of a generic drug, or the anticipated biosimilars, as alternatives to brands. However, careful consideration must be made: with just about any product, cheaper is not always better, and sometimes it is better to look at value over price. For example, although warfarin is a popular anticoagulant, it requires monitoring and may not be tolerated well by some patients. In this case, the costs associated with monitoring and follow-up care may exceed the costs of an alternate branded drug that is better tolerated by the patient and does not require monitoring. We anticipate the same argument may be presented with the onset of biosimilars, meaning that the costs of managing possible adverse events may surpass the costs of the branded reference drug.
An example of selecting value over price can be seen in relation to a bundled payment program. Suppose that research with a branded pain medication showed excellent patient satisfaction scores in post-acute care for joint replacements compared with cheaper generic alternatives. Normally, the decision to use a generic option would be fairly straightforward. However, within the new CMS Comprehensive Joint Replacement bundled payment program, one survey measure asks the patient to rate how the hospital staff helped in managing pain. While seemingly insignificant, this item is a part of a quality measure with a 40% weighting of a composite score tied to payment.6 This makes the better-tolerated branded pain medication possibly a better value if it is tied to higher patient satisfaction scores than the generic option.
There is one other unique scenario in which the value of prescription drugs can be demonstrated without cost being a factor: Medicare accountable care organizations (ACOs). The reason for this is that Medicare ACOs are not responsible for prescription drugs, because they are handled strictly through the Medicare Part D program.7 This means that, as each ACO establishes its benchmark cost and quality measures, the costs of prescription drugs are not included in the calculations. This allows the decisions of what drugs to use to be based more on value than on cost.
Almost as a direct response to the third component of the Triple Aim—improving the health of populations—the term “population health” is becoming more popular in the US health care system. With its origins in Canada, the term has a tendency to have varying definitions both in the United States and in Canada. However, if one definition appears to be most in favor, it is the Kindig and Stoddart definition: “the health outcomes of a group of individuals, including the distribution of such outcomes within the group.”8 In other words, population health is a broader perspective than “disease management,” encompassing wellness and preventative issues as well as delivery systems, both public and private, that support and provide health-related services.
For pharmaceutical manufacturers, entry into the population health space means even more pressure to reduce costs and articulate value to payers in terms of quality. Even more challenging is that different payers have different perspectives. For example, traditional payers are firmly focused on costs, whereas integrated delivery networks (IDNs) are focused more on the Triple Aim. In addition, population health also promotes the concept of quality-adjusted life years (QALYs), emphasizing a balance of cost and quality of care. Unfortunately, even in this environment, IDNs tend to focus first on costs, second on quality measures tied to payments, and a distant third on the QALYs and other population health measures.
It is no longer sufficient to focus just on efficacy, safety, outcomes, and cost when determining the placement of prescription drugs within clinical pathways. Some of the more forward-thinking pharmaceutical manufacturers are incorporating elements of the Triple Aim into their communication platforms. In fact, some drug companies are making the Triple Aim the platform for their work with customers in the managed markets space, including IDNs, patient-centered medical homes, ACOs, health plans, pharmacy benefit managers, and the Veterans Administration/Department of Defense. With that said, the manufacturer’s value propositions can no longer be a one-size-fits-all approach. Incorporating the Triple Aim and customizing it for different models of care are becoming the new normal as these factors become increasingly critical for placement in clinical pathways.
1. Berwick DM, Nolan TW, Whittington J. The triple aim: care, health, and cost. Health Aff (Millwood). 2008;27(3):759-769.
2. Agarwal A, Ressler D, Snyder G. The current and future state of companion diagnostics. Pharmgenomics Pers Med. 2015;8:99-110.
3. Goodman A. Blood test can predict high-risk cancers in patients treated with CAR-T. The ASCO Post. http://www.ascopost.com/issues/january-25-2016/blood-test-can-predict-high-risk-cancers-in-patients-treated-with-car-t/. Published January 25, 2016. Accessed March 31, 2016.
4. Cox C, Kamal R, Jankiewicz A, Rousseau D; for the Kaiser Family Foundation. Recent trends in prescription drug costs. JAMA. 2016;315(13):1326.
5. Centers for Medicare & Medicaid Services. Nation’s health dollar – where it came from, where it went. CMS web site. https://www.cms.gov/Research-Statistics-Data-and-Systems/Statistics-Trends-and-Reports/NationalHealthExpendData/Downloads/PieChartSourcesExpenditures2014.pdf. Updated December 3, 2015. Accessed April 2, 2016.
6. The Center for Medicare and Medicaid Innovation. CMMI Comprehensive Care for Joint Replacement Model: Quality Measures, Voluntary Data, Public Reporting Processes for Preview Reports. The CMS Innovation Center web site. Accessed March 11, 2016.
7. Centers for Medicare & Medicaid Services. Shared Savings Program. CMS web site. https://www.cms.gov/Medicare/Medicare-Fee-for-Service-Payment/sharedsavingsprogram/index.html?redirect=/sharedsavingsprogram/. Updated December 2, 2015. Accessed December 2, 2015.
8. Kindig D, Stoddart G. What is population health? Am J Public Health. 2003;93(3):380-383.