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The American Journal of Managed Care June 2016
Development of a Tethered Personal Health Record Framework for Early End-of-Life Discussions
Seuli Bose-Brill, MD; Matthew Kretovics, MPH; Taylor Ballenger, BS; Gabriella Modan, PhD; Albert Lai, PhD; Lindsay Belanger, MPH; Stephen Koesters, MD; Taylor Pressler-Vydra, MS; and Celia Wills, PhD,
The Value of Decreasing Health Cost Volatility
Marc Herant, PhD, MD, and Alex J. Brown, MEng, MBA
Variations in Patient Response to Tiered Physician Networks
Anna D. Sinaiko, PhD
Primary Care Appointment Availability and Nonphysician Providers One Year After Medicaid Expansion
Renuka Tipirneni, MD, MSc; Karin V. Rhodes, MD, MS; Rodney A. Hayward, MD; Richard L. Lichtenstein, PhD; HwaJung Choi, PhD; Elyse N. Reamer, BS; and Matthew M. Davis, MD, MAPP
Impact of Type 2 Diabetes Medication Cost Sharing on Patient Outcomes and Health Plan Costs
Julia Thornton Snider, PhD; Seth Seabury, PhD; Janice Lopez, PharmD, MPH; Scott McKenzie, MD; Yanyu Wu, PhD; and Dana P. Goldman, PhD
Risk Contracting and Operational Capabilities in Large Medical Groups During National Healthcare Reform
Robert. E. Mechanic, MBA, and Darren Zinner, PhD
The Evolving Role of Subspecialties in Population Health Management and New Healthcare Delivery Models
Dhruv Khullar, MD, MPP; Sandhya K. Rao, MD; Sreekanth K. Chaguturu, MD; and Rahul Rajkumar, MD, JD
When Doctors Go to Business School: Career Choices of Physician-MBAs
Damir Ljuboja, BS, BA; Brian W. Powers, AB; Benjamin Robbins, MD, MBA; Robert Huckman, PhD; Krishna Yeshwant, MD, MBA; and Sachin H. Jain, MD, MBA
Review of Outcomes Associated With Restricted Access to Atypical Antipsychotics
Krithika Rajagopalan, PhD; Mariam Hassan, PhD; Kimberly Boswell, MD; Evelyn Sarnes, PharmD, MPH; Kellie Meyer, PharmD, MPH; and Fred Grossman, MD, PhD
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Value of Improved Lipid Control in Patients at High Risk for Adverse Cardiac Events
Anupam B. Jena, MD, PhD; Daniel M. Blumenthal, MD, MBA; Warren Stevens, PhD; Jacquelyn W. Chou, MPP, MPL; Thanh G.N. Ton, PhD; and Dana P. Goldman, PhD
Adoption of New Agents and Changes in Treatment Patterns for Hepatitis C: 2010-2014
Xiaoxi Yao, PhD; Lindsey R. Sangaralingham, MPH; Joseph S. Ross, MD; Nilay D. Shah, PhD; and Jayant A. Talwalkar, MD

Value of Improved Lipid Control in Patients at High Risk for Adverse Cardiac Events

Anupam B. Jena, MD, PhD; Daniel M. Blumenthal, MD, MBA; Warren Stevens, PhD; Jacquelyn W. Chou, MPP, MPL; Thanh G.N. Ton, PhD; and Dana P. Goldman, PhD
Reducing lipid levels in high-risk patients can significantly reduce disease burden and, depending on final negotiated prices, PCSK9 inhibitors can make an economic contribution to this goal.
We estimated that by 2020, approximately 3.6 million MACEs would be averted if LDL-C levels were reduced by 50% among those treated with standard LLT but who still had LDL-C levels >70 mg/dL (Figure 1). Additionally, we estimated that approximately 1.9 million MACEs would be averted by then if LDL-C levels were reduced by 50% among those treated with standard LLT but who still had LDL-C >100mg/dL. By 2035, these figures were estimated to increase to 14.2 million and 7.5 million, respectively.

We also estimated that by 2020, approximately 400,000 CVD deaths would be averted if LDL-C levels were reduced by 50% among those treated with standard LLT but who still had LDL-C levels >70mg/dL (eAppendix Figure 1). Approximately 230,000 CVD-related deaths would be prevented by 2020 for those with LDL-C >100 mg/dL. These figures increased to 1.6 million and 900,000, respectively, by 2035.

We estimated that by 2035, the cumulative value of averted CVD deaths and MACEs associated with a hypothetical 50% LDL-C reduction would be $2.9 trillion among individuals treated with standard LLT but who still had LDL-C >70mg/dL and $1.6 trillion among individuals with LDL-C >100mg/dL.

Value of Averted MACEs and CVD Deaths Associated With PCSK9 Inhibitor Use in SBGs 1 and 2

We estimated that by 2035, approximately 17 million MACEs (eAppendix Figure 2) and 2 million CVD deaths (eAppendix Figure 3) would be averted under a conservative PCSK9 inhibitor efficacy scenario if all patients in SBGs 1 and 2 treated with standard LLT, but who still had LDL-C >70 mg/dL, used PCSK9 inhibitors. Under the high-efficacy scenario, which assumed a 50% reduction in CVD mortality with PCSK9 inhibitor use, PCSK9 inhibitor use would prevent approximately 19 million MACEs and 3 million CVD-related deaths. In patients treated with standard LLT, but who still had LDL-C >100 mg/dL, 100% uptake of PCSK9 inhibitors would prevent approximately 9 million MACEs and 1 million CVD-related deaths under the conservative-efficacy scenario, as well as 10 million MACEs and 1.5 million CVD-related deaths under the high-efficacy scenario.

For patients in SBGs 1 and 2 treated with standard LLT but who still had LDL-C >70 mg/dL, we estimated the cumulative value of averted deaths and MACEs by 2035 to be $3.4 trillion under the conservative scenario and $5.1 trillion under the high-efficacy scenario (assuming 100% PCSK9 inhibitor uptake) (Figure 2). For patients in SBGs 1 and 2 treated with standard LLT, but who still had LDL-C >100 mg/dL, we projected the cumulative value of averted deaths and MACEs by 2035 to be $1.9 trillion under the conservative-efficacy scenario and $2.7 trillion under the high-efficacy scenario (again assuming 100% PCSK9 inhibitor uptake).

We modeled how varying PCSK9 inhibitor uptake influenced our projections. In our low-uptake scenario (in which a maximum of 10% of eligible patients use PCSK9 inhibitors by 2035), we estimated the value of averted MACEs and CVD-related deaths by 2035 to be $300 billion in the conservative-efficacy PCSK9 inhibitor scenario and $430 billion in the high-efficacy scenario (Figure 3). In contrast, in a high-uptake scenario (in which a maximum of 30% of eligible patients by 2035 use PCSK9 inhibitors), we estimated the value of averted MACEs and CVD-related deaths by 2035 to be $830 billion in the conservative-efficacy PCSK9 scenario and $1.2 trillion in the high-efficacy scenario.

Per-Person Value of Averted MACE and CVD Deaths Associated With PCSK9 Inhibitor Treatment

In addition to estimating MACEs, CVD-related deaths, and the potential value of averted events associated with PCSK9 inhibitor use at the population level, we estimated the value per person-year of treatment with a PCSK9 inhibitor for patients in SBGs 1 and 2 (Table 2). For patients treated with standard LLT but who still had LDL-C >70 mg/dL, we projected the value per person-year of treatment to be $11,600 in the conservative-efficacy scenario and $17,100 in the high-efficacy scenario. At a goal LDL-C of ≤100 mg/dL, the estimated value per person-year of treatment with PCSK9 inhibitors was $12,600 in the conservative-efficacy scenario and $18,000 in the high-efficacy scenario. These estimates illustrate the potential range of social value per eligible patient treated with PCSK9 inhibitors.

DISCUSSION
We estimated the economic value to the United States over the next 20 years of reducing the burden of hyperlipidemia among patients in the highest ACC and AHA SBGs.3 We estimated that reducing LDL-C by 50% in SBG 1 and 2 patients who have been treated with standard LLT but still have LDL-C levels >70 mg/dL could avert approximately 14.2 million MACEs, including 1.6 million CVD-related deaths, by 2035.

Our study complements previous estimates of the economic value of LLT, including statin therapy, in the United States.12 Our estimates of the value of PCSK9 inhibitors are lower than the previously estimated social value of statins of approximately $51,000 per patient (in 2015 dollars).12 One factor that may partly explain this difference is that the growing use of percutaneous coronary intervention, P2Y12 inhibitors, angiotensin-converting enzyme inhibitors, aldosterone antagonists, and implantable cardiac defibrillators has led to dramatic improvements in CVD outcomes since statins were introduced.27-34 It is also possible that new CVD treatments produce smaller absolute benefits compared with those projected for statins, simply because overall morbidity and mortality for CVD were higher 20 years ago than today.

Although PCSK9 inhibitors may lower LDL-C and improve health outcomes for high-risk populations, policy makers and clinicians have raised concerns about the cost of these drugs to the healthcare system.16 The prices per patient-year of treatment were set recently at $14,100 and $14,600 per year for evolocumab and alirocumab, respectively. Some have estimated that systemwide costs for these drugs could be $150 billion annually.35 A critical question for patients and payers will be whether the value of PCSK9 inhibitor benefits outweighs their expense. Our estimates suggest that whether PCSK9 inhibitors deliver net social value (ie, generate benefits in excess of costs) depends on the assumptions about drug efficacy, the economic value of mortality improvements, which patient populations receive PCSK9 inhibitors, and the ultimate prices paid for these drugs. PCSK9 inhibitors would generate net positive social value for the average patients in SBGs 1 and 2, as long as the annual net price falls below $18,000 in a high-efficiency scenario or $12,000 in a conservative-efficiency scenario. According to a recent report, the average reported rebates for branded pharmaceutical drugs are around 30%,36 which would mean an average cost per year for PCSK9 inhibitors of around $10,000. Because our estimates do not include any benefits from increases in health-related quality of life from treatments or events avoided, our estimates may be conservative.

Our findings also add to our understanding of the size of the populations likely to receive PCSK9 inhibitors initially. Several current estimates assume that a majority of the 70 million patients in the 4 ACC/AHA SBGs (only 3 of which were included in this study) will eventually receive PCSK9 inhibitors, which may overestimate the actual societal cost of PCSK9 inhibitors if they are instead primarily prescribed to high-risk patients with poorly controlled LDL-C despite LLT—a small subset of all patients in these 4 SBGs. Our models for patients in the 2 highest SBGs (patients with ASCVD or LDL-C >190 mg/dL) were designed to reflect the reality that clinicians and payers are likely to focus initially on the smaller subset of patients who have up-titrated to maximal statins, but have still failed to achieve LDL-C reduction. For this reason, our projections of the potential benefits and value of PCSK9 inhibitors over the next 3 to 5 years may be more accurate than previous estimates. Nonetheless, even if PCSK9 inhibitors are prescribed only to patients in SBGs 1 and 2 who have been treated with standard LLTs but have not reached the LDL-C goal, some estimates suggest that healthcare spending on LLTs may increase by $10 billion annually over the next 2 decades.15

Limitations

Our study has several limitations. First, our estimated clinical impacts rely on epidemiologic models and evidence from studies of statins of the association among LDL-C levels, MACEs, and CVD-related deaths, which may differ in patients receiving PCSK9 inhibitors. Our estimated impacts assume a linear relationship among the levels of LDL-C reduction and MACEs and CVD-related deaths, which may not be the case at the lower levels of resulting LDL-C simulated in our model. In addition, our analysis of PCSK9 inhibitors used early clinical outcomes data for these drugs; relative risk reductions estimated from larger patient populations may differ from current estimates. Although existing studies suggest that PCSK9 inhibitors are associated with reduced MACEs and deaths, definitive evidence will not be available for several years. Furthermore, even definitive trial evidence may differ from real-world outcomes. Real-world efficacy patterns will be affected by patient treatment heterogeneity and physician decision making.

Second, we estimated the value of PCSK9 inhibitors among patients in the 2 highest SBGs who had been treated but were not at goal LDL-C, assuming that these patients would be the relevant treatment population who had been up-titrated to maximally tolerated doses for statins or other LLTs. In reality, many patients on LLT are not at maximally tolerated doses or are nonadherent with LLT even if they do not experience side effects from these agents.37 Optimizing LLT may reduce the size of the prevalent population who could potentially benefit from PCSK9 inhibitors, since the number of patients failing to achieve LDL-C goals would fall. Our estimates may therefore be an upper bound of the value of PCSK9 inhibitors in these populations because we assumed that LLT rates would remain at their current levels and not rise due to payer pressures. In addition, patients who are nonadherent with LLT may not be candidates for PCSK9 inhibitors even if their LDL-C is >70 mg/dL.

Third, we relied on parameter assumptions and outcomes data to estimate the impacts of LDL-C reduction and PCSK9 inhibitors. The Truven Marketscan database had inconsistent information across variables, particularly with regard to risk factors and cardiovascular events, and lacked information on blood pressure. We therefore may have underestimated cardiovascular events. In addition, the NHANES data used for mortality estimates lacked information on heart failure and transient ischemic attacks, leading us to potentially underestimate overall CVD burden. In our PCSK9 inhibitor analysis, we also assumed that the mean difference in LDL-C between those receiving LLT plus PCKS9 inhibitors versus LLT alone was 59%.13  We did not conduct a separate sensitivity analysis using the bounds of this confidence interval. Rather, we reported a broad range of estimates based on assumptions of conservative versus high efficacy of PCSK9 inhibitors.

 
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