A new study led by researchers at the USC Schaeffer Center for Health Policy & Economics found PCSK9 inhibitors- the newest drugs aimed at dramatically lowering harmful cholesterol- have the potential to offer a significant long-term net value for patients and the healthcare system. Uncertainty surrounding the efficacy and ultimate cost of these drugs has caused trepidation for payers and policymakers as they grapple with balancing patient access and health care spending. In the study published in Value in Health, Wei-Han Cheng, Etienne Gaudette, and Goldman used the Schaeffer Center’s Future Elderly Model (FEM) to evaluate what the long term costs and health outcomes are for individuals and the healthcare system overall from take up of PCSK9 inhibitors.
PCSK9 inhibitors are novel, cholesterol lowering specialty drugs which have been shown to significantly lower LDL-C levels in patients who haven’t responded to statins and lifestyle modifications alone. Though they have the potential to significantly improve cardiovascular disease outcomes, there is still a lack of consensus on whether measured clinical results translate into improved mortality rates.
To estimate the potential long-term health benefits and quantify their value, Cheng and her colleagues modeled two scenarios against the status quo. One scenario predicts the changes in trends using the current FDA eligibility. The alternative scenario expands eligibility to a larger portion of the population. Each of the scenarios is outlined below:
- Under the status quo, the researchers assumed treatment strategies for managing hypercholesterolemia in the United States before the introduction of PCSK9 inhibitors.
- The current eligibility scenario defined eligibility by current FDA-approved indications (those with familial hypercholesterolemia and pre-existing CVD). In addition, patients had to be 80 years of age or younger, currently be on a cholesterol-lowering therapy, and have failed to reduce the LDL-C level to 70 mg/dl or less.
- The expanded eligibility scenario extended access to patients without a history of CVD but with high-risk equivalents who are also eligible for treatment with PCSK9 inhibitors. High-risk equivalents were defined as people with diabetes aged 40 to 75 years or with an estimated 10-year ASCVD risk of more than 7.5 percent. In addition, like in the current eligibility scenario, patients had to be 80 years of age or younger, currently be on a cholesterol-lowering therapy, and have failed to reduce the LDL-C level to 70 mg/dl or less.
Three additional important details to note about their model: First, the researchers accounted for gradual adoption of the drug given current trends. They also assumed a discounted price due to the expected entry of other branded drugs and the patent protection expiration. Furthermore, the researchers conducted their analysis using the efficacy estimates from the meta-analyses of the earlier trials which demonstrated a substantial effect on mortality, before the most recent long-term trial data was available. The results of the more recent studies have added to the uncertainty about drug’s effects on improving mortality over the long-term.
PCSK9 Inhibitors Have the Potential to Significantly Improve Outcomes, Yield Considerable Net Benefits
The researchers found PCSK9 inhibitor use would increase both life expectancy and disability-free life expectancy, improve quality of life, and reduce the overall incidence of heart disease. According to their analysis, under the current FDA guidelines, patients taking PCSK9 inhibitors are predicted to live on average just over a year longer compared with the status quo. Life expectancy improves to 1.9 years longer under the extended eligibility scenario.
In their analysis, Cheng and her colleagues estimate the lifetime net value of PCSK9 inhibitors to be $5,800 per person under the current eligibility scenario (with a 95 percent confidence interval of -$1,100 to $13,700) and $14,100 under the expanded eligibility scenario (with a 95 percent confidence interval of $4,200 to $25,300).
All told, wide access to the drugs could pay enormous health dividends. By 2036, the cumulative net present value of the health benefits for the population aged 51 years and older is projected to be $540 billion under the current FDA eligibility and $900 billion if eligibility is expanded.
The full study is available at Value in Health.
Citation: Cheng, W. H., Gaudette, É., & Goldman, D. P. (2017). PCSK9 Inhibitors Show Value for Patients and the US Health Care System. Value in Health, 20(10), 1270-1278.