The Institute for Clinical and Economic Review (ICER) - an organization that produces reports that analyze the cost effectiveness of drugs and other medical services - has highlighted the improved cost-effectiveness of high cholesterol treatments inclisiran and bempedoic acid in patients who have both established atherosclerotic cardiovascular disease (ASCVD) and either Familial Hypercholesterolemia (FH) or who can't take statins due to side effects, recognizing the increased risk for further cardiovascular events for people with ASCVD and FH. The ICER report also recognized that individuals with FH are at higher risk for cardiovascular events earlier in life and the need for additional treatment options to improve outcomes for higher-risk individuals.
"I have FH and ASCVD. I survived at the age of 39. Contrary to the assumptions made in the report about quality of life after a heart attack, I can attest from personal experience and from the experiences of thousands of men and women who are members of the FH Foundation, that life does not return to its former state. Having a heart attack, bypass surgery, getting multiple stents, or having a stroke; becoming a cardiovascular disease patient in whatever form changes your life.
How you vote today on the key questions presented to you will matter to families across the United States, because we have created a system that is inherently challenging for Americans who are sick or need care to navigate."
– Katherine A. Wilemon
Full Remarks from Katherine Wilemon
On February 5th for ICER’s Virtual Public Meeting to review evidence included in its recent report on therapies for high cholesterol
My name is Katherine Wilemon and I am the Founder and CEO of the FH Foundation, a non-profit research and advocacy organization dedicated to improving the diagnosis and treatment of those born with a metabolic disorder that causes severe hypercholesterolemia and premature cardiovascular disease.
Thank you for the opportunity to comment today on the Report made by ICER on Therapies for High Cholesterol. We are grateful that ICER has recognized the important public health need for additional treatment options to improve outcomes for high-risk individuals.
Today we are wrestling with the question of how to value therapies that can further reduce the LDL-C burden on patients at high risk. Lowering LDL-C is the single most modifiable risk factor for cardiovascular disease. Even now as we live through a pandemic – cardiovascular disease kills and sickens more people than any other disease.
The FH Foundation appreciates ICER’s mission to bring data, analytics, and a consortium of stakeholders to vote on the question of value for novel therapies. This is a complicated question with significant implications for people with familial hypercholesterolemia and established heart disease.
We have witnessed ICER make substantive efforts to improve accuracy of the inputs and the quality of the assumptions in the model. They have a stellar team and we are grateful to have had real opportunity to share data, medical expertise, and the experiences of people living with ASCVD and FH in this most recent report. Nevertheless, the assumptions in the model and the process are still imperfect.
Specifically, I must point out that the assumption that all patients are taking statins and ezetimibe. This is a gross over estimation- for which there is data to support a more realistic estimate.
Another assumption that is misleading is that quality of life goes back to perfect or almost perfect health after a heart attack.
I have FH and ASCVD. I survived at the age of 39. Contrary to the assumptions made in the report about quality of life after a heart attack, I can attest from personal experience and from the experiences of thousands of men and women who are members of the FH Foundation, that life does not return to its former state. Having a heart attack, bypass surgery, getting multiple stents, or having a stroke; becoming a cardiovascular disease patient in whatever form changes your life.
How you vote today on the key questions presented to you will matter to families across the United States, because we have created a system that is inherently challenging for Americans who are sick or need care to navigate.
Let’s face it. In the United States, healthcare incentives are not aligned across stakeholders. The healthcare industry is big business, whether it is payers, pharmaceutical companies, or health systems, but every stakeholder in healthcare has a responsibility that can never be forgotten. We have to do the right thing for the people we serve.
Science has clearly elucidated the role of LDL cholesterol management in preventing cardiovascular disease. The sooner we lower someone’s high cholesterol and the more we lower someone’s cholesterol, the further we take them out of harm’s way. We must recognize that for some people, including those with FH, multiple therapies are required to reach recommended safe levels for LDL-C.
As the report calls out, the FH population is an extremely high-risk population that crosses the threshold of cardiovascular disease on average, 2 decades earlier than the rest of the population.
There are approximately 1.3 million men, women, and children in the US who have familial hypercholesterolemia. Much of the scientific understanding of how LDL-cholesterol drives the disease process of atherosclerosis has come from the study of this population. LDL-C runs on average twice the normal levels and the toxic impact on the vessels and is measurable in the FH population before puberty.
When you consider both the need and uptake of therapies, bear in mind that 9 out of 10 people with FH remain undiagnosed. Because we are undiagnosed, those of us who make it to the other side of the first heart attack become members of the ASCVD population.
This ICER report highlights the evidence that people with both FH and ASCVD are a higher-risk subpopulation for which there is a greater benefit from cholesterol lowering treatment for secondary prevention and recognizes that that the FH population will also benefit from primary prevention.
It would be unforgivable to suggest that people can only have access to safe and effective medications to lower their risk for cardiovascular events after they have had a first cardiovascular event.
That leads me to my final point. This ICER report recognizes that there are people who will benefit from these new treatments, and by extension, from the LDL lowering treatments that are already available but are underutilized. We know that there is a significant unmet need for LDL lowering in high-risk populations. We also know that past utilization management practices intended to limit the use of treatment and manage healthcare dollars have had the unintended consequence of denying, delaying, or making unaffordable treatment to people who would benefit.
FH Foundation research published in 2019 showed that people who were prescribed a PCSK9 inhibitor by their physician, but who did not get treatment because it was denied by insurance or they did not fill the prescription, which were most often Medicare patients who have high out of pocket costs – were significantly more likely to have a cardiac event within 12 months. These are devastating events that could be prevented. And sadly, women, blacks, and Asians, and Hispanics were more likely to be denied by insurers - an inequity that we cannot perpetuate.
I implore the insurance plans, manufacturers and others to find a way to make all cholesterol lowering treatments both affordable and accessible and to work together with patients and healthcare professionals to facilitate better care, rather than create undue burdens that stand in the way of better outcomes.
How you vote today on the key questions presented to you will matter to families across the United States, because we have created a system that is hard for Americans who are sick to navigate. Today you have an opportunity to earn the trust of the American people and invest in prevention of the immense burden of cardiovascular disease.
Founder and CEO
The FH Foundation