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12 Days of Misgivings About the Institute for Clinical and Economic Review’s Recent Draft Report on Alzheimer’s Disease

January 11, 2023   |   Sue Peschin   |   Alzheimer's Disease

The following 12 Days of Misgivings were originally posted to Sue Peschin’s LinkedIn.

Day 1

Starting today, I will present our “12 Days of Misgivings” about the Institute for Clinical and Economic Review (ICER) and its draft report on the economic value of new therapies for the treatment of mild cognitive impairment (MCI) and early-stage dementia due to Alzheimer’s disease (AD). Before anyone gets ahead of themselves, the Alliance for Aging Research does NOT endorse any medical products or take positions on whether the FDA should approve products in clinical development.

For those who have never heard of it, ICER is a non-governmental organization that issues cost-effectiveness reports on new medical treatments. Health insurance companies use ICER reports to help decide whether to cover the new treatments—and when a company offers coverage, whether certain access barriers ought to be put in place to control its overall costs. 

That all said, my first misgiving is this: Did you know traditional cost effectiveness assigns a value–called a “health state preference value”–to the group of patients a treatment is intended for? This is used as part of a calculation called the quality-adjusted life year (QALY), which I’ll talk about more tomorrow.

For now, check out the chart below (from the Value Our Health campaign) to see what I mean. Health state preference values are based on the perceived value of living with a given condition in comparison to being in “perfect health”—and you can see here these preference values assign a value to a human’s life on a scale between 0, which is dead; and 1, perfect health. As you can see here, it is possible to rate a health state as worse than death. Older age also ranks less than 1. 

The actual impact of this is really big. Because, if a treatment is for a group that is sicker, older, or includes people with disabilities, or is focused on a population that has historically faced inequities in society and in the health care system, the treatment is assessed as being less valuable–or another way to put it is that those groups are considered more expensive to treat. Imagine if a car dealer priced their car differently because you had a bum knee or were older and wouldn’t be driving it as long?  

Kinda’ weird, right? Tomorrow we’ll dig in more to see what this means for how ICER calculates the value of treatments for people with Alzheimer’s.

Day 2

Welcome to day 2 of our “12 Days of Misgivings” about the Institute for Clinical and Economic Review (ICER) and its draft report on the economic value of new therapies for the treatment of mild cognitive impairment (MCI) and early-stage dementia due to Alzheimer’s disease (AD).

Today’s misgiving: Why are quality-adjusted life years (QALYs) used by ICER? The QALY is a health economics measure, which (theoretically) represents the degree to which a treatment extends life and improves quality of life. 

Yesterday, I wrote about how QALY assessments assign a “health state preference value” to patients a treatment is intended for. The QALY is calculated by multiplying this value with the time the patient is likely to spend in that disease state.

As an example, if a 30-year-old and a 60-year-old receive identical treatments for a fatal disease, which result in a 100 percent cure and expectation of a lifespan to 80 years old, it is 2.5x more cost-effective to treat the younger patient than the older one. By definition, QALYs undervalue treatments for populations that have fewer expected years of life left or shorter average life spans than the overall population. The National Council on Disability, an independent federal agency, describes the discriminatory impact of QALY-based cost-effectiveness analysis and alternatives in its Nov 2022 brief.

I used the word “theoretically” above because health state preference values reflect the views/prejudices of the people who develop/rate them, e.g., people without the disease may be doing the rating. So, when someone claims a treatment is worth X amount per QALY, now you know it is likely discriminatory and arbitrary.

When QALYs are applied to value assessments for AD treatments, it presents a moral quandary due to older age and worse health status. Of the 6.2 million Americans ages 65+ w/AD, one-third are over age 75 and another one-third are over age 85, meaning Alzheimer’s treatments would be expected to receive lower QALY scores. Pile on co-morbid conditions such as diabetes, coronary artery disease, and heart failure and the preference value goes down further. QALYs also do not value symptomatic treatments that may only improve quality of life but not length of life. And to add insult to injury, QALYs also generally don’t recognize the significant burden of Alzheimer’s disease and other types of dementia on family caregivers and society, which means they also don’t recognize the value of treatments that alleviate these burdens.

If you want to nerd-out more, we have 2 reports: 1) one from 2021 with Milliman and 2) the Alliance for Aging Research‘s latest.

Day 3

Welcome to day 3 of our “12 Days of Misgivings” about the Institute for Clinical and Economic Review (ICER) and its draft report on the economic value of new therapies for the treatment of mild cognitive impairment (MCI) and early-stage dementia due to Alzheimer’s disease (AD).

Today’s misgiving is the last of my 3 installments on traditional health economics: Where do QALYs come from?

Use of QALYs was promoted more than 50 years ago when the UK was searching for ways to ration organ transplants and other emerging health care technologies. In 1999, Britain set up its National Institute for Clinical Excellence (in 2005, they changed their name to NICE – National Institute for Health and Care Excellence). NICE’s role is to perform QALY-based cost-effectiveness assessments for new medical treatments/tests and then develop guidelines for the National Health Service (NHS). The NHS is the UK’s public health insurer, and they use NICE’s recommendations to ration health care products and services among its citizens.

In 2005, NICE proposed banning cholinesterase inhibitor drugs (e.g., Aricept) for NHS patients living with AD on the grounds that their cost was too high and ‘‘outside the range of cost effectiveness that might be considered appropriate for the NHS.” The recommendation to ban the AD drugs was despite NICE’s admission that they were effective treatments, and despite NICE having approved far more expensive treatments for other diseases.

At the time, there were no other available therapies to treat AD. So, it wasn’t the drugs that were judged to be not cost effective when compared to rival treatments–it was the people living with AD who were being condemned as not cost effective to society. Why were AD patients considered not cost effective to treat? While not stated outright, the answer was NICE had determined that people living with AD were simply not worth helping.

When it comes to its own assessments of AD treatments, ICER is a fan of referencing NICE guidelines. So, now you know where I am going with this.

I’d prefer ICER choose to be kind, rather than NICE, wouldn’t you?

Day 4

Welcome to day 4 of our “12 Days of Misgivings” about the Institute for Clinical and Economic Review (ICER) and its draft report on the economic value of new therapies for the treatment of mild cognitive impairment (MCI) and early-stage dementia due to Alzheimer’s disease (AD).

Today’s misgiving is: Why doesn’t ICER ask people living with AD, family caregivers, and patient advocacy organizations for their opinions about the treatments under review?

ICER states in the draft review that they interviewed 13 people living with AD and 5 family caregivers. They asked the volunteers about their challenges with being diagnosed, coping and adjusting to a new way of living, impact on caregiver quality of life, general treatment concerns and hopes, and health disparities in clinical care and research. Basically, it was a general discussion about the experience of living with AD across a spectrum of 18 people.

Nowhere–in either the report section on patient and caregiver perspectives, or the methods supplement–does ICER state that their staff asked people living with AD and family caregivers (or advocacy groups) about the specific treatments under this review. Nothing was asked about whether the clinical results would be considered meaningful from their perspectives. No questions were raised about the benefit/risk of these therapies, including:
·       Views on each treatment’s effectiveness at delaying the progression of disease from MCI/mild to moderate stage. What would a delay like that mean to patients and family caregivers?
·       If the treatments stop working at the moderate stage of AD, would that change perspectives?
·       What were their thought about the assumptions ICER was making in their model?
·       What kinds of risk, and how much risk, would they be willing to tolerate with these therapies? What do they think about the risk of ARIA?
·       What do they think about ICER’s contextual contention that AD has a “moderate lifetime impact” on patients with the disease since it is not a lifelong condition? Do they agree with that assumption?

None of those types of questions were asked, yet the answers would surely impact a patient-centered estimate of treatment value. Do the folks at ICER aim to be patient-centered? If so, they seem to be missing the mark.

Day 5

Welcome to day 5 of our “12 Days of Misgivings” about the Institute for Clinical and Economic Review (ICER) and its draft report on the economic value of new therapies for the treatment of mild cognitive impairment (MCI) and early-stage dementia due to Alzheimer’s disease (AD).

Today’s misgiving is: Why does ICER bother with a “modified societal perspective” as a “co-base-case analysis” when it 1) ends up being dismissed, and 2) insults family caregivers?

First, let’s get the lingo down. ICER’s main conclusions come from its “base case”, which is the health care sector perspective–i.e., the traditional, QALY-based, cost-effectiveness analysis that is limited to the direct medical care costs/health outcomes of the patient (check out Days 1-3 of Misgivings for more info).

However, the total costs for AD care exist disproportionately beyond medical costs, with most of the burden falling on families. Available public health data show Alzheimer’s has extensive effects on both the patients and their family caregivers, including unintended job loss, reduction in income, behavioral and psychiatric consequences, and much more.

ICER relegates the costs of family caregiver health or work loss related to caring for loved ones to its subjective “modified societal perspective” as a “co-base-case analysis.” ICER states that the rationale for this additional analysis is due to “the large impact of AD on caregivers,” which makes it seem as though it would better account for the caregiver perspective–but nope.

Instead, ICER’s modified societal perspective PENALIZES the caregiver for the productivity and economic impacts of keeping a loved one at home, as the report bluntly states:
“In addition, keeping a patient in earlier AD states longer, which delays the transition to long-term care, can increase productivity losses for the caregiver. These countervailing factors reduce the spread between the cost-effectiveness results using the health care system and modified societal perspectives. This highlights the complexities of capturing caregiver perspectives in the modified societal perspective in that caregivers may prefer to keep loved ones at home, rather than in a long-term care facility, although doing so may increase the negative financial impact on the caregiver.”

From a patient, family caregiver, and societal perspective, there is significant value to prolonging independence and identity that is not reflected in medical costs or solely captured in caregiving burden. These factors provide immense intrinsic value to patients and their families that outweighs opportunity costs lost elsewhere. If this value is not reflected in the value assessment, that is a shortcoming of ICER’s model in accurately capturing and incorporating value, not of patients and caregivers in valuing non-monetary outcomes.

Day 6

Today’s misgiving is a general one about ICER’s process:

Why does ICER ask patient advocacy organizations to serve as stakeholders and give substantial time to reviews, only to be confrontational toward them at their public meetings?

Let’s back up a bit. With each treatment review, ICER hosts a public meeting at one of its three appraisal committees (in CA, the Midwest, and New England) to deliberate and vote on questions about the “net health benefit” of the treatments under review, as well as “contextual considerations” (although, these don’t count in the final analysis). One or more of the patient advocacy organizations that served as voluntary stakeholders throughout ICER’s time-consuming review process are chosen to provide public comments, which must be submitted to ICER ahead of the meeting.

Here are two questions to consider:

1) Why doesn’t ICER share its questions with patient advocacy organizations (and other speakers) ahead of time? ICER receives advance copies of all speaker comments, so they certainly have time to craft questions and share them. Instead, patient advocacy group representatives are often confronted by ICER President Steve Pearson at the live meeting with what he himself calls “tough questions.” Doesn’t that sound a little imbalanced?

2) Why does ICER have a different standard for industry funding to patient advocacy groups than they do for themselves? ICER receives substantial annual sponsorship funding from pharmaceutical, biomedical, and insurance companies; a large percentage of general operating support from an LLC (not the same as a foundation, BTW, but it is lumped in with foundations); some restricted funding from foundations; funding to assist the Veteran’s Health Administration with the VA formulary; as well as a few other supporters. At the start of its public meetings, Dr. Pearson asserts ICER’s “independence,” but does not afford patient advocacy groups the same courtesy. That also seems imbalanced, doesn’t it?

We think patient advocacy organizations should be more outspoken about these misgivings at ICER public meetings moving forward, starting with the one for Alzheimer’s treatments on March 17, 2023. Let’s be grateful for support from funders and recognize that we are ALL capable of independent thought. Go forth, friends!

In the next few days, we will discuss our misgivings about ICER inconsistencies on racial and ethnic equity issues; unusual framing of treatment harms; and a short list of what we consider ICER’s top offbeat model assumptions.

Day 7

It’s day 7 of our “12 Days of Misgivings” about the Institute for Clinical and Economic Review (ICER) and its draft report on the economic value of new therapies for the treatment of mild cognitive impairment (MCI) and early-stage dementia due to Alzheimer’s disease (AD).

Today’s misgiving pivots back to ICER methodology:
Why does ICER knowingly perpetuate a racist health economics model for use by payers?

Alzheimer’s disproportionately affects people of color, yet health disparities in Alzheimer’s treatment, research, and value assessment remain unaddressed. According to the Alzheimer’s Association®, Black Americans are approximately two times more likely, and Hispanic Americans are 1.5 times more likely to develop Alzheimer’s than non-Hispanic white people.

Overall, health economic models that rely on QALYs distort our ethical instincts by placing a lower value on people’s lives due to having less “life years” or “perfect health” ahead of them. This is a particular issue for underrepresented minority groups, which experience a higher prevalence of AD, are often diagnosed at later stages, and with more comorbidities–all factors that force the cost-effectiveness threshold for treatment to rise. QALYs also seek to generalize the population, which sounds like it would have an equalizing effect, but populations that experience social disadvantage are routinely considered more expensive to treat.

ICER has openly acknowledged in the “contextual considerations” sections of previous evidence reports that its traditional cost-effectiveness model cannot capture the impact of systemic issues such as racism on underserved populations. However, since “contextual considerations” don’t count in ICER’s final recommendations, it serves as a convenient cop-out on wider treatment access implications.

The “contextual considerations” in the ICER AD evidence report include a newfangled “Health Improvement Distribution Index.” ICER estimates that Black Americans would receive 1.6 times the health improvements from treatment as compared to the same sized group of Americans without regard to race–except, again, it’s contextual so it doesn’t count. What is the point of this exercise?

For better solutions, ICER should look to the new @PIPCpatients report, “Aligning Health Technology Assessment with Efforts to Advance Health Equity”. The report was co-authored with @GlobalLiver, @NMQF, and @ptechealth and it provides recommendations to assist ​organizations, health systems, payers, and policymakers that want to center their value assessment work on health equity. If you are not already doing so, please follow these fabulous, expert groups on social and check out their websites.

ICER, we know you can prioritize health equity and change the way value assessment is done!

Day 8

Thanks to everyone who has been tuning in! It’s day 8 of our “12 Days of Misgivings” about the Institute for Clinical and Economic Review (ICER) and its draft report on the economic value of new therapies for the treatment of mild cognitive impairment (MCI) and early-stage dementia due to Alzheimer’s disease (AD).

Today’s misgiving is:
What is the purpose of ICER’s “Potential Budget Impact” calculation and why should we question it?

Each ICER report includes a section that projects the impact that coverage of a new drug might have on an insurance company’s annual bottom line. ICER calculates this “Projected Budget Impact” by estimating the percent of patients who would get access to the treatment under review, at 3 different price point levels, before the percent hits a certain threshold. Not surprisingly, ICER generally concludes that its lowest recommended price point would cover the most patients.

For example, ICER’s AD report concludes that 100% of patients with MCI and early dementia due to AD would be able to be treated with the therapies under review if the drugs were priced at ICER’s lowest recommended price. If they are not priced at the lowest price, then insurance companies may need to identify strategies to limit access. Essentially, ICER’s budget impact calculation serves as justification for payers to ration coverage of new therapies for patients. It is meant to help payers, not patients.

And if you think ICER’s budget impact math seems unclear, you are not alone. In 2019 actuary experts at Milliman (who are a lot smarter than us) outlined several “disconnects” in ICER’s budget impact work, calling it “highly variable.” Their report notes that “ICER’s national-level analysis ignores many of the financial, operational, and clinical realities of today’s healthcare system.”

See you tomorrow for Day 9!

Day 9

It’s day 9 of our “12 Days of Misgivings” about the Institute for Clinical and Economic Review (ICER) and its draft report on the economic value of new therapies for the treatment of mild cognitive impairment (MCI) and early-stage dementia due to Alzheimer’s disease (AD). We’ve got a 1990s throwback vibe going on this evening, so please read to the end for a special treat!

Today’s misgiving is:
Does ICER believe they know better than the medical experts at the U.S. Food and Drug Administration (FDA) about biomedicine, clinical development, and regulatory review?

ICER draft AD report includes a section on “Comparative Clinical Effectiveness” that compares the treatments under review in addition to supportive care, versus supportive care alone. This is a term-paper-like, 20-page section that reviews separate studies on each treatment (i.e., making them not directly comparable).

In its review, ICER challenges the FDA’s congressionally authorized Accelerated Approval (“AA”) Program. The use of AA has been an important regulatory mechanism for the FDA to allow for earlier approval of drugs that treat serious and life-threatening illnesses than would occur through the traditional approval program. Under the accelerated route, a drug can win earlier approval if it shows an effect on a “surrogate endpoint”—in this case, amyloid plaque formation in the brain—that is reasonably likely to predict a clinical benefit to patients. Created in 1992, the AA Pathway was conceived as a direct response to patient therapy during the HIV/AIDS epidemic and in recognition of the urgency of access to new therapy needs facing patients with life-threatening illnesses.

By ICER’s analysis, most of the new drugs approved each year for rare diseases and cancer should not be covered by health insurance. ICER is not only directly challenging the FDA’s statutory authority it is missing the point of AA for people living with deadly diseases that have no other options. Plus, as we covered a few days ago, ICER doesn’t solicit patient preference data on the specific treatments under review, so how would they know what people living with AD would choose for themselves?

This is unacceptable. ICER is not intellectually or experientially equipped to be the arbiter of the safety and effectiveness of medical treatments or of who should be able to access them. It is reckless for them to even hint otherwise.

Seriously ICER, who do you think you are?

Day 10

It’s day 10 of our “12 Days of Misgivings” about the Institute for Clinical and Economic Review (ICER) and its draft report on the economic value of new therapies for the treatment of mild cognitive impairment (MCI) and early-stage dementia due to Alzheimer’s disease (AD).

Today’s misgiving has us questioning the overfocus on amyloid-related imaging abnormalities (ARIA) by ICER and others, and the unbalanced way this side-effect of the anti-amyloid therapies has been framed.

According to the Alzheimer’s Association®’s website, ARIA is “a common side effect that does not usually cause symptoms but can be serious. It is typically a temporary swelling in areas of the brain that usually resolves over time. Some people may also have small spots of bleeding in or on the surface of the brain with the swelling, although most people with swelling in areas of the brain do not have symptoms. Some may have symptoms of ARIA such as headache, dizziness, nausea, confusion and vision changes.”

There are risks with every single drug. Individuals faced with life-threatening conditions sit down with their health care providers, often alongside family members, to discuss the risks and benefits of whether to take a drug or not. It is not the job of ICER, private payers, or Medicare to determine individual risk or to exploit it in the narrow and flawed methodological context of value assessment, or the equivocal application of “reasonable and necessary.”

Alzheimer’s disease is an irreversible, degenerative brain condition that results in death—either directly from the disease itself, or from a related infection or other complication. Alzheimer’s first destroys memory and thinking skills, progresses to an inability to carry out even the simplest of daily tasks such as washing oneself or going to the bathroom. One or more mood and emotional changes, such as depression, anxiety, agitation, and psychosis—commonly co-occur in people with Alzheimer’s. In late-stage Alzheimer’s, people living with the disease can no longer walk, eat, talk, or swallow.

When deaths in a clinical trial are attributed to the study therapy before evidence is fully reviewed, with little to no mention of the therapy’s benefits to delaying disease progression, and the fatal risk of the disease itself is glossed over—that is imbalanced. When senior officials at Medicare characterize people in the early stages of Alzheimer’s as “healthy” (e.g., see CMS stakeholder call transcript from 4-11-2022), and the same agency applies “coverage with evidence development” (CED) as extreme utilization management to the entire class of anti-amyloid therapies—that is grossly irresponsible and goes beyond the pale. It seems clear there is a political preference to allow people with Alzheimer’s to die from their disease rather than have an option for treatment, all in favor of the almighty dollar.

That’s not okay with us. We’re going to fight it side-by-side with partner organizations and friends.

Day 11

It’s day 11 of our “12 Days of Misgivings” about the Institute for Clinical and Economic Review (ICER) and its draft report on the economic value of new therapies for the treatment of mild cognitive impairment (MCI) and early-stage dementia due to Alzheimer’s disease (AD).

Today’s misgiving is:
Who are ICER’s customers?

This series has gone over a number of problematic issues with ICER’s discriminatory methodology, perfunctory patient and family caregiver stakeholder engagement, unbalanced focus on treatment harms, and out-of-touch budget impact. We are not alone in making these criticisms, yet ICER seems perpetually unperturbed and unmotivated to change. Why?

The reason is because patients are NOT ICER’s customers. PAYERS are ICER’s customers and assessing value more accurately could negatively affect them. As a result, ICER has absolutely no incentive to mess with the status quo by fixing anything we’ve identified as problematic over the last 11 days.

While other independent groups have developed value frameworks to help with shared decision making or inform coverage and payment for insurers, ICER stands alone in what they do for health insurance companies. ICER’s value framework “includes a measure of affordability for new drugs within the conceptual core of an assessment of value to the health system.” Because ICER’s framework includes both cost-effectiveness and budget impact analyses, the conclusions will favor the financial interest of insurers.

For example, a new treatment could be calculated to be cost-effective for a patient, but if a large number of patients are likely to be prescribed the medication the budget impact will be larger and ICER will conclude that the expense will not be “affordable.” This is relevant to Alzheimer’s, since more than 6 million Americans are living with the disease. Even when a treatment targets a segment of a patient population, as with anti-amyloid therapies, if there remains a large enough volume of patients to hit ICER’s “budget threshold,” report recommendations will encourage strategies to ration care for the new therapy.

Patients need to understand the lens through which ICER views them, which is essentially as expensive-to-maintain products. ICER’s mandate is to reduce their customers’ product costs as much as possible.

In ICER’s analyses, payers almost always win because the customer is always right. 

Day 12

It’s day 12 of our “12 Days of Misgivings” about the Institute for Clinical and Economic Review (ICER) and its draft report on the economic value of new therapies for the treatment of mild cognitive impairment (MCI) and early-stage dementia due to Alzheimer’s disease (AD). Thanks to all who joined us for the “nerdy dozen”!

Today, the Alliance for Aging Research celebrates the FDA’s approval of a promising therapeutic that has illustrated the ability to help slow the progression of AD! The approval for lecanemab, a new treatment for people living with MCI and early dementia due to AD – an irreversible, degenerative, and fatal brain disease – builds on the promising data published in the NEJM and validated by the views of over 140 physicians who recently sent a letter supporting the science. Read our full statement here.

Today’s misgiving is:
What will happen with coverage of the new AD therapy, and what is ICER’s role?

For years, our nation’s Medicare program has gotten away with paying only a fraction of the costs for this fatal neurological disease that impacts more than 6 million Americans. Currently, Medicare pays just 16% of lifetime costs for a person with Alzheimer’s. The price tag for Medicare is so low because, without treatments, expenses are primarily for non-medical services — such as at-home help with bathing, eating, and using the bathroom. It is families that end up paying for a staggering 70% of overall costs. Medicaid picks up the remaining 14%, primarily for nursing home and related long-term care services.

Now, the next move is in the hands of the Centers for Medicare & Medicaid Services (CMS), and we will be watching closely. The Alliance calls on CMS to rescind its current extreme barriers to coverage for anti-amyloid therapies. As the predominant insurer for individuals with Alzheimer’s disease, it is imperative that CMS provide immediate national coverage for Medicare beneficiaries.

CMS claims that cost doesn’t factor into coverage decisions. In fact, CMS has previously included ICER reports as part of its Medicare coverage determination process, including last year’s decision to limit access to anti-amyloid treatments for the treatment of Alzheimer’s disease. This is wholly inappropriate, given that the ACA prohibits use of the QALY-based cost-effectiveness analysis in CMS coverage and reimbursement decisions.

Our nation’s healthcare system can’t succumb to the ICER’s pessimistic view that healthcare is a zero-sum game. Our national healthcare leaders are too smart, creative, and resourceful to fall back on lazy reasoning that ultimately values the lives of some individuals more than others.

In our view, it is high time that ICER – and the insurers that rely on them – stop telling older adults and people with disabilities that they are worth less.

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