The pace of cancer drug development may have accelerated over the past decade, but so too has the cost of care, which threatens to limit access for a large percentage of Americans in the decade to come. During the keynote lecture at the 2022 ASCO Quality Care Symposium, Ezekiel J. Emanuel, MD, PhD, FASCO, BA, Vice Provost for Global Initiatives at the University of Pennsylvania, examined these trends and the need for reform in U.S. health policy, including coverage and subsidies, drug costs, accelerated approval, and changes in care delivery.1
According to Dr. Emanuel, the Inflation Reduction Act of 2022, which was passed by Congress and signed into law in August, addressed two important factors that impinge on oncology: coverage and drug cost. Since 2021, said Dr. Emanuel, health-care coverage has increased by 5.8 million Americans, 80% of whom received coverage for less than $10 per month during a special enrollment period. In addition, Medicaid enrollment has increased by nearly 18 million since the start of the pandemic and now covers 89 million Americans.
Ezekiel J. Emanuel, MD, PhD, FASCO, BA
“The Congressional Budget Office estimates that nearly 5 million more people will get coverage over the next few years with expanded premium subsidies,” said Dr. Emanuel. He noted, however, these subsidies only go through 2025. “Right after the [2024] election, these [subsidies] must be renewed or made permanent.”
More on the Inflation Reduction Act
The Inflation Reduction Act also addressed prescription drug costs by giving the Department of Health and Human Services Secretary the power to negotiate prices. Although this authority will not take effect for another 4 years, in 2026, 10 drugs will be chosen by the Secretary to be regulated based on their impact on Medicare. The number of regulated drugs will increase to 20 in 2029.
“The Congressional Budget Office estimates that it will save over $250 billion over 10 years, which is approximately 15% of Health and Human Services’ total drug spend,” said Dr. Emanuel. “That’s a substantial amount of money.”
In addition, the Inflation Reduction Act requires drug companies to pay rebates if their prices rise faster than underlying inflation and caps drug spending for retail pharmacy (Medicare Part D) at $2,000 per year. Starting next year, it will also eliminate the 5% co-insurance on catastrophic coverage for drugs as well as the monthly payment that Medicare beneficiaries must make for insulin.
“Capping out-of-pocket expenditures at $2,000 for drugs is very relevant to cancer patients because out-of-pocket expenses often exceed $8,000 above the catastrophic threshold,” said Dr. Emanuel.
Accelerated Approval
According to Dr. Emanuel, the recent approval of aducanumab-avwa (Aduhelm), an Alzheimer’s drug that was brought to market by Biogen through the accelerated approval process, sparked massive public controversy for three main reasons: (1) the surrogate endpoint used was never correlated with cognitive or other clinical improvements in patients; (2) despite “meager data,” the drug was originally priced at $56,000 per year (although it has since come down); and (3) the legally required confirmatory trial was proposed to take 9 years, despite the fact that there are 5 million patients with Alzheimer’s disease in the country.
As Dr. Emanuel explained, beyond the Biogen controversy, there are important questions about the accelerated approval process. From its inception in 1992 to 2019, 194 cancer drugs were approved by this process for indications based on surrogate endpoints. However, 5 years after accelerated approval, approximately half of these drugs did not complete their confirmatory trials.
“The drug companies are not in any rush to complete the confirmatory trials because they have little to gain,” Dr. Emanuel explained. “If the drugs have a negative confirmatory trial, they have a reduction in price, but positive outcomes are not associated with a higher price.” What’s more, among the drugs that did complete their confirmatory trial, only 20% showed improved overall survival.
“These drugs are not actually getting us to the endpoint we want,” said Dr. Emanuel. “This is a market failure…. We’re getting a lot of drugs with minor therapeutic additions, which is not the point of accelerated approval.”
We’re getting a lot of drugs with minor therapeutic additions, which is not the point of accelerated approval.— Ezekiel J. Emanuel, MD, PhD, FASCO, BA
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To solve this problem, said Dr. Emanuel, surrogate or intermediate endpoints used for accelerated approval should be established as highly or moderately correlated with long-term outcomes that clinicians care about—ie, disease-free survival, overall survival, or improved quality of life.
“In addition, accelerated approval should not be granted until we’ve started recruitment on the confirmatory trial,” Dr. Emanuel continued. “Finally, we need transparency regarding the recruitment into the confirmatory trials, the progression of those confirmatory trials, and a firm deadline for completion…. This will put a lot of pressure on the drug companies to do what they have no incentive to do now, and that’s complete the confirmatory trial.”
Medicare Bankruptcy
Although rarely discussed, said Dr. Emanuel, another important issue is the impending bankruptcy of Medicare. According to a recent report released by Medicare trustees, the Medicare Trust Fund, which funds inpatient care (Part A), will become bankrupt over the next few years.
“This will make Medicare funding a political football,” said Dr. Emanuel. “Whoever’s not in power going into 2026, 2027, and 2028 will attack the administration for losing Medicare or having to bail it out.”
He added: “This is an issue that almost no one wants to pay attention to because the solutions are hard, but it’s going to become more politically salient in the next few years.”
Megatrends for Cancer Care Delivery
Finally, Dr. Emanuel outlined several trends in cancer care delivery that will become impactful over the next decade. For one, mobile administration and outpatient administration of chemotherapy were greatly improved during the COVID-19 pandemic.
“We’re going to see an acceleration of giving chemotherapy to patients at home,” said Dr. Emanuel. “I think many patients are going to prefer to receive treatment in an environment they are very comfortable with rather than to have to travel.”
A second major trend is expanded focus on improved delivery of end-of-life care. According to Dr. Emanuel, suboptimal end-of-life care has been a “sore point” for 40 years.
“Very frequently in the United States, patients in the last 6 months of life end up in the intensive care unit and get chemotherapy,” he said. “And if they get on hospice, it’s with only a few weeks left in life.”
As Dr. Emanuel explained, however, data have shown that the introduction of comprehensive home care can improve patients’ experience, increase their satisfaction, increase deaths at home, decrease emergency room visits, and decrease hospitalizations. “These programs require integration of multidisciplinary evidence-based components. As the [patient’s disease] progresses, the interventions progress,” said Dr. Emanuel.
“It also requires a proactive identification of patients,” he continued. “One of our biggest problems in end-of-life care is we don’t like to talk to patients about end-of-life care…, but this is going to become a greater focus with the improved ability to administer care out of the house.”
One of our biggest problems in end-of-life care is we don’t like to talk to patients about end-of-life care…— Ezekiel J. Emanuel, MD, PhD, FASCO, BA
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Finally, the COVID-19 pandemic highlighted mental health issues experienced by a large proportion of the population, especially anxiety and depression. According to Dr. Emanuel, patients with cancer are more susceptible to these mental health conditions, but the rate-limiting step is going to be mental health providers.
“We’ve tried mental health services online, which seems to work very well,” said Dr. Emanuel. “I think the shortage of mental health providers—we have fewer than 1 mental health provider for every 1,000 people in the United States—is going to force more online care and probably more group care, which could be a good thing.”
DISCLOSURE: Dr. Emanuel has a leadership role in COVID-19 Recovery Consulting, Embedded Healthcare, Oak HC/FT, Oncology Analytics, and Village MD; has served as a consultant to Clarify, JSL Health Fund, and Peterson Center on Healthcare; has received honoraria from CBI (a division of Informs), Healthcare Leaders of New York, Queens Health System, Massachusetts Association of Health Plans, MedImpact, Rightway, Rise Health, Signature Healthcare Foundation, The Galien Foundation, and WellSky; and owns stock in Cellares and Notable.
REFERENCE
1. Emanuel EJ: New directions for cancer care in the U.S.: Building a transformational research and development ecosystem and healthy payment landscape that better supports our patients. 2022 ASCO Quality Care Symposium. Keynote Lecture. Presented September 30, 2022.
