fbpx

Mental Health

Why We May Never Know Whether the $56,000-a-Year Alzheimer’s Drug Actually Works

The Food and Drug Administration’s approval in June of a drug purporting to slow the progression of Alzheimer’s disease was widely celebrated, but it also touched off alarms. There were worries in the scientific community about the drug’s mixed results in studies — the FDA’s own expert advisory panel was nearly unanimous in opposing its approval. And the annual $56,000 price tag of the infusion drug, Aduhelm, was decried for potentially adding costs in the tens of billions of dollars to Medicare and Medicaid.

But lost in this discussion is the underlying problem with using the FDA’s “accelerated” pathway to approve drugs for conditions such as Alzheimer’s, a slow, degenerative disease. Though patients will start taking it, if the past is any guide, the world may have to wait many years to find out whether Aduhelm is actually effective — and may never know for sure.

The accelerated approval process, begun in 1992, is an outgrowth of the HIV/AIDS crisis. The process was designed to approve for sale — temporarily — drugs that studies had shown might be promising but that had not yet met the agency’s gold standard of “safe and effective,” in situations where the drug offered potential benefit and where there was no other option.

Unfortunately, the process has too often amounted to a commercial end run around the agency.

The FDA explained its controversial decision to greenlight the Biogen pharmaceutical company’s latest product: Families are desperate, and there is no other Alzheimer’s treatment. Also, importantly, when drugs receive this type of fast-track approval, manufacturers are required to do further controlled studies “to verify the drug’s clinical benefit.” If those studies fail “to verify clinical benefit, the FDA may” — may — withdraw them.

But those subsequent studies have often taken years to complete, if they are finished at all. That’s in part because of the FDA’s notoriously lax follow-up and in part because drugmakers tend to drag their feet. When the drug is in use and profits are good, why would a manufacturer want to find out that a lucrative blockbuster is a failure?

Historically, so far, most of the new drugs that have received accelerated approval treat serious malignancies.

And follow-up studies are far easier to complete when the disease is cancer, not a neurodegenerative disease such as Alzheimer’s. In cancer, “no benefit” means tumor progression and death. The mental decline of Alzheimer’s often takes years and is much harder to measure. So years, possibly decades, later, Aduhelm studies might not yield a clear answer, even if Biogen manages to enroll a significant number of patients in follow-up trials.

Now that Aduhelm is shipping into the marketplace, enrollment in the required follow-up trials is likely to be difficult, if not impossible. If your loved one has Alzheimer’s, with its relentless diminution of mental function, you would want the drug treatment to start right now. How likely would you be to enroll and risk placement in a placebo group?

The FDA gave Biogen nine years for follow-up studies but acknowledged that the timeline was “conservative.”

Even when the required additional studies are performed, the FDA historically has been slow to respond to disappointing results.

In a 2015 study of 36 cancer drugs approved by the FDA, only five ultimately showed evidence of extending life. But making that determination took more than four years, and over that time the drugs had been sold, at a handsome profit, to treat countless patients. Few drugs are removed.

It took 17 years after initial approval via the accelerated process for Mylotarg, a drug to treat a form of leukemia, to be removed from the market after subsequent trials failed to show clinical benefit and suggested possible harm. (The FDA permitted the drug to be sold at a lower dose, with less toxicity.)

Avastin received fast-track approval as a breast cancer treatment in 2008, but three years later the FDA revoked the approval after studies showed the drug did more harm than good in that use. (It is still approved for other, generally less common cancers.)

In April, the FDA said it would be a better policeman of cancer drugs that had come to markets via accelerated approval. But time — as in delays — means money to drug manufacturers.

A few years ago, when I was writing a book about the business of U.S. medicine, a consultant who had worked with pharmaceutical companies on marketing drug treatments for hemophilia told me the industry referred to that serious bleeding disorder as a “high-value disease state,” since the medicines to treat it can top $1 million a year for a single patient.

Aduhelm, at $56,000 a year, is a relative bargain — but hemophilia is a rare disease, and Alzheimer’s is terrifyingly common. Drugs to combat it will be sold and taken. The crucial studies that will define their true benefit will take many years or may never be successfully completed. And from a business perspective, that doesn’t really matter.

Source

After Nearly 60 Years of Marriage, This Missouri Couple Stayed Together to the End

Arthur Kelley could barely raise his voice above a whisper last fall when he told a nursing assistant he never wanted his wife, Maggie, to be alone. After almost 60 years of marriage, five children and a lifetime filled with more victories than defeat, Kelley wanted to be there for his ailing wife, even if she didn’t know he was there.

He got to be there for her. But like so many other people who have died of covid-19, he died without his family.

Dementia had stripped Maggie Kelley of her memory, so her family had moved her into a nursing home in 2015. Arthur, who had received care for Parkinson’s disease at home, moved to the same facility in the St. Louis suburbs two years later to be closer to Maggie.

“It was a literal choice to go be there with Mom,” said their youngest son, Kevin Kelley. “He really desired to be there.”

Their parents shared meals, watched television and slept in the same room for three years. They were separated only once, when Maggie, 81, contracted an asymptomatic case of covid early in August.

“He protected her like Superman protects Lois Lane,” said their oldest daughter, Lisa Kelley-Tate. “That’s how he was with her.”

Arthur and Maggie Kelley celebrating an anniversary leading the Greater Faith Missionary Baptist Church. The couple died 30 days apart in 2020. A double funeral marked the end of nearly 60 years together as a married couple.(Derrick Varner)

Arthur, 80, would often ask when he could see his wife again.

“He wanted to make sure he didn’t pass before she did,” Kelley-Tate said a staffer at the nursing home told her. “It was his job to make sure he was there for her. Maybe he knew then that his time wasn’t going to be long.”

Maggie finished her quarantine and they reunited. But only briefly. She died of complications of dementia on Nov. 2.

That afternoon, Arthur held her hand as long as he could. When Kelley-Tate arrived, he was still holding on, so she took her mother’s other hand. She carefully painted Maggie’s nails red, her favorite color. But Arthur still wanted more time with Maggie.

“It took a while before he had me call the mortician to come pick her up,” Kelley-Tate recalled. “He said, ‘I want her here with me just a little longer.’”

Maggie and Arthur grew up together in Coffeeville, Mississippi, a small town about 90 miles south of Memphis, Tennessee. Maggie was the daughter of a teacher and a farmer. Arthur helped his family run its dry-cleaning business. He also learned to play the piano well enough to perform in juke joints and churches.

Their relationship bloomed in high school. Arthur took Maggie to the prom before they headed off to college. Maggie attended two historically Black colleges in Mississippi: what’s now known as Alcorn State University in Lorman and Rust College in Holly Springs. Arthur left the South for the Midwest, where he attended Southern Illinois University in Carbondale.

After their wedding on June 3, 1961, in Coffeeville — Maggie walked down the aisle in a lace dress with a sweetheart neckline; Arthur wore a white jacket and a wide grin — the couple decided to put down roots in St. Louis. Their lives revolved around the children they soon had, church and music. Maggie taught elementary school and took care of the children while Arthur studied speech pathology.

“They would always talk about how they would work together,” said their youngest daughter, Gina Kelley. “They worked as a team.”

The couple attended Central High School in Coffeeville, Mississippi, a small town about 90 miles south of Memphis, Tennessee. Maggie graduated with the class of 1956. Arthur earned his high school diploma in 1957. (Kelley Family)
Arthur Kelley became the pastor of Greater Faith Missionary Baptist Church in north St. Louis in 1977. Arthur and Maggie Kelley pose for a photo in the early 1990s at the church. (Erma Moore)

Arthur became the pastor of Greater Faith Missionary Baptist Church in 1977. He juggled life as a speech pathologist and minister, their children said. Maggie, who at this point was home raising the kids full time, established a routine for them that included prayer time, gospel music and home-cooked meals, including her beloved “Heath bar cake.”

Arthur and Maggie Kelley stayed dedicated to each other, in good times and bad. One of their toughest moments was the death of their 3-year-old son, Arthur Jr.

In their final years, both struggled with their health, but they never complained about their conditions. They leaned on their faith instead as he pushed through the challenges caused by Parkinson’s disease while her dementia progressed.

“At times, I said if my father had my mom’s body and my mom had my father’s brain we would be all good,” their son Kyle Kelley said.

After Maggie died, Arthur helped his children make funeral arrangements for her. He picked out her casket, and then he selected one for himself. Two of his children lifted him out of a chair so he could see the inside.

“He said, ‘I like that,’” Kelley-Tate recalled. “I said, ‘OK, we’ll keep that in mind,’ not thinking it would happen 30 days later.”

He too had contracted covid, one of the more than half-million nursing home residents nationwide to catch the contagious virus. Arthur wanted to attend his wife’s service, so his family decided to hold off on the funeral until he got better.

He never recovered. Exactly one month after Maggie’s death, he died in the covid ward of a nearby hospital. No family was allowed to be with him. A nurse called Kelley-Tate by video after he died.

But the family came together for what was now a double funeral with the caskets close to each other — the mauve one Arthur had picked for Maggie and the mahogany casket he had picked for himself.

4 Vital Health Issues — Not Tied to Covid — That Congress Addressed in Massive Spending Bill

Late last month, before President Joe Biden took office and proposed his pandemic relief plan, Congress passed a nearly 5,600-page legislative package that provided some pandemic relief along with its more general allocations to fund the government in 2021.

While the $900 billion that lawmakers included for urgent pandemic relief got most of the attention, some even bigger changes for health care were buried in the other parts of that huge legislative package.

The bundle included a ban on surprise medical bills, for example — a problem that key lawmakers had been wrestling with for two years. Starting in 2022, because of the new law, patients generally will not pay more for out-of-network care in emergencies and at otherwise in-network facilities.

But surprise bills weren’t the only health care issue Congress addressed as it ended a tumultuous year. Lawmakers also answered pleas from strained health facilities in rural areas, agreed to cover the cost of training more new doctors, sought to strengthen efforts to equalize mental health coverage with that of physical medicine and instructed the federal government to collect data that could be used to rein in high medical bills.

Here are some details about those big changes Congress made in December.

Rural Hospitals Get a Boost

Throwing a lifeline to struggling rural health systems — and, it appears, a bone to an outgoing congressional committee chairman — lawmakers gave rural hospitals a way to get paid by Medicare for their services regardless of whether they have patients in beds.

The law creates a new category of provider, known as a “rural emergency hospital.” Starting in 2023, some hospitals will qualify for this designation by maintaining full-time emergency departments, among other criteria, without being required to provide in-patient care. The Department of Health and Human Services will determine how the program is implemented and which services are eligible.

Medicare, the federal insurance program that covers more than 61 million Americans 65 and older or with certain disabilities, currently does not reimburse hospitals for emergency or hospital outpatient services unless the hospital also offers in-patient care.

That requirement has exacerbated financial problems for rural hospitals, many of which balance serving communities with fewer patients and less need for full in-patient services with the need for emergency and outpatient services. One study last year found 120 rural hospital facilities had closed in the past 10 years, with more at risk.

Hospital groups have praised the change, which was introduced by Sen. Chuck Grassley (R-Iowa), who has championed rural health issues and ended his term as chairman of the Senate Finance Committee this month. “I worked to ensure rural America would not go overlooked,” he said in a statement.

Medicare Invests in More Doctors

Hoping to address a national shortage of doctors that has reached critical levels during the pandemic, Congress created an additional 1,000 residency positions over the next five years.

Medicare will fund the positions, which involve supervised training to medical school graduates going into specialties like emergency medicine and are distributed among hospitals most in need of personnel, including rural hospitals.

Critics like The Wall Street Journal’s editorial board have noted this is Congress’ attempt to fix a problem it created in the late 1990s, when lawmakers capped the number of Medicare-funded residency positions in the United States, fearing too many doctors would inflate the cost of Medicare.

While Medicare is not the only source of educational funding and hospitals may add their own residency slots as needed, Medicare generally will reimburse hospitals for the number of residents they had at the end of 1996. Among other consequences of that 1996 cap, most Medicare-funded residencies are clumped at Northeastern hospitals, a 2014 study showed.

In contrast to the 1,000 positions created as part of the stimulus package, one bipartisan proposal in 2019 that was never enacted would have added up to 15,000 positions over five years.

Strengthening Mental Health Parity

The legislative package strengthens protections for mental health coverage, requiring federal officials to study the limitations insurance companies place on coverage for mental health and substance use disorder treatments.

In 1996 Congress passed the first law barring health insurers from passing along more of the cost for mental health care to patients than they would for medical or surgical care. The Affordable Care Act, building on earlier laws, made mental health and substance use disorder treatments an “essential health benefit” — in other words, it required most health insurance plans to cover mental health care.

But enforcing that standard has been a challenge, in part because violations can be hard to spot and the system has often relied on patients to notice — and report — them.

In December, lawmakers approved a measure requiring insurers to analyze their coverage and provide their findings to state and federal officials upon request.

They also instructed federal officials to request the findings from at least 20 plans per year that may have violated mental health parity laws and tell insurers how to correct any problems they find — under penalty of having insurer violations reported to their customers if they do not comply.

The law requires federal officials to publish an annual report summarizing the analyses they collect.

More Transparency in Cost and Quality

Americans often do not know how much they will be expected to pay when they enter a doctor’s office, an ambulance or an emergency room.

Taking another modest step toward transparency, Congress banned so-called gag clauses in contracts between health insurers and providers.

Among other things, these sorts of “gag” restrictions previously have prevented insurers and group health plans from sharing with patients and others — such as employers — information about a provider’s prices or quality. The December legislation also prohibited insurers from agreeing to contracts that prevent them from getting access electronically to claims and other information from providers on behalf of the insurer’s enrollees.

In 2018, Congress banned gag clauses in contracts between pharmacies and insurers or pharmacy benefit managers. Those gag clauses had prevented pharmacists from sharing cost information with patients, like whether they could pay a lower price for a prescription by paying out-of-pocket rather than using their insurance coverage.

The proposal approved in December’s legislation came from a big, bipartisan package of health care cost fixes passed in 2019 by the Senate Health, Education, Labor and Pensions Committee, but not by the rest of Congress. The committee’s Republican chairman, Sen. Lamar Alexander of Tennessee, retired from Congress this month. His Democratic partner on that package, Sen. Patty Murray of Washington, will take over the chairmanship as Democrats assume control of the Senate and has vowed to focus on health care affordability.

Consumers First, a health consumer-focused alliance of health professionals, labor unions and others, led by Families USA, praised the ban. The change is “a significant step forward” to stop “the abusive practices from hospitals and health systems and other segments of the health care sector that are driving up health care costs and making health care unaffordable for our nation’s families, workers, and employers,” it said in a statement.

KHN senior correspondent Sarah Jane Tribble contributed to this report.