Cancer Drugs Cost More Than Ever. They Often Don’t Extend Lives - Bloomberg
Medicare, required by law to cover cancer treatment, would have paid much of the rest of the cost. At the time, in 2020, the pill listed for a jaw-dropping $160,000 a year, rising more recently to $214,000.
Despite having a PhD in pharmaceutical science, Dusetzina couldn’t figure out whether swallowing those costs — potentially for years — would help her mom live any longer or any better. Her research showed that while the drug might shrink tumors, no clinical trials proved it would extend her mother’s life. At the time of her search, Ibrance had already been on the market for five years. “I could not find any satisfactory data where I could say this is worth the money,” said Dusetzina, whose mother ended up not using the drug because she had a less common type of cancer. “It was really frustrating.”
Ibrance is one of the biggest-selling drugs in a revolution that has made new treatments available to American cancer patients faster than ever before. Prodded by legislation backed by drugmakers that allowed the Food and Drug Administration to speed approvals, the regulator greenlit more than 200 cancer drugs over the past three decades. Cancer treatments generated at least $200 billion in worldwide sales for the pharmaceutical industry last year, turning the once-sleepy oncology business into a gold mine more than 10 times bigger than obesity drugs.
The profusion of new treatments is a source of hope for many patients. Some drugs are game-changers, especially new immune therapies that teach the body itself how to destroy cancer cells. But the downside of approving so many drugs with so few hurdles is now clear: Many don’t prolong life at all, or do so only modestly, with problematic side effects. Indeed, fewer than half of the cancer drugs approved since 2000 that Bloomberg News reviewed based on their FDA labels have ever been shown to extend patient survival for any of their approved uses. Even fewer have been shown to improve cancer-related symptoms or quality of life.
In the past decade alone, drugmakers have made more than $50 billion on cancer drugs that so far have demonstrated no survival benefit, Bloomberg’s analysis shows.
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| Sources: Bloomberg calculations using 3 Axis Advisors price data and FDA labels Note: Includes only cancer drugs for which pricing data around initial approval was available. Some drugs listed were later removed from the market. Some drugs have regimens as short as a couple months. Provenge price is for three doses over four weeks. Survival data for some drugs remain under FDA review. |
“There is a myth that these cancer drugs are saving enormous numbers of lives,” says Richard Sullivan, a professor of cancer and global health at King’s College London. “It is not true.”
Trials are still underway that, according to the companies, could one day prove their drugs extend lives. Some treatments for rare cancers have kept patients in remission for years, even when they don’t have formal data from controlled survival trials. The genuine successes have helped many thousands of cancer patients live longer. The American Cancer Society says US cancer death rates have plummeted 34% since peaking in 1991, thanks to early detection, advances in medicines and a decline in smoking. Immunotherapies revolutionized treatment of melanoma, while also extending survival more modestly in cancers of the lung and other organs. Drugs that block specific overactive proteins have improved the outlook for certain types of breast cancer, leukemia and myeloma.
Still, an analysis in JAMA Oncology of a subset of major solid tumors estimated that four out of five cancer deaths averted from 1975 to 2020 were due to improved screening and preventive health measures, not new treatments. Survival rates remain relatively low for some types of advanced cancer, including advanced colon cancer — the second-biggest killer — and cancers of the pancreas and ovaries.
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Sources: Bloomberg calculations using 3 Axis Advisors price data and FDA labels Note: Prices are inflation adjusted to 2023. Includes only cancer drugs for which pricing data around initial approval was available. Includes drugs that were later removed from the market. |
The rise of what some doctors now call the “cancer industrial complex” is in part a familiar story of a captured regulator that draws much of its funding from the industry it’s supposed to monitor. In exchange for drug companies and medical device makers paying fees that now bring in almost half of the FDA’s $7 billion annual budget, Congress effectively has handed them ever-more ways to shorten testing and get priority treatment from agency reviewers.
This new cancer capitalism, though, is also a little-known tale of scientific gamesmanship. An empire of drugs grew out of the companies’ ability to exploit one specific marker: a measure of tumor growth known as “progression-free survival.” An obscure term used primarily by doctors until a standardized definition appeared in an academic journal in 2000, it’s since become a common justification for the FDA’s expedited approval of treatments. Researchers assumed that if a drug delayed the growth of tumors, it would also extend life. Recent studies have found the link is not so clearcut, and even one of the originators of the measure calls it arbitrary.
Yet demonstrating a 10-month improvement on this metric turned Pfizer’s Ibrance into a blockbuster. One thing it’s never been proven to do: prolong women’s lives. Ibrance was the top-selling drug in the Bloomberg survey that fell short on this measure, long considered the gold standard of clinical trials. Notably, a 2022 study showed no statistically meaningful improvement in survival for more than 600 women who were initially diagnosed with advanced breast cancer, the drug’s main use. Women would have to read deep into the fine print on page 19 of the label to find this out.
“It’s incredibly confusing for patients and their families to know what they’re signing up for,” says Vanderbilt’s Dusetzina.Drug companies enhanced the groundswell for new treatments by working with patient advocacy groups, led by sympathetic spokespeople: cancer sufferers. The companies help fund the groups’ budgets, sponsor their conferences and sometimes pay members to consult on studies. Hospitals, for their part, share in the markups for administering the drugs and also get paid for bringing patients to trials.
The growing reliance on industry in recent decades has had other long-lasting effects. Once primarily sponsored and paid for by the government, cancer drug trials are now largely conceived, organized and paid for by companies. That means basic questions sometimes go unanswered: When should treatment stop, how long should treatment last and when is it not necessary at all? Emerging studies suggest that in some cases recommended dosages may be higher than required, and given for longer than needed — a potentially lucrative outcome for drugmakers who are paid per dose.
More than any part of the FDA, the cancer division has embraced the concept of allowing drugs to be approved based on preliminary data. It’s led in that effort by its longtime oncology chief, Richard Pazdur, who has attributed his own conversion from “regulator” to “regulator-advocate” in part to his wife’s death from ovarian cancer.
Moving faster is popular with many patient groups. It’s also led to products with uncertain benefits lingering on the market for years, saddling some patients with unnecessary treatments or, in some cases, life-threatening side effects. “They never look at the life within those days,” says Rachel Koven, whose husband died of gastroesophageal junction cancer in 2016. She says he spent much of his last four months in the hospital being treated for side effects of various treatments that didn’t work, keeping him away from home during the precious last months of his life. To this day, she’s not sure all the extra treatments were worth it.
The new Health and Human Services secretary, Robert F. Kennedy Jr., and Marty Makary, the newly appointed FDA commissioner, are themselves keen critics of the agencies they now lead. Kennedy has called the agency a “sock puppet” for industry and Makary is a prominent surgeon who’s written books on the absurdities of US medical economics, including The Price We Pay: What Broke American Health Care — and How to Fix It.
President Trump, of course, has long cast himself as an industry scourge; an executive order he issued this month seeks to force cuts of as much as 80% in drug prices. But a similar effort in his first administration died in the courts. And pharmaceutical stocks rose the day of his new order, signaling investor skepticism of any significant impact from it.
Far longer than Trump has commanded the national political stage, the FDA official guiding the cancer drug revolution has been Pazdur, a former gastrointestinal oncologist from the MD Anderson Cancer Center in Houston. He’s led the FDA oncology division since 1999 and is one of the few top agency officials who has remained in his post under the new administration. In a brief interview after receiving a lifetime achievement award at a cancer conference in Chicago last month, Pazdur jokingly called himself “the designated survivor” and said he doesn’t know if the new administration will seek changes in approval standards.
An FDA spokesperson says “drug development, especially in oncology, is not static” and that the accelerated approval program “attempts to strike a balance between the need for overall survival assessments and the need for patients to have timely access to novel drugs.”
Pazdur’s tenure stretches all the way back to a time when the FDA, stung by public criticism of its response to the HIV crisis, faced mounting pressure to get drugs on the market faster. A 2002 Wall Street Journal editorial painted the agency as a bureaucratic mess that held up promising treatments of all kinds, including for cancer. It was titled: “FDA to Patients: Drop Dead.”
Under Pazdur, the oncology division made more frequent use of an expedited approval program that relies on shorter-term clinical measures, such as tumor size, to assess potential treatments. The shift was welcomed by patients and especially by industry, which was increasingly responsible for, as one former FDA commissioner put it, “trying to keep the lights on” at the agency.
Congress in 1992 had approved a “user fee” program under which drug companies funded a share of the FDA’s budget. In return, the agency agreed to speed up reviews of some drugs it deemed priority applications to as little as six months. A 1997 law had further eased approval standards, allowing the agency to fast-track certain drugs without its traditional requirement of two “well-controlled trials.”
At the time, advances in genetics were opening exciting opportunities. Researchers started identifying mutated genes that drove cancer growth. It gave the drug companies specific molecular targets for the first time. Sensing an attractive new market, the companies focused on ways to get their drugs to market faster than the years-long survival studies.
They soon found one. In 2000, an international group of academics devised new standards for measuring tumors’ growth. Once they grew 20% in their longest dimensions, or any new tumor appeared, the disease was considered to have “progressed.”
The threshold was chosen because it was the smallest amount likely to indicate a treatment was no longer working, says Elizabeth Eisenhauer, a professor emerita at Queen’s University in Ontario who helped devise the standards. The idea was to assess whether an agent was promising or not, allowing doctors to quickly move into trials assessing survival, she says. “It was arbitrary,” says Eisenhauer, who cautions against reading too much into the measure. A drug might shrink tumors, only to have toxic side effects that hasten death.
Still, once the new standards were published in the Journal of the National Cancer Institute that year, drug companies seized on them. Progression-free survival was easier to demonstrate than actual survival; they just had to keep tumor growth below that magic 20% threshold for as little as one period of scanning, typically six to eight weeks. By the mid-2000s, the FDA increasingly started approving cancer drugs based on their ability to slow tumor growth, with the idea that the metric would reliably predict which drugs extended patients’ lives.
Sometimes it did. Often, it didn’t. A review in the European Journal of Cancer found that two-thirds of drug trials aiming to slow progression of solid tumors were deemed successful from 1999 to 2015. This turned out to be a surprisingly poor predictor of whether patients live longer — which happened in only 38% of those cases.
The manufacturers got an additional fast track with Congressional approval of another category known as “breakthrough therapy” drugs. The concept came from Friends of Cancer Research, a group founded by Ellen Sigal, a real estate developer with a Russian literature PhD whose sister had died of cancer. She co-authored a column for The Hill with a former FDA commissioner in April 2012, calling for the “breakthrough” designation for agents that looked promising early in testing.
The effort came at a time of particularly high enthusiasm for cancer drugs, after multiple medicines had bolstered survival in advanced melanoma, previously considered one of the toughest to treat.
Sigal’s group hosts regular roundtables bringing together companies and regulators; Pazdur is a frequent speaker at its events. About 64% of its $6.8 million in funding last year came from drug and biotech companies including Pfizer, Sanofi and Amgen Inc., according to Friends. “The challenges we aim to address arise and evolve due to the rapid pace of scientific progress,” a spokesperson says. “That is the source of our agenda and the projects which we take on, not directives from industry or any one party. No matter the topic we strive toward evidence-based solutions.”
The law Congress passed in 2012 required the FDA to consider requests for the new “breakthrough” designation within 60 days and to work with the sponsor on a trial design that would be “as efficient as practicable… such as by minimizing the number of patients.” The law also endorsed the concept of using short-term clinical data as the basis for expedited approvals. In the decade through 2020, 85% of the drugs granted accelerated approval treated cancer, many premised on preventing tumor growth.
It became a shortcut to a financial, not a medical, blockbuster that rewards investors faster, says Christopher Booth, an oncologist at Queen’s University in Ontario. “The bar is lower,” he says. “You just need to show that you can delay tumor growth by literally eight weeks without having the patient live any longer or feeling better. And that’s a multi-billion-dollar drug right there.”
In the program’s first full year, the FDA got more than 90 applications for the new breakthrough designation. Among them was a drug from Pfizer called palbociclib.
Researchers from the University of California at Los Angeles had been investigating a Pfizer compound, PD-0332991, in the lab since the mid-2000s. Now better known by its brand name, Ibrance, palbociclib appeared to target specific proteins, CDK4 and CDK6, that can become overactive and lead to the development of the most common type of advanced breast cancer. After a clinical trial at UCLA on 12 women, four had significant responses.
The researchers embarked on a Phase 2 trial of the drug that eventually included 165 patients. The FDA granted breakthrough status in April 2013 after initial results showed it significantly delayed the growth of tumors in combination with a standard hormone treatment.
Reviews with an agency team followed, as mandated by the new law. Some staffers pointed out that the pivotal study was small, according to meeting minutes later made public. They noted that it was hard to draw conclusions because a portion of the trial studied only women with certain genetic alterations. Pfizer hoped to get approval to use the drug more widely.
That May, a team from the FDA gathered with Pfizer executives in a conference room in Silver Spring, Maryland. An agency reviewer called the initial results encouraging and said it looked forward to seeing more comprehensive Phase 3 data, recalls Mace Rothenberg, then head of development for Pfizer’s oncology unit. That could add years. Then, he remembers, Pazdur’s voice boomed in from a loudspeaker on the ceiling: “I am not so sure I agree with that.”
The oncology chief hadn’t spoken until that point, but argued the trial challenges could be overcome. “We knew we had some traction,” Rothenberg recalls.
Pazdur’s wife, Mary, who’d worked at MD Anderson as an oncology nurse, was at the time herself suffering from ovarian cancer. That, and the new law, had helped motivate him: “I have been on a jihad to streamline the review process and get things out the door faster,” as he later told one interviewer.
Pfizer formally submitted its application the next year. The company was slated to defend the drug before an external panel of advisers for a vote the FDA suggested could take place in February 2015. Rothenberg says he assigned staffers to prepare hundreds of slides for potential use during the meeting. Then, late in 2014, Pazdur called and told him the agency had decided against the panel.
The drug won accelerated approval in the same month the advisory panel would’ve met. One condition was that Pfizer follow up with the results of its larger trial, including overall survival data by November 2020.
Ibrance was an almost immediate hit for Pfizer. It beat competing medicines from Novartis AG and Eli Lilly & Co. to the market by two years. At an investment conference in New York in February 2015, Lazard’s Stephen Sands congratulated its chief executive officer, Ian Read, on winning such rapid approval with preliminary results. “That was unexpected by the marketplace, I think everybody was surprised,” he said.
“We started delivering product the day of the launch,” Albert Bourla, who was then running Pfizer’s oncology and vaccines business, told investors a month later. “Our reps were trained immediately after the approval and we had increased our salesforce the year before, so they were ready.”
In October, Pfizer reported that quarterly growth in oncology revenues had reached 54%, driven by Ibrance, Analysts on earnings calls showered executives with compliments — “great launch” — over the next year as doctors issued tens of thousands of prescriptions.
Pazdur, too, won praise. In the five years following passage of the breakthrough law, the FDA approved almost 50 new cancer drugs, more than it had in the previous decade. Pazdur earned a new public profile: Fortune in 2015 put him on the magazine’s list of the “World’s 50 Greatest Leaders” for his work in getting more cancer medications to market. (Pope Francis and Apple Inc. leader Tim Cook were among the others.) In November that year, Pazdur’s wife died.
Ibrance sales topped $2.1 billion in 2016, and the FDA granted additional approval that year for use in women who had failed other treatments. After a larger trial confirmed the initial results showing delayed tumor growth, the FDA converted its accelerated approval to a full blessing in 2017.
Bourla was elevated to CEO in 2019. The next year, Ibrance sales reached $5.4 billion, making it one of the company’s top three products and accounting for 13% of revenue.
One Pfizer deliverable wasn’t forthcoming: The overall survival data it had originally promised to submit to the FDA by November of that year.
Its competitors had already raced ahead with additional studies of their own, even though they’d entered the market later. The Eli Lilly drug proved in a 2019 study to prolong survival in breast cancer patients who had failed other therapies. Two years later, the Novartis product showed it prolonged survival a full year when used as an initial treatment for advanced cancer — Ibrance’s biggest use. Finally, in 2022, Pfizer reported the results of its large trial of 666 women: There was no survival benefit for initial treatment.
A Pfizer spokesperson says the results took longer than planned because patients in the trial lived longer than anticipated, even those in the control group. Its trials were aimed at slowing progression and thus “not optimized” to detect a difference in survival, which can be hard to measure due to post-trial therapies and patient dropout, the spokesperson adds. The company presented a real-world analysis of 9,000 patients at a breast cancer conference late last year that found no statistical difference in survival rates among the drugs. (A Novartis spokesperson says that study had “design limitations” and its findings shouldn’t be used to directly compare the drugs.) Ibrance “remains a standard of care first-line treatment” that gives women with incurable cancer “more time without their disease worsening” and can delay the need for chemotherapy, the Pfizer spokesperson said.
Doctors are divided over what to make of the contrasting results.Julie Gralow, chief medical officer of the American Society of Clinical Oncology, considers Ibrance’s lack of survival data an “anomaly” that doesn’t reflect real-world practice. Gralow has found that Ibrance generally has fewer side effects than its competitors. “Getting promising drugs to patients sooner, particularly those who have run out of other options, is important,” she says. Others argue that the survival results reflect subtle but real differences that become apparent over time. Matthew Goetz, an oncologist at the Mayo Clinic in Rochester, Minnesota, says the benefits from Ibrance seem to fade more quickly than its competitors; he favors the other medicines.
Pazdur is convinced the FDA’s approach is correct. Borrowing a line from a Black Eyed Peas song, he says critics who focus on survival data are “so 2000 and late.” He suggests a number of reasons why companies commonly struggle to prove their drugs prolong life. Some treatments target rare types of cancer where there are few patients to study. In other cases, patients are living so much longer that confirming survival gains can take years. Some of them drop out of studies if they end up in a control group for an extended period of time.
For the patients, an effective drug means “I might be able to make a graduation” or “I may be able to make a wedding,” Pazdur says. “We’re working to get safe and effective therapies out to patients in the most expeditious fashion here.” He adds that in about 15% of cases, followup studies have failed to confirm the benefits of drugs approved early and the agency has pushed for their removal. “We interpret that as a reasonable figure if you’re taking the appropriate degree of risk,” he says.
In the view of a growing number of cancer specialists, the trouble with this approach is that too many mediocre drugs stay on the market for years as companies drag their feet in producing the evidence that might prove whether they actually work.
Booth, the Queen’s University doctor, helped found a group called Common Sense Oncology in 2023 that’s pushing for a return to higher standards in clinical trials. It’s drawn members from Canada, the US, India, Japan, Australia, the UK, Israel, Ghana, Rwanda and Brazil.
These doctors agree that new drugs give patients hope — it’s just that it sometimes turns out to be false hope. In the meantime, drug companies generate billions.
Takeda Pharmaceutical Co., for example, won approval for the $4,450-a-pill multiple myeloma treatment Ninlaro in 2015 after a trial showed it delayed the growth of bone marrow cancer by six months. Multiple follow-up studies failed to show Ninlaro extends patients’ lives. It also can cause severe diarrhea. Since that early trial, though, Takeda has made more than $4 billion by touting the pill as the “first and only” medication in its class that allows patients to avoid a trip to an infusion center.
Note: Participant numbers are from FDA label at time of approval. Includes only cancer drugs for which pricing data around initial approval was available. Includes drugs that were later removed from the market.
The other concern for cancer drugs is quantity. A study by Mark Ratain, a University of Chicago cancer researcher, identified dozens of cancer pills sold at unnecessarily high doses or frequencies, adding to cost and side effects. “The thirst for profits and revenues is harming patients at this point,” Ratain says.
Most mid-stage melanoma patients fare just as well with two doses of immunotherapy treatment prior to surgery, compared with the usual regimen of nearly a year of drugs such as Opdivo from Bristol Myers Squibb Co. after surgery, according to results published in the New England Journal of Medicine last year. Preliminary findings in the Netherlands suggested that lower dosing of Merck & Co.’s Keytruda was also effective at keeping lung cancer patients alive for a year. And in a 1,000-patient-study published in Nature, researchers at the Netherlands Cancer Institute showed that advanced breast cancer patients could safely postpone taking drugs such as Ibrance for more than a year without any difference in long-term progression, reducing side effects and saving millions.
The conflicting evidence often leaves patients and their families questioning everything. Crystal Vaagen, a children’s book writer in Fargo, North Dakota, says her mother was diagnosed with advanced breast cancer in July 2021 at age 74. Her mom was put on Ibrance three months later after a 20-minute appointment during which no other treatment choices were mentioned. Doctors didn’t tell her about the Novartis drug, says Vaagen, who went to all her mother’s appointments. Her mom remained on the drug until April 2024, when her tumors began spreading and she switched to chemotherapy. She died from a bloodstream infection, a possible chemotherapy complication.
Ibrance “did okay for her,” Vaagen says. But she wishes they had been told that other drugs had more proven life-extending benefits. “If I would have known there are different medicines out there, I would have fought a little harder,” she says. “I would have said, ‘Maybe we ought to switch, is there a way of doing that?’ But as a patient you are expected to know nothing.’’
Costs, meanwhile, radiate across the health system. An analysis of Medicare found that annual spending on oncology drugs more than doubled in the four years ending in 2020, to $53.9 billion. By comparison, Trump’s Health and Human Services Department has said it would save taxpayers $1.8 billion a year by reducing its workforce to 62,000 from 82,000.
One of FDA commissioner Makary’s first major hires, UC San Francisco cancer doctor Vinay Prasad, is a critic of the overuse of expedited approvals who has questioned the lack of survival evidence for some cancer drugs. As head of the FDA’s Center for Biologics Evaluation and Research, he won’t oversee most cancer treatments, which remain in Pazdur’s division. But he may have the commissioner’s ear.
Ibrance has now brought in more than $40 billion for Pfizer, a decade after the drug hit the market on the strength of a trial of tumor growth in 165 patients. Sales have slipped recently, though. Ibrance was among 15 drugs selected by Medicare this year for price negotiations under a 2022 law that capped out-of-pocket prescription costs at $2,000.
Bourla, the Pfizer chief executive, has recently touted a new breast cancer treatment that targets a similar protein. Derived from a Pfizer compound known as PF-07220060, this one is called atirmociclib.Early trials show an encouraging ability to shrink tumors.
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Methodology:
Drugs listed as shown to prolong life have statistically significant survival benefit in FDA label or were under review as of April 1, 2025. Prices shown are 30-day prices at or near the time of initial approval. For single-dose drugs, total price is shown. For weight or body-surface-area based drugs, dosages for 75 kg or 1.75 m2 adults are used; pediatric drug prices are based on age. Prices are rounded up to nearest single dose vial. Prices for multiweek cycles are prorated to create an exact 30 day price.
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