As cancer treatment advances, costs are spiralling, leaving healthcare systems struggling to keep up. Breakthrough drugs like Opdivo offer hope to patients like Linda Medendorp, who saw her terminal diagnosis reversed. Yet, the hefty price tags challenge healthcare budgets and raise ethical questions about access and affordability. With spending on oncology drugs expected to nearly double by 2028, policymakers face tough decisions in balancing innovation with sustainability.
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By Sarah Neville
Medical breakthroughs come with spiralling price tag, leaving policymakers with tough decisions ___STEADY_PAYWALL___
The day her son turned 18, Linda Medendorp was told she was going to die. After seeing a doctor about persistent problems swallowing her food, tests showed the then 51-year-old had incurable cancer that had already spread from her stomach and rectum.
“It was the biggest nightmare,” she said, but there was “one positive factor in all the bad news”. Her tumour’s characteristics meant she qualified for a Dutch trial of a checkpoint inhibitor, which removes brakes on the immune system that prevent it from attacking cancer cells. Suddenly she had a sliver of hope.
The drug she received, Bristol Myers Squibb’s Opdivo, is an immunotherapy — one of several classes of treatment to emerge in recent years that have made the outlook for cancer patients more promising than ever before.
Other potentially transformative advances include highly targeted treatments such as antibody drug conjugates, an advanced form of chemotherapy, and radioligands, which combine a nuclear isotope with an antibody to irradiate cancer cells.
But for health systems that were struggling to cope with rising and ageing populations even before Covid ravaged budgets, innovation cannot be divorced from its price tag — a year’s supply of Opdivo at its list price costs almost $200,000, according to a calculation for the FT by health policy consultancy ATI Advisory.
“All these drugs are extremely, extremely expensive,” said Denis Lacombe, chief executive of the European Organisation for Research and Treatment of Cancer.
A surge in innovative medicines, along with a global push to increase early diagnosis and expand access to treatment, raised global spending on oncology drugs to $223bn last year, according to research from the IQVIA Institute for Human Data Science. It expects the figure to balloon to $409bn by 2028.
The cost of cracking cancer: an FT series
This is the third in a three-part series looking at how an influx of money is changing prospects for patients but creating new challenges for healthcare systems. The other parts will be published in the coming days.
Part one: What the $1tn race for a cure leaves behind
Part two: The Covid-era tech that could reinvent cancer care
Part three: New cancer drugs add to strains on stretched health budgets
Clinicians are at the sharp end of the mismatch between resources and demand. As a researcher Lillian Siu, an oncologist at Toronto’s Princess Margaret Cancer Centre, is striving to break new ground in cancer care. But as a doctor she knows the pain of not always being able to offer the optimal treatment.
Just a day earlier, she said, she had unsuccessfully attempted to secure for a patient “off-label” access to a drug — reimbursement for its use beyond the application for which it had received regulatory approval.
“It is definitely not an easy discussion with our patients when you say you have evidence for a drug that may be helpful for their care, and yet you know they do not have coverage, whether private or public funding.” This was especially the case for rare tumours where drug approvals or relevant clinical trials were often not available, she said.
Even as governments and payers grapple with issues of affordability, the nature of many of the treatments is making it more difficult to adjudicate the crucial question of whether they offer value for money.
A large proportion of the new drugs fall into the category of “precision medicine”: tailored treatments for patients whose tumours display particular genetic characteristics. However, this niche approach means the number of patients who may potentially qualify is becoming “smaller and smaller”, according to Emile Voest, professor of medical oncology at the Netherlands Cancer Institute.
He said trials were increasingly being conducted as “single arm” studies — without a comparator group — creating “the inherent risk that you overestimate the value” of the medicine.
In order to compensate for the lack of data, in 2016 he established the “drug rediscovery protocol”, or DRUP, since replicated in a number of other European countries, whereby patients with advanced cancer are treated with off-label drugs matched to the genetic profile of their tumours.
Under a “personalised reimbursement model”, manufacturers will in some circumstances pay for the first four months of use. If the drug proves effective, the healthcare insurer will pick up the bill for continued treatment.
“In the absence of sufficient evidence, it bridges the risk between pharmaceutical companies and the payers,” said Voest.
Over the past eight years more than 1,500 patients have been treated under the scheme, and 37 drugs made available to patients outside their original “label”.
One beneficiary is Medendorp, who felt the effects of her treatment almost instantaneously, realising after only two infusions that she was able to eat more easily. “And it’s amazing, because I never had surgery. The medicine was like a starting sign for my immune system to clean up all the cancer,” she said.
The Netherlands is now recognised as an international leader in demonstrating the value of medicines. Six countries, including Norway, Finland and Denmark, are running similar trials, “and we’re working closely together to extend that to 15 countries in the EU, including the UK,” Voest said.
One drug has since been approved for reimbursement by payers based on the data gathered through the DRUP study and three others have been greenlit on the basis of a separate initiative overseen by Voest that gathers “real world evidence” of how a drug performs with a broader group of patients who might not have met the criteria for inclusion in a clinical trial.
In the US, payers have traditionally been reluctant to use “financial optimisation” approaches to control access to cancer drugs, said Sarah Emond, president of the Institute for Clinical and Economic Review, which measures the cost-effectiveness of drugs.
However, she noted that the Food and Drug Administration’s decision to approve many innovative treatments through an “accelerated access pathway” — which uses surrogate endpoints such as tumour shrinkage rather than proof of improved survival — meant drugs often entered the market despite relatively thin evidence they would extend lives.
She also said that while some manufacturers were fulfilling the “social contract” under which they carry out the necessary additional studies to confirm effectiveness once their drug was in use, “you also have ones that are . . . not doing the trials as quickly as they should”.
Commercial confidentiality means outcome-based arrangements, where they exist, are often shrouded in secrecy. However, one that came to public notice involved Pfizer, which in 2021 set up a programme linking payment to the performance of Xalkori, a drug to treat non-small cell lung cancer. Pfizer said it had always been intended as a pilot project, which ended in June 2022, but did not rule out extending the approach in future.
One concerning aspect of the newer oncology treatments, according to experts, is that despite huge recent advances in the understanding of human biology, there is often little clarity about if or when a treatment can safely be stopped, or whether the dose established through clinical trials is the ideal one.
The question is starting to attract the attention of regulators. Lacombe co-chairs the European Medicines Agency’s cancer medicine forum alongside Francesco Pignatti, the agency’s head of oncology. They are exploring clinical trials addressing “patient-centred questions” such as when treatment should be de-escalated and for which patients the benefits outweigh side effects such as toxicity — which can add to costs.
However, such trials are unlikely to be welcomed by an industry that may fear a loss of revenue, raising questions about how they would be funded — a conundrum faced by Yasushi Goto of the National Cancer Centre in Tokyo.
In Japan, he said, all drugs that receive regulatory approval must by law be reimbursed. But an ageing population was creating an unsustainable strain on the health insurance system, leading the government to cut the prices it is willing to pay — a move he suggests is deterring some smaller biotech companies from developing drugs for the Japanese market.
He and fellow researchers are now examining whether the overall drug bill could be reduced by proving immunotherapy can be given at reduced dosage, and for shorter periods, without sacrificing efficacy.
Securing funding and recruiting for trials to test these theses has been difficult but he is convinced the approach offers the best way of balancing breakthroughs with cost-effectiveness.
“I understand that innovation is very important . . . but I believe that cost and sustainability are too,” he said.
Ivana Cattaneo, who chairs the oncology platform at industry body the European Federation of Pharmaceutical Industries and Associations, is wary about reducing dosage or duration from those used in clinical trials without “the same level of evidence” that led regulators to approve the medicine in the first place.
Cattaneo, who is also executive director of oncology policy at Novartis, argues the way health systems and governments calculate value needs to change, recognising that some treatments can deliver cures that in time will save huge sums not just for health systems but for wider economies.
“People can go back to their life, can go back to work, be productive, and enjoy their family,” she said. The industry was “giving back a lot to society thanks to innovation” but was “still trapped with an old way of thinking” that assessed only immediate clinical benefit.
In the Netherlands, almost four years after her diagnosis, Medendorp believes her prognosis is now very good, but she continues to undergo regular tests. Suffused with gratitude for the medical advances that saved her, she is volunteering on a programme that encourages people living with cancer to engage in sport, and prioritising spending time with her family, with whom she recently enjoyed a trip to the Paris Olympics.
“Suddenly I can live again and feel the beauty of life.”
Read also:
- Redefining cancer as a metabolic disease: Dr Thomas Seyfried makes stunning revelations about new therapies that could transform cancer treatment.
- Female cancer treatment in South Africa: A regrettable shadow of neglect
- Blood tests detecting cancer earlier: a sign of hope for millions
© 2024 The Financial Times Ltd.
