Many European Cancer Drugs Offer No Added Benefit
Many cancer drugs approved by the European Medicines Agency (EMA) between 1995 and 2020 lack proof of added benefit, according to a new study from Utrecht University in the Netherlands.
Researchers reported that “a large proportion” of new drug approvals offered minimal or no added benefit, and that this particularly applied to those approved through expedited “fast track” pathways.
The costs of such drugs may be a burden to healthcare systems, and they give patients false hope, they said.
Lead researcher Francine Brinkhuis, a PhD candidate at Utrecht University, Utrecht, Netherlands, told Medscape Medical News: “Around half of all new drug approvals with a new active substance are oncology drugs, whether standard or conditional approvals.” Extensive drug development appears to be concentrated on specific cancer types, she explained. “This leads to many parallel and/or sequential drug approvals that are not necessarily incremental innovations.”
The study, published in the BMJ, retrospectively assessed 131 oncology drugs with 166 indications that had been evaluated for added benefit by at least one organization over the relevant period. It found that health technology assessments (HTAs) showed that the added benefits of the new drug were negative or non-quantifiable in two fifths of cases.
Median Time to Offset R&D Costs Is Just 3 Years
The researchers noted that the pharmaceutical industry claims that high drug prices are needed to offset research and development (R&D) costs. However, their analysis of publicly available revenue data compared with published estimates of R&D costs showed that the median time to offset median R&D costs of $684 million, adjusted to 2020 values, was just 3 years. Drugs with higher added benefit ratings generally had greater revenues.
In a linked opinion piece, the authors pointed out that the high numbers of innovative yet costly drugs entering the market were “leading to increasing budgetary distress.”
“Research into the rational use of expensive oncology drugs is needed to ensure that budgets of health systems are well spent,” they wrote. “Oncology drugs often not only reach the market while lacking proof of added benefit but also succeed in recovering their research and development costs in a relatively short period. All this raises serious questions about the alignment of marketing approval and reimbursement policies with the actual clinical benefit offered to patients.”
Better Alignment Needed
The team pointed out that differences in evidentiary requirements between the EMA and HTA bodies often lead to positive benefit-risk but negative added benefit assessments. They called for better alignment between regulatory and reimbursement processes, particularly for drugs approved through expedited pathways, to promote development of the most effective drugs for patients with the greatest needs.
Global spending on oncology drugs is projected to rise from $167 billion in 2020 to $269 billion in 2025. Still, the researchers wrote, oncology drugs are increasingly approved on “less robust evidence, raising concerns about misalignment of incentives in the pharmaceutical market with patient interests.”
Brinkhuis said: “There is a need to discuss in what situations there is a high unmet medical need that makes approval and reimbursement based on preliminary evidence, such as single-arm trials and surrogate endpoints, acceptable for both regulatory authorities and HTA bodies.”
She added that regulatory authorities, HTA bodies, and pharmaceutical companies should engage in joint discussions on development plans for new products.
New EU HTA regulation will include processes for patient involvement, with joint clinical assessments for oncology drugs beginning in 2025 to help better define and assess what matters most to patients, while also addressing potential conflicts of interest. “Both regulatory authorities and HTA bodies are more and more working toward actively involving the patient perspective and considering it in their decision-making,” Brinkhuis said.
Medscape Medical News has approached the EMA for comment.
