Many oncology drugs approved by the European Medicines Agency (EMA) between 2005 and 2020 provide ‘little or no added benefit’ for patients, according to new research.
This was particularly true for medications that had ‘fast-tracked’ approval pathways, the Dutch researchers said.
Many drugs with higher benefit ratings were associated with more significant revenues for the associated pharmaceutical companies, they added.
The study, published in the BMJ, highlights the need for better alignment between regulatory and reimbursement processes, enabling the most effective drugs to be developed and utilised.
The added benefit of a medication refers to its therapeutic value compared with other treatments, and it can aid decisions about treatment priorities and inform drug-related decisions.
Oncology drugs are increasingly being approved on less robust evidence, and as global spending on these drugs is projected to rise from $167bn in 2020 to $269bn in 2025, and concerns have been raised about the misalignment of incentives in the pharmaceutical market with patient interests.
This retrospective cohort study aimed to evaluate the added benefits and revenues of oncology drugs, explore their association and investigate potential discrepancies between added benefits and revenues across different approval pathways of the EMA.
The researchers collected ratings from evaluation reports from several organisations, including HTA agencies from Europe and the United States, medical oncology societies and a drug bulletin.
To assess growth revenues, they collected information on the financial performance of pharmaceutical companies and compared the revenue figures with previously reported estimates of research and development (R&D) expenses.
The revenue figures were correlated with the added benefit ratings to analyse the performance of the pharmaceutical company against the benefits of the medications they provide.
Of the 458 added benefit ratings identified, nearly two-thirds of the medications have limited or no added benefits. Some 41% of the drugs had a negative or non-quantifiable added benefit, and 23% had a minor added benefit. The remaining drugs had a major (13%) and a substantial added benefit (23%).
Compared with drugs approved under the standard pathway (SMAs), drugs which were approved under conditional marketing authorisation (CMA) and authorisation under exceptional circumstances (AEC) were more likely to have a negative or non-quantifiable added benefit rating, at 57% and 47% respectively compared to the SMA rate of 36%.
Revenues generated from the oncology drugs were generally found to outpace minimum R&D costs quickly, with median cumulative revenues exceeding minimum R&D costs of $166m within two years, the median R&D costs of $684m within three years, and the maximum R&D costs of $2,060m within just over five years after market entry. By eight years of market entry, 91% of drugs surpassed the median R&D costs.
The researchers found a correlation between the level of added benefit a drug had and the associated revenues. Cumulative revenues three years after market entry generally increased with the level of added benefit. For medications with major and substantial added benefit, cumulative revenues three years after market entry were $502m and $506m higher respectively than drugs without benefit.
The researchers stated that the findings underscore ‘the need for better alignment between regulatory and reimbursement processes, particularly for drugs approved through expedited pathways’.
They concluded: ‘It is crucial for policy makers to assess whether the current regulatory and reimbursement incentives are properly structured to promote and facilitate the development of the most effective drugs for patients with the greatest needs.
‘These findings could inform policy initiatives in the field of drug regulation and reimbursement, contributing to equitable and sustainable patient access to innovative treatments.’
In 2020, researchers found that while increasing numbers of oncology drugs are being approved my medicines regulators, cancer patients wait longer for innovative new cancer drugs than for more conventional treatments.
