Many new oncology drugs approved in the EU lack proof of added benefit

Many cancer drugs recently approved by the European Medicines Agency (EMA) lack clear proof of added benefit. This is particularly the case for drugs that are granted via special processes, intended to accelerate the approval of promising treatments. A research team from Utrecht University draws this conclusion today in the scientific journal The BMJ. Their study also indicates that almost all oncology drugs, including these fast-tracked drugs, manage to recover their substantial research and development (R&D) costs relatively quickly after approval.

A new study conducted by researchers from Utrecht University sheds light on the dynamics of added benefit and revenues of oncology drugs approved by the EMA between 1995 and 2020. The findings, published today, reveal significant insights. The research team consisted of Francine Brinkhuis, Wim Goettsch, Aukje Mantel-Teeuwisse, and Lourens Bloem, affiliated with the Pharmacoepidemiology and Clinical Pharmacology division at Utrecht University.

The study aimed to evaluate the added benefit and financial outcomes of oncology drugs, while exploring different EMA approval pathways. Using data from various organizations and publicly available financial reports, the researchers identified 458 evaluations of added benefit for 131 oncology drugs and revenue data for 109 oncology drugs.

Fast-tracked drugs

The researchers found that many of these drugs lack proof of added benefit. In fact, 41% of the 458 evaluations of added benefit were negative, indicating that no added benefit was (or could be) determined. This seemed particularly the case for medicines that were approved through special regulations called expedited pathways.

This is a system of regulations that facilitates a faster market entry for drugs intended for patients with an unmet medical need. To do so, less evidence is accepted at the moment of drug approval, provided that further studies are performed to gather additional evidence of efficacy and safety. This limited evidence can be one of the reasons that added benefit cannot be established.

Expedited approval may be a way to have patients benefit from a new drug, but we question whether added benefit can eventually be proven

The authors emphasize that approving drugs through these so-called expedited pathways is not necessarily a bad thing. “There is often a great need for new drugs”, says Lourens Bloem, one of the researchers who contributed to this study. “This is especially true in oncology, where the medical need is often high. Expedited approval may be a way to have patients benefit from a new drug but we question whether added benefit can eventually be proven, once additional data are available. Therefore, it is essential to continue to monitor the actual added benefit of these types of drugs, and assess the costs involved.”

Recovering R&D costs

The study reveals that more than half of the oncology drugs, including those with minimal or no added benefit, are able to recover their research and development (R&D) costs within three years. According to the researchers, this challenges the pharmaceutical industry's claim that high drug prices are necessary to offset R&D expenses.

While lower levels of added benefit might result in lower revenues, these are still sufficiently high to recover R&D expenses

Acceptable revenues

The analysis shows that medicines with higher levels of added benefit generally lead to greater revenues. “While lower levels of added benefit might result in lower revenues, these are still sufficiently high to recover R&D expenses”, the research team writes in The BMJ.  This may diminish the pharmaceutical industry’s incentive to develop high value drugs, the researchers argue, “because pharmaceutical companies might be satisfied with the revenue generated from lower value drugs”.

Reimbursement processes

Since global spending on oncology drugs is expected to rise significantly in the coming years, the authors argue that regulatory and reimbursement processes should be aligned better. This could help stimulate pharmaceutical companies to keep pursuing drugs that offer significant added benefits, and not settle for drugs that offer minimal added benefits but still generate an acceptable revenue.

“We urge policymakers to consistently reassess both ongoing and new initiatives aimed at ensuring fair, affordable, and sustainable patient access to innovative and expensive drugs”, the authors write in an opinion article, also published in The BMJ. “Additionally, we emphasise the importance of investigating and promoting the rational use of these drugs in clinical practice. This approach strives for a future where drug development and limited resources align more closely with real-world benefits to patients.”