London School of Hygiene & Tropical Medicine

Patients with cancer could be willing to wait for greater certainty of the benefit of new cancer drugs before they are formally approved by regulators, according to new survey results.

The study, led by researchers from the London School of Economics and Political Science (LSE) and the London School of Hygiene & Tropical Medicine (LSHTM), enrolled over 850 people in the United States (US) to take part in the survey, where participants were asked to make a series of hypothetical choices between potential cancer drugs.

In the US, new drugs or medicines are approved by the US Food and Drug Administration (FDA). As part of their evaluation, the FDA is faced with a trade-off between approving new drugs quickly or delaying approval until there is certainty about the clinical benefit they provide. In recent years, the FDA’s accelerated approval pathways have drawn criticism from some doctors and experts who have questioned whether the right balance is always struck between speed and certainty.

Previous research has also shown that most cancer drugs approved through the FDA’s accelerated approval pathway do not help patients with cancer to live longer.

Participants enrolled in the survey had either been diagnosed with cancer themselves or knew a close friend or family member previously or currently diagnosed with cancer. Researchers were able to model their choices to find out what was most important to individuals regarding new cancer drugs and how long they would be willing to wait to ensure a new drug provided meaningful clinical benefit.

The study, published in The Lancet Oncology, is the first to provide an estimate of the duration individuals would be willing to wait for greater certainty. Depending on their health status and other factors, individuals indicated they would prefer to wait up to 22 additional months to be sure a medicine worked before it was approved by the FDA.

Ajay Aggarwal, Consultant Clinical Oncologist and Professor of Cancer Services and Systems Research at LSHTM, and co-author of the study, said: “As an oncologist, providing new treatment options for patients with cancer that offer meaningful improvements in survival and quality of life is critical, however expediency should not come at the cost of robust evaluation especially when we know many drugs often fail to offer the benefits that patients themselves hope for.”

Robin Forrest, a researcher in LSE’s Department of Health Policy and lead author of the study, said: “People with experience of cancer value faster access to new cancer drugs, particularly when they have limited treatments available. But when faster access comes at the expense of certainty that a drug extends survival, then this might not always the case.”

Dr Huseyin Naci, Associate Professor of Health Policy at LSE and senior author of the study, said: “These results show that patients are not willing to access new cancer drugs at any cost. Some participants expressed their potential willingness to wait longer than necessary to run clinical trials and achieve greater certainty on the clinical benefit of new cancer drugs.”

Moving forward, the authors call for increased transparency and justification for the use of accelerated approval for cancer drugs, and a greater emphasis on outcomes that are most important to patients in the drug approval process.

Publication

Robin Forrest, et al. Preferences for speed of access versus certainty of the survival benefit of new cancer drugs: a discrete choice experiment. The Lancet Oncology. https://doi.org/10.1016/S1470-2045(24)00596-5

This news story is based on an original press release from the London School of Economics and Political Science (LSE).