Dr and patient Fernando Zhiminaicela from Pixabay
Patients with cancer could be willing to wait for greater certainty of the benefit of new cancer drugs before they are approved by the US Food and Drug Administration (FDA), suggests a new study published in The Lancet Oncology.
When evaluating new drugs, 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, FDA’s accelerated approval pathways has drawn criticism from some doctors and experts who have questioned whether the right balance is always struck between speed and certainty. Previous research has shown that most cancer drugs approved through FDA’s accelerated approval pathway do not help patients with cancer to live longer.
Researchers at LSE enrolled over 850 people in the United States to take part in a survey study. All participants had either been diagnosed with cancer themselves or knew a close friend or family member previously or currently diagnosed with cancer. In the study, participants made a series of hypothetical choices between potential cancer drugs. 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.
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.”
The study is the first to provide an estimate of the duration individuals would be willing to wait for greater certainty that a new cancer drug would provide meaningful clinical benefit. 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.
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.”
Professor Ajay Aggarwal, Consultant Clinical Oncologist and Professor of Cancer Services and Systems Research (London School of Hygiene and Tropical Medicine), co-author on the study added: “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.”
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.