Low benefits spur alternative drug cost proposals


It’s no secret that cancer drugs are among the most expensive medical treatments in the United States, and now, new research reveals some high-priced cancer drugs may yield little benefit for patients.

A recent analysis of 71 oncology indications approved by the Food and Drug Administration between 2011 and 2017 found that overall survival gains were marginal for drugs approved by the FDA based on overall survival (OS) data. The majority of the 71 indications (75%) demonstrated no statistically significant improvement in patient-reported outcomes (PROs), according to the study (JAMA Oncol. 2019 Jul 3 doi: 10.1001/jamaoncol.2019.1760).

More than half of the indications evaluated demonstrated neither an OS benefit nor a PRO improvement post approval, the study found.

While the researchers did not analyze cost, a number of the cancer drugs that demonstrated little benefit come with a high price tag. Cabozantinib (Cabometyx) for example, approved for the treatment of medullary thyroid carcinoma and advanced renal cell carcinoma (RCC) during the study period, did not demonstrate an overall survival benefit post approval, findings showed. Cabometyx, approved based on survival data, had a 2016 wholesale acquisition cost of $13,750 for a 1-month supply. Olaparib (Lynparza) meanwhile, approved for the treatment of advanced ovarian cancer during the study period, also showed no overall survival benefit post approval. The 2017 wholesale acquisition cost for olaparib was $13,482 for a 30-day supply.

Investigators noted in the study that evaluation of OS can be challenging or unfeasible in some instances and is complicated by factors such as use of crossover trial design.

The findings emphasize the need for a sharper eye on how regulatory authorities approve drugs, said Chadi Nabhan, MD, senior author of the study and chief medical officer at Aptitude Health based in Chicago.

Dr. Chadi Nabhan, chief medical officer at Aptitude Health speaks to a group of community oncologists in February 2019. Courtesy of Dr. Nabhan

Dr. Chadi Nabhan, chief medical officer at Aptitude Health speaks to a group of community oncologists in February 2019.

“We all want our patients to receive the best and the latest and the most important and innovative drug they can possible get, as long as these drugs show a benefit,” said Dr. Nabhan, a hematologist and medical oncologist. “We need to look critically at making sure drugs getting approved are truly helping patients by extending their lives or improving their quality of life.”

Growing questions about the benefits of some cancer drugs come as a push to reconsider the pricing of medications to better account for value gains momentum. A number of proposals have recently emerged that would revamp the current payment structure for prescription drugs with the aim of lowering costs and improving access. Value-based pricing proposals are not without challenges, namely defining what value truly means, said Leonard Saltz, MD, a medical oncologist at Memorial Sloan Kettering Cancer Center in New York.

Dr. Leonard Saltz, medical oncologist at Memorial Sloan Kettering Cancer Center, New York

Dr. Leonard Saltz

“We’re all quite clear there is a huge absence of connection between cost and value,” Dr. Saltz said in an interview. “There is also a real absence of the definition of, ‘What is value?’ I think that ultimately we have to rely on defining value by its absence. By that I mean, where do we say, this is insufficient value?”


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