Clinical Trial Data Often Flawed According to OHSU & Mayo Clinic AZ Researchers

May 4, 2019 | Approved Drugs, Cancer, Oncology, Standard-of-Care, Study Control Arms

The US Food and Drug Administration (FDA) may approve some cancer drugs on the basis of trials with suboptimal control arms according to a new research conclusion from investigators associated with Oregon Health & Science University and Mayo Clinic Arizona. According to the researcher’s abstract published in JAMA Oncology, there was no empirical evaluation of the quality of control arms involved with randomized clinical trials for anticancer drug approvals to date. This was a surprise to TrialSite News team.

The researchers sought to answer the question “how often does anticancer drugs approved by the US Food and Drug Administration (FDA) based on randomized clinical trials designed with a suboptimal control arm? The answer was surprising based on the research findings.  The OHSU & Mayo Clinic Arizona affiliated team designed a quality improvement study and reviewed 143 anticancer drug approvals issued by the FDA from January 1, 2013 to July 3, 2018. They discovered 16 (17%) of 96 anticancer drugs approved for market were approved based on comparing them with suboptimal control arms.

The team has concluded although the FDA is approving more cancer drugs, they may be doing so based on questionable assumptions—leading the inquiring mind to question whether the new drug is actually superior to an established specific standard-of-care therapy. The ultimate result is the physician and the patient may be unsure of the benefit introduced by these new agents.

We recommend other research groups also consider this methodology to determine what are the implications for existing cancer patients. We also suggest an economic study to determine what are the cost differentials involved for new agents that have been approved based on suboptimal control arms.  If new agents are being approved that are not superior to standard-of-care, yet cost more, then this is highly relevant for the medical establishment (not to mention consumers) to understand.  We include contacts for the researchers below.

Lead Research/Investigator

Hilal Talal, MD, Mayo Clinic, Phoenix, AZ, Division of Hematology and Oncology

Mohammad Bassam Sonbol, MD, MD, Mayo Clinic, Phoenix, AZ, Division of Hematology and Oncology

Vinay Prassad, MD, MPH, Oregon Health & Science University, Division of Hematology Oncology, Knight Cancer Institute


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