The Complex Relation Between Clinical Outcomes, Drug Approval, and Drug Price

Does an FDA approval guarantee clinical benefit of a drug? How does it influence drug price?

Does an FDA approval guarantee clinical performance of a drug? Likewise, should clinical performance dictate drug price? These were the questions that researchers of a paper published in the Annals of Oncology aimed to address.

The authors clustered 51 molecules that were approved by the FDA during a 15-year period from 2000 to 2015 for the treatment of advanced solid tumors. Using 2 value frameworks, 1 developed by the American Society of Clinical Oncology (ASCO) and the other by the European Society for Medical Oncology (ESMO), the clinical benefit of the drugs was graded. Approval information was sourced from publicly accessible FDA data and drug price was evaluated based on Medicare, US Veterans Health Administration, and United Kingdom market systems.

Only 73%, that is 37 of the 51 drugs could be evaluated using the 2 frameworks. The ESMO framework classified 5 drugs at the lowest grade (grade 1); 9 at grade 2; 10 at grade 3; 11 at grade 4; and 2 at grade 5 (highest grade). Only 13 drugs (35%) were found to have clinical benefit per ESMO’s clinical framework. The ASCO framework found a median drug value of 37 (range, 3.4 to 67). The authors conclude that their analysis failed to provide evidence of an association between the price charged for recently approved cancer drugs and their clinical benefit.

Clinicians around the globe are mounting a resistance against the incessant rise in drug prices. An article published in the latest issue of the journal Cell calls for creating a partnership between academic researchers and drug manufacturers to maintain the brisk rate of innovation, but at sustainable drug prices. They raise a particularly valid argument regarding immunotherapy drugs that are now being tested in combination clinical trials—with the average cost of the combination priced at $250,000, these treatments are unaffordable for most individuals and a tremendous burden on the healthcare system. In the United States, clinician researchers at MD Anderson Cancer Center and health policy researchers at Memorial Sloan Kettering Cancer Center have spearheaded the pushback against rising oncology drug prices.

Without a doubt, the success rate of some of the newer agents, many of which are “personalized” based on a person’s genetic makeup, would be high. Immuno-oncology agents like pembrolizumab and nivolumab have seen success in a growing number of tumor types, which increases the eligible patient population—however, the drug price have not dipped. As pointed out by authors in the August 2016 issue of Evidence-Based Oncology™, high costs invariably result in patient access issues.

The authors in the Cell paper sum their argument as, “With a lifetime risk of developing cancer of close to 40%, the problem is clear.”