Experts Suggest Prioritizing Price and Benefit, Allowing Negotiations for Targeted Cancer Drugs

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Experts recently proposed 3 steps to promote targeted cancer drugs that yield clinical benefits while reducing overall price growth.

In the United States, spending on cancer drugs continues to substantially increase and targeted cancer drugs contribute significantly to this growth. Experts recently proposed 3 steps to promote targeted cancer drugs that yield clinical benefits while reducing overall price growth.

“The distorted pricing of marginally effective drugs risks crowding out the capacity of the US health system to pay for highly effective cancer drugs or other therapies of public health importance, potentially jeopardizing valuable innovation and escalating out-of-pocket expenses,” the authors explained. “The combination of high prices and marginal effectiveness is unsustainable.”

First, the report suggested the FDA should develop guidance on minimum clinically meaningful effect sizes for cancer drugs. This would explain past FDA guidance and establish a consensus-driven definition of minimum clinically meaningful effect sizes. In order to achieve this, multidisciplinary advisory councils of scientists, oncologists, patient advocates, and industry representatives should work together.


The authors noted that clinical experts have already supported this principal, as the American Society for Clinical Oncology endorsed a minimum absolute improvement of 3 to 6 months in overall survival over best available treatment for drug trials among metastatic disease patients.

“Guidance could separately address cases in which, despite little or no change in median overall survival or hazard ratios, small proportions of patients experience large gains and the challenge of estimating benefits when pivotal trials involve head-to-head comparisons against active controls, thereby potentially underestimating the overall efficacy of novel agents,” the authors wrote. “By defining norms, the FDA would encourage manufacturers to design trials that demonstrate clinically meaningful benefits.”

The next step the experts proposed stated that Medicare should negotiate for targeted cancer drugs. Specifically, it was suggested that Congress could direct CMS to conduct a demonstration project. In this project, Medicare should negotiate the prices of targeted cancer drugs paid for by Parts B and D and it is also authorized to apply limited formulary tools to marginally effective targeted cancer drugs.

The last recommended step to promote targeted cancer drugs while reducing overall price growth was that guidelines should prioritize drugs by benefit and price. The authors explained that evidence-based guidelines are best suited to complete this and such guidelines should distinguish marginally effective drugs from highly effective drugs. These guidelines should also promote price transparency by reporting the estimated monthly price of cancer drugs.

“Successfully implementing steps to limit the use of high-priced, marginally effective drugs will be difficult; patients with life-threatening diseases may expect access to drugs despite their high costs and limited benefits,” the report concluded. “Nevertheless, the ultimate beneficiaries of these changes will be patients, who deserve the substantial efficacy, reduced toxicity, and enhanced value that were the original promise of targeted cancer drugs.”


Bekelman JE, Joffe S. Three steps toward a more sustainable path for targeted cancer drugs. JAMA. 2018;319(21):2167-2168. doi:10.1001/jama.2018.3414.