High Cost of Cancer Drugs Does Not Reflect Clinical Benefit

Roxanne Nelson, RN, BSN

May 13, 2020

The cost of cancer drugs continues to escalate with each new product that comes on the market, often presenting formidable challenges to both patients and healthcare systems. But a new analysis suggests these high costs cannot be justified with respect to clinical benefit.

The analysis considered 65 oncology drugs launched in the past decade and found no association between clinical benefit and monthly treatment cost.

The study was published online May 1 in the Lancet Oncology.

"Prices of cancer drugs should be better aligned with their clinical benefit to improve access to beneficial medicines and enable limited resources to be used for treatments that offer patients improved outcomes," said lead author Kerstin Noëlle Vokinger, MD, JD, PhD, LLM, assistant professor for public law and digitalization, health law, and regulatory sciences at the University of Zurich, Switzerland.

Clinician "Tough Discussions" With Patients

The findings of this study are similar to findings of previous studies, noted an expert who was approached for comment.

There is already considerable evidence that the price of new drugs does not reflect the drug's value, said Yousuf Zafar, MD, MHS, FASCO, associate professor of medicine and public policy at Duke University in Durham, North Carolina.

"Because price is not linked to value, manufacturers are incentivized to develop more drugs in an existing class or drugs with limited benefit," he told Medscape Medical News. "If value played a role in the drug pricing and approval process, we could disincentivize the production of the tenth checkpoint inhibitor or the drug that improves survival by just weeks."

Existing value frameworks (such as those used in the current study) provide a good start for this process, he noted, but payers will ultimately need to make difficult decisions about coverage.

"But that responsibility does not end with payers," Zafar emphasized. "Clinicians should have the tough discussion with patients about how much benefit is actually expected from the drugs we prescribe.

"In many instances, I have had patients decline drugs like regorafenib [Stivarga, Bayer] for colorectal cancer when I explain the magnitude of expected benefit vs the potential for toxicity ― both physical and financial," he added.

Details of the Findings

Drug prices have been a hot topic of discussion for some time, especially in the United States, where they are the highest in the world. Determining the value of a drug by pegging cost to benefit has been proposed by experts and the two major professional organizations.

The American Society of Clinical Oncology (ASCO) developed a value framework (ASCO-VF) that defines the value of a drug on the basis of clinical benefit, toxicity, and cost. The European Society of Clinical Oncology (ESMO) released a Magnitude of Clinical Benefit Scale (MCBS), which offers a "rational, structured, and consistent approach" to stratifying drugs' clinically meaningful benefits.

The new study used both of these frameworks to assess the association between the clinical benefit of approved cancer drugs and their cost in the United States and four European countries (England, Switzerland, Germany, and France).

The researchers identified all new drugs indicated for adult cancers that were approved by the US Food and Drug Administration between January 1, 2009, and December 31, 2017, and by the European Medicines Agency through September 1, 2019. Because the ESMO-MCBS tool can only be used with regard to solid tumors, the evaluation of clinical benefit was restricted to drugs indicated for nonhematologic cancers.

The median monthly treatment costs for the included cancer drugs was highest in the United States ($13,179), followed by England ($6202), Switzerland ($5696), Germany ($5121), and France ($4866).

When the team assessed the 46 drugs approved for solid tumors, there was no significant difference in monthly treatment costs between drugs that had high clinical benefit and those of low benefit (using the ASCO-VF, two-sided P = .25; using the ESMO-MCBS, P = .25).

As an example, for the treatment of prostate cancer, cabazitaxel (Jevtana, Sanofi-Aventis) had lower clinical benefit scores than abiraterone (Zytiga, Janssen) (ESMO-MCBS score, 2 vs 4), yet the cost of the two drugs was similar in the United States ($10,531 vs $10,887) and Germany ($3311 vs $3340) and was slightly higher in England ($4554 vs $3568) and Switzerland ($5292 vs $3475).

With regard to individual countries, no significant associations were observed between cost and clinical benefit using the ESMO-MCBS (for the United States, P = .16; for England, P = .98; for Switzerland, P = .54; for Germany, P = .52; and for France, P =.40). The same was true when the team used the ASCO-VF except for France, where there was a correlation (for the United States, P = .56; for England, P = .47; for Switzerland, P = .26; for Germany, P = .23; and for France, = .037).

The authors conclude that the prices of cancer drugs should correspond more closely with their clinical importance, "such as by prioritizing drugs with low or uncertain benefit for price negotiations, to improve access to beneficial medicines and enable finite resources to be used for treatments that offer patients improved outcomes and optimal value."

"I recognize two explanations for the lack of strong association between price and clinical benefit seen in this paper," said Scott Huntington, MD, MPH, of Yale School of Medicine, New Haven, Connecticut, who was approached for comment.

"First, this work and others provide compelling evidence that prices of recent cancer therapeutics are not strongly tied to clinical utility, particularly in the US, where payers have little leverage over coverage or pricing of cancer drugs," he told Medscape Medical News.

In countries such as the United Kingdom, formal cost-effectiveness analyses do inform negotiations, drug prices, and ultimately coverage decisions, thus, "we would expect cancer drug prices in the UK to be more closely associated with clinical benefit, but that is not seen in this analysis," Huntington pointed out. "It is likely that the cost measure — using monthly drug cost — and clinical utility — framework composite scores — in this study is less precise than what is typically used in formal cost-effectiveness analyses."

The bottom line, he added, is that it is probably safe to conclude that there's a long way to go before cancer drug prices are strongly associated with their clinical benefit.

"Incorporating the spirit of available value-based oncology frameworks, particularly concepts of treatment-related toxicity and degree of innovation, alongside traditional cost-effectiveness modeling is likely an important step towards value-based pricing of cancer therapeutics," he said.

The study was funded by the Swiss Cancer Research Foundation (Krebsforschung Schweiz). Vokinger received grants from Swiss Cancer Research Foundation during the conduct of the study. Several coauthors also report receiving various grants. Huntington is employed at an academic medical center with 340B status; he has consulted for Celgene, Bayer, Genentech, Pharmacyclics, and AbbVie and has received research (institutional) funding from DTRM Biopharma, Celgene, and TG Therapeutics. Zafar has relationships with Shattuck Labs, AIM Specialty Health, Copernicus WCG, Discern Health, Family Reach Foundation, McKesson, RTI Health Solutions, Vivor, and AstraZeneca (institutional).

Lancet Oncol. Published online May 1, 2020. Abstract

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