Our expanding knowledge of the molecular biology of malignancy, the related identification of therapeutically-important targets, and the subsequent development of systemic agents that inhibit critical kinases and pathways, have all contributed to great excitement and progress in cancer treatment. In kidney cancer alone—long considered a drug-resistant disease—over five agents have been approved by the US Food and Drug Administration since 2005. Not all new drugs for a cancer indication are necessarily blockbusters. The basis for their US Food and Drug Administration approvals vary—an improvement in overall survival compared with an existing therapy is not always required, and even when present, may be quite modest; other clinically important factors such as toxicity profiles and progression-free survival are also considered. However, what is remarkably consistent among these newer drugs regardless of the reasons for their ultimate approval (and as highlighted in the article by Kantarjian et al that accompanies this editorial) are their high prices. Table 1 in the article by Kantarjian et al lists 20 such agents and their REDBOOK costs—even the least expensive runs over $5,000 monthly or per cycle. While drug development and related research are costly, growing more so, and certainly contribute to escalating drug prices, the authors highlight that this is not the whole story. They argue that “what the market will bear” seems to be a central pricing consideration and priority among pharmaceutical companies. Even when more drugs become available in a particular therapeutic space suggesting a potential for increased competition surrounding the choice of agents, prices seem to remain high; similarly, there is no clear “correlation between the actual efficacy of a new drug and its price.” Other peer countries regularly pay less for the same agents without apparent differences in outcomes. The authors provide three examples to illustrate their assertions—tyrosine kinase inhibitors in the therapy of chronic myeloid leukemia; targeted therapies for metastatic solid tumors such as kidney cancer; and recently approved therapies for melanoma. They note that increasing regulatory burdens imposed on drug development, the growing presence and role of intermediaries between investigators and pharmaceutical companies, and substantial company budgets for marketing and related education efforts add to the cost of drug development with subsequent effects on drug pricing. In addition, they highlight two phenomena that contribute to higher drug prices but are not part of the drug development process: the prohibition of Medicare from negotiating lower prices for cancer drugs and pay-for-delay strategies where pharmaceutical companies with expiring drug patents pay fees to delay the introduction of competing generics. They propose sensible solutions to address these issues, including a more value-based system for determining initial drug price that considers cost-effectiveness data. The ultimate goal is to identify Justum Pretium – the Just Price. Articles in the lay press during the last several months highlight the timeliness of the Kantarjian et al article. Last October and as mentioned in their paper, Bach et al wrote in the New York Times regarding the decision at Memorial Sloan-Kettering Cancer Center not to add ziv-aflibercept to the hospital formulary for the treatment of colorectal cancer. The rationale provided was that it provided no apparent or significant advantage in efficacy (median survival advantage of approximately 1.5 months when added to chemotherapy), toxicity profile, nor ease of administration when compared with bevacizumab, but cost over twice as much. Of interest, in follow-up to the editorial, the cost of ziv-aflibercept was reduced. More recently, and perhaps encouraged by this outcome, specialists in chronic myelogenous leukemia published a similar commentary regarding the high price of therapy for that disease which received prominent coverage in the lay press. These two examples are notable given their grass roots nature; furthermore, they resonate with many at this time of growing appreciation and need for the documentation of value in health care. While there are drug-company sponsored programs to facilitate access to drugs for patients, of which the Merck-sponsored one to treat river blindness is a heroic example, overall such mechanisms are not a comprehensive solution for most cancer patients or scenarios. There is a need for better solutions, although the best strategy moving forward is controversial. Others have previously expressed concerns regarding the impact and sustainability of the growing cost of cancer care—of which the high price of related treatment and supportive care drugs is part of the picture—and similarly called for new strategies to contain costs without adversely affecting quality, safety or other outcomes. These and other experts and investigators provide some insights that complement and give additional perspective to those provided by Kantarjian et al. JOURNAL OF CLINICAL ONCOLOGY E D I T O R I A L VOLUME 31 NUMBER 28 OCTOBER 1 2013
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Nov 1, 2013
Michael Vassalos + 2
