Smileband health topics

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 al4that accompanies this editorial) are their high prices. Table 1 in the article by Kantarjian et al lists 20 such agents and their REDBOOK costs are 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.