In a recent commentary published in the journal Blood, an international group of more than 100 clinicians and scientists criticized pharmaceutical companies that market costly cancer drugs to treat chronic myeloid leukemia (CML). The authors argued that the prices of six CML drugs have reached unsustainable levels that jeopardize patients' treatments and economic well-being (http://dx.doi.org/10.1182/blood-2013-03-490003). The article has sparked reactions that underscore the complexity of the issue, which encompasses questions of how drug values are determined, where moral boundaries are drawn and what mechanisms can be employed to reduce the divide between commercial interests and patient welfare.

When the tyrosine kinase inhibitor imatinib (Gleevec/Glivec) was approved in the US in 2001, it cost $30,000 annually per patient. The initial price tag was expected to cover its development costs, and Novartis followed common marketing practice by fixing the price of imatinib slightly above that of interferon, then the standard of care for CML. However, the unexpected survival benefits conferred by imatinib prompted the company to hike the drug's price to $92,000 per annum by 2012. This probably set the pricing standard for newer drugs used to treat CML, including bosutinib (Pfizer), ponatinib (ARIAD Pharmaceuticals) and nilotinib (Novartis). The price hike also reflects a general trend, in which cancer drug prices on average have roughly doubled in the last decade. This rise places a growing toll on both healthcare systems, which carry the bulk of the costs, and on individual patients who may have to co-finance their treatments.

Drug companies argue that the inordinately high prices for CML drugs have to cover development costs, which for cancer drugs are estimated to average $1 billion. Industry representatives also noted to us that the price of CML drugs must offset a relatively small patient pool (∼26,000 in the US) and the cost of drug development failures. In addition, their pricing reflects the drugs' notable life-prolonging potential. In pharmacoeconomic terms, healthcare systems in many countries value a life year gained from a treatment at $50,000–100,000 (Value Health 12, 80–87, 2009). However, in recent years, the price of many newly marketed cancer drugs has far exceeded the actual clinical benefits they provide (J. Clin. Oncol. 29, 2543–2549, 2011).

In response to this disparity, some regulatory agencies and insurance companies have refused to approve payment for certain high-cost treatments. In the UK, the National Institute for Health and Care Excellence (NICE) evaluates the cost-effectiveness of drugs that have been approved by the European Medicines Agency (EMA) and issues funding guidance to the National Health Service (NHS). The NHS covers the cost of drugs that NICE approves for a given disease. In 2008, NICE declined to approve sunitinib for the treatment of kidney cancer, and in 2011 it similarly rejected erlotinib for the treatment of non–small-cell lung cancer, questioning its high cost and patient benefit. NICE has since revised both decisions following the manufacturers' agreement to provide the drugs to the NHS at a discounted price.

In the US, medical insurance companies have also refused to reimburse patients for some cancer drugs, including sunitinib, or to differentially reimburse costs for oral and intravenous cancer drugs. A bill recently approved by the General Assembly of North Carolina (House Bill 609) puts patient co-payments for oral cancer drugs, which make up about 25% of cancer medicines, on par with those for intravenously administered cancer drugs. Likewise, the proposed federal Cancer Drug Coverage Parity Act (H.R. 1801) would require insurance companies to cover intravenous and oral cancer drugs equivalently. Medical expenses are the most frequent cause of personal bankruptcy in the US, and the high cost of drugs is a factor in poor adherence and treatment failure. Ultimately, legislation that would allow healthcare agencies to negotiate directly with drug companies or that would cap drug prices might be needed to curtail further cost escalation of cancer treatments. Pharmaceutical companies realize that not everyone can pay for their drugs and have created patient assistance programs, some of them benefiting underdeveloped countries or underprivileged segments of the population. For example, the Max Foundation, together with Novartis, runs a global program, the Glivec International Patient Assistance Program (GIPAP), to distribute its CML drugs in poor countries. In the US, Novartis has reported that it provides 5,000 patients lacking sufficient insurance with free imatinib or nilotinib every year. These initiatives are laudable and highlight the impact of patient advocate organizations, but they solve only a small part of the problem of access to affordable medicines.

At least one medical center has also entered the fray. Last year, Sanofi and Regeneron cut the price of the colorectal cancer drug aflibercept in half after Memorial Sloan-Kettering Cancer Center publicized its decision not to use the drug because it cost twice the price of its predecessor, bevacizumab, without showing additional treatment benefits. The problem is clear and the stakes are high: drugs that are too costly may cost lives. The question is how to move from ad hoc solutions to a systematic approach to cancer drug valuation that won't break the bank.