Study Spotlights Debate on Comparative Effectiveness and Costs of New Cancer Drugs

Cost in the cancer care setting has gained immense importance in a highly payor-conscious drugs market. A commentary published in the Journal of the National Cancer Institute (JNCI), titled "How Much is Life Worth: Cetuximab, Non-Small-Cell Lung Cancer, and the US$440-Billion Question", has set out some of the issues surrounding this debate, questioning not only the link to costs, but also the benefits derived, and the role of the regulators, professional bodies, as well as healthcare professionals. Authors Tito Fojo and Christine Grady have focused on Bristol-Myers Squibb (U.S.) and Merck Serono (Switzerland)'s Erbitux, detailing the results of a European trial examining cetuximab plus cisplatin and vinorelbine as a treatment for non-small-cell lung cancer (NSCLC). The key takeaways from the report are as follows:

• Cost-Benefit: The cetuximab trial highlighted a 1.2-month prolongation of survival in NSCLC patients, raising the question of treatments with marginal benefits being sold at very high cost. Avastin's approval in combination with carboplatin and paclitaxel for metastatic NSCLC also added to this debate.
• PFS Versus Quality of Life: Lack of quality-of-life analysis is another area of concern contributing to the benefits debate. The emphasis on progression-free survival (PFS) is increasingly being used to demonstrate drug efficacy with not much detail on the tolerability profile of the cancer drugs.
• Role of Regulators and Physicians: Interestingly, the above areas have culminated in some recommendations from the authors, who call for: clear guidance from oncologists on the issue of cost of marketed products; and the discouragement of research studies towards survival advantage of two months or less, off-label use, and administering drugs that have a marginal benefit. They also highlight the role of the American Society of Clinical Oncology (ASCO) and the U.S. FDA in recognising and endorsing drugs with marginal benefit.

The study is available here.

Outlook and Implications

The report has highlighted some relevant issues in the cancer drug market pertaining to costs and survival benefits. Essentially, some of the main endpoints used to demonstrate drug efficacy have been questioned. The overall benefit element has been sidelined in favour of progression-free survival, which, although an important assessment, does not itself provide the full context. The occurrence of adverse events affecting quality of life is being ignored to present a more positive picture. Clearly, the authors' view will elicit some debate testing the question of "what constitutes benefit" in relation to costs.

The recommendations provide for a greater role for oncologists in the pricing of drugs. In examining the role of the FDA and ASCO, the authors have indicated that endorsement by these bodies have often been translated to the cost structure and the amount that patients are willing to pay for drug benefits. This is also highlighted in the off-label usage setting, where the study indicates that benefits, unless demonstrably outweighing the toxic effects, should not be reimbursed by insurance firms.