Eur Rev Med Pharmacol Sci 2011; 15 (11): 1355-1356

Grouping of molecularly targeted anti-cancer agents based on cost-effectiveness analysis

U. Tirelli, M. Berretta, A. Bearz, A. Carbone*

Department of Medical Oncology, National Cancer Institute, Aviano (Italy)
*Division of Pathology, National Cancer Institute, Aviano (Italy)

Corresponding Author: Umberto Tirelli, MD; e-mail:;

Dear Editor,
One of the challenges in clinical oncology today is how to cost-effectively treat patients with molecularly targeted anti-cancer agents. In the hope that the novel drugs may have higher activity and lower toxicity than “older” antiblastic agents, patients and the scientific and medical communities have pushed the health-care systems to accelerate their approval for clinical use. However, the high cost of molecularly targeted anti-cancer treatments constitutes a substantial obstacle to their widespread use, which poses significant challenges to clinicians who care for patients with cancer. For instance, the cost of FOLFOX (folinic acid, fluorouracil, and oxaliplatin) and bevacizumab for the treatment of a very common disease, such as metastatic colorectal cancer, has a considerable economic impact1-3.
To overcome this critical issue, one of the first goals is the identification of patients who are most likely to benefit from these treatments through predictive and prognostic factors. Global negotiations, such as those implemented by the European Medicines Evaluation Agency network (EMEA), will likely be more helpful and effective than those undertaken by individual groups. For instance, within the European Union, different countries have different attitudes to bevacizumab, although EMEA has approved it for treatment of colorectal cancer, non-small cell lung cancer, breast cancer and renal cancer: in Great Britain bevacizumab has not been approved for use against any of those neoplastic diseases (NICE guidelines)4, whereas in Italy the Italian Medicines Agency (AIFA) and Roche have recently negotiated a “risk-sharing” agreement for a 50% reimbursement of the cost of bevacizumab for the first 6 weeks of treatment5.
Molecularly targeted anti-cancer agents really differ based on their impact on survival in patients with different tumors.
“Imatinib-like drugs”, i.e., agents like Imatinib that are employed successfully against chronic myeloid leukemia6 and GIST7, have highly improved survival. “rituximab- and trastuzumab-like drugs” used for the treatment of lymphomas8 and breast cancer9 that have a rather good impact on survival.
Conversely, “erlotinib-, gefitinib-, cetuximab-, bevacizumab-like drugs used”, for example, against lung10 and gastrointestinal tumors11 have a medium impact on survival (a few months).
From an economic point of view, the cost of the treatment of patients with drugs in the last group will be relatively high because of their poor impact on cancer survival, whereas the treatment with drugs in the previous groups will be really cost-effective as a result of their higher impact on survival.
Then, we strongly suggest that the EMEA approves only drugs that have a favorable impact on survival by prolonging it of at least six months, and reject those that only have a poor impact on survival by prolonging it of just a few months or even weeks.
This theoretical proposal, that would be beneficial for ethical and economic reasons, would allow a higher number of European patients to be treated with the same drugs. At present access to treatment is not equally distributed across the EU, and some European citizens are prevented from receiving a new antineoplastic agent. Different local policies and costs are discriminating patients within the EU.
Moreover, we think that more information, regarding tumor biology characteristics, are needed to choose the right target anticancer treatment to obtain the best result, in terms of objective response, progression free survival and finally overall survival. We believe that this way is the only solution to optimize the pharmaceutical costs and to yield the financial of the public health sustainable.


1)    Omura K. Advances in chemotherapy against advanced or metastatic colorectal cancer. Digestion 2008; 77(Suppl 1): 13-22.
2)    Corral MJ, Clopès A, Navarro M, Germà JR, Borràs JM. Impact on budget of new drugs for colorectal cancer treatment. Med Clin (Barc) 2007; 129: 134-136.
3)    Schrag D. The price tag on progress–chemotherapy for colorectal cancer. N Engl J Med 2004; 351: 317-319.
6)    Leitner AA, Hochhaus A, Müller MC. Current treatment concepts of CML. Curr Cancer Drug Targets 2011; 11: 31-43.
7)    Blay JY. A decade of tyrosine kinase inhibitor therapy: Historical and current perspectives on targeted therapy for GIST. Cancer Treat Rev 2011; 37: 373-384.
8)    Saini KS, Azim HA Jr, Cocorocchio E, Vanazzi A, Saini ML, Raviele PR, Pruneri G, Peccatori FA. Rituximab in Hodgkin lymphoma: is the target always a hit? Cancer Treat Rev 2011; 37: 385-390.
9)    NCCN Clinical Practice Guidelines in OncologyTM, Breast Cancer, Version 2.2011,
10)    NCCN Clinical Practice Guidelines in OncologyTM, Non-Small Cell Lung Cancer, Version 1.2012,
11)    NCCN Clinical Practice Guidelines in OncologyTM, Colon Cancer, Version 1.2012,

To cite this article

U. Tirelli, M. Berretta, A. Bearz, A. Carbone*
Grouping of molecularly targeted anti-cancer agents based on cost-effectiveness analysis

Eur Rev Med Pharmacol Sci
Year: 2011
Vol. 15 - N. 11
Pages: 1355-1356