Current development of targeted therapy and multidisciplinary approach for new treatment modalities, has contributed to
increase the outcome in cancer patients. Based on the new knowledge of molecular features of the tumor, it is urgently needed
to clarify some aspects on new treatments.
Actually the innovative approach in oncology is represented by: a) the development of anticancer targeted drugs; b) growing
member of elderly cancer patients; c) constant augmentation of so-called “cancer survivors”; d) infection-related neoplasms
(HIV, HPV, EBV, HHV8 and Hepatitis Virus); and lastly, e) the colossal healthcare cost to support all these new treatments.
In order to comprehend well these “new” issues for the upcoming future, it is necessary to make some reflections [1]. The
end of the 2nd Millennium has been characterized by the introduction of new molecules in cancer treatment allowing “target
therapy”. Targeted drugs are principally small inhibitor molecules (i.e. erlotinib, imatinib etc.) and monoclonal antibody (i.e.
trastuzumab, rituximab, ipilimumab, nivolumab, etc.) that interfere directly in the molecular mechanism of proliferating
pathways, reducing traditional toxicities of antiblastic chemotherapy (AC).
The mechanism of action of these new targeted molecules is universal in all neoplastic cells carrying the corresponding
cancer target, but some of these have been approved by drug labeling studies. Efficacy failure is recorded when the therapeutic
indication has been extended to other types of cancer (e.g., erlotinib in metastatic pancreatic cancer). However, an appropriate
study on resistance, efficacy of long-term treatment and outcome is mandatory for tailored cancer therapies. Furthermore, new
drugs indication must only be approved by an appropriat clinical trial. Currently these issues exist for drugs with strong
evidence to improve personalized label or schedule (i.e., cetuximab used only in KRAS mutational status, duration of adjuvant
trastuzumab); or an existing anticancer agent for another therapeutic indication (i.e., imatinib in sclerodermatous). Often,
pharmaceutical corporations do not support these studies due to rarity of the disease. These problems occur recurrently in the
context of a low subset of targeted population (i.e., cancer with mutations frequency lower than 2%) [2].
Targeted therapy has introduced new economic reflections: for example substituting traditional chemotherapy (often needs
vascular access and intravenous infusions) to oral small molecule inhibitors, eliminates some costs by moving therapy from the
hospital to home [3]. On the other hand, if therapy includes monoclonal antibodies (mAbs), the costs dramatically increase
(Table 1) calculated that the cost of $30,790 of the eight weeks treatment regimens containing bevacizumab or cetuximab in
colorectal cancer was calculated, compared with $63 for the same period of 5-fluorouracil, leucovorin and oxaliplatin regimen
[4].
Current treatments of cancer are derived through validated clinical trials, that include, often, innovative patented drugs.
Nevertheless, global contemporary model of community healthcare systems highlights that new therapeutic care must be
carried out at an equal or lower cost. Besides, trials evaluating the precise economic impact of cancer treatments are still few.
Advanced methods to assess cost-effectiveness, cost-utility and cost-benefit in the cancer organizations have been introduced.
Such as the National Institute for Health and Clinical Excellence (NICE). NICE has established several clinical Advisory
committees, which stimulate Academic and Pharma societies to design studies, including economic models for personalized
healthcare [5]. Personalized medicine is a promising model which includes genomic tests due to specific tailored treatments [6].
It is well known that Pharmacogenomics tests, performed before drug treatment, minimize toxicity and maximize benefits and
provide higher quality of life [7]. NICE also provides a method to measure Quality-Adjusted Life-Years (QALY), combining
data on outcomes, analytical assay and cost-effectiveness for entire treatment.
The up-coming methods to measure the QALYs will lead to multidisciplinary treatment approach for decreasing the costs of
these treatments.
Progress in cancer treatment is driven by cumulative knowledge of the molecular cancer pathways, and the subsequent
development of systemic agents that inhibit critical neoplastic pathways. However, not all new molecules with biological target
are automatically approved. The base for their US Food and Drug Administration (FDA) approvals differ: an upgrading in
overall survival (OS) compared with a current therapy is not always required; [8] toxicity profiles and progression-free survival
are also considered as important factors [9].
Finally, we contemplate that the recent progresses have provided exceptional opportunities to identify prognostic and
predictive markers of efficacy of antiblastic treatments [10]. Genetic markers can be used to identify patients who will benefit
from therapy, excluding patients at high risk to develop severe adverse events, and adjust dosing [11].
Furthermore, trials evaluating the pharmacoeconomic impact of genotyping assay on cancer therapy are still few. Also, the
major issue to consider for genotyping is the need to interpret laboratory data results by an oncologist [12]. However,
inadequate education of the physicians regarding Pharmacogenomics field has been observed [13]. The contemporary
knowledge of healthcare professionals concerning pharmacogenomics is still limited, and teaching programs are poor in this
field. However, new method to measure cost-effectiveness in cancer therapy have already been proposed [14].
