Personalized medicine and economic evaluation in oncology: all theory and no practice?

The clinical definition of personalized medicine (PM) is closely related to that of pharmacogenomics. Ideally, PM could lead the pharmaceutical industry to differentiate products by subgroups of patients with the same pathology and find new gene targets for drug discovery. Here, we focus on the potential impact of PM on the design of clinical trials and economic evaluations limited to oncology (its first and main field of application). Then, we assess the European economic evaluations focused on trastuzumab and cetuximab, the two drugs usually mentioned as emblematic examples of targeted therapies. Clinical results of PM in oncology have not been as encouraging as hoped so far. Of course, economic evaluations on targeted therapies cannot help overcome the lack of clinical evidence for most of them. The two paradigmatic examples of cetuximab and trastuzumab indicate that the methodological implications on economic evaluations debated in the literature are more theoretical than practical.

Long-term modeling on HPV vaccination: do we really need any more?

The human papillomavirus (HPV) is closely related to cervical cancer. In 2007, the EMA approved two vaccines, a bivalent and a quadrivalent one, launched at three-dose schedules and very high prices worldwide. We describe what happened in the EU and what might change in the near future from an economic perspective. HPV vaccination is now established in most EU countries. The main target group of the programs is girls aged 10-14 years. Many western countries used competitive tendering to purchase the two vaccines, achieving considerable savings. The extension to males has been a hotly debated issue. The sex limitation implies that this vaccination cannot by definition achieve a 'herd immunity' effect. EMA recently approved a two-dose schedule for both vaccines that should lead to savings, although it is hard to predict how the forthcoming nonavalent vaccine will affect the market situation. Several economic evaluations based on long-term models have been published on the HPV vaccination in the recent years, using official list prices as a baseline. Most of these models can be considered mere exercises in long-term forecasting. Recently, further long-term models have been published with two- and three-dose schedules as alternatives, and the nonavalent vaccine. We wonder what added value they give for public policy purposes.

Cost-effectiveness of trastuzumab in metastatic breast cancer: mainly a matter of price in the EU?

Trastuzumab (TR), a monoclonal antibody approved by EMA in 2000 and one of the first examples of "targeted therapy", is indicated to treat human epidermal growth factor receptor 2 (HER2) positive breast cancer. TR, whose patent will expire in 2015 in Europe, has been judged positively for reimbursement by most public authorities in the EU. Here we critically review the existing evidence on TR in metastatic breast cancer (MBC), in line with the multidisciplinary health technology assessment (HTA) approach, to assess whether the existing evidence supports TR positive reimbursement decisions taken in MBC by EU health authorities. We did a literature search for the main HTA topics (efficacy, quality of life and ethics) on the PubMed international database (2000-2013). Then, we did a specific literature search to select the full economic evaluations (FEEs) conducted in EU countries focused on TR as first-line innovative therapy in MBC. We retrieved scant evidence in the literature to support TR reimbursement in MBC. We found only two clinical trials and their results were unclear because of the large proportion of patients who crossed over. Moreover, the quality of methods was poor in all four European FEEs selected. This example of HTA exercise on a mature monoclonal antibody in a specific indication casts doubts on how often the reimbursement decisions taken by EU health authorities in emotional pathologies like cancer are rational. These decisions should at least be reconsidered periodically on the basis of the latest evidence.

Vaccination planning and vaccine prices in a decentralizing country - Italy.

This paper gives an overview, in the era of regionalization, of vaccination planning and vaccine price management in the Italian National Health Service. In particular, we analyse the current National Vaccination Plan (NVP) and end with two "case studies" of the latest entries in the Italian vaccination calendar, comparing HPV and PCV vaccines, the most expensive ones in Italy at present. The present NVP put an end to the long period without official documents for vaccination planning, mainly reflecting the controversial relationships between the national and regional tiers. However, this document is not really useful for planning from the health professionals' point of view, lacking epidemiological information. Thorough systematic assessment of the new, expensive vaccines is becoming a real priority in the light of current financial difficulties. In this perspective, the two examples discussed have given different results so far, starting from a heterogeneous situation of potential market competition.

A critical systematic review of budget impact analyses on drugs in the EU countries.

BACKGROUND: Budget impact analysis (BIA) is a relatively recent technique that is supposed to be complementary to more established economic evaluations (EEs). OBJECTIVE: We reviewed the BIAs published on drugs in the EU since December 2008, to assess whether these studies have improved in quality in the last few years. METHODS: We conducted a literature search on the international databases PubMed and EMBASE. The selected articles were screened using a two-step approach to assess (1) their main methodological characteristics and (2) the level of adherence to the latest BIA definition. The assessment was made by two independent reviewers and any disagreement was resolved through discussion. RESULTS: Eventually, 17 articles were reviewed. Thirteen referred to a stand-alone BIA not accompanying a full EE, only nine focussed on a new treatment, 15 were sponsored by the manufacturer of the drug of reference, all but one claiming savings for healthcare budgets. The quality of methods was poor in many of the studies, and only a few of them attempted to estimate real local costs in a credible way. Therefore, the crucial items that in theory make a BIA different from other types of EEs were often the major points of weakness of the studies reviewed. CONCLUSIONS: Our review confirmed that the BIA is not yet a well-established technique in the literature and many published studies still fail to reach an acceptable quality. In particular, BIAs funded by pharmaceutical companies appear to be tailored to show short-term savings induced by new, highly priced products.