[Apropos of the exceptionality of new cancer drugs]

Given that new oncology drugs are frequently involved in departures from the normal technology appraisal processes, the main objective of this article is to analyze these decision-making processes, as well as their impact on the regulation of new approaches to selecting novel drugs and the regulatory steps to deal with risk-sharing arrangements. This analysis was carried out by selecting two drugs, trastuzumab and imatinib, both agents being representive examples of the innovative targeted therapies introduced in the last decade, and by reviewing some international experiences (particularly those of Australia, England, and Wales, pioneers in the application of technology appraisals), centered mainly - but not exclusively - on these two drugs. The review of these experiences indicated that other concerns apart from efficiency are especially important in the evaluation of this type of innovations. In England and Wales (where, in contrast to Australia, the "rule of rescue" has not been adopted) the controversial decision-making processes have resulted in new approaches to the appraisal of end-of-life treatments. Moreover, overlapping or sequential risk-sharing arrangements have led to regulatory steps, paying attention to possible problems of inconsistencies in decision-making and inequalities of access. The case of Spain is critically discussed, with special emphasis on the risk of favoring the application of micro formulae (such as risk-sharing arrangements) over macro formulae. Finally, a strategy is suggested, based on the recently approved Royal Decree-law 9/2011.