Clinical relevance of therapeutic benefit in anti-cancer drugs assessment

Abstract

The added values of new medical technologies can be defined in many different ways. Patient-oriented approach requires consideration of clinical relevance and cost necessary to obtain additional health effects. Well-designed clinical trials provide the highest standard in evaluating therapeutic effectiveness. Therefore, the American Society of Clinical Oncology has developed a document defining minimal — clinically relevant — therapeutic benefit offered to patients with selected tumors. Obtaining these values should be the aim for anti-cancer drug manufacturers and organizations designing clinical trials. However, reliable verification of patient’s therapeutic benefits seems to be particularly difficult in respect to the financing of drugs that are used off-label, novel therapeutics, and in rare cancers. In response to the growing needs for selection of the most valuable medical technologies that will also stay within a limited budget, some countries have designed mechanisms that are effective in describing the added value by assessing the efficacy, toxicity and cost of anti-cancer drugs. They initiate the discussion on optimization of value in cancer care.