title: Clinical and outcome research in oncology. The need for integration.
abstract: Cancer is one of the main healthcare problems in Europe. Although significant progress has recently been made, long-term survival is still disappointing for most common solid tumours. The explosion of information has strengthened the need to create and sustain coordinated interaction between technology, biology, clinical research, clinical practice and health policy. A simple process based on automatic and passive translation from bench to clinical research and eventually to the bed side is usually assumed but cannot be taken for granted. A critical role might be played by Outcome Research (OR), defined as the discipline that describes, interprets, and predicts the impact of various influences, especially interventions, on final endpoints (from survival to satisfaction with care) that matter to decision makers (from patients to society at large), with special emphasis on the use of patient-reported outcomes (PRO). Recently, under pressure from several parts of society, the FDA, recognizing the need for faster drug approval, has modified existing regulations and created new rules to allow anti-cancer drugs to be approved more quickly and, in certain but quite common circumstances, single arm trials and surrogate endpoints to be used as measures of clinical benefit. In this context, the faster approval process may lead to drugs being marketed without there being a complete picture of how effective or safe they are. The FDA move to speed up drug approval, together with the use of not fully validated surrogate endpoints, give OR the unique opportunity to help understand the value of drugs that have received accelerated approval. Despite this opportunity, OR has yet to demonstrate its role in this specific setting and provide proof of the validity, reliability and added value of its primary endpoint measures when evaluated in a broader context. The implementation of lines of OR in the development and evaluation of anti-cancer drugs hinges upon the availability of specific knowledge, methods, instruments and resources and upon their appropriate integration in the mainstream of clinical research. In the USA specific interdisciplinary projects have been launched by the NCI. In Europe there is a lack of such initiatives. The correct placement of OR in the anti-cancer drug development process will guarantee the highest possible standard of validity and reliability of OR at European level and better integration of both translational and outcome research in the mainstream of clinical research into anti-cancer drugs, thus speeding up the introduction of the results of patient-oriented translational clinical research into clinical practice.
authors.affiliation: Istituto di Ricerche Farmacologiche Mario Negri, Milan, Italy. apolone@marionegri.it
authors.forename: Giovanni
authors.initials: G
authors.lastname: Apolone
mesh_terms: D000970:Antineoplastic Agents / Q000627:therapeutic use*; D035843:Biomedical Research / Q000379:methods*; D002986:Clinical Trials as Topic; D017277:Drug Approval; D005060:Europe; D006801:Humans; D008495:Medical Oncology; D009369:Neoplasms / Q000188:drug therapy*; D017063:Outcome Assessment, Health Care / Q000379:methods*; D011788:Quality of Life; D014481:United States; D014486:United States Food and Drug Administration
chemical_list: D000970:Antineoplastic Agents