title: Which Metrics Are Appropriate to Describe the Value of New Cancer Therapies?
abstract: Patients with certain cancers are treated with curative intent, but for others the results are less favorable and different therapeutic approaches are needed. Early data suggest that new therapies, which modulate immune responses to cancers, may have potential for long-term survival in a proportion of cases. Therefore, it is timely to consider whether metrics generally used to describe the medical value of therapies for patients with common solid tumors remain appropriate for therapies with curative potential. Literature reviews were conducted to define how various stakeholders describe cure in oncology and to identify the endpoints used in clinical trials for selected solid tumors. The results showed that "cure" is described using various terms that can be divided broadly into lack of disease progression, eradication of cancerous cells, and survival. The review of trial endpoints showed frequent use of median overall survival (OS) and progression- and response-related endpoints. Because these endpoints were mainly described in the context of chemotherapies that are not generally curative, they may not adequately capture outcomes of new therapeutic modalities with potential for long-term survival. More appropriate endpoints may include mean OS, cure fraction, and OS rate at landmark time points.
authors.affiliation: Cancer Research UK Centre, University of Southampton, Southampton SO16 6YD, UK.
authors.forename: Peter
authors.initials: P
authors.lastname: Johnson
authors.affiliation: Department for Health Economics, University of Bielefeld, 33501 Bielefeld, Germany.
authors.forename: Wolfgang
authors.initials: W
authors.lastname: Greiner
authors.affiliation: PAREXEL International, 160 Euston Road, London NW1 2DX, UK.
authors.forename: Imad
authors.initials: I
authors.lastname: Al-Dakkak
authors.affiliation: Bristol-Myers Squibb, Route 206 and Province Line Road, Princeton, NJ 08543, USA.
authors.forename: Samuel
authors.initials: S
authors.lastname: Wagner
mesh_terms: D002986:Clinical Trials as Topic; D018572:Disease-Free Survival; D023381:Endpoint Determination; D006801:Humans; D009369:Neoplasms / Q000453:epidemiology* / Q000473:pathology / Q000628:therapy*; D016896:Treatment Outcome