Using patient-initiated study participation in the development of evidence for personalized cancer therapy.

Personalized cancer therapy offers the promise of delivering the right treatments to the right patients to improve patient outcomes and quality of life, while reducing exposure to ineffective therapies and the cost of cancer care. Realizing this promise depends in large part on our ability to generate timely and sufficiently detailed information regarding factors that influence treatment response. Generating this evidence through the traditional physician investigator-initiated clinical trial system has proved to be challenging, given poor recruitment rates and low compliance with requests for biospecimen collection. As a result, our current understanding of treatment response is inadequate, particularly for cancer therapies that have been in use for many years. Patient-initiated study participation may offer a new model for evidence generation that capitalizes on strong patient interest in furthering research to inform better and more tailored cancer therapies. In this approach, patients are engaged and recruited directly by the sponsor of an Institutional Review Board-approved study, and patients subsequently drive the participation of their health care providers to facilitate collection of required data and tissue samples. The ultimate goal of these studies is to generate evidence of sufficient quality to inform regulatory decisions (i.e., labeling changes for marketed therapies to reflect populations most likely to respond) and treatment selection. Here, we describe a hypothetical prospective observational study in non-small cell lung cancer that could serve as a model for patient-initiated study participation applied to understand molecular determinants of treatment response. Key elements discussed include study design, patient engagement, and data/biospecimen collection and management principles.