OBJECTIVES: To better understand the views of various stakeholders on patient involvement in pharmaceutical industry research and to describe the current state of knowledge of best practices. METHODS: A targeted review of the literature on patient involvement with pharmaceutical drug development and research. Approximately 150 articles were reviewed, including academic journal articles; corporate, foundation and government reports; and existing literature reviews. RESULTS: Preliminary results demonstrate a growing recognition within the industry of the value of understanding patient experiences and perspectives. Patient involvement in research varies tremendously, from superficial and tokenistic to highly substantive (e.g., patient involvement in every stage of the research process, from framing research questions to dissemination of results). Research on the outcomes of patient involvement is largely qualitative, making it difficult to arrive at generalizable conclusions. Positive results often are assumed without being empirically demonstrated. That said, the literature suggests patient involvement can lead to benefits for all stakeholders, including increased legitimacy and transparency, better alignment of research agendas with outcomes that patients value, higher data quality and better communication of results to lay audiences. Other sources, however, highlight barriers to incorporating a patient-centered perspective, such as resistance to diverting research resources, defensiveness relating to researchers’ expertise and traditional practices and the time and effort necessary to include patients in an already lengthy R&D process. CONCLUSIONS: While there is a growing literature on patient involvement in industry research, the current lack of robust evidence on the outcomes of patient involvement hinders effective inclusion of patients. Further development of a science of patient involvement is a necessary step for broadening and strengthening industry inclusion of patients in the drug development process.