Type: Peer Reviewed Publications

Abstract Purpose: Restrictive eligibility criteria induce differences between clinical trial and “real-world” treatment populations. Restrictions based on prior therapies are common; minimizing them when appropriate may increase patient participation in clinical trials. Experimental Design: A multi-stakeholder working group developed a conceptual framework to guide evaluation of prevailing practices with respect to using prior treatment as…

Abstract Purpose: In clinical research, eligibility criteria promote patient safety and optimize the evidence generated from clinical trials. However, overly stringent eligibility criteria, including laboratory requirements, may limit enrollment, resulting in delayed trial completion and potentially limiting applicability of trial results to a general practice population. Experimental Design: Starting in 2018, a working group consisting…

Timely access to therapies that treat serious illnesses is critical for patients, particularly for those with rare or serious disease types that have no current treatments. Congress and the Food and Drug Administration (FDA) have addressed this by periodically establishing programs designed to expedite different steps associated with the development and review of drugs and…

Abstract The field of cell therapy is rapidly emerging as a priority area for oncology research and drug development. Currently, two chimeric antigen receptor T-cell therapies are approved by the US Food and Drug Administration and other agencies worldwide for two types of hematologic cancers. To facilitate the development of these therapies for patients with…

Abstract Recent scientific progress is, in some cases, leading to transformative new medicines for diseases that previously had marginal or even no treatment options. This offers great promise for people affected by these diseases, but it has also placed stress on the health care system in terms of the growing cost associated with some new interventions. Effort has been taken to…