National Cancer Institute Formulary: A Public‐Private Partnership Providing Investigators Access to Investigational Anticancer Agents

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Access to and feasibility of genomic sequencing has moved personalized medicine from the realm of abstract theory into current practice. One example of bringing personalized medicine to oncology is a new clinical trial that the National Cancer Institute (NCI) recently launched. The NCI‐Molecular Analysis for Therapy Choice (NCI‐MATCH; NCT02465060) study is a precision medicine clinical trial that enrolls patients with solid tumors or lymphomas, irrespective of specific histology, who have progressed on standard therapy by assigning treatments based on whether individuals carry tumor mutations/amplifications/translocations in a genetic pathway for which an available, molecularly targeted therapy may provide clinical benefit. A targeted next‐generation sequencing panel, as well as a defined panel of immunohistochemistry assays, is performed to determine study eligibility. Each mutation‐agent pairing constitutes an independent phase II trial conducted under the single NCI‐MATCH umbrella.1
In addition to the many scientific challenges that developing the NCI‐MATCH trial involved, an extensive administrative and regulatory support structure was created to enable over 20 simultaneous phase II studies to proceed. This support structure facilitated the participation of many pharmaceutical partners who are providing targeted investigational and marketed agents for the NCI‐MATCH study.
As genomic sequencing of tumors becomes more prevalent, and as insurance companies begin to cover the costs of sequencing, requests for targeted agents for preclinical research, clinical trials, and expanded use have become more frequent.2 An efficient mechanism that could pair clinical investigators with pharmaceutical and biotechnology collaborators would benefit investigators, companies, and the public. Furthermore, there is reason to believe that somatic mutations in tumors evolve in response to therapy.3 Unfortunately, for these patients, there may not be standard therapeutic alternatives. Access to investigational as well as marketed agents for cancer clinical trials, particularly for combination studies targeting multiple molecular pathways with agents from different companies, is often difficult and time‐consuming, to the point that many critically important trials are never initiated. This situation presents a major challenge and an opportunity for NCI, pharmaceutical companies, and investigators.
In response to this problem, and in collaboration with the US Food and Drug Administration (FDA) and the Pharmaceutical and Biotechnology Industries as part of Vice President Biden's White House Moonshot initiative, NCI has created a virtual drug formulary to support novel clinical investigations. NCI's goal is to create a mechanism that leverages NCI drug distribution resources and collaborative agreements (providing a structured intellectual property environment and data access) to rapidly provide promising agents to academic investigators. The resulting trials will be conducted in NCI's extensive network of comprehensive cancer centers nationwide. These trials will include (but are not limited to) studies of novel agent combinations and molecularly targeted agents for patients whose tumors carry rare somatic mutations.
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