Proceedings - Pathways for Successful Translation of New Imaging Agents and Modalities: Phase III Studies.

Significant advances continue in the development of cancer-specific molecular imaging agents and modalities for diagnosing, staging, and treating various cancer types. For these advances to successfully translate to widespread clinical use, both regulatory approval by the U.S. Food and Drug Administration (FDA), and completion of the reimbursement approval process with the Centers for Medicare & Medicaid Services (CMS), must be achieved. To this end, methods for expediting these processes are being addressed by both agencies. This report summarizes a one-day meeting held May 15, 2017 with officials from the National Cancer Institute (NCI), FDA, and CMS; members of the Society of Nuclear Medicine and Molecular Imaging (SNMMI), the World Molecular Imaging Society (WMIS), the American College of Radiology (ACR), the Radiological Society of North America (RSNA), the International Society for Strategic Studies in Radiology (IS3R), the Medical Imaging and Technology Alliance (MITA), molecular imaging scientists from some major hospitals/institutions and industry representatives, who joined together to learn about the pathways of approval, coverage, and payment decisions. The meeting's focus was three-part: i) to broaden the conversation to other agents/modalities and address both the reimbursement and regulatory components; ii) to present a current view of FDA regulatory innovations in cancer imaging development, a past and present view of PET/MI agent approval processes, and a current view of CMS reimbursement protocols; and iii) to discuss scientific issues involved in orchestrating agency-required Phase III clinical trials as presented through three case examples: a PET probe targeting the prostate specific membrane antigen (PSMA) for imaging prostate cancer, an optical antibody probe for image-guided head and neck cancer surgery targeting epidermal growth factor receptor (EGFR), and ultrasonography with targeted microbubbles against a neovasculature target, kinase insert domain receptor (KDR), for ovarian cancer. Additionally, the meeting served to provide more clarity to researchers on trial design, to delineate more uniformly required steps investigators should take to successfully translate their innovations. Reported here are the meeting's discussion items and recommendations for continued honing of the approval and reimbursement processes investigators must navigate.