| Abstract: |
The development of radiopharmaceuticals is essential for nuclear medicine, implementing new findings in molecular biology, drug development, and in many fields of clinical specialities, increasing its pace in particular in the field of PET and therapeutic applications. New radionuclides are emerging; an increased need of radiopharmaceuticals as companion diagnostics and new insights in targeting of in particular tumor cells open new applications in diagnosis and therapy involving radiopharmaceuticals. A major bottleneck in the process of translating new radiopharmaceuticals into clinical applications lies in the increasing regulatory burden in the pharmaceutical field in general and radioactive drugs in particular. In almost every country today, radiopharmaceuticals are considered as drugs or “medicinal products,” as the European regulations define it, whereby similar principles apply. A major principle of pharmaceutical development is the implementation of clinical trials before any new drug is considered appropriate for its intended use, aimed to ensure efficacy and safety of the product. Even though some countries allow the use of new drugs outside clinical trials, in a kind of “compassionate use” way, which has in the past been one of the major routes to bring new diagnostic and therapeutic radiopharmaceuticals into clinical application, it is not clearly regulated and has become less applicable over the last decade. Clinical trials also provide data that are recognized by authorities and also by other clinical specialities outside nuclear medicine. Such data are also an absolute requirement for commercialization of new drugs and have to be generated in a very documented way within the commercial development. Therefore, it is of ever more importance in the development of new radiopharmaceuticals to follow controlled clinical trials in the translational process from the preclinical phase to routine patient application. In this chapter the major steps required for initiating clinical trials with radiopharmaceuticals are described. As there are some formal differences between two major regulatory frameworks, Europe and the United States, recommendations are given on how to conduct clinical trials in these two regions. This chapter will cover the major regulatory background in this scenario and describe the documentation requirements and major hurdles in this process focusing on pharmaceutical issues involved. © 2025 Elsevier B.V., All rights reserved. |
