EMEA's draft guideline "The Requirements for First-in-Human Clinical Trials for Potential High Risk Medicinal Products"

The Biotechnology Industry Organization (BIO) submits these comments on the European Medicines Agency’s (EMEA’s) draft guideline The Requirements for First-in-Human Clinical Trials for Potential High Risk Medicinal Products. BIO represents more than 1,100 biotechnology companies, academic institutions, state biotechnology centers and related organizations across the United States and 31 other nations. BIO members are involved in the research and development of healthcare, agricultural, industrial and environmental biotechnology products. Our members invest heavily in the research and development of biotechnology and pharmaceutical products in the European Union (EU) and elsewhere, and employ thousands of highly skilled persons in the EU. We appreciate the opportunity to submit comments on the draft guideline.

The draft guideline highlights some of the key points to consider when taking Investigational Medicinal Products (IMPs) that “have a potential for high risk in firstin-man administration” into clinical testing. We agree that some new IMPs can be classified as highly novel molecules which show a high degree of species specificity and for which there is little or no prior knowledge of the risk/benefit ratio in humans. These molecules require special attention in defining and communicating the risk management strategy. However, the appropriate requirements for entering first-in-human (FIH) clinical trials may not be equally applicable to all such IMPs, and many of the most important requirements will be equally applicable to both “high-risk” and non-high-risk products. Consideration should be given to re-focusing the guideline to a ‘points to consider’ document on risk management strategies and dose-setting for all FIH clinical trials. The emphasis of the guideline should be more focused on risk mitigation strategies through the integrated analysis of all pre-clinical data and the appropriate design of clinical trials. This would remove the need for a definition of “high risk”, while still addressing appropriate risk management strategies.