Challenges of translational research in cutting edge medical technology: A case of first-in-human (FIH) trials of medical applications of nanotechnology

Brief Summary

The translation of discoveries in basic science into safe and effective clinical applications is essential for improving health care. Unfortunately, translational research is a long, arduous and resource intense process filled with high levels of uncertainty and renown for resulting in a series of failures before succeeding in its goal. This is particularly true when cutting-edge medical technologies are translated into improved diagnostic tests or better and safer therapeutic options. The aim of this doctoral research project was to describe the challenges involved in the translational research of medical applications of nanotechnology with a particular focus on first-in-human (FIH) trials.

This exploratory research project was situated in a larger study and included in-depth qualitative interviews to gain insight into the perspectives of various stakeholders involved in planning, conducting or evaluating FIH trials in nanomedicine. Translational research is a multi-stakeholder enterprise and often requires the negotiation of various conflicting values and goals. FIH trials mark the moment in translational research when an experimental intervention is tested in human beings first time and poses the highest level of uncertainty with regard to the safety and efficacy. In-depth interviews conducted with expert stakeholders based in Europe and North America led to a greater understanding of the various challenges in translational nanomedicine and potential solutions.

The key results of this research project indicate a number of challenges in the current practices of translational research. The results are mainly focused on insights obtained from stakeholder interviews and point to issues such as (1) diverse definitions of nanomedicine and its impact on the translational research, (2) financial, ethical and regulatory challenges in the translational research, (3) inconsistent disclosure practices with regard to the ‘nano’ nature of investigational products in patient information sheets and informed consent forms, (4) implications of the current practices of the researchers of including patients with end-stage cancer and no effective treatment options in FIH trials on the scientific value of the early phases of the translational research and (5) factors such as public health emergencies that can accelerate decision making and mobilize resources to initiate FIH trials with investigational products despite significant uncertainty related to their safety and efficacy.

To understand all the complexities of translational research in nanomedicine, there is a need to further investigate the role of various regulatory guidelines and to define ‘meaningful’ public engagement in science and drug development. Both these aspects critically hinge upon scientific integrity and the public’s trust in science and regulatory mechanisms. Although these topics were not investigated in this research project, our results clearly indicate the need to explore them further. We conclude that while discussing the regulation of nanotechnology, careful attention must be paid to each application on a case-by-case basis. We argue for the critical examination of current procedures in regulatory assessments rather than creating new and special regulations for nanomedicine. Finally, we believe that the challenges in translational nanomedicine discussed in this project are also applicable to any cutting-edge medical technology.

Dr Priya Satalkar

Institute for
Biomedical Ethics

Universität Basel
Bernoullistrasse 28
CH-4056 Basel

Tel. +41 61 207 17 82
Fax +41 61 207 17 80