By: Michael Kurilla, M.D., Ph.D., NCATS Director of the Division of Clinical Innovation
As a result of the release of the latest CTSA suite of FOAs, there has been vigorous discussion regarding the usage of ‘translational science’ and ‘translational research.’ And while some use these terms interchangeably, others regard these concepts as distinct as geology versus philosophy. Here then is the NCATS perspective on this topic. Beginning with the NCATS Strategic Plan (https://ncats.nih.gov/strategicplan/introduction), we define ‘translation’ as “the process of turning observations in the laboratory, clinic and community into interventions that improve the health of individuals and the public — from diagnostics and therapeutics to medical procedures and behavioral changes.” I tend to shorten this to simply ‘turning science into health.’ Translational science then is “the field of investigation focused on understanding the scientific and operational principles underlying … the translational process.” There is a spectrum within translational science that seeks to traverse the path from initial basic research activities through preclinical and clinical activities to implementation within routine health care and finally public health outcomes at the population level. Those activities across this spectrum comprise ‘translational research.’ A couple of examples will be provided to highlight this distinction.
During my time at NIAID, I was suborned upon numerous taskforces. One such taskforce was the NIH Nanotechnology taskforce. The Office of Science and Technology Policy (OSTP) had concerns that the US was not doing enough to be a world leader in nanotechnology. We met with both academic and industrial communities. One particularly enlightening conversation was with scientists from Dupont which was the world leader in automotive coatings. They described related activities where they were developing coatings for airplane wings that provided advantages in terms of performance (less drag), improved strength, more durability, etc. They were utilizing nanotech, but they lamented the total lack of any scientific principles underlying the research they were conducting. Minute changes in design could result in massive alteration in performance. As such, their efforts were all trial and error requiring shipping numerous prototypes to airline companies for testing in wind tunnels. Even when a desirable prototype was identified, any subsequent minor modification could have disastrous results. Their plea was for the USG to target resources (to others) to understand the underlying scientific principles so that they could proceed in a more directed manner towards practical products. In this case, Dupont was conducting nanotech translational research, while asking for someone else to study the translational science of nanotech.
For an example closer to home, consider clinical trials. The basic framework for conducting clinical trials is well established and the conduct of clinical trials comprises a large component of clinically focused translational research activities. The vast bulk of clinical trial experience has been gained through drug and vaccine trials. For other applications such as non-pharmaceutical interventions, the standard approaches do not always work well. Furthermore, when approaching rare diseases, there may simply be an insufficient number of subjects to adequately power a trial to allow traditional statistical analyses to be performed. It might be easy to conclude that we need to wait for more patients with that rare disease in order to develop an intervention, but looking at this through a translational science lens, the question becomes one of addressing a clinical question that does not rely on large numbers of subjects. Note that this question is not unique to the specific rare disease under study. Developing and validating ‘N-of-1’ trial methodology, employing alternative statistical analyses (Bayesian perhaps), or developing and validating alternative endpoints may offer practical and implementable options compared to the traditional large number, frequentist approach to trials. In addition, methodologies – while focused on a particular target – are likely to find broader applicability. Especially as the regulatory science base expands, this will preclude others from needing to ‘reinvent the wheel’ for their own unique applications.
In summary, as our name suggests, NCATS’ mission is to ‘advance translational science.’ The research activities will always include a generous amount of translational research, but the goal is to promote and disseminate those precious new kernels of translational science principles that have broad applicability to a wider community and finding more ways to ‘turn science into health.’
I learned that you can’t choose the ways in which you’ll be tested.
– Robert J. Sawyer, Calculating God
This Mike’s Blog was featured in September 2022’s Ansible. Read other full-length stories from the September 2022 Ansible.