Letter From the President
Over the last few months, we have seen extraordinary efforts to get well designed clinical trials done on an informative timeline, and we have seen seismic change in how we approach such things as consent and collection of follow up data. Necessity has truly been the mother of invention as we strive to implement studies differently. I applaud our community for rising to the challenge. I am amazed, though, at how little attention we are paying to the way we design our trials. In the setting of COVID-19, I continue to see outcomes that ignore information about disease progression, sample sizes with planned accrual rates that will give us answers two or three years from now, and designs that have no plans for learning from the data along the way. Beyond revolutionizing how we implement trials, a major opportunity of the current pandemic is to rethink traditional trial designs to focus on the goal of producing meaningful results in a relevant timeframe.
As one example, I have been heartened by recent conversations that have emphasized the importance of meeting accrual goals while there are still cases of COVID-19 to study. I am equally disheartened by the notion that we can’t predict accrual even as a study is running, and so truly holding ourselves accountable to the idea of timely enrollment is somehow abhorrent. Rationalizing that position is a challenge. For every patient we enroll in a trial that knowingly has no chance of producing useful information, we are asking for consent to the possibility of being randomized to an unproven therapeutic with unclear side effects with the goal of providing inconclusive information. Not to mention the opportunity cost for not enrolling in a potentially fruitful trial, the financial cost of doing the research itself, and the potential for lives lost and harm done by coming to an inconclusion too late. The common approach of enrolling to the maximum sample size unless there is beyond clear evidence of benefit or harm along the way without considering that we have finite cases and finite time is nonsensical at best.
Another major trial design feature that I am finding problematic is our selection of outcomes. We continue to stick to the tried and trusted measurements. We are very comfortable with 30-day mortality, and time to hospitalization. We are less comfortable with incorporating information about how outcomes change over time. We are even less comfortable when we try to combine safety and efficacy. Yet, if a therapy causes major harm to some while saving the lives of others, the relative weight of risk v benefit will continuously be debated. Why not put the fact of harm and the fact of death on the same scale and model them together? We should certainly eschew outcomes that lose information, and thus statistical power. No matter what our statistical philosophy or our clinical trial pedigree, aren’t we obligated to minimize the number of participants we expose to harm?
These are two essential trial design features – choosing good outcomes and evaluating for futility along the way. There are many more to consider. Different types of clinical research may not carry some of the same formalities, but similar principles apply. Changing from tradition is difficult. Overturning years of training that has been confirmed by peer review and adverse funding decisions is difficult. It takes stepping outside of our comfort zones. As the saying goes, “A comfort zone is a beautiful place, but nothing grows there”. Together, let’s push aside tradition and be inventive in our thinking. It is, after all, necessitated by the context of a global pandemic.
Christopher John Lindsell, PhD
Translational Science 2021
Save the date for Translational Science 2021, the scientific meeting that brings together the brightest trainees, junior faculty, and senior scientists, taking place March 30 - April 2, 2021, and make an investment in your professional development.
There's still time to get involved! Submit a self-nomination to serve on our TS21 Planning Committee or nominate a colleague who you think would help us push the envelope. Nominations should include a proposed topic for the meeting (maximum of 100 words) and an NIH biosketch. The proposed topic should fit the theme for the meeting: "Breaking Barriers, Building Bridges." Think of diversity, translation and team science. Specifically, in your nomination, please answer the following questions:
In what way will this topic break barriers and build bridges?
Why is this topic critical for translational science in 2021 and beyond?
What virtual and in-person activities could support your topic?
Please send nominations to firstname.lastname@example.org by this Friday, August 14.
Regulatory Science SIG Survey
We invite you to please complete this brief survey designed to gather information on the existing landscape of regulatory science-focused training (e.g., fellowships, degrees, programs). This effort is being led by the ACTS' Regulatory Science Special Interest Group.
This survey should be completed by an individual in your program who is best able to provide detailed program information, such as program coordinators or program directors. If you feel that a colleague would be better able to respond to these items, please forward as appropriate. We anticipate that completion of these questions should take no more than 20 minutes.
For the purposes of the survey, we utilize the definition of "regulatory science" developed by the U.S. Food and Drug Administration (FDA): "Regulatory Science is the science of developing new tools, standards, and approaches to assess the safety, efficacy, quality, and performance of all FDA-regulated products." Please note that this is different from programs that exclusively focus on regulatory affairs (ensuring compliance with current FDA regulations).
Thank you for your willingness to contribute to this survey and we look forward to sharing the overarching findings with the community!
Thank you to everyone that attended the webinar Current Trends in Qualitative Methodology for Translational Science Research and Evaluation on July 8. The session recording is now available in the Learning Library. In this one-hour webinar, five experts demonstrated the application of current qualitative methods to evaluate clinical and translational science programs, organizations, and enterprises. Over 300 registrants attended the live session.
This session was originally scheduled for Translational Science 2020 and was co-sponsored by the ACTS Evaluation SIG and the American Evaluation Association (AEA) Translational Research Evaluation Topical Interest Group (TRE TIG).
Journal of Clinical and Translational Science
Volume 4 / Issue 4 of the Journal of Clinical and Translational Science and the 2020 Abstract Supplemental are now available online!
JCTS's mission is to provide a forum for the rapid communication of topics of interest and relevance to the large and diverse community of clinical and translational scientists with the goal of improving the efficiency with which health needs inform research and new diagnostics, therapies, and preventive measures reach the public. The Association for Clinical and Translational Science has partnered with the American Physician Scientists Association (APSA) and the Clinical Research Forum (CRF) to support the growth and development of JCTS.
Submit your article today to be featured in future issues of JCTS! Please also visit the JCTS website for information on our themed issue related to Design, Development, Evaluation, and Dissemination of Team Science Interventions in Clinical and Translational Research.
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