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Dear Reader,

One is the loneliest number that you’ll ever do… (with apologies to Three Dog Night)

N-of-1 trials formally assess the impact of an intervention in a single patient.   A classic example of an N-of-1 trial is the ABA or reversal design. In an ABA study, the pre-intervention/baseline status is assessed (condition A), a treatment/intervention is introduced and status re-assessed (condition B), and then the intervention is withdrawn (A) and status is assessed again.  Observing an effect of treatment followed a return to baseline constitutes a positive study.

N-of-1 trials have been around for many years. However, their utilization has been mostly limited to rare diseases or informal settings. Given the increasing focus on personalized medicine and the realization that population level data may be missing the mark for many patients, it is time to consider expanding the use of these designs.   

Going from population-based to “personalized-based” (N-of-1) clinical trials doesn’t have an “easy button” and its adoption has been slow and painful.   Successful examples of N-of-1 studies have been achieved in oncology, where data from a single patient (including genetic, environmental, and phenotypic) is routinely utilized to accurately predict which therapies are likely to be more successful and which ones to avoid for that individual, with the potential for direct benefit at both the individual participant and other, similar patients. Furthermore, combining multiple N-of-1 studies, could result in enough information for the generalization of therapeutic safety and efficacy data in a specific sub-population, providing additional justification from both a public health and healthcare cost perspective.

Currently there are several areas where the CTSA Program has helped improve the translational process that could also help catalyze the design, development and completion of N-of-1 studies, including: IRB reliance agreement through SmartIRB for agile protocol development and validation of multiple N-of-1 studies and reliance across participating institutions, EHR based study feasibility and enrollment capabilities through Accrual to Clinical Trials (ACT) for participant screening, uniform electronic data collection forms using tools such as REDCap for data aggregation and analysis.  Other areas, such as standardized clinical trial protocol using NIH and FDA standard protocol template can also be used in the design of N-of-1 studies.

Additionally, some areas where the CTSA Program can further impact:

  • Training and decision-making tools to assist in the design and implementation of successful N-of-1 trials.
  • Seamless alignment of medical care and clinical research to improve study protocol adherence and the collection of meaningful Real World Data.
  • Sharing of expertise, as well as, dissemination and implementation of  innovative resources in non-traditional biostatistical approaches at the consortium level.

The above list is not exhaustive but rather a start to the conversation about how to do more with less traditional trial designs, such as N-of-1, in the development of new therapies that can get to patients more efficiently.

Looking forward to seeing everyone at the end of the month.

Monthly musing:  

“It is said that power corrupts, but actually it’s more true that power attracts the corruptible. The sane are usually attracted by other things than power."

– David Brin

Dr. K
Michael G. Kurilla, M.D., Ph.D.
Director of the Division of Clinical Innovation, NCATS

 

THE SPOTLIGHT


CLIC Website Gets a Makeover

The CLIC website got a new look! The upgrade did not change any of the key functions of the website but some menu items have moved. We are excited for you to explore the new site and check out the re-designed interface, which provides several functional improvements under the hood. If you have any questions or concerns regarding the new site, please reach out to contact@clic-ctsa.org.


WHAT'S NEW


Request for Information (RFI): Enhancing the Clinical and Translational Science Awards (CTSA) Program NOT-TR-19-027

The National Center for Advancing Translational Sciences (NCATS), part of the National Institutes of Health (NIH), seeks comments from all key stakeholder communities on how it can strengthen the Clinical and Translational Science Awards (CTSA) Program to meet its broad scientific mission to improve the efficiency and effectiveness of clinical research and translational science. Key stakeholders include academic health centers, scientific research organizations, healthcare delivery organizations, and healthcare providers; individuals involved in the translational pipeline to improve health in the community including those public and private partners who fund such research and services; those who provide education and training in the translational science process; and members of the public who are advocates, clinicians, patients, and community leaders seeking better diagnostics, treatments, preventions, cures, and overall general good health.

All comments must be submitted online at https://grants.nih.gov/grants/rfi/rfi.cfm?ID=90.

Responses must be received by October 25, 2019. 

 

It’s Here! Transition from DTFs to ECs.

As discussed during our August CTSA Program Webinar, the transition from the previous Domain Task Forces to the Enterprise Committees is in full swing.  At this point all the DTFs' summary reports have been reviewed by the CTSA Program Steering Committee. We also received requests for extensions of six to 12 months for three of the existing Working Groups (two Bioinformatics and one Methods and Processes). The Steering Committee decided that those WGs should propose a new WG and/or transition to a Discussion Forum in January 2020.  New WG applications may be submitted from Oct. 1 to Oct. 31. WG application forms will be available online during the month of October. For questions about the new structure and deadlines please feel free to contact us at contact@clic-ctsa.org.

Deadline to Upload Final Poster for Program Meeting Extended

The deadline to upload your hub’s final poster to the CLIC website for the Fall 2019 CTSA Program Meeting Networking & Poster Session has been EXTENDED to Sept. 16. Hubs are responsible for printing their own poster and bringing it to the Program Meeting, but in order for it to be available electronically on the CLIC website, you must upload it by Sept. 16. You will need to be logged on to the CLIC website in order to upload your poster. Electronic versions of the poster will be available before the meeting, for those submitted on time. If you have any questions or need additional information, please contact the CLIC Meeting Planner, at program_meeting@clic-ctsa.org.

Synergy Paper RFA Due Oct. 2, 2019

Do you have a topic related to machine learning and/or artificial intelligence that you are looking to write a manuscript on? Apply today to receive project management support from CLIC, as well as funds toward copy editing and publishing costs. A Synergy Paper is a collaborative manuscript developed by a multi-translational stage group of individuals from at least three CTSA Program hubs with the purpose of addressing substantial challenges in clinical or translational research. To learn more about the application requirements or to submit an application click here. Applications for this RFA are due Oct. 2, 2019 and support will be provided to the selected team for one year, starting in January 2020.

Collaborative Lunch Session Between IDeA-CTRs and CTSA Program Hubs

Discussion Moderator: Ming Lei, Ph.D. (NIH – NIGMS) and Michael Kurilla, M.D., Ph.D. (NIH – NCATS) 

Room: Washington B 

The CTSA Program PIs and the IDeA-CTR Program PIs have been encouraged to work together to address translational science barriers. One opportunity for collaborative efforts is the acceleration of clinical and translational research to address health disparities and the significant burden of conditions that disproportionately affect rural, minority, and other underserved populations (NOT-TR-19-015). To help facilitate the sharing of ideas, a collaborative lunch session is planned for those who are interested. The session is scheduled to take place on Friday, September  27, from 11:45 a.m. - 12:45 p.m. in the Washington B room, Hyatt Regency Crystal City, 2799 Jefferson Davis Highway. The format of the session will be open mic with the opportunity to talk about existing collaborations or new opportunities to collaborate between CTSA hubs and IDeA-CTRs of collaborations. Visit the 2018 IDeA-CTR Directory and the CTSA Program hubs Directory for a complete list of institutions and for more information.

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