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Re-engineering Precision Therapeutics Through N-of-1 Trials

Award Number: R01LM012836
ORGANIZATION: NATIONAL LIBRARY OF MEDICINE
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 09/01/2017
PERIOD OF PERFORMANCE END DATE: 05/31/2024

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Re-engineering Precision Therapeutics Through N-of-1 Trials - The objective of this proposal is to develop, test, and implement an innovative technology platform for conducting N-of-1 trials that transforms precision therapeutics. Right now, clinicians are engaging in clinical encounters at which they are trying to determine the best therapy for individual patients. These encounters are likely to be unsuccessful. Clinicians rely on the best available evidence (e.g., results from phase III randomized clinical trials; RCTs) for recommending therapies to a patient. Yet, conventional, between-patient RCTs only provide estimates of the effect of therapies on the average patient in those trials. Individual patients, however, often respond differently than the average patient in the phase III RCTs, and thus, heterogeneity of therapy response plagues these clinical decisions every day. The most scientifically rigorous-- and potentially transformative--method for determining optimal therapy for apatient is a single-patient (N-of-1) trial. N-of-1 trials are multiple crossover trials, usually randomized, and often masked, conducted within a single patient, with data collected objectively, continuously, and in the real-world, for a sufficient time period to determine whether the therapy, compared to a placebo or other active therapy, is optimal for a particular patient. They also yield information on off-target actions, such as side-effects, so that a more complex picture can emerge about the overall benefits and harms of a therapy for an individual patient.Clinicians and patients do not routinely engage in this type of scientific endeavor because they lack the tools. In this proposal, we will create an electronic platform that will allow clinicians and/or patients to order and conduct a single-patient trial. We will then collect RCT data to be able to estimate the benefit (if any) from using this approach. To do so, we will conduct 3 experiments using our platform for 3 different health conditions, each of which has high public health burden, high heterogeneity of therapy response, and high priority for a precision therapeutics approach as determined by previously interviewed clinicians and patients. For each, we will randomize 60 patients to receive an N-of-1 trial, or to receive usual care. We will then test a pragmatic design that simulates how the platform will be implemented in clinical practice after its release date. The application will be embedded in the clinical workflow, with clinicians having the capability of referring and tracking their patients in N-of-1 trials directly through the electronic health record. In this experiment, we will randomize 200 patients to receive the pragmatic N-of-1 trial, or to receive usual care. We will thus be able to compare our new precision therapy approach to the way therapies are typically determined for a patient. This N-of-1 trials platform will also facilitate a paradigm-shifting approach to discovery of therapeutic response phenotypes, as we will build a public facing registry of N-of-1 protocols and tools and a repository of de-identified data from N-of-1 trials. This international database of N-of-1 trial results can then be mined to identify phenotypes categorized by treatment responsiveness.


Issue Date FY	Funding FY	Legal Entity Name	Legal Entity Address	Legal Entity City	Legal Entity State	Legal Entity Zip Code	Legal Entity COUNTY	Legal Entity COUNTRY	Assistance Listing	Award Code	Budget Year	Action Date	Action Type	Action Amount

Issue Date FY: 2025 ( Subtotal = -$64,083 )
2025	2023	THE FEINSTEIN INSTITUTES FOR MEDICAL RESEARCH	350 COMMUNITY DR	MANHASSET	NY	11030	NASSAU	USA	Trans-NIH Research Support	000	7	10/17/2024	NON-COMPETING CONTINUATION	-$64,083
														Subtotal = -$64,083

Issue Date FY: 2023 ( Subtotal = $1,295,650 )
2023	2023	FEINSTEIN INSTITUTES FOR MEDICAL RESEARCH, THE	350 COMMUNITY DR	MANHASSET	NY	11030	NASSAU	USA	Trans-NIH Research Support	000	7	7/13/2023	NON-COMPETING CONTINUATION	$1,295,650
														Subtotal = $1,295,650

Issue Date FY: 2022 ( Subtotal = $1 )
2022	2022	FEINSTEIN INSTITUTES FOR MEDICAL RESEARCH, THE	350 COMMUNITY DR	MANHASSET	NY	11030	NASSAU	USA	Trans-NIH Research Support	004	6	7/29/2022	NON-COMPETING CONTINUATION	$1
2022	2019	FEINSTEIN INSTITUTES FOR MEDICAL RESEARCH, THE	350 COMMUNITY DR	MANHASSET	NY	11030	NASSAU	USA	Trans-NIH Research Support	003	3	10/29/2021	CHANGE OF GRANTEE / TRAINING INSTITUTION / AWARDING INSTITUTION	$2,137,877
2022	2018	TRUSTEES OF COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK, THE	630 W 168TH ST FL 4	NEW YORK	NY	10032	NEW YORK	USA	Medical Library Assistance	002	2	10/29/2021	SUPPLEMENT FOR EXPANSION	-$404,982
2022	2018	TRUSTEES OF COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK, THE	630 W 168TH ST FL 4	NEW YORK	NY	10032	NEW YORK	USA	Trans-NIH Research Support	001	2	10/29/2021	NON-COMPETING CONTINUATION	-$1,336,914
2022	2017	TRUSTEES OF COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK, THE	630 W 168TH ST FL 4	NEW YORK	NY	10032	NEW YORK	USA	Trans-NIH Research Support	000	1	10/29/2021	NEW	-$395,981
														Subtotal = $1

Issue Date FY: 2021 ( Subtotal = $1,467,835 )
2021	2021	FEINSTEIN INSTITUTES FOR MEDICAL RESEARCH, THE	350 COMMUNITY DR	MANHASSET	NY	11030	NASSAU	USA	Trans-NIH Research Support	000	5	6/4/2021	NON-COMPETING CONTINUATION	$1,467,835
2021	2019	FEINSTEIN INSTITUTES FOR MEDICAL RESEARCH, THE	350 COMMUNITY DR	MANHASSET	NY	11030	NASSAU	USA	Trans-NIH Research Support	004	3	8/9/2021	CHANGE OF GRANTEE / TRAINING INSTITUTION / AWARDING INSTITUTION	-$2,137,866
2021	2018	TRUSTEES OF COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK, THE	630 W 168TH ST FL 4	NEW YORK	NY	10032	NEW YORK	USA	Medical Library Assistance	003	2	8/9/2021	SUPPLEMENT FOR EXPANSION	$796,464
2021	2018	TRUSTEES OF COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK, THE	630 W 168TH ST FL 4	NEW YORK	NY	10032	NEW YORK	USA	Trans-NIH Research Support	002	2	8/9/2021	NON-COMPETING CONTINUATION	$1,336,914
2021	2017	TRUSTEES OF COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK, THE	630 W 168TH ST FL 4	NEW YORK	NY	10032	NEW YORK	USA	Trans-NIH Research Support	005	1	8/12/2021	NEW	$0
2021	2017	TRUSTEES OF COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK, THE	630 W 168TH ST FL 4	NEW YORK	NY	10032	NEW YORK	USA	Trans-NIH Research Support	001	1	8/9/2021	NEW	$4,488
														Subtotal = $1,467,835

Issue Date FY: 2020 ( Subtotal = $181,313 )
2020	2020	FEINSTEIN INSTITUTE FOR MEDICAL RESEARCH, THE	350 COMMUNITY DR	MANHASSET	NY	11030	NASSAU	USA	Trans-NIH Research Support	003	4	6/22/2020	NON-COMPETING CONTINUATION	$181,313
2020	2019	FEINSTEIN INSTITUTES FOR MEDICAL RESEARCH, THE	350 COMMUNITY DR	MANHASSET	NY	11030	NASSAU	USA	Trans-NIH Research Support	002	3	12/9/2019	CHANGE OF GRANTEE / TRAINING INSTITUTION / AWARDING INSTITUTION	$4,488
2020	2019	FEINSTEIN INSTITUTE FOR MEDICAL RESEARCH, THE	350 COMMUNITY DR	MANHASSET	NY	11030	NASSAU	USA	Trans-NIH Research Support	000	3	11/13/2019	SUPPLEMENT FOR EXPANSION	$0
2020	2017	TRUSTEES OF COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK, THE	630 W 168TH ST FL 4	NEW YORK	NY	10032	NEW YORK	USA	Trans-NIH Research Support	001	1	12/9/2019	NEW	-$4,488
														Subtotal = $181,313

Issue Date FY: 2019 ( Subtotal = $1,644,714 )
2019	2019	FEINSTEIN INSTITUTE FOR MEDICAL RESEARCH, THE	350 COMMUNITY DR	MANHASSET	NY	11030	NASSAU	USA	Trans-NIH Research Support	007	3	9/4/2019	SUPPLEMENT FOR EXPANSION	$181,312
2019	2019	FEINSTEIN INSTITUTE FOR MEDICAL RESEARCH, THE	350 COMMUNITY DR	MANHASSET	NY	11030	NASSAU	USA	Trans-NIH Research Support	002	3	7/19/2019	CHANGE OF GRANTEE / TRAINING INSTITUTION / AWARDING INSTITUTION	$1,463,402
2019	2019	FEINSTEIN INSTITUTE FOR MEDICAL RESEARCH, THE	350 COMMUNITY DR	MANHASSET	NY	11030	NASSAU	USA	Trans-NIH Research Support	006	3	8/1/2019	CHANGE OF GRANTEE / TRAINING INSTITUTION / AWARDING INSTITUTION	$2,133,378
2019	2018	TRUSTEES OF COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK	630 W 168TH ST FL 4	NEW YORK	NY	10032	NEW YORK	USA	Trans-NIH Research Support	000	2	7/18/2019	NON-COMPETING CONTINUATION	$0
2019	2018	TRUSTEES OF COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK	630 W 168TH ST FL 4	NEW YORK	NY	10032	NEW YORK	USA	Medical Library Assistance	001	2	7/18/2019	SUPPLEMENT FOR EXPANSION	$0
2019	2018	TRUSTEES OF COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK	630 W 168TH ST FL 4	NEW YORK	NY	10032	NEW YORK	USA	Trans-NIH Research Support	004	2	8/1/2019	NON-COMPETING CONTINUATION	-$1,336,914
2019	2018	TRUSTEES OF COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK	630 W 168TH ST FL 4	NEW YORK	NY	10032	NEW YORK	USA	Medical Library Assistance	005	2	8/1/2019	SUPPLEMENT FOR EXPANSION	-$404,982
2019	2017	TRUSTEES OF COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK	630 W 168TH ST FL 4	NEW YORK	NY	10032	NEW YORK	USA	Trans-NIH Research Support	003	1	8/1/2019	NEW	-$391,482
														Subtotal = $1,644,714

Issue Date FY: 2018 ( Subtotal = $1,741,896 )
2018	2018	TRUSTEES OF COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK	630 W 168TH ST FL 4	NEW YORK	NY	10032	NEW YORK	USA	Trans-NIH Research Support	000	2	5/26/2018	NON-COMPETING CONTINUATION	$1,336,914
2018	2018	TRUSTEES OF COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK	630 W 168TH ST FL 4	NEW YORK	NY	10032	NEW YORK	USA	Medical Library Assistance	001	2	9/5/2018	SUPPLEMENT FOR EXPANSION	$404,982
														Subtotal = $1,741,896

Issue Date FY: 2017 ( Subtotal = $1,220,046 )
2017	2017	TRUSTEES OF COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK	630 W 168TH ST FL 4	NEW YORK	NY	10032	NEW YORK	USA	Trans-NIH Research Support	000	1	8/1/2017	NEW	$1,220,046
														Subtotal = $1,220,046

Grand Total All Awards = $7,487,372

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Re-engineering Precision Therapeutics Through N-of-1 Trials

Award Number: R01LM012836

ORGANIZATION: NATIONAL LIBRARY OF MEDICINE

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)

PERIOD OF PERFORMANCE START DATE: 09/01/2017

PERIOD OF PERFORMANCE END DATE: 05/31/2024

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