Early-Stage Technology Development Research Tony Dickherber, Ph.D. - PowerPoint PPT Presentation

Title of Presentation Evaluation of NCI’s Strategy for Early-Stage Technology Development Research Tony Dickherber, Ph.D. Center for Strategic Scientific Initiatives, NCI August 2017

1. Overview of the IMAT Program 2. Evaluation Design/Approach 3. Evaluation Findings Outline 4. NIH-wide Technology Development 5. Conclusions and Next Steps 2 2

Title of Presentation “Progress in science depends on new techniques, new discoveries and new ideas; probably in that order.” -Sydney Brenner

NCI Support for Technology Development Title of Presentation Advanced Technology Prototyping & Development Scaling/Optimization Hardening and Concept Feasibility Dissemination Development towards Context of within Context of Use Validation Demonstration Pipeline Use Typical NIH barrier for technology Nano (IRCN-U01 & Alliance-U24) Clinical Proteomics Technology Assessment (U01, U24) Academic-Industrial Partnerships (R01) ITCR (R21, U01, U24) Low Cost Global Health Technologies (UH2/UH3, R01, R03) SBIR/STTR (R41-44) Bioengineering Research Grants (R21, R01) Genome Technology Program (R21, R01, SBIR/STTR) Common Fund Initiatives (e.g. Single Cell Technologies or Tissue Chip)

IMAT Program Structure Title of Presentation Program Mission : To support the development, maturation, and dissemination of novel and potentially transformative next-generation technologies through an approach of balanced but targeted innovation in support of clinical, laboratory, or epidemiological research on cancer. Advanced Technology Prototyping & Development Scaling/Optimization Hardening and Concept Feasibility Dissemination Development towards Context of within Context of Use Validation Demonstration Pipeline Use Typical NIH barrier for technology • Feasibility/Proof-of-principle study ≤$400k over 3 years • Highly innovative technology R21 direct cost support • No preliminary data required • Advanced development • Demonstration of transformative utility ≤$900k over 3 years R33 • Requires proof of feasibility direct cost support R43 R44 ≤ $225k over 6m total ≤ $1.5M over 2 years • Development & (regulatory) validation cost support total cost support • Manufacturing & marketing plan • Feasibility study • Requires proof of feasibility and Two Tracks: 1. Molecular/Cellular Analysis Technologies (MCA) • Clear commercial Fast-Track commercialization plan • Demonstration of transformative utility potential 2. Biospecimen Science Technologies (BST)

What is a “biospecimen science” technology? Title of Presentation • Sample Quality Control ( e.g., RNALater) – Focus on preserving the biological integrity of the molecular and cellular targets to be assessed – Spans the preanalytical time period from patient management variables, through sample procurement, immediate handling and preservation, and processing and sample preparation in advance of analysis • Sample Quality Assessment ( e.g., RIN) – Focus on verifying/assessing the biological integrity of the molecular and cellular targets to be tested/measured

Distinguishing Features of IMAT Title of Presentation • Solicitation: – Emphasis on innovative technology with transformative potential ( i.e. high-risk, high-impact) – Focus exclusively on technology development ( NOT biological/clinical hypothesis-driven research ) – 100% investigator-initiated research grants • Review: – Special emphasis panels recruited based on focus of submissions, drawing heavily from former IMAT grantees – Milestone-based applications to quantitatively assess the capabilities of the technology ( e.g. , specificity, sensitivity, and speed) and characterize the improvement over state-of-the-art

IMAT New Award Distribution by FY Title of Presentation BST: Biospecimen Science Technologies MCA: Molecular/Cellular Analysis Technologies BST R33 BST R21 MCA R33 MCA R21 Success Rate 45 45% 40 40% 35 35% Success Rate Total # of Awards 30 30% 25 25% 20 20% 15 15% 10 10% 5 5% 0 0% Fiscal Year Issued as PAR 8

General Breakdown of the IMAT Portfolio Title of Presentation Technologies for Clinical Treatment and Diagnosis • Drug screening platforms • Patient-derived tumor modeling • Diagnostic imaging agents Cancer Biology Technologies • Cancer-targeting • Molecular fingerprinting (-omic discovery) • Drug delivery vehicles • Molecular interactions 40% • Point-of-care diagnostics • Cancer modeling • et cetera… • Imaging/spectroscopy probes 47% • Sample preparation • Mechanobiololgy/microrheology • et cetera… 9% Early Detection Screening 4% • Point-of-care detection • Field sample collection and storage Molecular Epidemiology Tools • Liquid biopsy platforms • Population-scale analysis • et cetera… • Low-resource setting point-of-care screening • et cetera…

Active IMAT Portfolio Title of Presentation Application & Validation of Emerging Technologies for Cancer Research (R33) Innovative Technologies for Cancer Research (R21) o Optimization/scaling or other further development o Initial proof-of-concept o Analytical/technical validation in biological context of use o Quantifiable milestone driven development plan Current R21 Portfolio Current R33 Portfolio clinical diagnostics (75 Active Projects) drug screening (49 Active Projects) epigenomics 2 1 8 genomics 1 4 2 6 glycomics 5 4 5 imaging 2 1 immunotherapy 3 liquid biopsy 4 6 4 metabolomics 2 modeling 5 3 novel biosensor 2 4 pathway tools 3 proteomics 4 3 6 sample prep 1 6 3 sample QA 4 6 1 single cell 2 3 8 transcriptomics treatment

1. Overview of the IMAT Program 2. Evaluation Design/Approach 3. Evaluation Findings Outline 4. NIH-wide Technology Development 5. Conclusions and Next Steps 11

IMAT FOA & Evaluation History Title of Presentation IMAT RFAs Approved for 3 RFAs Renewed for 5 years RFAs Renewed for 3 years IMAT PAR Released • 3 R21 (1 is a 3-yr award) • 2 R21 (3 yr awards) years • 1 R21/R33 • 3 R21/R33 • 2 R33 • 2 R33 • 1 STTR/SBIR • 2 STTR/SBIR • 2 STTR • 2 SBIR RFAs Renewed for 1 year RFA Renewed for 3 years • 2 R21 (3 yr awards) IMAT PAR Renewed • 2 R21 • 2 R21/R33 • 2 R33 • 2 R33 • 1 STTR/SBIR • Competitive Revisions RFAs Renewed for 2 years • 2 R21 (both 3-yr awards) IMAT PAR Renewed • 2 R33 • 2 R21/R33 • 2 STTR/SBIR Ongoing Evaluation Evaluative Update Evaluation Feasibility Study Full Program Targeted Evaluation Evaluation Full Program (until FY2016) Evaluation

Evaluation Criteria Title of Presentation • number of publications that cite a specific IMAT award number; • number of patent applications submitted to the US Patent & Trademark Office (USPTO); • number of patent applications granted or approved by the USPTO based on patent applications that cite a specific IMAT award number in one of four government interest fields; number of IMAT ‐ funded technologies now used in other NCI and NIH strategic • initiatives; and follow ‐ up case studies on previously funded technology development projects and • platforms, including their current use by and utility to the extramural scientific and clinical communities. 13

2015-16 IMAT Evaluation Overview Title of Presentation • Group: Ripple Effect Communications • Design: From Macro International during a prior Evaluation Feasibility Study for the IMAT program (2007) – Key: Follow each technology from before target award to after to understand how the program/intervention affected the outcome • Focus: Outcomes for all IMAT project prior to 2014 – 705 unique awards (including SBIR and STTR) – archival data records, web-surveys, and phone interviews – Included web-survey of and archival data analysis for a comparison group and phone interviews with IMAT technology end-users 14

Logic Model (Conceptual Framework) Title of Presentation Courtesy Ripple Effect Communications 15