1. Life Sciences Accelerated R&D Services The Science of Getting Products to Patients Faster Study Data Standardization Plan Use Case Experience Dave Izard May 14, 2015

2. Client Use Case for Study Data Standardization Plan PreNDA Meeting Briefing Package for NCE –First discussion of clinical data details w/ FDA @ PreNDA meeting Thirteen Studies –10 phase 1 (7 early, 3 recent) –1 phase 2 –1 phase 3 –1 phase 3 extension Compound purchased from another company following 1 st phase 1 study © 2015 Accenture All Rights Reserved. 2

3. Goals for Study Data Standardization Plan Convey standards utilized at study level for clinical studies Rationalize reason for limited legacy conversion Rationalize decision for limited / no pooling of data © 2015 Accenture All Rights Reserved. 3

4. Section 2 – List of Studies (2.2 Clinical Studies) Study Status –No formal definitions available for planned, ongoing & completed –Agency decisions made based on concrete study start date –Sponsor wanted “Study Start Date” and “Study End Date” with available check boxes of “planned” & “actual”, coupled with a formal agency definition for “Study End Date” in order to use this table © 2015 Accenture All Rights Reserved. 4

5. Section 2 – List of Studies (2.2 Clinical Studies) Study Design / Trial Blinding Schema / Diagnosis Group / Indication Type / Study Type –Protocols & CSRs already written using company / industry conventions –No desire or willingness to restate these items using controlled terminology after the fact –Concern about potential requirement to write this way in the future © 2015 Accenture All Rights Reserved. 5

6. Section 2 – List of Studies (2.2 Clinical Studies) Submit Study Datasets –Decision to submit study datasets is independent of the characteristics of a study –A simple “yes/no” response is of limited value Multiple submissions for ongoing studies? Submission w/ CSR vs. SPA vs. NDA vs. 120DSU © 2015 Accenture All Rights Reserved. 6

7. Section 4 vs. Table Ultimately Produced © 2015 Accenture All Rights Reserved. 7

8. Section 4 vs. Table Ultimately Produced © 2015 Accenture All Rights Reserved. 8

9. Section 4 vs. Table Ultimately Produced © 2015 Accenture All Rights Reserved. 9 CRF annotation standards need to be taken into consideration… Data definition file standards could be different across data types…

10. Section 4 vs. Table Ultimately Produced © 2015 Accenture All Rights Reserved. 10

11. Section 4 vs. Table Ultimately Produced © 2015 Accenture All Rights Reserved. 11 Does this add value? Transparent rationale for use of legacy standards…

12. Observations © 2015 Accenture All Rights Reserved. 12 Should probably remove hard version of Study Data TCG from this section of document…

13. Observations This table only holds the information pertaining to the data utilized to support the original CSR Any assets created as the result of legacy conversion for other purposes are documented in a separate table © 2015 Accenture All Rights Reserved. 13

14. Observations Need to add context (e.g., Tabulation Data, Adverse Event Coding) between Study Identifier & Expected Standard © 2015 Accenture All Rights Reserved. 14

15. Study Data Standardization Plan No mention of if / how / when pools will be developed, submitted and potentially updated over time No mention of if / how / when individual studies will be submitted and potentially updated over time © 2015 Accenture All Rights Reserved. 15 Observations Actions to Consider Get FDA buy in that pooled data standardization should be recorded in this same document Separate standardization versus submission strategy