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25-Apr-2014 FDA/DCDISC Workshop. ›Introductions ›Housekeeping Rules ›Thanks –Colleen Ratliffe ›What is DCDISC? Welcome.

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Presentation on theme: "25-Apr-2014 FDA/DCDISC Workshop. ›Introductions ›Housekeeping Rules ›Thanks –Colleen Ratliffe ›What is DCDISC? Welcome."— Presentation transcript:

1 25-Apr-2014 FDA/DCDISC Workshop

2 ›Introductions ›Housekeeping Rules ›Thanks –Colleen Ratliffe ›What is DCDISC? Welcome

3 ›9:00-9:15 – Gathering & Welcome ›9:15-9:30 - FDA/CDER State of Data Standards –Mary Ann Slack, Ron Fitzmartin - CDER ›9:30-9:45 - FDA/CBER State of Data Standards –Lise Stevens, CBER ›9:45-10:45 –Challenges with Incoming Data –Doug Warfield, Lisa Lin – CDER, eData Team ›10:45-11:00 – Break ›11:00-11:40 – “The Importance of CDASH – A Statistical Reviewer’s Perspective” –Ben Vali, CDER ›11:40-12:15 –Challenges with Submission Data –DCDISC Team ›12:15-1:00 – Lunch/Networking Agenda

4 Ingeborg Holt Jane Diefenbach Alan Meier Challenges with Submission Data

5 Ingeborg Holt Booz Allen Hamilton Technology Provider Perspective

6 ›The ideal conditions for this are: 1.The problem is clearly defined 2.The input sources are known and have stable formats 3.The desired functionality and output are distinctly described Technology Provider’s Task: Apply Knowledge in Order to Solve a Problem Problem: High incidence of spilt milk Input: Milk Functionality: (1) Allow drinking of milk from the cup (2) Minimize spilling when the cup isn’t oriented properly

7 1.The problem: Can it be determined from the submission data if the drug under review is safe and effective? 2.The input: Submission Data 3.The functionality: The analyses that need to be performed to determine the quality and completeness of the data and the safety and efficacy of the drug. Technology Provider’s Task and Submission Data

8 ›Data standards (CDISC SDTM and ADaM) –Organize submission data in a systematic way –Control terminology –Reduce the work needed to locate and understand the data and create functionality –Increase the potential to reuse functionality The Role of Data Standards is to Regulate the Input

9 ›Success using data standards has expanded the scope of the problem to be solved The Problem Definition has Changed Is this drug safe and effective? How can the question “Is this drug safe and effective” be answered in the more efficiently and effectively? How can data collected for answering the question “Is this drug safe and effective” be reused to inform drug safety and public health in other ways? How can submission data be better modeled for its intended use, effective reuse in drug safety and public health AND assist drug development the pre- competitive space? time

10 ›Data standards change and grow as the problem changes and grows –Adherence to principles of the standard –Early communication ›Correct implementation of the standard –Requires ›Sponsors understand which standards are accepted ›Data validation requirements are unambiguous – Single Set of Validation Rules ›OpenCDISC rules, FDA rules, rules from other organizations ›Rules based on IG versus common sense rules – Checks between standards (SDTM, ADaM) ›The implementation guides are as specific as possible and cover expected scenarios – Pharma SUG 2012 paper (Octagon) ›EX domain often implemented incorrectly ›Time and practice: implementation is improving Challenges to Standardized Submission Data

11 ›Analyses for safety are well defined –Therapeutic area domains may require special analyses for safety ›Efficacy analyses, particularly those using therapeutic area domains, may be complex and present new challenges ›G uidance needed for derived variables –How much support should be provided for missing values that can be derived (eg imputing BLFL, STDY) –Should derived SDTM variables be validated? Well Defined Functionality and Output

12 Adherence of submission to data standards is improving Many of the new challenges result from an expanded problem space There are areas that need attention and work Technology Provider Perspective on Submission Data

13 ›Good Technology Choices –Designing tools to handle expected change ›Terminology changes, variable additions/deletions, validation rule additions/changes ›Anticipating therapeutic area domains, interoperability needs –What is the role of the Pre-competitive Community (FDA, CDISC and other Standard Development Organizations, Third Party Data Providers, Technology Providers, Sponsors,…) ›Should information technology and data standards usage decisions involve all stakeholders and open discussion about risk/benefit issues – Metadata Repository – Changes to Standards Technology Provider’s Responsibility

14 Adventures in CDISC Consulting and eCTD Preparation Jane Diefenbach, PharmaStat LLC DCDISC Implementation Network at FDA April 25, 2014

15 Preparing for a data submission 18 to 24-month lead time Legacy studies and Phase III may just be starting Everything is changing: people, processes, knowledge of the product, often even the company structure When standards change too it can be hard to accommodate them in this environment

16 Google Images for eCTD Preparation

17 What needs to happen Planning – What studies? What data formats? What standards? Production – legacy studies – Align the studies with a unified modeling plan – Perform the conversions, verify with reanalysis Production – current studies – Real time conversion and linear data flow is difficult Documentation, maybe most important

18 Planning the Submission: Company XYZ Data Flow ADaM eCTD SDTM Studies : CDMS 101, 102, 103 Analysis CSR Item 11 eCTD 201, 202 CDMS Analysis CSR eCTD SDTM 20 3 CDMS ADaM CSR eCTD SDTM 301 Integrated Data for ISS 10 4 CDMS ADaM CSR eCTD SDTM CDMS ADaM CSR eCTD SDTM ADaM eCTD ISE Credit John Brega 2014

19 Experience to Date Work volume is astronomical, easy to underestimate Clarity of purpose is sometimes absent – Standards are overwhelming; sponsors don’t know what is most important – We imagine perfection at kick off; eventually just need to get done – Downstream changes to standards, modeling are costly; sometimes we need to say Next time

20 What makes it easier? Use of CDASH in data collection Predictability of new standards, fair warning OpenCDISC – has done more to standardize implementation than anything Electronic distribution of linked standards (SHARe) and a data standards warehouse Widespread adoption

21 What makes it easier? Communication! – The more we know about how you interact with the data the better we’ll do – Guesswork leads to busy work and busy work does not advance Science or Health

22 Google Images for CDISC Consulting

23 Alan Meier MedImmune LLC Sponsor Company

24 ›Implementation/Acceptance of Standards by Agency –Set time period for accepting submissions in new version ›Allows sponsor time to decide when to upgrade –Work with CDISC to implement changes rather than publishing guidance ›Saves time searching through documents –Set policy on use of provisional / sponsor-defined domains ›Are sponsor-defined non-X_, Y_, Z_ domains acceptable ›Will provisional domain templates be accepted Sponsor Company

25 ›ADaM vs Sponsor-Formatted Analysis Data Sets –Will agency be requiring ADaM ›Versioning of CDISC Standards –Some products take 10+ years to develop –Acceptance of submission data in multiple versions Sponsor Company

26 ›Can we get an FDA representative on the Steering Committee DCDISC

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