regulatory guidance: the elephant in the room … · regulatory guidance: the elephant in the room...

Upper Providence SDE 2015 | 1

Regulatory Guidance:The Elephant in the Room

Upper Providence, PAJuly 16th 2015

2 | Upper Providence SDE 2015 Upper Providence SDE 2015 | 3

WelcomeDear Single Day Event Attendee,

PhUSE would like to welcome you to Upper Providence, Pennsylvania, which is part of the greater Philadelphia metropolitan area. One of the most historic cities in America, Philadelphia’s population is second only to NYC on the east coast. Philadelphia boasts many landmarks including Independence Hall, the Liberty Bell and Valley Forge National Historical Park, and the best cheese steaks in the world (is it Geno’s, Pat’s, or Jim’s?). Only eight miles from Upper Providence, you can easily get to Valley Forge National Historical Park and King of Prussia Mall (the largest mall in America by the end of summer 2015).

Philadelphia might be most famously known as the birthplace of American democracy, but you’ll find it’s an ideal destination for discovery, as you explore its colorful neighborhoods. Over 50 small and large biopharmaceutical companies call this city home. We are pleased to welcome you to the City of Brotherly Love.

The pharmaceutical and biotech industry are bound by regulatory guidelines, but often we tend to lack clarity and/or knowledge about those guidelines. The theme of today’s event “Regulatory Guidance: The Elephant in the Room” will cover some of these guidelines and shed some light on how best to implement them.

Our morning session will focus on de-identification, with two speakers offering different viewpoints. The first session will discuss the right way to share patient-level data and set expectations with outside requestors along with new SDTM versions of data de-identification documents. This will be followed by a presentation on the Six Ws, which will cover a Model Approach of how to anonymize individual patient data.

Our afternoon comprises of six speakers within three sessions that will focus on FDA-endorsed submission plans and documents articulated through recently issued guidance. The first session will discuss the four plans and documents that are expected by FDA reviewers during submission planning, at the time of submission and how PhUSE are working to support the development and maintenance of templates, guidelines and examples. The second session takes a deeper

dive into the Study Data Reviewers Guide, discussing the importance of this document in achieving clarity of the SDTM data assets, how to use the template and guideline, and real world examples. The final presentation of the day highlights the Analysis Data Reviewers Guide by detailing who, what, when, where and how a company should develop this document for analysis data assets during a submission.

We would like to thank our host GlaxoSmithKline and other event sponsors DataCeutics, d-Wise, GCE Solutions and Theorem Clinical Research for their support.

We hope you find the day both informative and enjoyable.

Terek Peterson & Asif KarbhariPhUSE Upper Providence Single Day Event Co-Chairs

You’ll find Philadelphia is an ideal destination for discovery, as you explore its colorful neighborhoods

AgendaTime Title and Speaker08:30 - 09:00 Welcome Refreshments09:00 - 09:15 Welcome & Introduction

Event Co-Chairs09:15 - 10:00 An Overview of PhUSE

Chris Hurley, PhUSE US Events Director 10:00 - 10:15 PhUSE CSS Update

Chris Hurley, PhUSE US Events Director 10:15 - 10:45 Considering De-identification? Legacy Data Approach

Kymberly Lee, AstraZeneca 10:45 - 11:15 Morning Break

Sponsored by d-Wise11:15 - 12:00 Data Transparency – The Six Ws

Natalie Martinez, Lilly 12:00 - 13:00 Lunch13:00 - 13:30 Tools to Plan, Deploy and Document the Use of FDA-Endorsed Study Data Standards

David Izard, Accenture13:30 - 14:00 Achieving Clarity through Proper Study Documentation:

An Introduction to the Study Data Reviewer’s Guide (SDRG)Terek Peterson, Joseph Kovach & Michael Stackhouse, Theorem Clinical Research

14:00 - 14:15 Afternoon Break14:15 - 14:45 Who, What, When, Where and How: Basics of the Analysis Data Reviewer’s Guide (ADRG)

Cathy Michalsky & Mark Gummel, Theorem Clinical Research15:00 - 16:00 Panel Discussion

All Speakers16:00 - 16:30 Closing Remarks

Co-Chairs

Terek Peterson Theorem Clinical Research

Asif Karbhari AstraZeneca


10.15 - 10:45 Considering De-identification?Legacy Data ApproachKymberly Lee, AstraZeneca

AbstractComplying with the current data privacy regulations and processes of de-identifying patient level data to share clinical data with outside requestors, legacy data and CDISC compliant data files could be a little challenging and approaches may differ based on your company’s needs and industry regulations. This presentation will give a quick explanation of clinical data sharing, clinical data privacy and clinical transparency. It will also discuss a previously used and proven approach to ensure the protection of patient level data with legacy data, versus CDISC complaint data, concerns over variables needed, variable naming processes, documentations after the de-identification work is completed and relations with clinicaltrials.gov.

BiographyKymberly Lee is currently employed at AstraZeneca as a global programming lead within the CNS Therapeutic Area. She moved from the RTP, NC area to Gaithersburg, Maryland towards the end of 2014. She has held various different functional roles and titles within the industry and brings with her over 18 years’ pharmaceutical/clinical trial experience, strong leadership and management skills, data quality assurance experience, ECG and Holter laboratory and data management experience as well as expertise in CDISC and ADaM developments.

11:15 - 12:00 Data Transparency – The Six WsNatalie Martinez, Lilly

AbstractData transparency and access to patient-level data has come to the forefront with the EMA’s policy on Publication of Clinical Data and PhRMA/EFPIA’s Principles for Responsible Clinical Trial Data Sharing: Our Commitment to Patients and Researchers. This presentation will discuss TransCelerate’s white paper on Data De-identification and Anonymization of Individual Patient Data in Clinical Studies – a Model Approach, as well as the corresponding documentation provided by sponsor companies.

BiographyNatalie Martinez has 14 years’ experience in the pharmaceutical industry. She joined Lilly in 2001 as an SAS programmer responsible for the creation and support of clinical trial data reporting systems in the neuroscience therapeutic area. Natalie has held roles as the process owner and lead for clinical trial integrated databases (IDBs) across the entire portfolio, as well as responsibility for the delivery of many key study, submission and IDB deliverables that supported US and OUS submissions for multiple compounds and therapeutic areas during her career. In her current role, Natalie is responsible for processes, research, inventory and delivery of clinical trial data for Lilly’s data transparency initiative. She is actively involved with three external industry groups. Natalie co-wrote a white paper on Clinical Trial Data Integration and presented it at the 2014 Pharmaceutical SAS Users Group (PharmaSUG) Conference. She chaired a sub-team in the Optimizing the Use of Data Standards Working Group for the FDA/PhUSE Computational Science Symposium, serves on the PharmaSUG Conference Committee and is a member of TransCelerate’s Clinical Data Transparency Data Anonymization Sub-Team.

13:00 - 13:30 Tools to Plan, Deploy and Document the Use of FDA-Endorsed Study Data StandardsDavid Izard, Accenture

AbstractThe FDA’s Computational Science Center’s (CSC) mission is to support CDER in continually improving the drug evaluation and review processes across the drug lifecycle through innovation, supporting the submission and use of high-quality data, and providing access to high-end analytical tools and training. High-quality data is the key to enabling regulatory reviewers to fully utilize the Computational Science Center’s tools and services to support decision-making. In order to achieve this goal, the FDA, as articulated through recently issued guidance documents and supporting resources, wants sponsors and service providers alike to plan for the use of FDA-endorsed study data standards, deploy these standards during the design and execution of clinical and non-clinical trials and subsequently document how successful you were in achieving this plan. The recently issued FDA guidance documents and supporting material define four new tools to support planning, deploying and documenting the use of FDA-endorsed data standards:• Study Data Standardization Plan• Study Data Reviewer’s Guide• Analysis Data Reviewer’s Guide• Legacy Data Conversion Plan & Report.

This presentation will describe the details for each tool, as laid out in the FDA materials, and discuss how various PhUSE efforts are supporting the development and maintenance of templates and associated completion guidelines and how examples are progressing.

BiographyDave Izard frequently finds himself at the intersection of clinical data standards, regulatory expectations and sponsor organization needs and desires. A pharmaceutical professional since 1997, he currently serves as the Clinical Data Consulting Lead for Accenture’s Accelerated Research & Development Shared Services after leading Octagon Research Solutions/Accenture’s SDTM practice for six years. Prior to joining Octagon/Accenture, he served in a variety of clinical programming leadership roles at both GSK and Shire. He has served as a paper author & presenter, seminar instructor and section chair at industry conferences including the Pharmaceutical SAS Users Group (PharmaSUG), the Society of Clinical Data Management (SCDM) and various local and regional SAS meetings. His current PhUSE efforts include supporting the development of the Study Data Standardization Plan and Legacy Data Conversion Plan & Report templates as well as the revised Site Selection/OSI BIMO guidance. He holds bachelor and master of science degrees in computer science from Bucknell and West Chester University respectively.

Speakers and Abstracts


Speakers and Abstracts13:30 - 14:00 Achieving Clarity through Proper Study Documentation: An Introduction to the Study Data Reviewer’s Guide (SDRG)Terek Peterson, Joseph Kovach & Michael Stackhouse, Theorem Clinical Research

AbstractWith the ever-growing standardization requirements of pharmaceutical submissions to the FDA, it can sometimes be difficult to understand where exactly you place non-conformant, essential information that does not have a home within other submission documentation. Now, with the development of the Study Data Reviewer’s Guide (SDRG), you can find this information a home and easily communicate it in an effective manner. This document provides reviewers with the key details they will need to perform a thorough review of the data without the need to search through all of the individual study material. Using the SDRG, FDA reviewers should be able to review the full documentation package of your clinical trial quicker, with less questions. PhUSE has provided a step-by-step template that helps you understand what to report, where to report it, and makes sure nothing is lost so that traceability is clear to both you and your FDA reviewers. This presentation will help you understand why it is important to think about the SDRG from the start, how to use these documents to your advantage, and provide real examples of their flexibility and utilization.

BiographyTerek Peterson is Senior Director of Global Statistical Standards at Theorem. He brings over 18 years’ experience in the pharmaceutical industry. As a leader in data standards, Terek participates as a member of the CDISC Advisory Committee, the ADaM team, the ADaM compliance sub-team and the ADaM integration sub-team. In his role, Terek consults with sponsors in order to create and implement strategies around the development, use and commitment to standards within and across strategic partnerships. He provides consultation services to clients conducting data submissions to regulatory groups, and furthered the development of standards with organizations such as CDISC, the FDA and PhUSE. He is a prolific presenter on standards and SAS programming-related topics.

Michael Stackhouse graduated from Arcadia University with a bachelor’s degree in business administration, with a concentration in economics and a minor in statistics. Michael has CDISC experience in both mapping and programming of SDTM, and creation of Define.xmls for both SDTM/ADaM standards. Past therapeutic areas of experience for Michael include asthma, COPD, ophthalmology, psychiatric disorders and oncology. Michael is a statistical programmer at Theorem Clinical Research.

Joseph Kovach is a statistical programmer at Theorem Clinical Research. He obtained a bachelor’s degree in applied statistics from West Chester University, and is currently pursuing a master’s degree in applied statistics. Joe has extensive experience with SDTM/ADaM, including specification creation, data set production and validation, as well as define.xml. Previous therapeutic areas of experience for Joe include neurology (ADHD), acute and chronic pain (diabetic neuropathy), cardiovascular system (heart failure), and idiopathic pulmonary fibrosis.

14:15 - 14:45 Who, What, When, Where and How: Basics of the Analysis Data Reviewer’s Guide (ADRG)Cathy Michalsky & Mark Gummel, Theorem Clinical Research

AbstractThis presentation will describe the basics of the Analysis Data Reviewer’s Guide – an overview of who should submit an ADRG, what is an ADRG, when it should be developed and how it should be structured including a review of required content when utilizing CDISC standards and the PhUSE template. Prior to the release of the PhUSE ADRG package, the structure and contents of the ADRG varied from company to company. We will provide examples of the ADRG used in previous successful regulatory submissions as well as review the key components of the PhUSE ADRG template. This presentation should provide a solid foundation for the understanding of when an ADRG is required and what should be contained in the ADRG.

BiographyCathy Michalsky is a director of statistics for Theorem Clinical Research and represents the Biometrics Department on project teams of major, multi-study projects of diversified scope. Cathy has over 15 years’ experience in the biopharmaceutical industry. She has worked across all study phases in multiple therapeutic areas including pain, respiratory, dermatology, cardiovascular, and renal failure. She received her MSc in Applied Statistics and BSc in Mathematics from Villanova University.

Mark Gummel is a manager in statistics, with over 16 years’ experience in the pharmaceutical industry. He has extensive hands-on experience with both SDTM and ADaM data set creation and validation. Mark holds a master’s degree in applied statistics, and has a background in many facets of pharmaceutical research including pre-clinical, clinical data management and biometrics in both a large pharmaceutical company and a CRO setting. He was formerly a member of both the SDTM virology sub-team and the DelVal CDISC Users Group. Mark is currently employed at Theorem Clinical Research based in King of Prussia, PA.


General Contributors

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ContributorsVenue Contributor Break Contributor

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PhUSE Annual Conference

October 11th-14th 2015InterContinental Wien HotelVienna, Austria

www.d-wise.com

A Balanced Approach that Scales to Meet your Needs

United StatesPhone: (919) 600-6230

1500 Perimeter Park Dr.Suite 150Morrisville, NC 27560

United KingdomPhone: +44 (0)161 209 3670

Peter House,Oxford Street,Manchester, M1 SANUnited Kingdom

Blur is the industry-leading solution for delivering de-identified clinical trial data and protecting patient privacy.Increased efficiency - Blur reduces the cost and effort of de-identifying data from individual trials, and brings scalability to the clinical trials de-identification process.

Designed for clinical trials - Embedded capabilities for clinical trial data structures (like CDISC and SAS), workflow, industry de-identification rules and regulatory practices enable Blur to match your business expectations.

Buy instead of build - Blur is an off-the-shelf software solution, developed by industry experts, that requires far less internal development, maintenance and support than SAS-based macros and programs.

12 | Upper Providence SDE 2015

Clinical Data Science –From Data to Knowledge

Conference Highlights• Inspirational Keynote Speakers• All-New Sunday Evening Welcome Event

& Poster Session• New-Look Management Stream• Over 100 Presentations Across

15 Streams and Discussion Clubs • PhUSE Scriptathon

Three Hands-on Workshops to Attend• Business & Decision Life Sciences Workshop

'Using Data Standards from Data Collection to Analysis to Submission'

• SAS Workshop 'Applying Advanced Analytics on Real-World Data: Patient-Centric Focus in Practice'

• Pharmacokinetics Workshop 'Introduction to Pharmacokinetics and Associated Data Analysis'

Workshops are free to attend although places are limited.

Keynote Speakers

Alex FlynnParkinson's Sufferer

John SallCo-Founder and Executive Vice President of SAS

Simon Weston OBEFalklands War Veteran

Vienna 2015InterContinental Wien Hotel, ViennaOctober 11th – 14th

To book a place at the Conference, visit phuse.eu/annual-conference.aspxConference Chair: Chris Price (Roche)Conference Co-Chair: Åsa Carlsheimer (Ferring Pharmaceuticals)

regulatory guidance: the elephant in the room … · regulatory guidance: the elephant in the room...

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