This document summarizes the changing landscape of clinical trial data transparency in the pharmaceutical industry. It discusses how patient-level clinical trial data was previously kept private but is now being shared more openly. Major players in the industry have implemented different approaches to sharing historical trial data through independent review boards. While the EMA supports more transparency, the FDA has not taken an official position and views data sharing as between companies and patients. Overall the industry is moving towards greater openness, though approaches and eligible trials vary by company.

1. © d-Wise 2013 Page 1
Seeing is Believing: How Clinical Trial
Data Transparency is Changing How an
Entire Industry Thinks about Data
Dave Handelsman
Senior Director, Industry Strategy
15-Oct-2014

2. The good ole’ days…
 Patient-level clinical trial data, summary
tables, study reports and other associated
evidence are delivered to regulatory
authorities as part of the drug approval
process.
 Some evidence is additionally shared on sites
like clinicaltrials.gov (summary results,
synopses, etc.)
 Patient-level clinical trial data is not shared
with anyone except the regulators.
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3. What changed?
http://www.ema.europa.eu/docs/en_GB/document_library/Other/2013/06/WC500144730.pdf Industry’s initial reaction
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4. Industry’s Thoughtful Response
http://phrma.org/sites/default/files/pdf/PhRMAPrinciplesForResponsibleClinicalTrialDataSharing.pdf
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5. Transparency in the News
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6. Transparency:
The Basic Process
Proposal RequestSite
ReviewPanel
Transparency
Analytics Repository
Proposal
request Approved
Rejected
Re-apply?
Approved
research
artifacts
https://clinicalstudydatarequest.com/
(for manycompanies)
Data
Documents
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7.  For processing investigator requests:
– Many, but not all, sponsors are using
www.clinicalstudydatarequest.com
– JnJ/Janssen is using YODA (Yale Open Data Access)
– Bristol Myers Squib using DCRI (Duke Clinical Research
Institute)
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8.  For determining what trials to include:
– Much less agreement between organizations
– Some companies going back very early – e.g.,
late 1990’s
– Some companies will only include patient-level
data from new trials started after the decision
to be transparent
– Others are somewhere in the middle
 Key challenges:
– Informed consent – widely varied
interpretations
– Locating and delivering data
– Data de-identification / anonymisation /
privacy
Go to
www.clinicalstudydatarequest.com
and click on a sponsor
to learn more
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9. Is there a standard industry approach?
(Well, sort of….)
 Varied paths are being taken for…
– Organizing the independent review
boards
– Defining the business rules regarding
how analyses and other artifacts are
extracted from the system to support
publications and claims
 Key differences that influence
participation:
– Executive commitment
– Legal perspective
– Availability of resources
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10.  How will the data be delivered
– CDISC (mostly, probably, hopefully)
– Consistently de-identified
 Why is this important?
– Efficient analysis and review by the
independent researcher
– Patient privacy protection
– Pooling clinical trial data across
multiple companies
Dave’s initial reaction
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11.  There are no required data standards for the Transparency
Analytics Repository
 CDISC is a likely approach but researchers will have to be
ready to interpret / understand / prepare the available
transparency data to investigate their research proposal
It’s already happening….
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12. Amgen
Sources: Applied Clinical Trials compilation of company web site data, June 30, 2014
Sea Change in Open Science and Data Sharing: Leadership by Industry, Circ Cardiovasc Qual Outcomes, published online June 2, 2014;
http://www.forbes.com/sites/harlankrumholz/2014/06/02/time-to-assess-pharma-progress-in-data-sharing/
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Email request Internal review, and “as appropriate” Not included in Forbesanalysis
Bayer www.clinicalstudydatarequest.com Independent centralized reviewpanel
• http://www.forbes.com/sites/harlankrumholz/2014/06/0i2nd/ictaimtiones-tthoat-ahasvseebseesn-paphparorvmedab-yEU
for further regulatory review orsubmissions
Company Research request type Review Board AvailableData
AbbVie Email request Internal review, with appeal All data for medicines and indicationsapproved
Sources: independent; external board for final in the US and the EU
• Applied Clinical Trials compilatiode
ncis
oio
fn
companyweb site data, June 30, 2014
• Sea Change in Open Science anidndDepaentdaenSthexaterrinnagla:dLviesoarsdership by Industry, Circ Cardiovasc
AstraZene
Qca
ualONo
ut
tp
co
ost
med
es,published onlinN
e/A
June 2, 2014; Not statedonline
Jan 2014 – present; Data on new medicinesand
progress-in-data-sharing/ and/or US regulatory agencies without plans
Biogen Idec Email request (developing a Not posted Not included in Forbesanalysis
request/approvalportal)
Boehringer
Ingelheim
www.clinicalstudydatarequest.com Independent centralized reviewpanel Not included in Forbesanalysis
BristolMyers
Squibb
Company-specific requestportal External scientific review provided by
DCRI facultymembers.
Jan 2008 – present
Celgene Email request External Scientific Review Board Not included in Forbesanalysis
Eli Lilly www.clinicalstudydatarequest.com Independent centralized reviewpanel Interventional clinical studies forapproved
indications of medicines on the market in the
US andEU
EMDSerono Not posted External scientific review board Not included in Forbesanalysis
GlaxoSmithKline www.clinicalstudydatarequest.com Independent centralized reviewpanel Dec 2000 – present; All global interventional
clinicalstudies

13. Patient-Level Trial Transparency Status
Top Pharma
Sources: Applied Clinical Trials compilation of company web site data, June 30, 2014
Sea Change in Open Science and Data Sharing: Leadership by Industry, Circ Cardiovasc Qual Outcomes, published online June 2, 2014;
http://www.forbes.com/sites/harlankrumholz/2014/06/02/time-to-assess-pharma-progress-in-data-sharing/
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Company Research requesttype ReviewBoard AvailableData
Janssen
Pharma
Internal requestportal External review provided by Yale Schoolof
medicine’s Open Data AccessProject
(YODA).
Data from interventional trials for productsand
indications approved in both the EU and US(older
studies may be difficult toaccess)
Lundbeck Not posted. “Lundbeck and a thirdparty
will be subject to a formal agreementthat
will address ownership and access to
data.”
Specific criteria listed onsite Not included in Forbesanalysis
Merck Emailrequest Internal review, external review boardas
needed
Sept 2007 – present; Trials approved by EU andUS
regulatory agencies and accepted for publication;
Trials for which results are posted onClinicalTrials.gov
Novartis www.clinicalstudydatarequest.com Independent centralized review panel Jan 2014 – present; Phase II – III trials for new
medicines or indications approved by the EU andUS
regulatoryagencies
Novo
Nordisk
Emailrequest Independent review board, dataaccess
granted within a web-basedsystem.
Not included in Forbesanalysis
Otsuka Not yetdeveloped. Not yetdeveloped. Not included in Forbesanalysis
Pfizer Internal requestportal Independent review panel Global trials that ended after Sept2007;
Trials for which results are posted onClinicalTrials.gov
Purdue
Pharma
Not posted; PhRMA-certified tofollowing
jointprinciples.
Information notavailable Not included in Forbesanalysis
Roche www.clinicalstudydatarequest.com Independent centralized review panel Jan 1999 – present; Trials from terminatedprograms;
Phase II – III trials, and some Phase IV trials, for new
medicines that have been approved by the EU andUS
regulatoryagencies
Sanofi www.clinicalstudydatarequest.com Independent centralized review panel Jan 2014 – present
Trials approved by EU and US regulatory agencies and
accepted forpublication

14. What is the FDA saying about Patient-Level
Clinical Trial Data Transparency?
 FDA has been not posted guidance or regulation or anything
official
 At conferences, when asked about patient-level clinical trial
data, FDA has been consistent in their response
Dave’s paraphrasing of FDA:
Decisions regarding patient-level clinical
trial data are between the pharmaceutical
company and the patient. FDA has no
intention of making submitted patient-level
trial data available as the EMA has
indicated, and has no intention of requiring
pharma to make that data available.
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15. http://w
3/06/WC5
ww.ema.europa.eu/docs/en_GB/document_library/Other/201
00144730.pdf
What is the EMA saying about Patient-Level
Clinical Trial Data Transparency?
http://www.ema.europa.eu/docs/en_GB/document_library/Other/2014/10/WC500174796.pdf
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16. What’s next?
 Biopharmaceutical companies will continue to implement
Clinical Trial Data Transparency solutions
 Many companies will converge on a single stack of technologies
 TransCelerate has recently gotten involved from a CSR redaction
and patient privacy perspective
 There are divergent opinions regarding the types of trials to
include, risks associated within including trials associated with
compounds not submitted for approval, business process
consensus, etc.
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17. Questions ?
Contact information:
Dave Handelsman
dave.handelsman@d-wise.com
919 5920607
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