AllTrials AAAS 2015 - Access to anonymised patient level data
1. Access to Anonymised Patient Level Data:
Experience from GSK
Frank W Rockhold, PhD
Senior Vice President, Global Clinical Safety and Pharmacovigilance
GlaxoSmithKline
Adjunct Professor of Biostatistics and Bioinformatics, Duke University
School of Medicine
Affiliate Professor of Biostatistics, Medical College of Virginia
AAAS Annual Meeting, San Jose, CA February 15, 2015
2. 1. Why is GSK
Making Clinical Trial
Data Available?
3. 2004 2006 2008 2010
Registration
of result summaries
Seek publication
(abstract or manuscript)
in the scientific literature
Registration of protocol*
and result summaries
Seek publication as
manuscripts in
the scientific literature
+ Post full protocol
when study published
Patient clinical trials
of marketed
medicines; other
studies (exploratory,
healthy volunteer,
observational) when
relevant for patient
care
All clinical trials
of marketed medicines
+ phase III of
discontinued
compounds and
phase II if discontinued for
safety related to
mechanism
+ observational studies of
safety that impact label
All human subject
research that
evaluates marketed or
terminated medicines
- all clinical trials
- observational
studies
- meta-analyses
/pooled analyses
All human subject
research that
evaluates
medicines
- Timelines
related to the
completion of
studies (not
approval or
termination)
Registration of protocol and
result summaries
Registration of
protocol & results
2011
Seek publication (abstract
or manuscript) in the
scientific literature
*All patient clinical trials expanded to all clinical trials in June 2006
Evolution of GSK's Policy
2014
4. Public Disclosure of Clinical Trial Information
Protocol
summary
posted
Study
Start
Study
Completion
Result
summary
posted
Manuscript
submitted
Full protocol &
clinical study report*
posted on the GSK
Clinical Study Register
All human subject research studies that evaluate investigational or approved
medicinal products (phase I-IV, meta-analyses, observational studies)
8-12/18
months
18-24
months
Time of
publication
* CSR posted after approval or termination of the medicine
5. So Why are We Doing More?
Result summaries and publications have limitations
– Summarise data from the study population with statistics to compare
treatment groups.
– Do not include the data from each research participant.
Primary Efficacy Results: Total Population
Emetic Episodes Day 1 To Day 5 Dose 1 Dose 2 Dose 3
Complete (0 Episodes) 7 (19) 8 (22) 10 (31)
Major (1-2 Episodes) 10 (28) 14 (39) 10 (31)
Minor (3-5 Episodes) 0 1 (3) 0
Failure (>5 Episodes/Rescued) 19 (53) 13 (36) 12 (38)
Dose 2 vs Dose 1 0.848
6. Access to Patient Level Data to Examine the Data
More Closely or to Combine Data in Meta-analyses
7. Benefits
– Enables further research to benefit medical research and patient care.
– Enables the review of results from individual clinical trials to validate the
results.
– Helps avoid duplication of research, unnecessarily enrolling patients into
clinical trials and exposing them to risks.
– Strengthens trust in clinical research through enhanced openness and
transparency.
8. Main Issues
– Protecting the privacy and confidentiality of research participants.
– Ensuring the data are used for valid scientific investigation.
– Practicalities of anonymising data and providing data in ways that
enable external researchers to understand and navigate the information.
10. Our Vision
The aim is to help realise a broad,
independent solution to allow access
to data from clinical trials conducted
by multiple companies and
organisations.
11. GSK took a first step in 2013
– In May 2013, GSK launched a system to provide greater access to
anonymised patient level data from our clinical trials.
12. The Clinical Study Data Request System
– In Jan 2014, a new multi-sponsor request site launched:
www.clinicalstudydatarequest.com.
– Three components:
Multi-sponsor
request site
Independent
review panel
Secure access
system
13. Benefits of a common system
– Advantages for researchers and the panel: requests for data from
multiple sponsors require one proposal, one review and one set of
communications.
– Cost effective:
– Request site developed (it works and is used).
– Panel member agreements and panel charter developed.
– Work flows and communications developed for administration system.
– Multi-sponsor data access system developed by SAS.
15. GSK’s commitment
– All interventional trials started from January 1 2013.
– Trials are listed after the primary manuscript has been accepted for
publication AND the medicines has been approved / terminated.
– Goal: All interventional trials that were ongoing or started after the
formation of GSK in 2001. This listing of 2000+ trials will complete in 2015
– Currently over 1000 GSK trials are listed for request.
– Researchers can enquire about the availability of data from other non-
listed studies.
16. What information do we make available?
The following anonymised data and documents (with personally identifiable
information redacted) are made accessible in the secure system:
– Raw dataset
– Analysis-ready dataset
– Protocols with any amendments
– Annotated case report form
– Reporting and analysis plan
– Dataset specification
– Redacted Clinical Study Report including modular appendices
(potentially identifiable information, including patient level data and patient narratives are removed)
17. How it works for the researcher
Select studies that are
needed for the
research
Submit a research
proposal
Review of proposal by
independent review
panel
Agree: where
approved, a data
sharing agreement
is signed
Access data in secure
access system
Register research,
conduct and publish
18. Perspective
Data Sharing, Year 1 — Access to Data from Industry-Sponsored Clinical
Trials
Brian L. Strom, M.D., M.P.H., Marc Buyse, Sc.D., John Hughes, B.Sc., and
Bartha M. Knoppers, Ph.D.
October 15, 2014DOI: 10.1056/NEJMp1411794
The NEW ENGLAND JOURNAL of MEDICINE
The Clinical Study Data Request System One Year on...
20. GSK Metrics – (May 2013-Dec 2014)
Proposals Submitted IRP Review Using Data in Access System
•79 proposals submitted (7 are joint
with another sponsor)
•58 through requirements check (7
failed due to request for non de-
identified data and 4 withdrew
proposal)
•120 enquiries (80% approved)
•54 approved or
approved with conditions
•4 rejected with option to
resubmit
•36 research team using data
(having completed signing of
DSA)
21. Next steps
1. We hope that other industry and academic sponsors will join.
2. The Wellcome Trust is in advanced discussions about the possibility of
running the Independent Review Panel for the system.
3. We have recently surveyed researchers accessing GSK data to
understand their experience of the access system. The feedback was
positive and will inform future improvements.
23. GSK Metrics – submitted proposals (May 2013-Dec 2014)
– Total metrics for all sponsors: www.clinicalstudydatarequest.com
– For GSK studies: Total number of research proposals submitted = 79
– 72 = GSK or ViiV only (ViiV is a joint GSK/Pfizer venture)
– 7 = joint proposals request data from GSK and another sponsor.
– Valid proposals = 58 (+ 9 in process of ‘requirements check’)
– 5 withdrawn by requestor
– 7 failed check e.g. not requesting anonymised patient level data.
24. GSK Metrics – approved proposals (May 2013-Dec 2014)
– Review by the Independent Review Panel:
– Approved or approved with conditions = 54 proposals.
– Rejected with option to re-submit = 4 proposal
– Following signed data sharing agreement, further details on each project
added to site = 44 proposals
– Data preparation complete and data accessed = 36 proposals
– In addition, enquiries for >120 GSK studies not listed on site, with >80%
positive response. Researchers can add studies to future proposals.
26. Visitor Interests and demographics
– Most downloaded file DSA followed by ‘user guide’
– Most popular page after landing page was ‘view available
studies’
Editor's Notes
1
405 v1 Effective June 2004
405 v2 Effective March 2006
405 v 3 Effective June 2006
408 v3 Effective Jan 2009 (405 and 408 combined)
408 v 4 Effective July 2011
10
Of the 54 approved proposals, 17 proposals were ‘approved with conditions’. This includes requirements to add example.
4 proposals were rejected with option to be re-submitted. Reasons for this included: too little information provided on the aim of the analysis or who will be doing the analysis.
Enquiries:
- In addition to submitting research proposals for studies listed on the site, researchers are able to ask about the availability of data from GSK studies they have not yet been listed.
- Reasons for enquiries being rejected include the study has not yet been accepted for publication or GSK does not have the legal authority to provide the data e.g. if the data are owned by a third party.
Additional detail:
Metrics for all sponsors using www.clinicalstudydatarequest.com are regularly updated and available on the site.
Metrics on slide are for GSK and ViiV studies.
Research proposals are first checked to make sure the information is complete and that they meet the requirements of this initiative and the sponsor’s requirements for informed consent. Seven research proposals were withdrawn by the requestor or identified as not valid e.g. were not requesting anonymised patient level data.
Of the 54 approved proposals, 17 proposals were ‘approved with conditions’. This includes requirements to add example.
4 proposals were rejected with option to be re-submitted. Reasons for this included: too little information provided on the aim of the analysis or who will be doing the analysis.
Details added to site following signed data sharing agreement include lay summary, requested studies, aim and methods
Enquiries:
- In addition to submitting research proposals for studies listed on the site, researchers are able to ask about the availability of data from GSK studies they have not yet been listed.
- Reasons for enquiries being rejected include the study has not yet been accepted for publication or GSK does not have the legal authority to provide the data e.g. if the data are owned by a third party.
