The document discusses the legal and policy issues surrounding clinical trial data sharing, emphasizing the importance of transparency, autonomy, and public health. It outlines the benefits of data sharing, such as promoting new discoveries and minimizing duplicative trials, while also addressing the ethical considerations of participant consent and privacy. The author advocates for a balanced approach that respects individual autonomy while maximizing the utility of clinical data for the public good.

1. Transparency and Clinical Trial Data Sharing:
Legal and Policy Issues
Autonomy versus Public Health
Barbara E. Bierer, MD
Faculty Co-Director, Multi-Regional Clinical Trials Center of Brigham
and Women’s Hospital and Harvard (MRCT Center)
Program Director, Regulatory Foundations, Law and Ethics Program
Harvard Catalyst | the Harvard Clinical and Translational Science
Center, Harvard Medical School
Professor of Medicine, Harvard Medical School
April 28, 2017 Petrie-Flom Center Annual Conference

2. Disclaimer
• The opinions contained herein are those of the authors and are not
intended to represent the position of Brigham and Women's Hospital or
Harvard University.
• The MRCT Center is supported by voluntary contributions from
foundations, corporations, international organizations, academic
institutions and government entities (see www.MRCTCenter.org) and well
as by grants.
• We are committed to autonomy in our research and to transparency in
our relationships. The MRCT Center—and its directors—retain
responsibility and final control of the content of any products, results and
deliverables.
• I have no personal conflicts of interests with this presentation.
3/29/17 © MRCT Center 2

3. Openpaymentsdata.cms.gov
© MRCT Center 3

4. Sharing Clinical Trial Data Speeds Science
Data sharing is making research data available to other
investigators, researchers and the public.
Enables new discovery and new research questions by allowing more
scientists to use and combine existing data
Validates findings by making it easier to re-analyze and review prior results
Prevents unnecessary repetition of trials – and the waste and risk to
additional patients -- when data already exist
Data sharing makes the best use of participant’s volunteerism and
contributions, and of society’s investment, in clinical trials, and
enables research using modern ”big data” methods
© MRCT Center 4

5. Increasing transparency over time
• Evolution of clinical trials to determine safety and efficacy of
investigational products in absence of bias
• Clinical trials registration (www.ClinicalTrials.gov;
www.who.int/ICTRP, other)
• Result reporting as a requirement of the Final Rule (2016)
• Sharing of IPD
5
Randomized
Clinical Trials
Clinical Trials
Registration
Clinical trials
Results
Reporting
Data Sharing:
Individual
Patient Data
(IPD)
© MRCT Center

6. Geographic distribution of ~250,000 clinical trials
registered on ClinicalTrials.gov
6© MRCT Center

7. Registered Studies
7© MRCT Center

8. Studies with Results Reporting
8© MRCT Center

9. Scope of Personal Data
Any information:
• Relating to a natural, living person
• Who can be identified, directly or indirectly, in particular
by reference to an identifier such as:
– a name
– an identification number
– location data
– online identifier
• or to one or more factors specific to the physical,
physiological, genetic, mental, economic, cultural or
social identity of that person.
© MRCT Center 9

10. Anonymization
• Different methodologies
• In US, either “strip” of 18 identifiers or utilize statistical
methods to demonstrate that the risk is low
– How low is low enough?
• Ex US, HIPAA method is insufficient
• Variable methods utilized, but none are perfect—and if
perfect, the data is not informative
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The risk of re-identification is not zero

11. Why share?
“Publicly funded research data are a public good,
produced in the public interest, which should be made
openly available with as few restrictions as possible in a
timely and responsible manner”*
• Enables new discovery
• Prevents duplicative trials when unnecessary
• Avoids putting patients/participants at risk
• Allows replication and promotes data integrity
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* RCUK Common Principles on Data Policy. Research Councils UK, revised July 2015.
Available at http://www.rcuk.ac.uk/research/datapolicy/, accessed 05/03/2017.
© MRCT Center

12. Why IPD?
• Dr. Eric Bomberg is a
pediatric endocrinologist at
UCSF.
• He wants to explore using
GLP-1 agonists to reduce
obesity and prevent Type 2
diabetes in children
• But which children would
lose weight on GLP-1?
12© MRCT Center

13. Valuable Clinical Trial Data Can Help
• There have been 11 completed GLP-1
trials on a total of 21,196 children
• Dr. Bomberg wants to find predictors
of which children will lose weight on
GLP-1 agonists
• To do this, he wants to
– Mine the 21,196 patient records to
identify predictors
– Combine the trial data with
electronic health record data to
verify that the predictors work in
routine care
13
n = 18,000
3,100
301
29
7
14
2
10
97
952
113
67283
exenatide studies
liraglutide studies
19,985
1,211
© MRCT Center

14. Detailed IPD is necessary
14
Privacy Transparency
Maximize Data Utility
Optimize Anonymization
© MRCT Center
Utility and IntegrityRespect

15. Autonomy and (versus?) Public Health
• Every participant in a clinical trial must give voluntary
informed consent for participation in the trial.
• But what about consent for data sharing?
• Must participants consent to data sharing?
• Should participants consent to IPD data sharing?
• If you know you will anonymize the data, should you tell
potential participants that you will do so?
• Can they refuse?
© MRCT Center 15

16. Autonomy and (versus?) Public Health
Participant consent to data sharing
• Assume data will be anonymized
• Assume consent will be as complete as possible
• Ethically appropriate, but is there an obligation to do so?
• If consent is declined, can the data be anonymized and
used anyway?
• Note: If dataset is incomplete, the dataset itself is less
useful for discovery, replication
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17. Autonomy and (versus?) Public Health
• If consent is necessary, how broad should the consent
be, or how explicit to purpose?
• Can broad consent ever be “informed”?
• If an opt-out sufficient or must the consent be
affirmative?
• Opt-out in many EU countries is not considered valid
consent.
• Future of data privacy is, at best, unpredictable (e.g.
General Data Protection Regulation (GDPR, EU
2016/679)
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18. Autonomy and Data Sharing
• Can data sharing be an inclusion criterion for the study?
• Can it be implied as a condition of participation in the
trial?
• Some have argued that requiring data sharing as a
condition of participation is “coercion.”
• Does it matter if the trial may have the possibility of
direct benefit?
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19. Consequence of misuse
• Currently basis for data re-use is informed consent
• Informed consent, however, does not protect against
intentional misuse
• In the US (at least), few consequences for re-
identification and/or misuse
• Arguably, a different paradigm could be envisioned:
– Transparency in intent, Information and education
– Civil (and/or criminal) penalties for misuse
– Oversight is difficult
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20. Closing Remarks
• Risk minimization as a principle
– Depending on sensitivity of data, access only through secure
channels with validation of user
– Encryption etc.
• Initiate program of education regarding utility of clinical
data for the public good
• Information transparency and insofar as possible,
standardization of information
• Legal consequences for misuse of data
• If decision is consent, then appreciate limitations of
consent 20© MRCT Center

21. Thank you
Discussion
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