Principles for Responsible
Clinical Trial Data Sharing
Our Commitment to Patients and Researchers

Biopharmaceutical companies are committed to enhancing public health through responsible sharingof clinical trial data in a manner that is consistent with the following Principles:•Safeguarding the privacy of patients•

Respecting the integrity of national regulatory systems•

Maintaining incentives for investment in biomedical researchCompanies routinely publish their clinical research, collaborate with academic researchersand shareclinical trial information on public websites at the time of patient recruitment,after new drug approvaland when investigational research programs have been discontinued.
Biopharmaceutical companies will apply these Principles for Responsible Clinical Trial Data Sharing
as a common baseline on a voluntary basis, and we encourage all medical researchers, including
those in academia and in the government, to promote medical and scientific advancement byadopting and implementing the following commitments:

1. ENHANCING DATA SHARING WITH RESEARCHERS
Biopharmaceutical companies commit to sharing upon request from qualified scientific and medicalresearchers patient-level clinical trial data, study-level clinical trial data and protocols from clinical trials
in patients for medicines and indications approved in the United States (U.S.) and the European Union(EU) as necessary for conducting legitimate research. Companies will implement a system (that could
include consortiums, multi-sponsor data sharing platforms or use of third-party vendors) to receive andreview research proposals and provide applicable data and protocols to help facilitate such scientificand medical research.
Each company will establish or secure access to a scientific review board. In all cases, reviewers will
include scientists and/or health care professionals who are independent of the company. Members of
the scientific review boards will participate in the review of data requests to determine whether theymeet the criteria described below regarding the qualifications of the requestor and the legitimacy of
the research purpose, unless a company makes an initial determination on its own to share applicableclinical trial data. Companies will publicly post their data request review process and ensure that theidentities of the external scientists and health care professionals who participate in the scientificreview board are disclosed, including any existing relationships with external board members.

1Companies will provide access to patient-level data and other clinical trial information consistent
with the principle of safeguarding patient privacy; patients’ informed consent provided in relation totheir participation in the clinical trial will be respected. Any patient-level data that is shared will beanonymized to protect personally identifiable information. Companies will not be required to provide
access to patient-level data if there is a reasonable likelihood that individual patients could be
re-identified. In addition, clinical data, in some cases, have been collected subject to contractual
or consent provisions that prohibit transfer to third parties. Such restrictions may preclude granting
access under these Principles. Where co-development agreements or other legal restrictions prevent
companies from sharing particular data, companies will work with qualified requestors to provide
summary information where possible.
Data requestors will be required to submit a research proposal to document the legitimacy of theresearch question and the qualifications of the requestor. Research proposals should include and will
be evaluated against the following: a description of the data being requested, including the hypothesis
to be tested; the rationale for the proposed research; the analysis plan; a publication and posting plan;qualifications and experience of the proposed research team; a description of any potential conflictsof interest, including potential competitive use of the data; and the source of any research funding.
Researchers who are provided access to company data will be encouraged and expected to publish theresults of their analysis so that it may be peer-reviewed and to consider publication via an open accessformat. Researchers must agree not to transfer the shared data or information to parties not identified
in the research proposal, use the data for purposes not contained in the research proposal or seek tore-identify research participants.

2. ENHANCING PUBLIC ACCESS TO CLINICAL STUDY INFORMATION
In order to help patients and health care professionals understand the results of clinical trials and theevidence used to approve a new medicine, following approval of a new medicine or new indication for
an approved medicine in the U.S. and EU, biopharmaceutical companies will make publicly available,
at a minimum, the synopses of clinical study reports (CSRs) for clinical trials in patients submitted tothe Food and Drug Administration (FDA), European Medicines Agency (EMA) or national competentauthorities of EU Member States. Companies will make this information available consistent with theneed to protect patient privacy, publication rights and confidential commercial information throughappropriate redaction.In addition, companies will evaluate requests for full CSRs, including patient-level
andstudy-level data, and share them under the terms of commitment 1 above. Companies will makeavailable technical results or CSR synopses for studies filed with regulators on or after January 1, 2014;
such results will be made available to the public (e.g., on a public registry or company website) withina reasonable period of time after approval of the product and indication.

3. SHARING RESULTS WITH PATIENTS WHO PARTICIPATE IN CLINICAL TRIALSIn order to help inform and educate patients about the clinical trials in which they participate,biopharmaceutical companies will work with regulators to adopt mechanisms for providing a factualsummary of clinical trial results and make the summaries available to research participants.

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4. CERTIFYING PROCEDURES FOR SHARING CLINICAL TRIAL INFORMATION
Companies following these Principles for Responsible Clinical Trial Data Sharing will certify on apublicly available website that they have established policies and procedures to implement thesedata sharing commitments.

5. REAFFIRMING COMMITMENTS TO PUBLISH CLINICAL TRIAL RESULTS
All company-sponsored clinical trial results should be considered for publication in the scientific
literature irrespective of whether the results of the sponsors’ clinical trials are positive or negative sothat it may be peer-reviewed. Companies should consider using an open access format. At a minimum,
results from all phase 3 clinical trials and any clinical trial results of significant medical importanceshould be submitted for publication. This commitment also pertains to investigational medicines
whose development programs have been discontinued.
Implementation of these commitments will begin on January 1, 2014.

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Principles for Responsible
Clinical Trial Data Sharing
Questions and Answers

Q: What type of information arein interpreting the outcome of a clinical study.

biopharmaceutical companies prepared to share

Study-level data present clinical trial data in anwith qualified medical and scientific researchers

objective manner, without subjective analysis

under commitment 1?

or interpretation, usually in tabular, graphic or

A: The biopharmaceutical industry is committing
to sharing with qualified medical and scientificresearchers patient-level data, study-level dataand clinical study designs and protocols.

statistical form showing, for example, averaged,
stratified or patterned presentations of study data
gathered. Examples would include a table that
presents cross-patient data on baseline patientcharacteristics (demographic and disease-related),

Patient-level data refer to information on

patient disposition (i.e., numbers/percentages ofindividual patients collected during a clinicalpatients who completed or discontinued the trial),

study, including: demographic data, lab results,endpoints (primary, secondary and other), study

baseline characteristics, drug concentration,

drug exposure, adverse events, vital signs andbiomarker and pharmacogenetic data and adverse

laboratory and other safety measures provided forevents experienced. Such information has beenthe overall study population and by subgroups.

gathered and recorded on case report forms(CRFs) or captured electronically and inputted
into electronic databases, where it can be readilyorganized into patient-level listings and datasets.
This information is created through what theInstitute of Medicine (IOM) has described as aprocess by which data in a clinical study originate
with CRFs, either handwritten or electronic, thengo through several stages of auditing, queries
and refinement by original investigators and study
staff to resolve ambiguities, and then ultimatelyClinical study design information and protocols
direct investigators how to run a particular study.
Protocols give instructions to the investigatorson, for example, what drug to give and when, whatstudy measurements to take and when and how
to record them and how to treat and record
adverse events.

Q: What is the rationale for providing the synopsisof CSRs in commitment 2?

yield “individual participant data.”1

A: Given the volume of data contained inStudy-level data consist of patient-level dataof pages—companies commit to publishing athat have been amalgamated, compiled andtabulated, manipulated, stratified or otherwiseorganized into study-level data sets to be used

regulatory submissions—often running to millions
synopsis after marketing approval in the U.S.,
EU or member states. The synopsis will provide
patients and their physicians with enhanced

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information about the results of clinical trials

Q: Under commitment 1, are companies

and the evidence used to approve a new medicine.

committing to share patient-level data and

The synopsis is a part of the CSR and is reviewed

other proprietary information with competitors?

by the FDA and EMA as part of their approval.
In order to accelerate research and advancescientific understanding, companies will also
evaluate requests for full CSRs, including patientlevel and study-level data, and share them under
the terms of commitment 1. Companies may
also or instead publish technical results, also
known as a results file, to ensure the results ofin-scope studies are made publicly available to allinterested parties.

A: No. Discovering and developing a new
medicine is a long, complex and costly process.
Today, bringing a new medicine from concept
to market can take an average of 10—15 years.
As a result, the average cost to develop a new
medicine has grown from $179 million in the1970s3 to an average $2.6 billion today including
the cost of failures,4 with overall development
costs more than doubling in the last decadedue to growing complexities.5 The risks involved

In addition to providing the synopsis, somein developing new drugs are also substantial.

companies may choose voluntarily to provide

For every single approved medicine, tens ofto the public additional parts of CSRs redacted

thousands of compounds have been screened

to protect patient privacy and confidential

during the research and development process.

commercial information.

Even medicines that reach clinical trials have a

Q: Why may it be necessary to limit theavailability of patient-level data for clinical trials
conducted involving patients whose data arelikely to be re-identified?

A: Protecting the privacy of patients who
participate in clinical trials is a critical obligation,
both ethically and legally, as defined in global
privacy laws such as the European Union
Global Data Protection Regulation (EU GDPR),of biopharmaceutical companies that sponsor
and conduct medical research. It may be possible
even for “anonymized” patient-level data to
be re-identified using modern data mining
techniques.2 For this reason, companies generally
withhold patient-level information from disclosure
when there is a reasonable possibility that patient
privacy could be jeopardized. The risk of “reidentification” is significantly higher when thenumber of patients is small, such as is typicallythe case for trials involving patients with rarediseases, which may include as few as 25 orfewer patients.

less than 12% chance of being approved,6 andonly 2 out of 10 approved drugs produce revenues
that match or exceed average research anddevelopment costs.7
Biopharmaceutical companies are dedicatedto fostering a sustainable research ecosystemthat protects the ability of companies to makeextremely costly investments to discoverand develop new medicines. One of the risks
to innovation is disclosure to competitors ofcompanies’ trade secrets and proprietary
information that could allow others to “free ride”
off of the substantial investments of innovators.
Such an environment will not foster the abilityof companies to make decades-long investments
in new medical technology. Therefore, in asustainable research ecosystem, companies mustbe certain that their proprietary information willremain secure from disclosure to competitors.
That is why commitment 1 calls for a company
to share patient-level data and other confidentialcommercial information—which could be used tohelp gain approval of a competing medicine—only5

for legitimate scientific and medical research.

and other legal permissions, such as privacy

Commitment 1 reflects these concerns by allowing

authorizations (e.g., HIPAA in the United States)

companies to consider requests for release of

and/or data use agreements. With respect to theseclinical information in light of potential conflicts

commitments to patients, any patient-level dataof interest, including any potential competitive usethat can be shared will, therefore, be “anonymized”

of the data.

in accordance with applicable legal requirements

Under commitment, companies will evaluate,
among other things, whether the research
proposed has a legitimate scientific or medical
purpose, including whether there is any potential
conflict of interest between the data requestor
and the company or competitive use of the datafor ongoing development activities and/or global
regulatory activities outside the EU and U.S. In
the latter case, it may be assumed that the data
requestor may intend to use the company’s
patient-level data or other information to help
gain approval of a potentially competing medicine.

to protect personally identifiable information.
Companies will not provide access to patientlevel data when there is a reasonable likelihoodthat individual patients could be re-identified. In
addition, where co-development agreements orother legal restrictions prevent companies fromsharing particular data, companies will workwith qualified requestors to provide summary
information if feasible.

Q: Will there be any other restrictions on use ofdata provided under commitment 1?

While companies may enter into agreements to

A: Each company will determine the best

co-develop medical products, these data sharing

method for safeguarding the privacy of patients

Principles are not intended to allow free-riding or

and ensuring that access to patient-level

degradation of incentives for companies to invest

data does not jeopardize incentives for future

in biomedical research. Accordingly, it would be

investment in biomedical research. Commitment

appropriate under commitment 1 for companies

1 requires that data requestors must agree not toto refuse to share proprietary information withtransfer shared data to parties not identified intheir competitors.

the research proposal, use the data for purposes
not contained in the research proposal or seek

Q: How will companies determine who canto re-identify research participants. Companies

receive patient-level data or other proprietary

may also require that the data are only used forinformation?

non-commercial purposes. Additional conditions

A: Each company will implement a system forreviewing research proposals and the credentials
of requesting researchers to determine that theproposed research is bona fide. Companies may
choose to implement these systems individually,
through consortiums, multi-sponsor data sharing
platforms or use of third-party vendors and/orwith centralized scientific review boards. Amongthe considerations for protecting patient privacy
are the research participants’ informed consent

may include granting access to the data only on
a company’s information system and/or requiringthat data requestors notify the company of any
safety finding that may be reportable to regulatory
authorities or of other significant results.

Q: Other than patient privacy information,
what type of information could be withheld from
CSR information provided to the public undercommitment 2?

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A: In order to maintain incentives for future

Q: If a company chooses, may it share moreinvestment in biomedical research, individual

clinical trial information than is described incompanies may choose at their discretion tothese commitments?

withhold from public access to CSRs various
business and analytical methods; manufacturing
and pre-clinical information or other confidentialcommercial information; any information notdirectly related to the conduct of the study orthat could jeopardize intellectual property rights;
or information that the company has no legal rightto share (e.g., due to an existing co-development
agreement).

A: Yes. Companies will make their own
determinations regarding how to implement
these commitments and whether to exceed
these common commitments to responsible data
sharing. For example, companies may choose toprovide voluntarily to members of the public themain body of CSRs redacted to protect patient
privacy or confidential commercial information.

Information withheld from public access toCSRs may nevertheless be available to qualified
researchers under the terms of commitment 1.

Sources1

Institute of Medicine, Sharing Clinical Research Data: A Workshop Summary 10 (2013).

2

See Melissa Gymrek et al., Identifying Personal Genomes by Surname Inference, 339 SCIENCE 6117 321-324 (2013).

3

J. A. DiMasi, R. W. Hansen, and H. G. Grabowski, The Price of Innovation: New Estimates of Drug Development Costs,
Journal of Health Economics 22 (2003): 151—185.

4DiMasi JA, Grabowski HG, Hansen RW; Tufts Center for the Study of Drug Development. Innovation in thepharmaceutical industry: new estimates of R&D costs. In: Briefing: Cost of Developing a New Drug.

5Previous research by DiMasi and Grabowski estimated average R&D costs in the early 2000s at $1.2 billion inconstant 2000 dollars (see DiMasi JA, Grabowski HG. The cost of biopharmaceutical R&D: is biotech different?
Managerial and Decision Economics. 2007;28: 469-479). That estimate was based on the same underlying survey
as the author’s estimates for the 1990s to early 2000s reported here ($800 million in constant 2000 dollars), but
updated for changes in the cost of capital.

6DiMasi JA, Grabowski HG, Hansen RW; Tufts Center for the Study of Drug Development. Innovation in thepharmaceutical industry: new estimates of R&D costs. In: Briefing: Cost of Developing a New Drug.

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J. A. Vernon, J. H. Golec, and J. A. DiMasi, Drug Development Costs When Financial Risk Is Measured Using the FamaFrench Three-Factor Model, Health Economics Letters (2009).

June 21, 2023

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