Principles Regarding The Use of Real World Evidence
Medical device regulators have shown increased interest in the use of real world evidence(“RWE”) in pre- and post- market regulatory decision making. As RWE collection
methods are developed, and platforms become available to regulators, manufacturers,academics, and providers alike, we believe that certain foundational principles arenecessary with respect to the use of RWE, and to guide the governance and operation of asystem that may be established to collect, disseminate and evaluate RWE.
I.

BACKGROUND

RWE has been defined as evidence derived from the aggregation and analysis of realworld data (“RWD”). 1 RWD, in turn, has been defined as data collected from sources
outside of traditional clinical trials. RWE may consist of information from many data
sources, including but not limited to:
1. Registries;
2. Patient Communities;
3. Public and Private Health Plans;
4. Manufacturers;
5. Delivery Systems, including electronic health records (EHR);
6. Regulators;
7. Safety surveillance systems; and8. Observational studies and scientific literature.

GMTA is committed to the principles of appropriate analysis of medical technologies.
Stakeholders, such as patients, their caregivers, providers, payers, regulators, andmanufacturers share in the commitment to improve the quality and increase the efficiency
1See, e.g., U.S. Food and Drug Administration Draft Guidance: Use of Real-World Evidence to Support
Regulatory Decision-Making for Medical Devices (July 27, 2016), available athttps://www.fda.gov/downloads/medicaldevices/deviceregulationandguidance/guidancedocuments/ucm513
027.pdf.

of healthcare. As medical device manufacturers, we recognize the need to ensure
adequate and accurate information concerning the safety, effectiveness, and impact of
medical interventions to guide healthcare decision-making. We further recognize there
may be a number of benefits with respect to the use of RWE, including the following:
1. Improve patient care and outcomes;
2. Improve patient access to new therapies and diagnostics by efficiently
collecting data to support regulatory submissions for expanded use andindications or expanded coverage;
3. Support device modifications;
4. Support patient access through expanded coverage, reimbursement, and valueanalysis based on RWD evaluation and collection;
5. Evaluate the “real-world” safety and/or effectiveness of products outside ofrandomized controlled clinical trials or other clinical study designs;
6. Meet regulatory requirements for post-market data collection;
7. Provide regulators with alternative methods to monitor the performance oftechnologies to reduce existing pre- and post- market data collection burdens;
8. Develop hypotheses for further clinical and economic evaluation; and9. Aid in the development or assessment of care guidelines.
Although we recognize these potential benefits as possible outcomes, by definition
RWD is not data collected from a clinical investigation. Therefore, it is importantthat this information be appropriately analyzed and validated prior to dissemination
and use.
In addition, we believe protections must be in place to prevent the spread of incorrect,incomplete, biased or misleading information. The public health relies on thecommunication and dissemination of accurate and reliable information from publichealth agencies. In this regard, we provide below key principles with respect to theuse of RWE, and to guide the governance and operation of a system that may beestablished to collect, disseminate and evaluate RWE.
II.

PRINCIPLES

1. Systems designed to collect RWE must include a clear purpose, objective, andparticipation requirements for promoting the use of RWE to facilitate
understanding of medical technology and innovation.

2

a. Criteria for an RWE system’s partners and sources of data must be
established to ensure partners providing information are committed to thesystem goals and that data are relevant and useful for RWE purposes.
b. RWE system partners must establish sufficient safeguards to ensure thatRWE data are of appropriate quality for the intended use of the data.
c. An RWE system must be validated for each purpose before its intended use.
d. Data integrity and security must be maintained across an RWE system bysystem partners.
e. RWE system partners must agree to follow data governance standards.
2. Before the launch of an RWE system, a data governance committee (thatincludes medical device manufacturers) must be formed and written proceduresfor data ownership, data access, data use, and participation must be established.
a. An RWE system must collect sufficient data to identify, consider and allow
risk adjustment for modifiable risk factors such as social, demographic anddisease-related factors, including changes in applicable evidence based
practice guidelines over time.
b. An RWE system governance committee must establish procedures forreviewing proposed research protocols that would use RWE or RWD
obtained through the system.
c. Findings derived from RWE or RWD obtained through an RWE system must
be reviewed by a data governance committee or through an appropriate peer
review committee prior to release. Manufacturers of the technologies studied
must be part of the review process as the manufacturer may have relevant
information to any conclusion. Manufacturers, however, do not need toapprove the conclusion.
d. An RWE system’s data governance committee must establish a process toensure transparency of the process for proposing, conducting, and releasing
reports based on RWE.
3. Regulatory authorities must have clear policies regarding the use of data froman RWE system, if applicable. Use of data from an RWE system to make aregulatory determination of device safety or effectiveness must only be
conducted by regulatory authorities in accordance with their statutory
authorities.
a. Regulatory authorities should seek input from and share relevant information
with manufacturers prior to taking any regulatory action based upon data
derived from an RWE system.
b. Regulatory authorities should actively work with manufacturers to identify
possible uses of RWE derived from an RWE system to address pre-market
requirements in a least burdensome manner.
c. In the interest of reducing healthcare costs, device surveillance systems
derived from an RWE system should be developed to replace, rather thansupplement, existing passive surveillance systems.
d. An RWE system must make clear that RWD and RWE analysis plans are notclinical investigations.
3e. Manufacturers should be able to communicate truthful and non-misleading
information about analyses of RWD, provided research methods are soundand well-described.
4. Access to data through an RWE system should be granted only upon request byqualified scientific, medical and economic researchers for purposes benefiting
public health or patient care. Procedures should be developed and employed bya data governance committee to receive and review data access anddissemination requests prior to approving the release of any data.
a. Requests for data access must be based on clear criteria established by a data
governance committee, including the validity of the hypothesis, whether thedata requested and analysis plan will address the hypothesis, and thequalifications of the requestor.
b. A data governance committee must establish a process for adequate dataprotection, including a process to ensure agreement from individuals granted
system access not to transfer the data or information to parties not identified in
the research proposal.
c. An approved analysis plan must address how data on patient characteristics,
patient medical conditions and co-morbidities, facility characteristics,
physician experience, interventional technique and associated parameters, anddevice characteristics (including unique device identifiers, if applicable) will
be used to identify potential factors that affect study conclusions.
d. Research partners must define a prospective process for considering changes
in the analysis plan after initiation, including items such as data collection andprotocol revisions.
e. Device manufacturers must be allowed full and timely access to data on theirproducts.
f. Data requesters may be charged reasonable fees.
5. An RWE system must comply with all applicable laws and regulatory
requirements.
a. Patient privacy must be protected, and any required consent must be obtained.
b. All confidential manufacturer, physician, and hospital data must be identified
as confidential and protected from release.
c. Data provided to an RWE system does not absolve facility, physician or
manufacturer obligations to file reports required under applicable laws orregulations.

4