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Posted 02 November 2020 | By Kari Oakes 

Sponsors get RWE guidance from MHRA

2705 The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has issued a draft guidance on the use of real-world evidence in clinical trials. The guidance, which remains open for consultation for 6 weeks from its 30 October publication date, is intended for sponsors planning clinical research in support of regulatory decisions.
MHRA notes that real-world evidence (RWE) may help investigators get at how a treatment will really work when it is in community use than data generated by a conventional clinical trial; also, RWE may be more generalizable. Though there are some scenarios where MHRA is more likely to look with favor on a RWE approach than others, “nothing is completely ruled out on principle, including the investigation of new drugs,” said the agency.
Using real-world data sources in clinical trials “has the potential for increasing the speed and reducing the cost of development programs, which would see effective medications being approved more quickly, or even programs which were previously thought to be unfeasible becoming feasible, with the consequent benefit to public health,” said MHRA.
This guidance, focused only on clinical trials of investigational medicinal products (CTIMPS), is the first in a series planned by MHRA to address the use of RWE by sponsors. When planning prospective clinical trials that will incorporate real-world data (RWD), sponsors should use protocols that incorporate all the elements of a conventional randomized clinical trial (RCT), “including pre-specification of the objectives, data to be collected, primary and secondary endpoints and analysis methods,” said MHRA.
However, trials conducted completely in the real-world setting with no additional data do not involve patient blinding as to treatment allocation. Although patients still must provide consent before being enrolled in such a trial, the experience of a patient enrolled in such a trial would not differ from someone who is not a trial participant, noted MHRA.
This type of trial, said the agency, “is likely to be classed as a Type A trial,” and such a trial, if well-designed, would be “equally acceptable” as a conventional open-label RCT. In some cases, a trial might need to be blinded, as when comparative safety criteria are an important aspect of the trial.
Hybrid trials are also possible, where patients and health care professionals provide such data as patient-reported outcomes or clinical assessments, in addition to the incorporation of RWD.
Using a RWE approach to clinical trials does not obviate the need for appropriate safety monitoring, with a risk-proportionate approach being advised for Type A trials.
The guidance also reviews authorization considerations for CTIMPs that involve RWE generation, including adverse event reporting requirements, which will depend in part on risk assessment.
In terms of regulatory acceptability, MHRA is agnostic as to whether a randomized trial uses only RWE, employs a hybrid data collection method, or is run as a conventional clinical trial. “The important thing is that the trial is designed in a way which allows it to provide the evidence required to answer the regulatory question,” said the agency.
It is true, said MHRA, that a key benefit of the RWE approach is the reduced burden for patients who participate in the program: “ The more involvement outside of routine care that is required, the smaller the advantages of running a RWD based trial become.” Accordingly, studies supporting labeling changes for use of a medication in a different population, for a dose or route of administration change, or for a new indication are most likely to be appropriate for RWE approaches.
The guidance provides an outline of what data characteristics mean a trial database will be of “sufficient quality;” also included are examples of RWD study types and a discussion of such aspects of these studies as endpoints derived from electronic health records and digital health technologies.
The draft guidance announcement also points sponsors to appendices that provide more technical information, and to the response form for consultation.


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