FDA finalizes guidances on PROs, patient engagement in device studies

Regulatory NewsRegulatory News | 25 January 2022 |  By 

The US Food and Drug Administration (FDA) issued two final guidance documents on Tuesday that aim to help sponsors better incorporate and capture patients’ experiences in medical device clinical studies.
One guidance addresses best practices for selecting and modifying patient-reported outcomes (PROs) in medical device evaluations while the other document focuses on increasing patient engagement in clinical trials. Both contain minor revisions from earlier drafts.
The guidance on PROs describes best practices for incorporating these instruments in clinical trials to better assess the patient experience in clinical studies.
“Patient-reported outcome (PRO) instruments facilitate the systematic collection of patient perspectives as valid scientific evidence to support the regulatory and healthcare decision-making process,” FDA said.
Such instruments may be used to “inform a patient’s eligibility for inclusion within a study, to capture safety or effectiveness outcomes, and may be aligned as primary or secondary endpoints or used as a stand-alone outcome assessments or component of a composite endpoints.”
The guidance revises the draft version issued in August 2020 by providing more information on what constitutes “fit for purpose” PROs, providing additional information on considering the patient burden, and providing more information on PROs’ connection with the total product lifecycle. (RELATED: FDA releases draft guidance on PROs for device manufacturers, Regulatory Focus 31 August 2020)
In the section on fit-for-purpose, the following is added: “PRO instruments should not include items that could be misinterpreted or that are not applicable to the intended use population.”
There is also new language in the section on ensuring PROs are understandable to patients: “FDA recommends that sponsors also consider the impact on patients in responding to PRO instruments with the goal of reducing unnecessary burden. For example, sponsor may choose to use a well-developed short form version of a PRO instrument when available.”
Additionally, text has been added encouraging sponsors to engage FDA throughout the total product life cycle of the device: “During the study design stage, prior to the Investigational Device Exemption (IDE) submission or conducting of the pivotal study, sponsors are encouraged to engage FDA regarding the relance and suitability of a proposed PRO instrument to the benefit-risk assessment.” FDA encourages sponsors to use the Q-Submission process to obtain this feedback.
Patient engagement
The guidance on patient engagement recommends a framework outlining how sponsors can improve the design and conduct of clinical studies by better incorporating patient input. The document was prompted by a recommendation of FDA’s Patient Engagement Advisory Committee (PEAC).
The guidance defines the term patient engagement and discusses the approaches sponsors can use to engage patient advisors to inform the design and conduct of device clinical studies, when such patient input can be gathered, the roles of institutional review boards (IRBS) and other groups in patient engagement, as well as how sponsors can receive feedback on their patient engagement plans with FDA.
The guidance revises a draft version released for comment in 2019 by providing more background on FDA’s patient engagement. (RELATED: Patient Engagement in Device Trials: FDA Drafts Guidance, Regulatory Focus, 23 September 2019)
For example, the update makes reference to a public workshop convened by the FDA and the Clinical Trials Transformation Initiative in March 2019 to discuss best practices and key considerations when receiving patient perspectives on clinical trial access, design, conduct and post-trial follow-up.
The final guidance also provides more information on how sponsors can obtain feedback from FDA on patient engagement plans and patient-centered study designs.
PRO guidance
Patient engagement guidance


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