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Regulatory Focus™ > News Articles > 2020 > 2 > Industry Groups Pitch FDA on Upcoming Clinical Outcome Assessments Guidance

Industry Groups Pitch FDA on Upcoming Clinical Outcome Assessments Guidance

Posted 07 February 2020 | By Michael Mezher 

Industry Groups Pitch FDA on Upcoming Clinical Outcome Assessments Guidance

In response to the US Food and Drug Administration’s (FDA) public workshop on incorporating clinical outcome assessments (COAs) into endpoints for regulatory decision-making last December, industry groups have submitted suggestions for what the agency’s upcoming guidance on the topic should cover.
 
Background
 
The workshop was the fourth in a series held by FDA to meet its commitments under the latest reauthorization of the Prescription Drug User Fee Amendments (PDUFA VI) to develop guidance documents aimed at creating “fit-for-purpose tools to collect meaningful patient and caregiver input for ultimate use in regulatory decision making.”
 
The first three workshops focused on collecting comprehensive and representative patient input, methods to identify what is important to patients and selecting, developing or modifying fit-for-purpose clinical outcome assessments.
 
The first two guidances in the series have already been published in draft form, while FDA has until the end of FY2020 and FY2021 to publish drafts of the final two guidances.
 
Comments
 
In its comments, the Pharmaceutical Research and Manufacturers of America (PhRMA) agrees with the agency that sponsors should discuss the use of COAs with FDA early in the drug development process.
 
And the association requests that FDA expand its thinking on how COA data and other patient experience and preference data can be used in regulatory decision-making, pointing out that a slide in FDA’s presentation on the benefit-risk framework for PFDD left the “risk and risk management” section blank.
 
“Specifically, PhRMA recommends that the guidance make clear that patient experience data could be used to inform decisions regarding endpoint relevance, benefit-risk evaluation, and labelling, amongst others,” the comment says.
 
PhRMA also suggests that FDA “issue a draft guidance as early as possible in 2020,” rather than by the end of FY2021 as set out in the PDUFA VI goals letter.
 
In separate comments, the Biotechnology Innovation Organization (BIO) asks FDA to include a discussion of how COA information can be communicated to healthcare providers and payors.
 
“BIO requests that FDA expand the guidance to provide the agency’s views on how and where information COAs can be best incorporated in the label and/or be included in other communications to disseminate patient-centric outcomes to other key stakeholders,” BIO writes.
 
BIO and PhRMA stress that the draft guidance should provide more detail on the types of regulatory decisions in which FDA will consider patient experience data.
 
Additionally, BIO and PhRMA each recommend FDA ensure that the guidance is consistent with the International Council for Harmonisation (ICH) E9(R1) guideline with respect to handling intercurrent events, missing data and treatment policy.
 
The two industry groups ask FDA to clarify which portions of the guidance apply only to specific phases of clinical development, such as the section on pre-defining within-patient thresholds, which PhRMA notes would only be feasible for late-stage trials.
 
PhRMA also requests that FDA standardize the language it uses to describe meaningful change throughout the guidance, such as “minimally clinically important difference” and “minimum important difference,” to ensure clarity, and asks that FDA include two additional terms, “no change” and “reduced rate of deterioration” in the definition of meaningful within-patient change.
 
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