FDA Releases the Pilot Version of the Clinical Outcome Assessment Compendium for Public Comment

by barbarabauer | January 14, 2016 2:54 pm

The Purpose of the COA Compendium
The pilot COA Compendium (view PDF[1]) is part of FDA’s efforts to foster patient-focused drug development.  The COA Compendium is intended to facilitate communication and to provide clarity and transparency to drug developers and the research community by collating and summarizing clinical outcome assessment (COA)2[2] information for many different diseases and conditions into a single resource. It can be used as a starting point when considering how certain clinical outcome assessments might be utilized in clinical trials and will likely be most informative in early drug development.

The COA Compendium is a table that:

FDA is seeking public comment and feedback about the pilot COA Compendium through establishment of a docket, as announced on January 13, 2016 in the Federal Register[4].  Comments and recommendations received will be reviewed by the Agency as we consider developing future iterations of the COA Compendium.

Key Consideration of the COA Compendium
The COA Compendium (PDF) is not a comprehensive list of clinical outcome assessments and is not intended to replace either existing disease-specific guidance or key interactions with FDA concerning drug development (e.g., during pre-IND meetings).  Inclusion of a clinical outcome assessment in the COA Compendium is not intended to indicate that the measure is or should be the sole (or primary) determinant of a clinical benefit in a clinical trial.

Drug sponsors are strongly encouraged to seek advice from the relevant Office of New Drug (OND) review division early in drug development to discuss the selection and implementation of the clinical outcome assessment specific to their program, irrespective of whether the disease, condition, indication, claim, or clinical outcome assessment is included in the COA Compendium.

Some of the clinical outcome assessments listed in the COA Compendium may be protected by proprietary rights, and in some cases, a royalty and fee may be charged by the copyright owners for their authorized use.  The inclusion of a clinical outcome assessment in the COA Compendium does not equate to an endorsement by FDA.

How Clinical Outcome Assessments Are Selected for the Pilot COA
Clinical outcome assessments for the pilot version of the COA Compendium have been selected from two sources:

  1. CDER’s DDT COA Qualification Program[5]
  2. Drug Labeling Approved From 2003 to 2014 (New Molecular Entity (NME) labeling)[6]

CDER’s DDT COA Qualification Program
The COA Compendium lists clinical outcome assessments that have been qualified,3 as well as those currently in the process of being developed and assessed,4 under CDER’s DDT Qualification Program[7].  Qualified clinical outcome assessments are publicly available for clinical trial use and have an associated FDA guidance describing their qualified Context of Use[8].  Clinical outcome assessments that are currently being developed within the qualification program  may not yet be publicly available for use but are described in the COA Compendium to encourage collaborative efforts and to be transparent about current efforts to develop clinically relevant measures across a variety of diseases or conditions.  Our guidance for industry and FDA Staff—Qualification Process for Drug Development[9] Tools—contains more information about the qualification program.
Drug Labeling Approved From 2003 to 2014 (NME labeling)

The COA Compendium lists clinical outcome assessments from approved drug labeling as described below.  Generally, the CLINICAL STUDIES section of labeling summarizes the clinical trials that provide the primary support for the effectiveness of a drug and that may be relevant for clinical decision making while prescribing, including what end points were used in the clinical trials.

In constructing the pilot COA Compendium, FDA reviewed the CLINICAL STUDIES sections of drug labeling for NMEs approved from 2003 to 2014 and any pertinent medical reviews of those NMEs. Clinical outcome assessments that were discussed in labeling (specifically NME labeling from 2003 to 2014) and used as part of primary or secondary endpoints in the controlled clinical trials that provided evidence of efficacy for the approved indication(s) were included in the COA Compendium with a few exceptions, such as when FDA issued guidance recommending the use of different, more recent, or more appropriate clinical outcome assessments for the subject condition.  Also, in a few cases, outcome assessments were excluded from the COA Compendium because the assessments were too complex to be amenable to a description in the COA Compendium’s tabular format and currently there were no corresponding guidance references available for additional information.

How Listing Labeled Clinical Outcome Assessments in the COA Compendium Complement the Qualification Program
The inclusion of labeled clinical outcome assessments in the COA Compendium is not intended to minimize the value of the CDER’s DDT Qualification Program in the collaborative development of clinical outcome assessments, especially in response to unmet measurement needs.  COA qualification is a regulatory conclusion that a specific clinical outcome assessment can be relied upon to have a specific interpretation and application in drug and regulatory review.  Once a clinical outcome assessment has been qualified, developers and CDER reviewers can feel confident using it within the qualified Context of Use and not have to reconfirm its utility.5   Because it is not feasible (due to the time and resources necessary for qualification) or necessary to qualify all clinical outcome assessments, the inclusion of labeled clinical outcome assessments in the COA Compendium may serve as an additional method of identifying clinical outcome assessments that may be appropriate for clinical trials to support labeling claims.

What the Pilot COA Compendium Looks Like
The pilot COA Compendium (PDF) is a table organized by CDER’s OND offices and review division assignments (e.g., the Division of Cardiovascular and Renal Products within the Office of Drug Evaluation I).  The table alphabetically lists conditions or diseases based on each review division’s therapeutic assignment.  The table rows are color coded—specifically, the shaded rows describe information relating to a COA qualification project, whereas unshaded rows describe information relating to clinical outcome assessment from previous labeling.

Click here to view a 6 column sample of the COA Compendium.

Limitations of the COA Compendium

Article courtesy FDA, January 14, 2016.

For more information on the Clinical Outcome Assessment Compendium pilot,
visit the FDA Website:
http://www.fda.gov/Drugs/DevelopmentApprovalProcess/DevelopmentResources/ucm459231.htm#Labeling[10]

 

Endnotes:
  1. view PDF: http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/UCM481225.pdf
  2. clinical outcome assessment (COA)2: http://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugDevelopmentToolsQualificationProgram/ucm370262.htm
  3. Drug Development Tool (DDT): http://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugDevelopmentToolsQualificationProgram/default.htm
  4. Federal Register: https://s3.amazonaws.com/public-inspection.federalregister.gov/2016-00529.pdf
  5. CDER’s DDT COA Qualification Program: http://www.fda.gov/Drugs/DevelopmentApprovalProcess/DevelopmentResources/ucm459231.htm#Qualification
  6. Drug Labeling Approved From 2003 to 2014 (New Molecular Entity (NME) labeling): http://www.fda.gov/Drugs/DevelopmentApprovalProcess/DevelopmentResources/ucm459231.htm#Labeling
  7. DDT Qualification Program: http://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugDevelopmentToolsQualificationProgram/default.htm
  8. Context of Use: http://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugDevelopmentToolsQualificationProgram/ucm370262.htm
  9. Qualification Process for Drug Development: http://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugDevelopmentToolsQualificationProgram/ucm370262.htm
  10. http://www.fda.gov/Drugs/DevelopmentApprovalProcess/DevelopmentResources/ucm459231.htm#Labeling: http://www.fda.gov/Drugs/DevelopmentApprovalProcess/DevelopmentResources/ucm459231.htm#Labeling

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