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COA Compendium Frequently Asked Questions (FAQs)

Scope and Purpose of the COA Compendium:

Key Considerations and Limitations of the COA Compendium:

Updates and Contact information for the COA Compendium:

SCOPE AND PURPOSE OF THE COA COMPENDIUM:

How were COAs selected for the pilot and the updated COA Compendium?
For the pilot COA Compendium, the labeled COAs were selected from the following sources:

  • Labeling of new molecular entities (NME) drugs whose original new drug application (NDA) or biological license application (BLA) was approved from 2003-2014
  • Measures qualified by CDER’s Clinical Outcome Assessment DDT Qualification Program
  • Submissions that were under review in CDER’s Clinical Outcome Assessment DDT Qualification Program

The first update of COA Compendium since the launch of the pilot in January of 2016 is an extension of the original document, and includes clinical outcome assessments from three major sources:

  • Labeling of NME drugs whose original NDA or BLA was approved from 2003 to June 20171
  • Efficacy supplements that were approved from January to June 2017 that pertain to new indications or new populations
  • Measures qualified by CDER’s Clinical Outcome Assessment DDT Qualification Program

For NME approvals and efficacy supplements, FDA reviewed the CLINICAL STUDIES sections of their labeling and pertinent medical reviews. COAs that were discussed in labeling and used as endpoints in the clinical trials to provide evidence of efficacy for the approved indication(s) were included in both the pilot and the updated COA Compendium. However, there are few exceptions such as when outcome assessments were too complex to be amenable to a description in the COA Compendium’s tabular format and when there were no corresponding guidance references available for additional information, or when there is a currently available FDA-issued guidance recommending the use of different, more recent, or more appropriate COAs for the subject condition.

The updated COA Compendium no longer includes submissions that are currently under development or review in CDER’s Clinical Outcome Assessment Qualification Program. Instead, in the updated COA Compendium, only qualified COAs are included. These COAs are publicly available for clinical trial use and have an associated qualification statement describing their qualified context of use.  To learn more, please review: COA DDT Qualification Program and COA DDT Qualification Program FAQs

KEY CONSIDERATIONS AND LIMITATIONS OF THE COA COMPENDIUM:
Does the COA Compendium contain a comprehensive listing of COAs?

No. It does not contain a comprehensive listing of all medical conditions or COAs that could potentially support labeling claims. It is limited in scope as it is primarily based on the retrospective review of select NDA, BLA and efficacy supplement labeling approved within a pre-determined timeframe. COAs that are not included in the COA Compendium should also be considered, as appropriate, especially those supported by data, literature and good measurement principles.

Do I still need to discuss my COA measurement strategy (e.g., COA development, selection) and plans with the FDA?

Yes. The COA Compendium is intended to be a communication tool and does not represent an endorsement of the COAs or agency guidance.  It also does not replace key FDA drug development interactions (e.g., pre-IND meetings). For example:

  • The COA Compendium omits critical aspects of how a listed COA could be implemented in a clinical trial (e.g., clinical trial design)
  • Inclusion of a COA in the COA Compendium does not indicate that the COA is or should be the primary or sole determinant of effectiveness in a clinical trial nor is it indicative of endpoint positioning (e.g., primary or secondary)
  • Endpoint hierarchy and selection of key outcome assessments should be discussed with the appropriate review division prior to initiating clinical trials

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 COA(s) specific to their program, irrespective of what is listed in the COA Compendium.

Are all COAs listed in the COA Compendium publicly available?     

FDA did not attempt to limit the compendium to publicly available COAs. Some listed COAs may be protected by proprietary rights. Additionally, copyright owners may charge a royalty fee for the authorized use of their COAs listed in the COA Compendium.

Are COAs listed in the COA Compendium endorsed by the FDA?
No.  The inclusion of a COA in the COA Compendium does not equate to FDA endorsement and COAs should be discussed with the appropriate review division early in drug development and prior to initiating clinical trials.

UPDATES AND CONTACT INFORMATION FOR THE COA COMPENDIUM:
Will the COA Compendium be periodically updated?

Yes, periodic updates are anticipated.

Who should I contact for any questions or feedback about the COA Compendium?

Questions and feedback on the COA Compendium should be sent to: COACompendium@fda.hhs.gov.

1 This adds information from labeling approved from 2015 to June 2017.

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