When it comes to developing Clinical Outcome Assessment (COA) strategies in the drug approval journey, the industry and researchers are often faced with many barriers. The challenges include selecting the right tool to assess the target concepts of interest for the intended population, limiting burden on patients and missing data, analyzing and interpretation of COA data, and meeting regulators’ COA requirements.

Over the last years, the FDA and EMA have worked on clarifying their expectations to better guide drug developers towards a successful regulatory decision-making.

A major milestone was the June 2020 release of the first guidance of the FDA’s “Patient-Focused Drug Development Guidance Series for Enhancing the Incorporation of the Patient’s Voice in Medical Product Development and Regulatory Decision Making”.

In parallel, the EMA and FDA also provide valuable advice to support the qualification of Clinical Outcome Assessments for a specific intended use in the context of research:

  • The FDA COA qualification program aims at providing a regulatory conclusion on the reliability of a COA, as stated on the FDA’s website in October 2023 “the program represents a conclusion that within the stated context of use, results of assessment can be relied upon to measure a specific concept and have a specific interpretation and application in drug development and regulatory decision-making.” (accessed on November 18, 2023)
  • The EMA qualification program intends at publishing an opinion on novel methodologies, as stated on the EMA’s website “the program represents the opinions on the qualification of innovative development methods and letters of support for novel methodologies that have been shown to be promising in the context of research and development into pharmaceuticals.” (accessed on November 18, 2023)

 

FDA & EMA qualification programs at a glance

 

FDA qualification program

EMA qualification program

Qualification definition

COA qualification is a regulatory conclusion that the COA is a well-defined and reliable assessment of a specified concept of interest for use in adequate and well-controlled studies in a specified context of use

  • Qualification opinions: opinion on the acceptability of a specific use of a method
  • Letters of support: proposed when the novel methodology under evaluation cannot yet be qualified but is shown to be promising based on preliminary data

 

Acknowledged COAs

  • 9 qualified COAs (all PROs*)
  • 41 COAs accepted in the qualification process (Letter of Intent accepted)
  • 4 qualification opinions (1 PerfO*, 1 ClinRO*, 2 PROs*)
  • 11 Letters of support

Major therapeutic areas covered by acknowledged COAs

  • Digestive System Diseases (eg. Pediatric Eosinophilic Esophagitis Symptom Severity Module - PEESS)
  • Respiratory Tract Diseases (eg. Community-Acquired Bacterial Pneumonia Symptom Diary - PneumoPRO©)
  • Mental Disorders (eg. Symptoms of Major Depressive Disorder Scale - SMDDS)
  • Digestive System Diseases (eg. Pediatric ulcerative colitis activity index - PUCAI)
  • Respiratory Tract Diseases (eg. Pulmonary Arterial Hypertension-Symptoms and Impact Questionnaire - PAH-SYMPACT®)
  • Mental Disorders (eg. ABS-II Adaptive Behavior Composite - VABS-II-ABC)

Major concepts assessed by acknowledged COAs

Symptoms, pain, and physical functioning

Symptoms, social and emotional functioning

*PRO: Patient-Reported Outcome, PerfO: Performance-Reported Outcome, ClinRO: Clinician-Reported Outcome

 

PROQOLID™: FDA/EMA qualified COAs at your fingertips

PROQOLID™ is a unique and comprehensive online database providing information about the development, the validity, the content, the translations, the copyright and the conditions of use of 6,000+ COAs.

You can now easily identify FDA/EMA qualified COAs in PROQOLID™ through newly developed search filters (“FDA qualified COA”, “COA accepted in the FDA qualification program”; “COA with an EMA opinion” and “COA with an EMA letter of support”) and retrieve a full set of key information about the instruments.

To get access to the full list of qualified COAs, subscribe to PROQOLID™ in the ePROVIDE™ platform.

 

Visit ePROVIDE™