Core Oncology Outcome Strategies: Do They Offer an Opportunity for Simplicity and Consistency, or Pose a Risk to Comprehensive Understanding of Patient Experiences?

Author(s)

Gaspar L1, Bondugula S2, Aubert F3, Bellet A3, Sylvester J4
1IQVIA, Reading, RDG, UK, 2IQVIA, Bangalore, Bangalore, India, 3IQVIA, Paris, Paris, France, 4IQVIA, San Diego, CA, USA

OBJECTIVES: The FDA proposes a core set of clinical outcomes to define treatment benefit across oncology indications during drug/biologic development: symptoms, symptomatic adverse events, physical and role functioning. While there is appeal in such consistent approach, it somewhat conflicts with the concept of patient-focused drug development which highlights the importance of comprehensively understanding and measuring the most important concepts for a target population. This study examined conceptual models from 5 oncology indications to explore whether the FDA’s proposed outcomes were important to patients in all 5 indications. Further, it was examined whether a general measurement approach could be considered across indications.

METHODS: IQVIA’s COA Accelerator (COAA) was used to auto-generate conceptual models based on existing literature in 5 indications: melanoma, breast, prostate, head & neck and hepatocellular cancer. COAA overlaid the models and evaluated similarities and differences across the five sets of outcomes.

RESULTS: Across the 5 conceptual models, 167 unique concepts were identified pertaining to signs, symptoms and impacts of cancer and cancer treatment. All models included concepts that aligned under FDA’s core set of clinical outcomes. All models also included concepts not included in FDA's core outcomes, such as clinical signs of disease (e.g. weight change) and emotional and social impacts. When examining the specific concepts, only 30 concepts (18% of total) appeared in all 5 models; 2 disease-related symptoms (fatigue, nausea), 4 symptomatic adverse events (fatigue, nausea, general pain, loss of appetite), and 24 impacts (covering physical, role and emotional functioning).

CONCLUSIONS: While the core set of clinical outcomes seems appropriate for measuring aspects relevant across indications, it would benefit from including emotional functioning. Specific concepts related to these core outcomes differ across indications and thus research is still required to understand the specific concepts of interest and measure them in an indication-targeted way when developing trial outcome strategies.

Conference/Value in Health Info

2023-11, ISPOR Europe 2023, Copenhagen, Denmark

Value in Health, Volume 26, Issue 11, S2 (December 2023)

Code

PCR244

Topic

Clinical Outcomes, Patient-Centered Research

Topic Subcategory

Clinical Outcomes Assessment, Patient-reported Outcomes & Quality of Life Outcomes

Disease

Oncology

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