PATIENT-CENTERED ENDPOINT STRATEGY FOR BIOLOGIC TRIALS IN SEVERE ASTHMA: INTEGRATING REGULATORY, PAYER, AND PATIENT PRIORITIZED ENDPOINTS
Author(s)
Setareh A. Williams, PhD1, Russell V. Becker, MS2, Catherine E. Hueston, PhD3, Charles D. Williams, BS1;
1Star Biopharma Consulting, LLC., Health Economics and Outcomes Research, Malvern, PA, USA, 2Star Biopharma Consulting, LLC., Health Economics and Outcomes Research, Mobile, AL, USA, 3Star Biopharma Consulting, LLC., Regulatory Science, Malvern, PA, USA
1Star Biopharma Consulting, LLC., Health Economics and Outcomes Research, Malvern, PA, USA, 2Star Biopharma Consulting, LLC., Health Economics and Outcomes Research, Mobile, AL, USA, 3Star Biopharma Consulting, LLC., Regulatory Science, Malvern, PA, USA
OBJECTIVES: Recommend a stakeholder-aligned patient-reported outcome (PRO) strategy and assessment schedule for phase III biologic trials in severe asthma that supports regulatory claims and payer requirements while meeting patient-prioritized endpoints for symptom control, quality of life (QOL) and treatment experience.
METHODS: We conducted a targeted landscape assessment: (1) review of severe asthma phase III biologic trials; (2) HTA reviews of biologics, including comparative effectiveness and economic evaluations; and (3) synthesis of measurement properties, respondent burden and interpretability of candidate PROs, including utility estimation and mapping to economic models. Recommendations for core PRO selection and assessment frequency balanced early signal detection, maintenance of treatment effect and operational feasibility in global phase III programs.
RESULTS: Symptom diaries remain foundational and are supported by regulatory bodies; additional PROs are needed for asthma control, QOL, utilities, work productivity, and treatment experience. ACQ is prioritized as the core control measure given alignment with exacerbation endpoints and common use in comparative effectiveness frameworks. Asthma Quality of Life Questionnaire (AQLQ) is recommended at milestone visits to capture sustained QOL, and EQ-5D aligned with AQLQ to support mapping for HTA submissions. SGRQ is not recommended due to length, overlap with AQLQ, multiplicity concerns, and limited HTA utility given mapping constraints. For populations with comorbid CRSwNP, SNOT-22 provides incremental patient-centered evidence, with timing aligned to dosing frequency. To differentiate extended dosing frequency, WPAI/CIQ at baseline, mid-term and end of trial are recommended, with TSQM to further quantify convenience and perceived benefit. Given the mental health burden in severe uncontrolled asthma, baseline HADS screening is recommended for subgroup analysis.
CONCLUSIONS: A fit-for-purpose PRO battery should combine qualified symptom diaries with ACQ, AQLQ, and EQ-5D, supplemented by modules for comorbidity impacts (e.g., SNOT-22, HADS), productivity impairment (WPAI/CIQ), and treatment satisfaction (TSQM); baseline HADS supports subgroup analysis of lower treatment response.
METHODS: We conducted a targeted landscape assessment: (1) review of severe asthma phase III biologic trials; (2) HTA reviews of biologics, including comparative effectiveness and economic evaluations; and (3) synthesis of measurement properties, respondent burden and interpretability of candidate PROs, including utility estimation and mapping to economic models. Recommendations for core PRO selection and assessment frequency balanced early signal detection, maintenance of treatment effect and operational feasibility in global phase III programs.
RESULTS: Symptom diaries remain foundational and are supported by regulatory bodies; additional PROs are needed for asthma control, QOL, utilities, work productivity, and treatment experience. ACQ is prioritized as the core control measure given alignment with exacerbation endpoints and common use in comparative effectiveness frameworks. Asthma Quality of Life Questionnaire (AQLQ) is recommended at milestone visits to capture sustained QOL, and EQ-5D aligned with AQLQ to support mapping for HTA submissions. SGRQ is not recommended due to length, overlap with AQLQ, multiplicity concerns, and limited HTA utility given mapping constraints. For populations with comorbid CRSwNP, SNOT-22 provides incremental patient-centered evidence, with timing aligned to dosing frequency. To differentiate extended dosing frequency, WPAI/CIQ at baseline, mid-term and end of trial are recommended, with TSQM to further quantify convenience and perceived benefit. Given the mental health burden in severe uncontrolled asthma, baseline HADS screening is recommended for subgroup analysis.
CONCLUSIONS: A fit-for-purpose PRO battery should combine qualified symptom diaries with ACQ, AQLQ, and EQ-5D, supplemented by modules for comorbidity impacts (e.g., SNOT-22, HADS), productivity impairment (WPAI/CIQ), and treatment satisfaction (TSQM); baseline HADS supports subgroup analysis of lower treatment response.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
PCR148
Topic
Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, SDC: Respiratory-Related Disorders (Allergy, Asthma, Smoking, Other Respiratory), STA: Biologics & Biosimilars