Endpoints That Matter: A Framework for Identifying and Integrating Patient-Relevant Outcomes Across Evidence Development and Assessment

Session Overview

In oncology, evidence generation has long been anchored to overall survival (OS). OS is a hard, unambiguous endpoint that matters to every stakeholder. But a registrational trial cannot always deliver a reliable OS result. In multiple myeloma, subsequent therapies, treatment sequencing, longer post-progression survival, and competing mortality from age or comorbidities all erode the ability to attribute an OS effect to the treatment under study. Many deaths in this population are driven by comorbidity, not disease progression. In this therapeutic context, disease control is not a secondary consideration: delaying progression, symptoms, complications, and further burdensome treatment, while preserving how patients function and live day to day, has direct value to patients, carers and clinicians. Despite this, patient-relevant outcomes remain inconsistently identified, inadequately measured, and insufficiently considered by many health technology assessors.

This webinar introduces a practical, four-stage framework to more comprehensively incorporate patient relevant endpoints in the evidence generation and assessment of novel health technologies: 1) Identify What Matters, 2) Prioritise stakeholders’ evidence preferences, 3) Measure Outcomes, and 4) Attribute Value. This framework is designed to help evidence generation teams embed patient-relevant endpoints into a medicine’s development programme from early clinical planning through to HTA assessment.

The session draws on clinical, patient advocacy, industry, and health economic perspectives. A brief, public-domain comparison between MM and a late metastatic setting will show concretely how clinical context shapes the relevance of alternative endpoints.

By the end of this session, participants will be able to:

1. Distinguish between an endpoint, the patient-relevant outcome it measures, and the value of the treatment effect.
2. Articulate why alternative endpoints, in defined clinical contexts, are essential to a complete assessment of patient relevant benefit
3. Use a four-stage framework to identify, prioritise, measure, and communicate patient-relevant endpoints across a development programme.
4. Describe how clinical teams, patient organisations, and industry can integrate alternative endpoints into evidence plans from early development through HTA submission.

Lumanity is proud to be a Corporate Partner of ISPOR. This session was originally presented in partnership with ISPOR on October 6, 2026.

Panelists

• • Daniel Gladwell, PhD
    Chief Scientific Officer (HEOR), Lumanity

• • Professor Åse Bratland, PhD, MD
    Oslo University Hospital

• • Antonella Cardone, MBA
    CEO, Cancer Patients Europe
  • Jose Diaz, MSc, MD
    Global Health Systems Economics and Value, BMS