This white paper has been developed as part of an EFPIA project to drive awareness of the use of oncology-relevant endpoints in HTA body / payer decision-making. Oncology-relevant endpoints refer to all endpoints used in oncology clinical trials to measure outcomes relevant to patients. These can include, in addition to overall survival, patient-reported outcomes as well as progression-free survival in some metastatic settings, pathological complete response or event-free survival in early oncology settings. The white paper was written with the support of L.E.K. Consulting and was informed by a literature review as well as 13 qualitative interviews with clinicians, patient advocates and former HTA bodies / payers to better understand the value of oncology-relevant endpoints and the challenges facing their adoption. The findings were discussed and refined at three roundtables with the project’s sounding board. 

The white paper highlights that across settings, increased adoption of oncology-relevant endpoints beyond OS in HTA body / payer decision-making can help to provide timely access to life-improving or life-prolonging medicines, ensure optimal outcomes for patients and and potentially reduce healthcare costs. Continued progress in incorporating oncology-relevant endpoints beyond OS depends on the collaboration of all stakeholders to overcome barriers and to ensure that HTA body / payer decision-making can result in the best outcomes for patients. 

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