Relevant endpoints in oncology are used in clinical trials to assess the most meaningful treatment outcomes for patients, including patient-reported or disease-free survival in certain metastatic forms, pathological complete response, etc.
A white paper published by EFPIA takes stock of the value of these types of endpoints and the challenges for their implementation, from the perspective of health technology assessment (HTA) bodies and payers. The white paper is based on a literature review and a dozen qualitative interviews conducted with various types of stakeholders.

The paper discusses the current standard of assessment, overall survival (OS) from the time of patient assignment to the clinical trial to death, and its limitations, and then goes on to analyse the possible types of endpoints relevant in oncology. The latter measure effects of oncology treatment that go beyond survival alone, and are thus proposed to support better evaluation of new products by HTAs and payers for the purpose of pricing and reimbursement. Oncology-relevant endpoints can also, in some cases, support an assessment of treatment efficacy, for example with reference to time without disease progression. In contrast to overall survival, they can also capture data on the patient’s quality of life, or on particular events such as disease relapse or the occurrence of metastases. Another difference from the traditional approach emphasised by EFPIA’s paper is the fact that relevant endpoints in oncology usually refer to a single line of therapy (e.g. first-line surgery), in contrast to overall survival which takes into account all subsequent lines with which the patient has been treated. This could, according to EFPIA, facilitate a clearer measurement of therapeutic benefit and patient relevance, as many confounding factors could be avoided.

The white paper also indicates that relevant endpoints in oncology should always be considered by tumour type and stage, both from the point of view of the patient and the treating physicians. The difficulties identified in the adoption of this type of measurement refer to uncertainties due to a lack of evidence and a mismatch between physicians/patients and regulatory and HTA bodies regarding the relative value to be attributed to specific outcomes for each treatment setting.
The white paper also outlines some actions that EFPIA believes could help resolve these uncertainties, starting with the creation of a portfolio of fit-for-purpose endpoints by tumour type and stage. Evidence should also be gathered to support the value of these endpoints – both standalone and surrogate – and key outcomes by treatment setting and the methodologies used to collect them should be standardised.