Event

11:00AM–1:00PM Eastern Daylight Time (EDT)

15:00PM–17:00PM Coordinated Universal Time (UTC)

17:00PM–19:00PM Central European Summer Time (CEST)

16:00PM-18:00PM British Summer Time (BST)

 

In recent years there has been increasing recognition of the importance of capturing the patient voice in clinical trials, underpinned by the development of 4 new patient-focused drug development guidance documents from the FDA. Clinical outcome assessment (COA) instruments, such as patient-reported outcomes, are widely used to capture the patient voice in clinical trials. A COA strategy refers to the process of deciding which COA instruments to include in a clinical trial.

A typical COA strategy tends to focus on meeting the evidentiary requirements for FDA or EMA labeling language. Subsequently, the byproduct of a labeling strategy is assumed to meet the evidence needs and interests of remaining stakeholders. However, a sole focus on FDA/EMA labeling is not sufficient to meet the informational needs of health technology assessment (HTA), healthcare providers, patients, and policymakers.

Beyond labelling, COA evidence can support the benefit-risk evaluation and decisions of all the aforementioned stakeholders. The availability of COA data for these decisions can ensure that treatments with most humanistic benefits can reach the patients in need. In addition, considering the evidence requirements of all stakeholders from the outset allows for potential efficiencies in the collection of COA data. This is crucial for minimizing patient burden, particularly in rare diseases, where a limited pool of patients and individuals may have many competing demands.

This short course will discuss the value of COA data in the context of patient-focused drug development and outline the many uses of COA data that can be leveraged beyond a typical COA strategy. This includes how COA data have the potential to influence overall licensing and HTA decisions, as well as policy decisions, clinician prescribing, and patient treatment choices. The audience will hear about an optimal approach to ensure that a COA strategy accounts for the needs of all stakeholders.

PREREQUISITE: Some familiarity with patient-reported outcome (PRO)/clinical outcome assessment (COA) data and tools would be preferable, though not required. Participants may wish to use a laptop, but it is not essential.