Table 4 Summary of how-to guide for PE in the development of a COA strategy

STEP 1: Preparation for partnership and collaboration with patients

Focus is on engaging patients, establishing relationships, understanding patients’ views on participating, and defining the expectations of all parties

Prepares stakeholders for participation in the program by:

● Educating patients on COA instruments and strategy (to the required level depending on anticipated input)

● Educating the study team on the importance of patient input in the development of a COA strategy

● Preparing the study team for a meaningful, effective, and respectful interaction with patients and giving them tools to build a mutually beneficial relationship

● Establishing the foundation for a long-term, collaborative patient–sponsor relationship

STEP 2: Identification of relevant concepts (i.e., the burden of disease)

Starting point of any COA strategy

● Gaining a clear understanding from the patient (and/or carer) of their experience with the disease (and its treatment) and its impact on their life and well-being

● Identification and understanding of the signs, symptoms, and treatment burden patients experience and how these affect patients’ day-to-day functioning and quality of life to inform the COA strategy and ensure that the selected COA instrument(s) include(s) concepts that are relevant and important to patients

STEP 3: COA selection

Undertaken after identifying concepts that are clinically relevant, important for patients, and likely to be affected by the drug

Describes how to secure patient input and collaboration to:

● Review existing COAs to determine which measure(s) provides the best insight(s) into the key domains of interest

● Determine the relevance of the measure(s) for use within a clinical trial

● Assess if the measure(s) is/are appropriate for its intended use and context of use (e.g., measurement properties, targeted study design and objectives, and targeted patient population)

STEP 4: COA development/revisions

Undertaken if the evaluation done under Steps 2 and 3 reveals that no existing measure meets the selection criteria that are relevant to the target population and context of use

● Describes how to secure patient input and collaboration to develop new COA tools or modify existing ones in accordance with available guidelines to meet regulatory requirements

STEP 5: COA implementation within clinical trials

Patients’ roles in reviewing the trial protocol and advising on elements, including (but not limited to)

● COA end points

● Feasibility for trial participants to complete the COA as described in the protocol

● Information provided to the trial participants with regard to the rationale for completing the COA instrument and how/when these data will be used

STEP 6: COA data interpretation

Collecting patient insights to provide a valuable perspective on what constitutes clinically meaningful change for COAs

● Conduct qualitative studies (e.g., in-trial exit interviews), surveys, or stakeholder meetings to discuss clinically meaningful change

● Patient experts involved in the study may provide input and facilitate communication with stakeholders to support the interpretation and importance of patient-relevant end points

STEP 7: COA communication

Communicating COA data to augment limited product labeling and potentially hard to access scientific communication

● Incorporating PE to improve the clarity and meaningfulness of communications around COA data by providing input (e.g., development, review, approval) on the information that will be disclosed before its publication