Setting International Standards in
Analysing Patient-Reported Outcomes
and Quality of Life Endpoints

Work Packages

SISAQOL-IMI aims to develop international consensus recommendations for the design, analysis, interpretation, and presentation of PRO data in cancer clinical trials (CCTs). To reach this goal, SISAQOL-IMI is organized into five scientific work packages (WP 2, 3, 4, 5, 6), and 3 cross-cutting work packages (WP 1, 7, 8).

Main objective

This WP will support the overall coordination of the scientific, operational, and administrative aspects of the project including administrative and financial management, compliance with scientific and ethical standards, compliance with general data protection regulations and communication with IMI. The EORTC infrastructure and its processes will serve as a framework for this project.

Sub-objectives

  1. Effective overall coordination and management of the project, including an efficient management structure.
  2. Promote communication and collaboration among the various stakeholders and partners providing support, monitoring, and coordination of their work.
  3. Ensure efficient administrative and financial management.
  4. Prepare communications to be provided to the IMI JU executive office and assist Coordinator in their responsibility to serve as liaison between IMI and project partners.
  5. Mediate disagreements among partners or in cases when partners do not meet their deliverables.
  6. Set-up an independent Scientific Advisory Board and an Ethics Advisory Board.
  7. Organise the kick-off and four consensus meetings and report on them.
  8. Develop and implement a sustainability plan.

Main objective

This WP will support the overall coordination of the scientific, operational, and administrative aspects of the project including administrative and financial management, compliance with scientific and ethical standards, compliance with general data protection regulations and communication with IMI. The EORTC infrastructure and its processes will serve as a framework for this project.

Sub-objectives

  1. To provide the needed methodological work to improve current scientific practice by determining consensus standards on the design, analysis, and interpretation of PRO data in cancer RCTs. This includes obtaining the patient perspectives and treatment outcomes that would result in meaningful treatment effect estimates.
  2. To identify and recommend similar principles meeting those set standards that can be applied to RCTs across various cancer indications in order to gain efficiency within cancer RCTs and to enhance comparability across cancer RCTs.
  3. To build on and continue the development of the initial SISAQOL-IMI work, particularly with attention to incorporation of the estimand framework.

Main objective

The overall objective of WP 3 is to investigate the feasibility of, and develop recommendations for, the use of PRO measures for non-RCTs in cancer studies with a specific focus on single-arm studies.

Sub-objectives

  1. Identify the scientific issues and needs of various stakeholders when assessing PRO measurements in non RCTs.
  2. Perform a critical review on current practices of PRO analysis in single-arm studies.
  3. Formulate valid PRO objectives for single-arm studies and translate them into estimands.
  4. Evaluate which aspects of the RCT recommendations can be applied to single-arm studies and develop specific recommendations for single-arm studies.
  5. Address specific issues of study design, analysis, and potential risks of bias in single-arm studies.
  6. Set criteria to evaluate the robustness of PRO findings from single-arm studies.

Main objective

The objective of WP 4 is the development of the recommendations for the graphical presentation of PRO findings from cancer clinical trials, including templates for graphs and tables for each objective identified in WP 2 and WP 3. These graphical presentation templates will create standards that promote interpretable and meaningful visualisation of PRO data from clinical trials.

Sub-objectives

  1. Aiming to consider the interests and needs of a broad range of stakeholders, a participatory design approach will be used to gain an in-depth understanding of how they perceive, use or wish to engage with PRO data.
  2. Development of templates according to the results of the literature review and interviews/focus groups.
  3. Heuristic evaluation of the stakeholder-specific templates for graphical PRO data presentation and the dynamic webtool.
  4. Development of a dynamic webtool to make the newly developed graphical templates publicly accessible.
  5. WP4 work is also aligned with WP8 for communication and dissemination purposes.

Main objective

The overall aim of WP 5 is to evaluate the preliminary recommendations developed in the scientific working groups (WP 2, RCTs; WP 3, non-RCTs) with respect to writing the part of the study protocol regarding PRO analysis and the PRO statistical analysis plan (SAP), as well as the preliminary recommendations for the presentation and visualisation of PRO results (WP 4).

Sub-objectives

  1. Address the effectiveness of using the preliminary recommendations for developing the PRO part of a research protocol with a well-defined PRO objective fitting the research question, given the design for a new trial, which can subsequently aid in the development of the SAP for PROs.
  2. Address the effectiveness of using the preliminary recommendations for reproducing comparable SAPs, for the PROs, PRO results, and interpretation of PRO findings, given a protocol with a well-defined PRO objective and a dataset of a case study.
  3. Address the effectiveness of using the recommendations for reproducing comparable presentation and visualisation of PRO results, given a well-defined PRO objective and a dataset of a case study.

Main objective

WP 6 is responsible for harmonising the terminologies and definitions related to clinically meaningful change in cancer clinical trials. This is important for patients as statistical significance does not always provide information on what is clinically relevant or meaningful for patients

Sub-objectives

  1. This WP initiates discussions with stakeholders to identify their current practices and challenges when deciding about “what is clinically meaningful.”
  2. WP6 will be matching terminologies and definitions to the appropriate PRO objective in WP2 and in WP3 and will ensure close collaboration with WP 2 and 3.
  3. It will also identify best practices in the development of PRO objectives using relevant thresholds for clinically meaningful change.
  4. Assess terminology and definitions of clinically meaningful change and related concepts as used in the development and evaluation of most frequently used PRO instruments in RCTs.
  5. WP6 will create a draft report which details recommendations for a harmonised terminology (including definitions for key concepts) and recommendations on how to apply these concepts in the context of the possible PRO objectives and statistical methods defined in WPs 2 and 3. The report will also contain a section with an explanation of the terminology for laypersons to facilitate accessibility of the document by patient groups.

Main objective

This WP is responsible for the development and implementation of the consensus process to ensure that the resulting recommendations are representative of the voice of key stakeholder groups (including methodological experts), and laypersons including patient representatives. They will ensure that the final recommendations address the needs of various stakeholder groups. They will also be responsible for the adaptation of the recommendations into two levels: (a) for experts and professionals, with a complementary technical/statistical section; and (b) for laypersons including patients, patient representatives and the general public.

Main objective

This WP is responsible for the development and implementation of the communication and dissemination plan, including scientific communication and information to health care professionals, patients, and laypeople. They will also be responsible for developing recommendations for dissemination strategies and educational programmes geared towards improving patient understanding of the use of PRO measures in CCTs.

Sub-objectives

  1. Support the wide and clear dissemination of the SISAQOL-IMI activities and results and to ensure timely and international promotion of the project objectives, activities, and outcomes.
  2. Create high-visibility and wide impact in the community involving relevant stakeholders and final users, including patient groups and the broad public.
  3. Ensure timely and international promotion of the project objectives, activities, and outcomes.
  4. Provide advice and assistance on the appropriate involvement of patient representatives in each part of the SISAQOL-IMI work programme.
  5. Ensure a clear, consistent, and identifiable brand for SISAQOL-IMI.
  6. Ensure the timely flow of information across SISAQOL-IMI work packages.
  7. Understand and document patient needs and priorities when it comes to their understanding and use of patient reported outcomes in their clinical care.

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