Periodic Reporting for period 3 - SISAQOL-IMI (Establishing international standards in the analysis of patient reported outcomes and health-related quality of life data in cancer clinical trials)
Período documentado: 2023-01-01 hasta 2023-12-31
Measuring and quantifying how a patient feels or functions during treatment is an important endpoint in cancer clinical trials. It is generally accepted that the collection of PRO data in cancer clinical trials allows the inclusion of the patient’s voice in the risk-benefit assessment of therapies. However, no standards exist on how to analyse, interpret or report health-related quality of life (HRQOL) and other patient-reported outcomes (PROs). SISAQOL-IMI’s goal is to pursue efforts in addressing the urgent need for standardization, by setting clear and robust standards that are tailored to and endorsed by all relevant stakeholders. With a strong international and multi-stakeholder Consortium, the initiative aims at finding consensus on suitable methods to analyse valid PRO objectives in cancer randomized clinical trials (RCTs) and ways to communicate these PRO findings in a standardized way that is understandable to all.
To achieve this aim, SISAQOL-IMI will identify valid PRO research objectives and match these with appropriate statistical methods for PRO analysis in cancer RCTs. Translation to the estimands framework will be provided. Furthermore, the possibility of extending these recommendations to single-arm trial designs will be explored. Recommendations on clinically meaningful change for PRO instruments, as well as design considerations and ways for assessing quality of collected PRO data will be developed, and tools and templates for presentation and visualization of PRO findings freely made available. Strong emphasis is put on continuous collaboration with patient advocacy representatives throughout the project.
Increased interpretability, adoption and full use of PRO outcomes for all stakeholders is expected by providing consensus-based and validated recommendations and communication tools for PRO data, ultimately resulting in better communication and shared decision making, improved outcomes, treatment satisfaction and care.
To achieve this aim, SISAQOL-IMI will identify valid PRO research objectives and match these with appropriate statistical methods for PRO analysis in cancer RCTs. Translation to the estimands framework will be provided. Furthermore, the possibility of extending these recommendations to single-arm trial designs will be explored. Recommendations on clinically meaningful change for PRO instruments, as well as design considerations and ways for assessing quality of collected PRO data will be developed, and tools and templates for presentation and visualization of PRO findings freely made available. Strong emphasis is put on continuous collaboration with patient advocacy representatives throughout the project.
Increased interpretability, adoption and full use of PRO outcomes for all stakeholders is expected by providing consensus-based and validated recommendations and communication tools for PRO data, ultimately resulting in better communication and shared decision making, improved outcomes, treatment satisfaction and care.
The SISAQOL-IMI Consortium generated its final set of recommendation statements in 2023. This final set of recommendation statements were initially voted through an online survey and then re-voted during the Consensus meeting #3 which took place on 23 – 24 May 2023. Out of 79 statements, 77 were accepted by 2/3 majority.
In the remaining part of the year, the Consortium focused on refining and finalizing the statements. This involved several processes that were run in parallel: receiving feedback from the Independent Scientific Advisory Board, beta-testing of the recommendations through independent validation, obtaining EMA feedback through the qualification advice and the harmonization of recommendations from WP2 (Randomized Controlled Trials) and WP3 (Single-Arm Studies).
The results of these processes became available at the end of 2023, and the WP leaders have started evaluating and responding to feedback in preparation for Consensus process #4. The revisions and responses proposed by the WP leaders will be voted on and discussed in the next consensus meeting in May 2024. The Consortium has also started working on its key project outputs, such as the web tool (interactive table), which will allow future users to access a subset of recommendations relevant to their trial objective and endpoint at hand. The first drafts of the graphical templates that will guide visualizations of PRO data from cancer clinical trials have also been circulated within the Consortium and are currently being revised to iron out any discrepancies. Following Consensus meeting #4, the Consortium will work on the finalization of these key outputs and work towards the journal publication with the final recommendation statements.
The Consortium has initiated discussions on the sustainability plan for the SISAQOL-IMI recommendations. A virtual sustainability workshop was held on Dec 5, 2023. During this workshop, stakeholders presented their views on the future of the SISAQOL-IMI Consortium, including how it has responded to their needs and what further steps are necessary to address those needs.
The dissemination of SISAQOL-IMI has been gathering momentum even though the recommendation statements have not been finalized and therefore cannot be shared outside the Consortium. However, there has been great interest in the SISAQOL-IMI recommendations from different stakeholders, and there has been specific requests to access to the draft recommendation statements. Alternatively, the presentations given by the Consortium members in 2023 focused on certain aspects of the work but not directly on the recommendation statements. For example, literature review results from WP2 were presented at the ISOQOL Conference in Calgary, Canada. WP1 co-lead Madeline Pe gave a presentation introducing SISAQOL-IMI and its objectives, and highlighting the key challenges the Consortium handles with this project at the ESMO-Magnitude of Clinical Benefit Scale (ESMO-MCBS) session at European Society for Medical Oncology (ESMO) Congress 2023 in Madrid. Madeline Pe and several other Consortium members presented as keynote speakers at a session on “The emerging trend of incorporating PROs into the Oncology trials” at the ASA Biopharmaceutical Section Regulatory-Industry Statistics Workshop.
Two manuscripts have been published in the Lancet Oncology in 2023. The first manuscript is titled “Setting International Standards in Analysing Patient-Reported Outcomes and Quality of Life Endpoints in Cancer Clinical Trials - Innovative Medicines Initiative (SISAQOL-IMI): Stakeholder Views, Objectives, and Procedures”. It introduces the Consortium, explains its goals, and presents results from the priority setting survey from the kick-off meeting. The second manuscript is a literature review by WP3 titled “Single-arm studies involving patient-reported outcome data in oncology: a literature review on current practice”. Two additional papers have been drafted by WP2 and WP3, one of which is currently being reviewed by the Consortium members.
SISAQOL-IMI organized an educational workshop for the WECAN Academy 2023 Training Event for Educating Cancer Patient Advocates from Around the World. The session was titled “Patient Reported Outcomes in Oncology Studies: Intercurrent Events. The session was led by Silene ten Seldam, Elektra Papadopoulos, Madeline Pe, Ahu Alanya from the SISAQOL-IMI Consortium.(https://wecanadvocate.eu/featured/wecan-academy-2023-recap/)
Patient engagement activities within the Consortium continued in in the third year with a patient workshop before Consensus meeting #3 in Portugal where a small group of experts discussed the recommendation statements with the patients/patient representatives and responded to their questions before the discussions and re-voting takes place during the meeting.
In the remaining part of the year, the Consortium focused on refining and finalizing the statements. This involved several processes that were run in parallel: receiving feedback from the Independent Scientific Advisory Board, beta-testing of the recommendations through independent validation, obtaining EMA feedback through the qualification advice and the harmonization of recommendations from WP2 (Randomized Controlled Trials) and WP3 (Single-Arm Studies).
The results of these processes became available at the end of 2023, and the WP leaders have started evaluating and responding to feedback in preparation for Consensus process #4. The revisions and responses proposed by the WP leaders will be voted on and discussed in the next consensus meeting in May 2024. The Consortium has also started working on its key project outputs, such as the web tool (interactive table), which will allow future users to access a subset of recommendations relevant to their trial objective and endpoint at hand. The first drafts of the graphical templates that will guide visualizations of PRO data from cancer clinical trials have also been circulated within the Consortium and are currently being revised to iron out any discrepancies. Following Consensus meeting #4, the Consortium will work on the finalization of these key outputs and work towards the journal publication with the final recommendation statements.
The Consortium has initiated discussions on the sustainability plan for the SISAQOL-IMI recommendations. A virtual sustainability workshop was held on Dec 5, 2023. During this workshop, stakeholders presented their views on the future of the SISAQOL-IMI Consortium, including how it has responded to their needs and what further steps are necessary to address those needs.
The dissemination of SISAQOL-IMI has been gathering momentum even though the recommendation statements have not been finalized and therefore cannot be shared outside the Consortium. However, there has been great interest in the SISAQOL-IMI recommendations from different stakeholders, and there has been specific requests to access to the draft recommendation statements. Alternatively, the presentations given by the Consortium members in 2023 focused on certain aspects of the work but not directly on the recommendation statements. For example, literature review results from WP2 were presented at the ISOQOL Conference in Calgary, Canada. WP1 co-lead Madeline Pe gave a presentation introducing SISAQOL-IMI and its objectives, and highlighting the key challenges the Consortium handles with this project at the ESMO-Magnitude of Clinical Benefit Scale (ESMO-MCBS) session at European Society for Medical Oncology (ESMO) Congress 2023 in Madrid. Madeline Pe and several other Consortium members presented as keynote speakers at a session on “The emerging trend of incorporating PROs into the Oncology trials” at the ASA Biopharmaceutical Section Regulatory-Industry Statistics Workshop.
Two manuscripts have been published in the Lancet Oncology in 2023. The first manuscript is titled “Setting International Standards in Analysing Patient-Reported Outcomes and Quality of Life Endpoints in Cancer Clinical Trials - Innovative Medicines Initiative (SISAQOL-IMI): Stakeholder Views, Objectives, and Procedures”. It introduces the Consortium, explains its goals, and presents results from the priority setting survey from the kick-off meeting. The second manuscript is a literature review by WP3 titled “Single-arm studies involving patient-reported outcome data in oncology: a literature review on current practice”. Two additional papers have been drafted by WP2 and WP3, one of which is currently being reviewed by the Consortium members.
SISAQOL-IMI organized an educational workshop for the WECAN Academy 2023 Training Event for Educating Cancer Patient Advocates from Around the World. The session was titled “Patient Reported Outcomes in Oncology Studies: Intercurrent Events. The session was led by Silene ten Seldam, Elektra Papadopoulos, Madeline Pe, Ahu Alanya from the SISAQOL-IMI Consortium.(https://wecanadvocate.eu/featured/wecan-academy-2023-recap/)
Patient engagement activities within the Consortium continued in in the third year with a patient workshop before Consensus meeting #3 in Portugal where a small group of experts discussed the recommendation statements with the patients/patient representatives and responded to their questions before the discussions and re-voting takes place during the meeting.