Periodic Reporting for period 2 - GetReal Initiative (The GetReal Initiative)
Reporting period: 2019-06-01 to 2021-04-30
Real-world data from clinical practice can help to determine the effectiveness and safety of medicines in a real-world setting; it can help regulatory bodies and health technology assessment (HTA) agencies to identify which medicines have most added value for patients, and can assist doctors in determining which treatment would be most suited for a patient.
The objective of this project was to drive the sustainable adoption of tools, methodologies and best practices from IMI GetReal, which will increase the quality of real-world evidence generation in drug development and regulatory/health technology assessment processes across Europe.
The objective of this project was to drive the sustainable adoption of tools, methodologies and best practices from IMI GetReal, which will increase the quality of real-world evidence generation in drug development and regulatory/health technology assessment processes across Europe.
- Think Tank
A think tank has been established that brings together thought leaders and in order to discuss key barriers to and opportunities for the use of real-world evidence (RWE) in medicines development and healthcare decision making in Europe. The Think Tank has been instrumental in:
a) Providing a platform for open stakeholder dialogue;
b) Positioning the GetReal Initiative as an international leader in research to define best practices for RWE generation and evaluation;
c) Demonstrating how the tools, methodologies and approaches generated in IMI GetReal and by the GetReal Initiative Task Forces can facilitate the generation and use of RWE;
d) Further enhancing the credibility and acceptance of the deliverables of the original IMI GetReal project leading to long term sustainability of all GetReal tools; and
e) Shaping the business plan for the GetReal Institute, including organisational design, and initial focus areas, objectives, and deliverables.
- Pragmatic trial tool
Pragmatic clinical trials (PCT) are designed to evaluate the effectiveness of interventions in real-life routine practice conditions, whereas more traditional explanatory trials aim to test whether an intervention works under optimal/controlled conditions. Pragmatic trials offer an opportunity to generate high-quality evidence on relative effectiveness prior to and after launch.
The GetReal Initiative has developed a tool to support clinical trial teams design in the implementation and interpretation of the results of pragmatic trials. The GetReal Trial Tool offers step by step guidance to evaluate the options and implications of introducing real world elements in clinical trial design. The tool is open access. See: https://getrealtrialtool.eu
- Key paper on statistical issues in pragmatic clinical trials
Work was conducted to identify statistical issues for pragmatic clinical trials and to identify issues specifically for the ‘augmented trial’ methodology. The articles in a focused literature review were analysed, and statistical issues were extracted and categorized. A modified Delphi method was used to produce the set of Best Practice Recommendations for the statistical approaches to PCTs, through a focused literature review and using expert’s insights. This has resulted in a multi-stakeholder paper entitled "Best practice recommendations for the design and statistical analyses of pragmatic clinical trials: a modified Delphi approach" which is currently under review in a scientific journal.
- Landscaping tools:
A simple to navigate online tool has been developed aiming to give guidance on how to identify key questions that should be addressed early in the development path and provide outlines of possible (RWE) study designs to help researchers to make an informed decision on what design is most suitable to answer their research question. Topics covered are Knowledge Gaps and associated research questions, lifecycle stage of compound, study designs, including respective pros and cons, impact on relevant stakeholders and available data sources. See: https://rwestudydesign.com/home. A White Paper is in the final drafting stages.
- Pragmatic trial training
A course on the topic of pragmatic trials, in which the participants get acquainted with the tool and its uses, has been developed as part of the project and will become part of the offerings of the GetReal academy education and training platform.This self-paced course will provide an understanding of methodology, statistics and operation of pragmatic trials and concepts and impacts of Estimands, for the use of real-world evidence. Participants are distributed over the whole medicine development chain: from academic researchers to industry, regulatory and other professionals. See: https://www.getreal-academy.org/courses/methodology-statistics-and-operation-of-pragmatic-trials/
- ADDIS tool update
Further development took place on the ADDIS tool as part of the GetReal Initiative project. Aggregate Data Drug Information System (ADDIS) is an evidence-based decision support system for health care policy decision making that concerns alternative treatment options. ADDIS is largely feature complete. Recent improvements that were part of the GetReal Initiative: improvement of ADDIS regarding usability, stability and marketability, and the MCDA too. A newly updated tool has been launched and ADDIS is part of the CHMP Work Plan. See: https://www.drugis.org/index.
- Benefit risk assessment training
A self-paced course will provide an understanding of current techniques, opportunities and challenges of structured benefit risk assessment including different preference elicitation techniques. Although combining key benefits and risks into a single metric have historically only been conducted for the “average” patient, such aggregate assessments may also provide helpful information on the level of an individual patient. Furthermore, there is an increasing interest on the use of real‐world data to substantiate benefit–risk assessment and support a more patient-centered approach and the value of a new treatment options in daily practice. See: https://www.getreal-academy.org/courses/benefit-risk-assessment-in-decision-making/
- Business plan and a sustainable GetReal Institute institute
To continue the activities in real-world evidence space, and based on the support of the existing partners and other stakeholders in the field, a business plan was developed for a 'GetReal Institute'. This institute was launched in April of 2021 as a member association based in The Netherlands. The GetReal Institute brings together a wide variety of stakeholders to drive the sustainable development and adoption of tools, methods and best practices in the generation and use of RWE for better health care decision-making. See: www.getreal-institute.org.
A think tank has been established that brings together thought leaders and in order to discuss key barriers to and opportunities for the use of real-world evidence (RWE) in medicines development and healthcare decision making in Europe. The Think Tank has been instrumental in:
a) Providing a platform for open stakeholder dialogue;
b) Positioning the GetReal Initiative as an international leader in research to define best practices for RWE generation and evaluation;
c) Demonstrating how the tools, methodologies and approaches generated in IMI GetReal and by the GetReal Initiative Task Forces can facilitate the generation and use of RWE;
d) Further enhancing the credibility and acceptance of the deliverables of the original IMI GetReal project leading to long term sustainability of all GetReal tools; and
e) Shaping the business plan for the GetReal Institute, including organisational design, and initial focus areas, objectives, and deliverables.
- Pragmatic trial tool
Pragmatic clinical trials (PCT) are designed to evaluate the effectiveness of interventions in real-life routine practice conditions, whereas more traditional explanatory trials aim to test whether an intervention works under optimal/controlled conditions. Pragmatic trials offer an opportunity to generate high-quality evidence on relative effectiveness prior to and after launch.
The GetReal Initiative has developed a tool to support clinical trial teams design in the implementation and interpretation of the results of pragmatic trials. The GetReal Trial Tool offers step by step guidance to evaluate the options and implications of introducing real world elements in clinical trial design. The tool is open access. See: https://getrealtrialtool.eu
- Key paper on statistical issues in pragmatic clinical trials
Work was conducted to identify statistical issues for pragmatic clinical trials and to identify issues specifically for the ‘augmented trial’ methodology. The articles in a focused literature review were analysed, and statistical issues were extracted and categorized. A modified Delphi method was used to produce the set of Best Practice Recommendations for the statistical approaches to PCTs, through a focused literature review and using expert’s insights. This has resulted in a multi-stakeholder paper entitled "Best practice recommendations for the design and statistical analyses of pragmatic clinical trials: a modified Delphi approach" which is currently under review in a scientific journal.
- Landscaping tools:
A simple to navigate online tool has been developed aiming to give guidance on how to identify key questions that should be addressed early in the development path and provide outlines of possible (RWE) study designs to help researchers to make an informed decision on what design is most suitable to answer their research question. Topics covered are Knowledge Gaps and associated research questions, lifecycle stage of compound, study designs, including respective pros and cons, impact on relevant stakeholders and available data sources. See: https://rwestudydesign.com/home. A White Paper is in the final drafting stages.
- Pragmatic trial training
A course on the topic of pragmatic trials, in which the participants get acquainted with the tool and its uses, has been developed as part of the project and will become part of the offerings of the GetReal academy education and training platform.This self-paced course will provide an understanding of methodology, statistics and operation of pragmatic trials and concepts and impacts of Estimands, for the use of real-world evidence. Participants are distributed over the whole medicine development chain: from academic researchers to industry, regulatory and other professionals. See: https://www.getreal-academy.org/courses/methodology-statistics-and-operation-of-pragmatic-trials/
- ADDIS tool update
Further development took place on the ADDIS tool as part of the GetReal Initiative project. Aggregate Data Drug Information System (ADDIS) is an evidence-based decision support system for health care policy decision making that concerns alternative treatment options. ADDIS is largely feature complete. Recent improvements that were part of the GetReal Initiative: improvement of ADDIS regarding usability, stability and marketability, and the MCDA too. A newly updated tool has been launched and ADDIS is part of the CHMP Work Plan. See: https://www.drugis.org/index.
- Benefit risk assessment training
A self-paced course will provide an understanding of current techniques, opportunities and challenges of structured benefit risk assessment including different preference elicitation techniques. Although combining key benefits and risks into a single metric have historically only been conducted for the “average” patient, such aggregate assessments may also provide helpful information on the level of an individual patient. Furthermore, there is an increasing interest on the use of real‐world data to substantiate benefit–risk assessment and support a more patient-centered approach and the value of a new treatment options in daily practice. See: https://www.getreal-academy.org/courses/benefit-risk-assessment-in-decision-making/
- Business plan and a sustainable GetReal Institute institute
To continue the activities in real-world evidence space, and based on the support of the existing partners and other stakeholders in the field, a business plan was developed for a 'GetReal Institute'. This institute was launched in April of 2021 as a member association based in The Netherlands. The GetReal Institute brings together a wide variety of stakeholders to drive the sustainable development and adoption of tools, methods and best practices in the generation and use of RWE for better health care decision-making. See: www.getreal-institute.org.
The GetReal Initiative has enabled interaction and research partnership on science and innovation in RWE involving regulators, HTA bodies, academia, patients, physicians and industry. This will facilitate researchers from across all sectors of the healthcare ecosystem to come together around key challenges facing the generation of RWE and its adoption into drug development, regulatory and HTA decision making as well as physician and patient treatment decisions.
The continued value of the GetReal collaboration is showcased by the launch of the GetReal Institute (www.getreal-institute.org) a self-sustinaing entity which brings together a wide variety of stakeholders to drive the sustainable development and adoption of tools, methods and best practices in the generation and use of RWE for better health care decision-making.
The continued value of the GetReal collaboration is showcased by the launch of the GetReal Institute (www.getreal-institute.org) a self-sustinaing entity which brings together a wide variety of stakeholders to drive the sustainable development and adoption of tools, methods and best practices in the generation and use of RWE for better health care decision-making.