Periodic Reporting for period 1 - PROMINENT (PRECISION MEDICINE PLATFORM IN NEURODEGENERATIVE DISEASE)
Okres sprawozdawczy: 2023-05-01 do 2024-04-30
Neurodegenerative diseases are one of the most important contributors to morbidity and mortality in the elderly. In Europe, over 14 million people are currently living with dementia, at a cost of over 400 billion EUR annually. Comorbidities with these conditions are frequent and a major obstacle to optimal diagnosis and management. Recent advances in diagnostic technologies and the advent of disease-modifying therapies (DMT) for Alzheimer’s disease (AD), the most common aetiology of dementia, heralds the beginning of precision medicine in this disease area. PROMINENT will develop a digital platform for precision medicine that will remove barriers that currently exists for leveraging these technological advancements in the routine care of patients with neurodegenerative disorders and co-morbidities. The platform gives clinicians access to prediction models leveraging multimodal diagnostic data automatically derived from multiple sources (imaging repositories, medical records, mobile devices), helping them choose optimal care pathways and improving diagnostic precision. It will provide personalized, relevant and meaningful information on diagnosis and prognosis in a format understandable by patients and care partners. Further, it will support the introduction of new health technologies such as DMT for AD, by ensuring adherence to appropriate use guidelines and facilitating the prospective collection of data on real-world usage, safety and effectiveness. The expected impact of the project is to increase diagnostic accuracy and optimized use of existing and new treatment options. It will empower patients and caregivers by engaging them in more person-centric health care decisions, leading to improved adherence and patient experience. Ultimately this is expected to lead to cost-effective care, improved health outcomes and quality of life.
The primary goal of PROMINENT is to deliver a digital platform for implementing precision medicine in neurodegenerative disorders. The aim is to develop a clinical decision support system that improves patient management, and to validate the system in a prospective clinical study. The system is developed by refining and adding functionality to existing platforms and tools from consortium partners.
PROMINENT has conducted a Europe-wide survey among specialist physicians on their views on what functionality should be included in a clinical decision-support system, and what value this would bring in clinical practice. Results from the survey were used to inform next steps, and a manuscript is now being submitted for publication. During the first year of the project, PROMINENT has made important additions to the digital platform, including functionality for automated analysis, interpretation and reporting of MRI, PET and DaTSCAN images, and integration of digital cognitive testing (cCOG) into the system.
PROMINENT has also made progress on developing novel prediction models for differential diagnosis and prognosis, which will later be added to the platform. A literature review on existing multimodal prediction models has been completed, establishing the data management and sharing procedures within the consortium, and developing a concept for combining predictions of test results into optimized diagnostic pathways.
A protocol for the validation study has been developed and finalized through multiple rounds of discussions between project participants. The study will focus on demonstrating the clinical value of the decision support system through increasing diagnostic confidence and optimizing the efficiency of the diagnostic process in terms of resource use, costs and time.
Additionally, the project aims to deploy the system so that new DMTs for AD can be introduced. In anticipation of the introduction of new DMT, the consortium has consulted key stakeholders on what evidence will be required to support decisions on pricing, reimbursement and implementation. A report on a survey among European HTA bodies on evidence requirements has been completed and the planning for how the PROMINENT platform will contribute to evidence generation on DMT as used in routine care have started.
A key factor for the project success is the meaningful involvement of patients and their caregivers in the majority activities across PROMINENT. The project has established a Public Involvement board and held two online consultations. Patients and caregivers have been consulted to review and comment on mock-ups of individualized reports from the decision support system, and their feedback is considered in subsequent updates.
PROMINENT has conducted a Europe-wide survey among specialist physicians on their views on what functionality should be included in a clinical decision-support system, and what value this would bring in clinical practice. Results from the survey were used to inform next steps, and a manuscript is now being submitted for publication. During the first year of the project, PROMINENT has made important additions to the digital platform, including functionality for automated analysis, interpretation and reporting of MRI, PET and DaTSCAN images, and integration of digital cognitive testing (cCOG) into the system.
PROMINENT has also made progress on developing novel prediction models for differential diagnosis and prognosis, which will later be added to the platform. A literature review on existing multimodal prediction models has been completed, establishing the data management and sharing procedures within the consortium, and developing a concept for combining predictions of test results into optimized diagnostic pathways.
A protocol for the validation study has been developed and finalized through multiple rounds of discussions between project participants. The study will focus on demonstrating the clinical value of the decision support system through increasing diagnostic confidence and optimizing the efficiency of the diagnostic process in terms of resource use, costs and time.
Additionally, the project aims to deploy the system so that new DMTs for AD can be introduced. In anticipation of the introduction of new DMT, the consortium has consulted key stakeholders on what evidence will be required to support decisions on pricing, reimbursement and implementation. A report on a survey among European HTA bodies on evidence requirements has been completed and the planning for how the PROMINENT platform will contribute to evidence generation on DMT as used in routine care have started.
A key factor for the project success is the meaningful involvement of patients and their caregivers in the majority activities across PROMINENT. The project has established a Public Involvement board and held two online consultations. Patients and caregivers have been consulted to review and comment on mock-ups of individualized reports from the decision support system, and their feedback is considered in subsequent updates.
The PROMINENT digital platform will be initially deployed at specialised memory clinics and has a potential to improve the diagnostic workflow of neurodegenerative disorders and provide more detailed and confident information on prognosis for patients and their care partners. PROMINENT have devised a novel approach to diagnostic algorithm development, taking into account the sensitivity and specificity of individual diagnostic tests, as well as their resource requirements and cost. The method will allow clinicians to identify the optimal sequence of diagnostic tests for an individual patient.
Moreover, the project has made an impact on clinical use as cMRI has been extended by the Parkinson+ report, cPET and cDAT have been launched. The system has already received positive feedback about the new features and products.
The project also aims to design, implement and evaluate a process for capturing RWE from the digital platform to evaluate the usage, safety, effectiveness and cost-effectiveness of disease-modifying therapies for AD. The tasks carried out during the first year of the project to determine which type of data is preferred by the European HTA organisations will be used to develop a framework and protocol for how the PROMINENT digital platform can be used for capturing RWE to meet these reimbursement requirements.
The evaluation and validation studies will majorly contribute to the quantification of project impact on the diagnostic and prognostic precision by 1) providing data on the usability of a CDSS and how it supports clinicians in the diagnostic process, 2) analysing the impact of the CDSS on diagnostic confidence and assessment of DMT eligibility, and 3) analysing the prognostic accuracy of the CDSS after one year.
The study design of the validation study has been carefully developed to be able to compare time and monetary resources required for the diagnostic pathway with and without use of the CDSS, thereby delivering estimates on potential savings in resources due to “unnecessary” diagnostic procedures without reducing diagnostic accuracy. This research on optimal diagnostic pathways is of major importance due to limited capacities of specialized memory clinics in the face of an increasing rate of older individuals in European societies, thereby combining both economic and societal impacts.
In addition, the relevance of efficient identification of individuals who are eligible for DMTs will increase further once DMTs become available in Europe. The validation study has been designed in a way to contribute toward the project’s impact on optimized introduction of DMTs and potential reduction of CSF tests.
Moreover, the project has made an impact on clinical use as cMRI has been extended by the Parkinson+ report, cPET and cDAT have been launched. The system has already received positive feedback about the new features and products.
The project also aims to design, implement and evaluate a process for capturing RWE from the digital platform to evaluate the usage, safety, effectiveness and cost-effectiveness of disease-modifying therapies for AD. The tasks carried out during the first year of the project to determine which type of data is preferred by the European HTA organisations will be used to develop a framework and protocol for how the PROMINENT digital platform can be used for capturing RWE to meet these reimbursement requirements.
The evaluation and validation studies will majorly contribute to the quantification of project impact on the diagnostic and prognostic precision by 1) providing data on the usability of a CDSS and how it supports clinicians in the diagnostic process, 2) analysing the impact of the CDSS on diagnostic confidence and assessment of DMT eligibility, and 3) analysing the prognostic accuracy of the CDSS after one year.
The study design of the validation study has been carefully developed to be able to compare time and monetary resources required for the diagnostic pathway with and without use of the CDSS, thereby delivering estimates on potential savings in resources due to “unnecessary” diagnostic procedures without reducing diagnostic accuracy. This research on optimal diagnostic pathways is of major importance due to limited capacities of specialized memory clinics in the face of an increasing rate of older individuals in European societies, thereby combining both economic and societal impacts.
In addition, the relevance of efficient identification of individuals who are eligible for DMTs will increase further once DMTs become available in Europe. The validation study has been designed in a way to contribute toward the project’s impact on optimized introduction of DMTs and potential reduction of CSF tests.