Table 2.
ROADMAP key deliverablesa
| Defining a minimum set of measurable real-world AD outcomes. |
| This was met in WP2 by developing a matrix of prioritized outcomes according to stakeholder group and, in collaboration with WP3, mapping them against the data available from DPUK cohorts and other relevant European datasets. |
| Developing recommendations on RWE-appropriate, AD-related cognitive, functional, and behavioral outcomes. |
| This was met by work conducted under WP2, WP3, and WP4 using cohorts and other data sources to identify intermediate (and possibly theragnostic) markers that may be transferable to clinical practice. |
| Identifying data sources and outlining a data strategy for RWE outcomes. |
| This was met in WP3 by providing an overview of data sources available throughout Europe (not just within the ROADMAP consortium), the tools available for identifying and combining these data, and opportunities and strategies for pooling data. |
| Developing new methods for collecting RWE to improve how new treatments can be valued in AD. |
| This was met in WP2, WP3, and WP5-8 through engagement with regulators, payers, HTA bodies, patients, caregivers, industry, and researchers. The use of smart devices for self-report, social media, and direct objective assessment applications was of particular interest. |
| Providing recommendations for disease progression modeling. |
| This was met in WP4 using both hypothesis-driven and machine-learning approaches. Both approaches were informed by a review of current models of AD pathology, and by using available datasets for validation of selected models. |
| Developing the concept of an integrated core health economic model of healthcare use/costs and health outcomes in AD. |
| This was met in WP5 in collaboration with WPs 2-4 and 6. It was evidenced on quality of life, resource utilization, and costs associated with AD, and previous AD economic modeling studies, and used systematic reviews to improve the evidence base of long-term AD cost-effectiveness modeling. |
| Identifying guiding principles on the use of RWE in AD in the regulatory/HTA context. |
| Partners in WP6 collected and collated regulatory and HTA-related data to identify possible recommendations for the development and incorporation of RWE into clinical and market-access development plans for AD. |
| Developing requirements for an ethical, legal, and social issues framework for RWE in AD. |
| Partners in WP8 developed a report on ethical, legal, and social issues arising from the combination of datasets from multiple countries for disease, health economic, and treatment modeling for AD, including requirements set out in national ethical and legal frameworks governing local ethics approval. |
aSee Table 1 for Work Packages. AD, Alzheimer’s disease; DPUK, Dementias Platform UK; HTA, health technology assessment; RWE, real-world evidence; WP, Work Package.