D6.3 Guiding principles and recommendations for the development and incorporation of RWE into clinical and market access development plans for AD

Although there are a growing number of well-reported late stage clinical trial failures in Alzheimer’s disease (AD), the introduction of a disease modifying therapy within the next 5 years may be anticipated. These treatments are likely to target AD in the earlier disease stages, unlike drugs that are currently available that treat symptoms of moderate to severe dementia. Therefore, there is a need to establish a consensus on regulatory and health technology assessment (HTA) requirements for AD as a new drug will have to go through regulatory and HTA assessments before it becomes available to patients. This paper reports the discussions and activities of the regulatory and HTA expert advisory group (EXAG) of the 2-year ROADMAP (real-world outcomes across the AD spectrum – a multimodal data access platform) project. The EXAG discussions identified a lack of consensus on validated outcomes in the earliest AD disease stages, the need for filling gaps between outcomes used across clinical trials and real-world settings, and the role real-world evidence might have in characterising the impact of a possible disease-modifying therapy on caregivers, resource use, and long-term outcomes.