The ROADMAP expert advisory group (EXAG) has met on four occasions and discussions have covered a number of topics including the prioritisation of outcomes for Alzheimer’s disease (AD), disease progression models and health economic modelling considerations.
One of the EXAG members, Raj Long (Gates Foundation (UK)) offers her views on the discussions to date and the value that having HTA/regulatory involvement has brought to the project.
“There is still a lot that we don’t understand about the natural history of AD, in particular, we lack data and evidence which covers the full disease spectrum from pre-dementia to dementia. This is a challenge for HTA and regulatory agencies who will require a strong evidence base to convince them of the validity of a disease progression model. There also needs to be transparency in the way that the different stages of the disease are modelled and then integrated in clinically meaningful way.”
“From a regulatory perspective, outcomes related to cognition and function are important. HTA agencies will also be interested in the economic value proposition. Beyond the clinical drivers of the patient themselves (largely cognitive and functional measurements), the only other area that would drive up costs is the societal impact. This needs to include the impact on carers’ and the costs associated with both formal and informal caregiving. We may also need to take a more holistic approach and consider other outcomes that haven’t traditionally been used in the past.
Given the challenges associated with AD, the EXAG has provided a valuable forum for discussions on disease progression and economic modelling for AD which should help to enable readiness for access when a disease-modifying product goes for regulatory and HTA assessment.”
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Find out more about the EXAG here.