Abstract
The global impact of Alzheimer’s disease (AD) continues to increase, and focused efforts are needed to address this immense public health challenge. National leaders have set a goal to prevent or effectively treat AD by 2025. In this paper, we discuss the path to 2025, and what is feasible in this time frame given the realities and challenges of AD drug development, with a focus on disease-modifying therapies (DMTs). Under the current conditions, only drugs currently in late Phase 1 or later will have a chance of being approved by 2025. If pipeline attrition rates remain high, only a few compounds at best will meet this time frame. There is an opportunity to reduce the time and risk of AD drug development through an improvement in trial design; better trial infrastructure; disease registries of well-characterized participant cohorts to help with more rapid enrollment of appropriate study populations; validated biomarkers to better detect disease, determine risk and monitor disease progression as well as predict disease response; more sensitive clinical assessment tools; and faster regulatory review. To implement change requires efforts to build awareness, educate and foster engagement; increase funding for both basic and clinical research; reduce fragmented environments and systems; increase learning from successes and failures; promote data standardization and increase wider data sharing; understand AD at the basic biology level; and rapidly translate new knowledge into clinical development. Improved mechanistic understanding of disease onset and progression is central to more efficient AD drug development and will lead to improved therapeutic approaches and targets. The opportunity for more than a few new therapies by 2025 is small. Accelerating research and clinical development efforts and bringing DMTs to market sooner would have a significant impact on the future societal burden of AD. As these steps are put in place and plans come to fruition, e.g., approval of a DMT, it can be predicted that momentum will build, the process will be self-sustaining, and the path to 2025, and beyond, becomes clearer.
Highlights
Increasing life expectancy has produced a dramatic rise in the prevalence, and impact, of aging-associated diseases including dementia
Accelerating research and clinical development efforts and bringing disease-modifying therapies (DMTs) to market sooner would have a significant impact on the future societal burden of Alzheimer’s disease (AD)
Improved mechanistic understanding of disease development and progression is critical to more efficient AD drug development and will lead to improved therapeutic approaches and targets
Summary
Increasing life expectancy has produced a dramatic rise in the prevalence, and impact, of aging-associated diseases including dementia. IMI projects include: the Prediction of cognitive properties of new drug candidates for neurodegenerative diseases in early clinical development (PharmaCog) [55], to increase the ability to predict new medicines from laboratory studies and clinical models; the European Medical Information Framework Platform (EMIF) [56], to develop a framework for evaluating, enhancing and providing access to AD data (including CSF data, MRI scans, PET scans, plasma samples, DNA samples and RNA samples) from across Europe using Electronic Healthcare Record databases, and to identify biomarkers of AD onset in the preclinical and prodromal phase as well as for disease progression and to identify high-risk individuals for prevention trial participation; and the European Prevention of Alzheimer’s Dementia (EPAD) Initiative [57], to provide an environment for testing interventions targeted at delaying the onset of clinical symptoms with the aim of establishing a European-wide register of 24,000 participants. Lengthy AD drug development shortens the time from approval to patent expiration, reducing the likelihood of recouping the expense of drug development, including failed efforts; additional patent life and tax incentives for developing treatments for chronic neurological disorders might make AD drug development more attractive to additional industry investors
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