RESUMEN
The IMI public-private partnership between the European Commission and the European Federation of Pharmaceutical Industries and Associations (EFPIA) was launched in 2008 with an initial budget of 2 billion. Aiming to accelerate the development of innovative medicines for areas of unmet clinical need, the IMI has committed over 380 million to projects on neurodegenerative disorders (NDD), catalyzing public-private collaborations at scale and at all stages of the R&D pipeline. Because of this vast investment, research on neurodegenerative diseases has made enormous strides in recent decades. The challenge for the future however remains to utilize this newly found knowledge and generated assets to develop better tools and novel therapeutic strategies. Here, we report the results of an integrated programme analysis of the IMI NDD portfolio, performed by the Neuronet Coordination and Support Action. Neuronet was launched by the IMI in 2019 to boost synergies and collaboration between projects in the IMI NDD portfolio, to increase the impact and visibility of research, and to facilitate interactions with related initiatives worldwide. Our analysis assessed the characteristics, structure and assets of the project portfolio and identifies lessons from projects spanning preclinical research to applied clinical studies and beyond. Evaluation of project parameters and network analyses of project partners revealed a complex web of 236 partnering organizations, with EFPIA partners often acting as connecting nodes across projects, and with a great diversity of academic institutions. Organizations in the UK, Germany, France and the Netherlands were highly represented in the portfolio, which has a strong focus on clinical research in Alzheimer's and Parkinson's disease in particular. Based on surveys and unstructured interviews with NDD research leaders, we identified actions to enhance collaboration between project partners, by improving the structure and definition of in-kind contributions; reducing administrative burdens; and enhancing the exploitation of outcomes from research investments by EU taxpayers and EFPIA. These recommendations could help increase the efficiency and impact of future public-private partnerships on neurodegeneration.
RESUMEN
Aims: To facilitate regulatory learning, we evaluated similarities and differences in evidence requirements between regulatory and health technology assessment (HTA) bodies of Alzheimer's disease (AD) approved products. Methods: The European marketing authorisation application dossiers and European public assessment reports (EPARs) of the licensed AD drugs were screened to identify the phase III randomised controlled trials (RCTs) and outcomes used. We also screened the assessment reports of the National Institute of Health and Care Excellence (NICE, England) and the National Health Care Institute (ZiN, the Netherlands) to identify the studies and outcomes used in HTA assessments. Results: The application dossiers of donepezil, galantamine, rivastigmine, and memantine contained 16 phase III RCTs in total. These trials were also included in HTA assessments except that NICE excluded studies that were not published (n = 2) or trials that included patients with other types of dementia (n = 3). In the regulatory assessments the focus was on cognitive and global outcomes, and to some extent on function. In the HTA assessments of clinical effectiveness other domains were also covered including: function, behaviour and mood, and, occasionally, quality of life. In the economic analyses of NICE the domains cognition, function, and quality of life were included. Conclusion: There was a large overlap in inclusion of trials in regulatory and HTA assessments, although the focus on specific outcomes slightly differed. Understanding the methods and perceptions of both authorities can stimulate regulatory and HTA cross-talk and further alignment, and therefore more rapid patient access to new treatments.
RESUMEN
Although there are a growing number of well-reported, late-stage clinical trial failures in Alzheimer's disease, the introduction of a disease-modifying therapy within the next 5 years may be anticipated. These treatments are likely to target Alzheimer's disease 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 requirements for Alzheimer's disease, as a new drug will need to undergo regulatory and health technology assessments before it becomes available to patients. This article reports the discussions and activities of the regulatory and health technology assessment expert advisory group of the 2-year ROADMAP (real-world outcomes across the Alzheimer's disease spectrum: a multimodal data access platform) project. The expert advisory group discussions identified a lack of consensus on validated outcomes in the earliest Alzheimer's disease stages, the need for filling gaps between outcomes used across clinical trials and real-world settings, and the role that real-world evidence might have in characterising the impact of a possible disease-modifying therapy on caregivers, resource use and long-term outcomes.