Institutional Positioning: On the Clinical, Healthcare and Organizational Challenges Associated with New Disease-Modifying Therapies for Alzheimer’s Disease

The introduction of the first disease-modifying therapies for Alzheimer’s disease is a moment of special relevance for the future of care for people living with this disease. At Ace Alzheimer Center Barcelona, ​​we recognize the complexity of an evaluation process that must combine scientific evidence, patient safety, system sustainability, and equity in access to therapeutic innovation. However, we believe that people with Alzheimer’s disease should be able to benefit from therapeutic advances that demonstrate a favorable benefit-risk balance, within a framework of responsible, sustainable use based on the best available scientific evidence. We believe that the current debate transcends the financing of two specific drugs. These first disease-modifying therapies represent the beginning of a new stage in the approach to Alzheimer’s, characterized by early and biological diagnosis, precision medicine, and the progressive incorporation of new therapeutic alternatives. The fundamental question is how the National Health System will approach this transformation. We defend the study of formulas for gradual, conditional, and evaluable access, adapted to the evidence and the characteristics of each treatment. These formulas should include common eligibility criteria, mandatory registries, centers with accredited experience in administering and monitoring of treatments, clinical and radiological monitoring, agreements based on results, and periodic reviews of financing conditions. The objective is not to anticipate the result of the economic evaluation or to presuppose a net saving. It is to ensure that, in addition to the pharmaceutical cost, the analysis incorporates the possible effects on autonomy, dependency, health, and social resources, quality of life, and the burden on families and caregivers. Above all other considerations, the new paradigm must aim to preserve the dignity and identity of each person, their autonomy, their capacity to decide, and their life purpose for as long as possible. Spain has highly qualified professionals, centers with extensive clinical and research experience, and an international leadership position in Alzheimer’s disease research. This scientific capital offers an opportunity to develop a responsible implementation model, aligned with the principles that inspire the Pharmaceutical Strategy for Europe: promoting innovation, ensuring equitable access, and strengthening the resilience of healthcare systems. We consider that innovation and sustainability are fully compatible objectives. To this end, we propose to promote a national strategy for progressive implementation based on the following principles:

1. Access, safety, and evaluation

• Define homogeneous clinical, diagnostic, and safety criteria for each treatment, which guarantee appropriate selection and rigorous monitoring of patients.
• Select centers with diagnostic, therapeutic, and monitoring capacity, and establish territorially equitable referral circuits.
• Create mandatory registries that integrate data on effectiveness, safety, quality of life, patient-reported outcomes, caregiver burden, and economic and social impact, and that allow the generation of Real-World Data and Real-World Evidence.
• Provide for periodic review mechanisms and, where feasible, conditional or results-based funding agreements.

2. Diagnostic expertise and care capacity

• Guarantee equitable access to excellent biomarker-based diagnostics, through centers or institutions with consolidated diagnostic expertise.
• Strengthen the role of primary care and facilitate effective coordination with specialized units, expert centers, social services, and community resources.
• Plan the training needs of professionals, especially in the areas of nursing, day hospitals, case management, information systems, and the necessary infrastructure.
• Combine pharmacological and non-pharmacological interventions within an integrated care model.

3. Prevention, families, and equity

• Promote brain health policies throughout life, focusing on modifiable risk factors, cardiovascular health, physical activity, nutrition, sleep, cognitive activity, and social participation.
• Ensure that place of residence, socioeconomic status or ability to access certain centers do not unduly condition diagnosis or treatment.
• Integrate support, training, respite, psychological support, and social protection measures for caregivers and families.
• Incorporate patient and caregiver organizations in a stable manner in governance, circuit design, and outcome evaluation.

4. Research, data, and responsible innovation

• Generate quality real-world data and evidence (Real World Data and Real World Evidence) through secure, interoperable platforms subject to ethical and data protection criteria.
• Promote clinical and translational research, clinical trials, and the development of new therapeutic strategies, including combined interventions.
• Incorporate digital health, artificial intelligence, and advanced data analysis with criteria of security, transparency, equity, and human supervision.
• Periodically publish aggregate indicators of access, safety, effectiveness, territorial equity, patient experience, and healthcare impact.

For all that has been stated, we make available to the system our more than thirty years of experience in care, research, clinical trials, diagnosis using biomarkers, and generation of scientific evidence, aiming to contribute to the design of a model for the responsible incorporation of disease-modifying therapies. In this context, we request an institutional meeting with the competent bodies to share these proposals and jointly explore measures that will allow the National Health System to prepare for the incorporation of current and future modifying therapies. We are convinced that collaboration between administrations, scientific societies, patient organizations, and expert centers constitutes the best guarantee for scientific progress to be translated into real benefit for people with Alzheimer’s disease and their families. Signing: Lluís Tárraga Mestre President Ace Alzheimer Center Barcelona Miren Jone Gurrutxaga Telleria General Director Ace Alzheimer Center Barcelona Mercè Boada Rovira Co-founder & Senior Medical Advisor Ace Alzheimer Center Barcelona Xavier Montalban Medical director Ace Alzheimer Center Barcelona