BEat Project: Integrated Models for Cardiomyopathy

This is the general population prevalence of hypertrophic obstructive cardiomyopathy (HOC), a complex disease that can manifest itself in a wide variety of ways: from asymptomatic patients to those with disabling symptoms, to cardiac arrest, generally in adults, and more rarely in children. This variability makes it difficult to ensure consistent treatment pathways across the country.

To address this challenge, the BEat project was born, bringing together clinicians, institutions, patient associations, and the pharmaceutical industry to redesign care for patients with CMIO in Italy. Developed with the unrestricted contribution of Bristol Myers Squibb, the final document proposes: a path aimed at overcoming current fragmentations and building an integrated and person-centered system, recognizing patient associations like AICARM as leading figures: not passive actors, but active partners in building more inclusive care pathways, training caregivers, and representing real needs, serving as an essential bridge between patients, healthcare professionals, and institutions.

The analysis conducted by the BEat project highlights a strong territorial heterogeneityFew centers are effectively able to offer multidisciplinary expertise and comprehensive access to advanced diagnostic technologies, such as multimodal imaging or new drugs, such as cardiac myosin inhibitors. Mavacamten has demonstrated efficacy comparable to invasive therapy in controlling symptoms and improving quality of life in a significant percentage of patients. However, while it represents a promising therapeutic innovation, it also requires rigorous follow-up, which is often made difficult by territorial fragmentation. Even more critical is access to interventional therapies such as surgical myectomy, available only in a very few highly specialized national centers.

A particularly innovative aspect concerns the redefinition of treatment objectives. The document openly criticizes the tendency to focus exclusively on traditional parameters such as reduction of ventricular obstruction or five-year survival. These indicators may not fully reflect the perceived quality of life, the improvement or decline in functional capacity, or the psychological and social impact of the disease.

The heart of the BEat project is the proposal of an organizational model based on the model hub-and-spoke, or  Highly specialized centers collaborate synergistically with territorial structuresPeripheral centers manage the patient's daily clinical care, while centers of excellence, or hubs, are involved in critical decisions, such as risk assessment or the recommendation for complex interventions. To ensure uniform quality standards, the document proposes rigorous accreditation criteria for referral centers: the presence of an in-house genetics service, access to multimodal imaging (e.g., cardiac MRI, coronary CT, bone scan, etc.), a dedicated cardiomyopathy clinic, and a multidisciplinary team with proven experience. These are not bureaucratic requirements, but concrete guarantees of competence and experience.

The economic impact of CMIO is not negligible. International studies demonstrate that symptomatic patients They have significantly higher rates of hospitalizations, emergency room visits, and diagnostic procedures than the general population, with costs that increase exponentially with the severity of symptoms. The document highlights how each advanced stage of the disease entails greater costs, making early and targeted intervention crucial.

But the most innovative aspect of the proposed approach is perhaps the cultural one: from fragmented and reactive management to an integrated, proactive and person-centered modelA paradigm shift based on collaborative networks between reference and peripheral centers, data sharing, ongoing training, and a national governance capable of consistently guiding innovation. The document outlines a action plan The project is structured into several phases: from validation of the national reference document to its adaptation to specific regional contexts, and finally, its translation into concrete guidelines for the Regions. This process will integrate pharmacoeconomic analyses and epidemiological data, ensuring the qualified participation of clinical experts and patient associations in the decision-making process.

The stated goal is ambitious but necessary: ​​to influence regulatory authorities to adequately recognize CMIO as a disease that, while not formally classified as rare, fully shares its diagnostic complexity and the need for specialized expertise.
Not just a technical document, but a strategic vision to radically transform patient care with CMIO in Italy. In a healthcare system under pressure, where resources are limited and regional disparities are enormous, this initiative represents a model that can be replicated for other complex pathologies.

As the document's conclusions emphasize, only through strategic alignment at the national and regional levels will it be possible to implement efficient diagnostic and therapeutic pathways, ensuring timely access to innovative therapies and the overall sustainability of the system. An ambitious challenge, certainly, but for thousands of Italian patients and families, it represents a concrete hope for better and more equitable care.