Challenges in Supportive and Palliative Neuromuscular Care in Developing Countries 

A report on a presentation from the International Congress on Neuromuscular Diseases in Florence, Italy.

By Jorge A. Bevilacqua

Jorge A. Bevilacqua

Palliative care is an essential component of comprehensive health care systems; however, its availability remains severely underdeveloped and markedly inequitable across regions and income groups.^{1, 2, 3, 4} Although Europe and the Americas have the greatest concentration of palliative care services, approximately 80% of the global need occurs in low- and middle-income countries (LMICs), which account for only 30% of existing services. These substantial disparities underscore the urgent need to strengthen palliative care capacity and improve equitable access, particularly in LMICs.¹

Although medical science has advanced rapidly, health care systems have struggled to ensure equitable access to diagnosis, treatment, and comprehensive patient care. Neuromuscular diseases (NMDs) add a layer of complexity to the supportive and palliative care needs of affected patients.^5,6 

Diagnostic delay and misdiagnosis — resulting in unacceptable delays in accurate diagnosis, prognosis, genetic counseling, and appropriate care — are not unique to LMICs. However, their prevalence in these settings remains unjustifiably high. Addressing these challenges requires sustained commitment from health care systems and the implementation of coordinated strategies to improve access to appropriate care.

Approximately 40 million people per year require palliative care worldwide, 78% of whom live in LMICs. Yet only 14% of the global need for palliative care is currently met.² In high-income countries, the likelihood of receiving palliative care is approximately 50%, compared with only 4% in LMICs. This disparity is even greater for neurological and neuromuscular diseases.^1,2 In India, for example, only 1%-2% of the estimated 5.4 million patients requiring palliative care each year receive such care.^7,8

A similar picture can be observed in other regions of the world. In Egypt, although multidisciplinary care is increasingly available, families pay up to 90% of medical costs out of pocket, with physiotherapy representing the second largest financial burden. Patients living in cities with access to specialized multidisciplinary care, including physiotherapy, have also been shown to maintain ambulation longer than their rural counterparts.⁹ In a recent study from Latin America, out-of-pocket expenses for patients with myasthenia gravis were found to substantially exceed the minimum wage.¹⁰ 

Amyotrophic lateral sclerosis (ALS) is the neuromuscular disease most associated with palliative care. Increasing recognition of its benefits has led to recommendations for earlier and greater integration of palliative care into ALS management.^5,6,11 However, an international survey of 21 countries across six continents demonstrated significant disparities in access to ALS/motor neuron disease (MND) care. 

Economic, cultural, and religious factors emerged as important determinants of access. Several resource-limited countries had virtually no public awareness of ALS and extremely limited access to patient care. Interestingly, according to the report, “these results were often a reflection of whether a country was WHO-designated high-, middle-, or low-income.”¹²

These inequities disproportionately affect patients with rare neuromuscular disorders because their diseases often require lifelong multidisciplinary care and expensive interventions.⁶

The Main Critical Areas and Barriers

Several interconnected barriers create what may be termed a circle of neglect for rare NMDs in LMICs. Their low visibility at the organizational level results in limited awareness, inadequate disease registries, and insufficient inclusion in medical curricula. This contributes to a shortage of trained health care professionals and, consequently, limited investment in specialized infrastructure, logistics, and services. 

These deficiencies, in turn, contribute to delays in diagnosis and treatment. Lack of diagnosis also affects the perceived prevalence of these diseases, perpetuating their neglect. Supportive and palliative care form part of this same circle. Although many of the limitations are shared with other areas of medicine, they may be particularly consequential in NMDs.

Understanding Supportive and Palliative Care

Definitions of supportive and palliative care are frequently used ambiguously and are often conceptualized primarily from an oncological perspective.^13,14 Globally, palliative care can be described as active, holistic care for individuals experiencing serious health-related suffering. It focuses on preventing and relieving physical, psychological, social, and spiritual distress without intending either to hasten or postpone death.¹⁵

Supportive care encompasses a broader spectrum, extending across diagnosis, rehabilitation, survivorship, secondary prevention, and end-of-life care.^4,14 Within this framework, palliative care represents one component of comprehensive supportive care; thus, supportive care encompasses palliative care.¹⁴

This distinction is particularly relevant in chronic hereditary neuromuscular diseases and other nononcological conditions. Patients with chronic inherited neuromuscular disorders must live with the consequences of their disease, which can impose substantial physical, emotional, and financial burdens on patients, families, and caregivers. Patients may experience one or more of the following:

• respiratory insufficiency and cardiac complications
• dysphagia and dysarthria, with consequent nutritional and communication difficulties
• immobility, chronic fatigue, and pain
• psychological and existential suffering 

These challenges extend well beyond traditional neurological management and require comprehensive supportive care throughout the disease trajectory.^5,6,16

Systemic and Infrastructural Barriers

In most LMICs, specialized services are largely concentrated in tertiary referral centers, typically located in major cities and often far from the patients who require them.^17,9 Patients may therefore need to travel long distances to access specialized care, while primary and secondary health care systems remain poorly equipped to manage chronic neuromuscular diseases. Consequently, patients frequently reach referral centers without an adequate diagnosis and at advanced stages of disease, when therapeutic options are increasingly limited.

This creates a cascading failure: Already scarce tertiary centers become overburdened with patients with complex or advanced disease, families face catastrophic out-of-pocket expenses, and the health care system becomes increasingly inefficient.¹⁷ Some of these pressures could be mitigated through better distribution and optimization of supportive and palliative care services closer to patients and their communities.

Diagnostic Delay and Misdiagnosis

Although approximately 15 million people worldwide live with inherited NMDs, access to precision molecular diagnosis remains highly unequal. Modern DNA sequencing enables accurate diagnosis and, increasingly, access to gene-specific therapies.¹⁸ Nevertheless, most affected families live in LMICs, while genetic reference databases remain heavily biased toward populations of European ancestry and those from developed regions. Molecular diagnosis therefore remains inaccessible to many patients.^19,20

As noted above, delayed diagnosis postpones appropriate therapy while potentially exposing patients to ineffective or even harmful treatments.

This challenge is directly linked to workforce limitations. Shortages of trained professionals exist across many developing countries because of factors related to training pathways, financial pressures, and increasing demand for specialized expertise. In supportive and palliative care, contributing factors include inadequate education in neuromuscular medicine, limited awareness of palliative care as a professional field, fragmentation between specialties, and insufficient multidisciplinary collaboration.^17,21,22 As a result, referrals are delayed, symptom control is inadequate, and tertiary hospitals become increasingly overburdened.

Limited Access to Therapy

Access to therapy deserves particular attention, as discussions often focus on novel, high-cost treatments. However, in LMICs, access is also limited for basic and essential interventions, including supportive and palliative care.³⁷

This limitation becomes even more evident when access to modern therapies is considered. Highly effective treatments now exist for spinal muscular atrophy (SMA), Duchenne muscular dystrophy (DMD), Pompe disease, and several autoimmune conditions, including immune-mediated neuromuscular junction disorders and myopathies.²¹ Yet these therapies remain inaccessible to most patients worldwide.^10,19,23

There is a widespread perception within both the medical and patient communities that when a disease-specific therapy becomes available, supportive and palliative care become less necessary. This notion is incorrect. On the contrary, the introduction of a disease-modifying therapy increases the importance of optimal supportive care to give patients the best possible chance of benefiting from treatment.

Moreover, the existence of novel high-cost therapies may make supportive and palliative care even more relevant when advanced treatments cannot be accessed. In other words, sophisticated, high-cost therapies cannot achieve their full potential when delivered in communities lacking adequate supportive care.

Why NMDs Require a Different Approach

NMDs should not be viewed solely as rare neurological disorders. They are chronic, progressive conditions, often associated with a shortened life expectancy. They require continuous multidisciplinary management that addresses physical, psychological, social, and existential needs.^5,6

NMDs require a model of supportive and palliative care that differs from systems derived primarily from oncology, which remain the prevailing models in many developing countries.^13,14 

NMDs differ substantially from cancer because they often follow prolonged disease trajectories, involve progressive disability rather than a predominantly terminal course, and require long-term ventilatory, nutritional, rehabilitation, and communication support. They may also present unique psychological and mental health challenges, particularly when cognitive impairment occurs in certain NMDs.^5,6,18

Consequently, cancer-centered models should not simply be transferred to NMD without appropriate adaptation.

Promising Strategies for Improvement

Several approaches may help overcome these challenges, including community-based models of care. Community- and home-based care can substantially improve access while reducing hospital utilization. The Kerala “Neighborhood Network” in India provides an example of a volunteer-supported model of home-based palliative care.²⁴ Telemedicine and wearable technologies may also provide opportunities to extend specialist expertise to underserved regions.²⁵

Education and Professional Development

Education and professional development are essential to achieving sustainable improvements in neuromuscular care. Based on the author’s experience in Latin America, examples of specialized educational initiatives in the neuromuscular field include the Paris Summer School of Myology (AcadeMYO).²⁷ This initiative subsequently inspired similar programs adapted to different regions, including the Euro Latin America Summer School of Myology (EVELAM),²⁸ which promotes education in rare NMDs, including the principles of supportive and palliative care and practical workshops.

Scientific societies can also contribute by organizing educational and scientific meetings, such as those promoted by the Latin American Society of Neuromuscular Disorders (SOLANE),²⁹ and by supporting specialized training programs such as the World Muscle Society SEED Project.³⁰ Collectively, these initiatives have trained thousands of health care professionals across Latin America and other developing regions.

Precision Diagnosis and Genomic Diversity

Regional genomic initiatives such as Latin Seq³¹ and sponsored diagnostic programs demonstrate how international collaboration can expand access to molecular diagnosis while increasing the representation of diverse populations in genetic databases.^32,33 Such projects can contribute to a better understanding of the genetic profiles of patients with NMDs in LMICs, facilitating earlier diagnosis and enabling more appropriate disease management.^34,35,36

Integrated Multidisciplinary Care

All of these approaches should ultimately converge toward what is clearly indispensable: integrated multidisciplinary care. Effective neuromuscular care requires coordinated collaboration among neurologists, rehabilitation specialists, palliative care clinicians, respiratory physicians, nutritionists, psychologists, speech and language therapists, nurses, and social workers. Depending on the context, patient associations, nongovernmental organizations, and other stakeholders can also play an important role.

Conclusion

Although remarkable therapeutic advances have transformed the outlook for several NMDs, these innovations have also widened disparities between countries with and without access to precision medicine. As a result, supportive and palliative care has become more important.

Treatment does not necessarily equal cure. Health care systems therefore have an ethical responsibility to provide integrated, patient-centered supportive care throughout the disease trajectory. Achieving this goal requires earlier diagnosis, multidisciplinary collaboration, expanded education, stronger health policies, and equitable access to both modern therapies and high-quality supportive care.

In essence, supportive care for NMDs in LMICs is a field in transition. Although it remains severely underresourced compared with high-income countries, there is growing recognition of the problem and increasing momentum toward practical, culturally adapted solutions aimed at maximizing quality of life despite resource limitations. •

Jorge A. Bevilacqua, MD, PhD, is professor at the Departamento de Neurología y Neurocirugía, Hospital Clínico Universidad de Chile. 

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