14 July 2022

Readiness is about making sure health systems can integrate new components of care appropriately as well as responding to evolving evidence and social changes. This is necessary to ensure health systems can always deliver efficient and equitable care.

What is ALS?

ALS (amyotrophic lateral sclerosis) is a motor neurone disease that affects the nerve cells in the brain and spinal cord. This causes loss of muscle mass and weakness in the limbs, which leads to a lack of muscle control. Over time, this can make it difficult for a person to speak, eat, move and even breathe; life expectancy is typically two to five years from when symptoms start to occur. The causes of ALS are unknown, but evidence suggests that both genetic and environmental factors may be involved.

ALS is classified as a rare disease, but incidence is expected to increase. The average global incidence rate of ALS is 1 in 50,000 and in Western countries the prevalence of the condition is approximately 1 in 20,000. By 2040, the number of people with ALS is predicted to rise by 69% owing to ageing populations in middle- and high-income countries. Health systems will therefore need to adapt to be ready to manage the growing number of people living with ALS and to accommodate increasing demand for care.

There is no long-term treatment for ALS

There is no cure or approved long-term treatment for ALS. The current treatment for ALS focusses on managing symptoms with physical, occupational and speech therapy, or using one of the two approved short-term treatments, but their impact on disease progression is limited. An array of treatments with a variety of therapeutic mechanisms are currently under investigation in clinical trials.

Any treatments that could improve life expectancy may increase the number of people using services for ALS. Moreover, long-term treatments could drastically alter the management of ALS. However, careful systems-level planning is needed to ensure that people with the condition benefit from such therapies.

The development of new therapies for ALS is not straightforward. A number of previous trials for ALS treatment have failed due to issues with clinical trial design and challenges with the therapies. The development of treatments has also been hindered by the lack of diagnostic markers, varied disease progression and vastly different patient groups.

Multidisciplinary care teams are needed to provide care for people with ALS

Caring for people with ALS necessitates the cooperation of a multidisciplinary care team. Any developments in treatments for ALS will require careful planning to ensure there are properly trained clinical staff who understand the disease area and can work with others to provide treatment. Careful consideration is needed to highlight the workforce areas that need further development and support, and subsequent training takes time. Policymakers need to plan ahead to ensure there can be the appropriate integration of care pathways that can be executed by well-trained staff as part of a multidisciplinary care team.

ALS has a profound impact on people’s lives; new innovations are on the horizon, but it is important to consider how ready our health systems are to integrate new therapies. With a huge unmet need for treatments for people with ALS, a number of therapies under development and a growing disease burden, now is right time to think about health system readiness for ALS.

This is the second in a series of opinion posts about increasing health system readiness for different disease areas– read the previous instalment here.

Emily Medhurst, Project Officer at The Health Policy Partnership