This article first appeared in Forum, The Edge Malaysia Weekly on December 23, 2019 - December 29, 2019

When we talk about health or healthcare, we often forget about the ripple effect it has on our everyday lives. This is especially true of any condition that is chronic and debilitating as it can dramatically change not just one but multiple lives simultaneously. The challenges faced by individuals with long-term health conditions are exacerbated when there is inadequate social support in place. For a government to adequately support patients with complex health needs, there has to be inter-ministerial collaboration when formulating strategies.

Rare or orphan disease is one that affects a very small percentage of the population. There are around 7,000 diseases, the majority of which are genetically inherited. Of these, only 5% have an existing treatment option available. In Malaysia, many cases of rare diseases are left untreated and undetected with little knowledge when it comes to choices of treatment available. Rare diseases are often incurable and crippling, which spell a long-term psychological, medical and financial burden. Adapting to the demands of living with rare disease can be incredibly difficult, especially when treatment comprises only a fraction of the story.

Over the last year, the Institute for Democracy and Economic Affairs’ (IDEAS) Championing for the Rare project has aimed to highlight the voices of patients and advocate for a national policy for rare disease. A policy can pave the way for strategic planning for the management of rare disease in Malaysia and improve the patients’ quality of life. Deputy Minister of Health Dr Lee Boon Chye recently announced that a National Framework for Rare Disease has been developed to integrate its management in Malaysia. This initiative by the Ministry of Health is a step in the right direction to improve access to healthcare services for rare disease patients.

According to the minister, the short-term goals of the framework include deciding on a definition of rare disease and strengthening the governance of the relevant services and laboratories. Apart from that, public health advocacy on rare disease will be stabilised. Lee also noted that in the long term, the committee aims to create policies and better strategies to improve access to orphan products and aspires to set up a registry to collect and analyse patient data. These are all positive initiatives to improve the treatment and management of rare disease in Malaysia.

However, it is important to recognise that the social and everyday care of people with rare disease and their families is beyond the responsibility of the Ministry of Health. People diagnosed with rare disease often lack information about their disease or rights, and have a high level of social, psychological and economic vulnerability. This often extends to the main caregivers of the patient and cannot realistically only be supported through the Ministry of Health and requires collaboration with other relevant ministries.

In line with this, as announced by Lee, the national rare disease governance committee will also be represented by other ministries, including the Ministry of Education and Ministry of Women, Family and Community Development. But how can these ministries contribute to a more holistic framework for rare disease that will improve the well-being of these patients in a sustainable manner?

Under the Championing for the Rare project, there were numerous opportunities to interact with multiple patients with rare disease and their caregivers in the country. From these interactions, one could surmise that among the struggles faced by the caregivers, the most common was the lack of social support services.

A position paper by EURORDIS outlined the importance of holistic person-centred care for patients with rare disease. People living with rare disease and their caregivers should have access to a social worker and to adequate social protection and inclusion. Malaysia is currently facing a shortage of capacity with a social worker-population ratio of 1:8,576.

Without adequate support, caregivers are likely to burn out quickly, affecting their ability to take charge of treatment regimes or show better medication compliance. The national framework for rare disease needs to include mechanisms for patients and caregivers to access respite care to better handle life stressors associated with rare disease. Alongside efforts to coordinate policies and programmes to build the general capacity of social workers in Malaysia, the Ministry of Women, Family and Community Development can also engage existing rare disease organisations to provide such care.

Apart from that, patients struggle with a lack of information and awareness regarding their disease. The lack of healthcare professionals working on rare disease and the lack of awareness among existing practitioners can cause delayed diagnosis. This, in turn, can cause long-term complications for patients and increased anxiety for caregivers. Capacity building for critical areas of expertise for rare disease, including clinical geneticists and genetic counsellors, within the public healthcare system is vital and requires financial support from the Ministry of Finance.

Given the right access to quality education, patients with rare disease are able to function as independent adults and contribute to society through formal employment. Appropriate adjustments to employment and education systems can improve inclusivity for individuals living with rare disease.

Encouraging steps were taken in this regard by the Ministry of Education earlier this year through the introduction of the Zero reject policy coupled with increased budgets to improve school infrastructure to be more disabled-friendly. Steps in this direction are encouraged and anticipated to allow children with rare disease to have equitable access to education.

Nevertheless, there is currently no policy or framework in place that supports their access and retention in the labour market beyond education. This should include mechanisms to ensure reasonable accommodation and national level legislation to guarantee no form of discrimination based on health or disability status. To design and implement these policies, there needs to be active engagement by the Ministry of Human Resources within the national governance committee.

Alongside access to timely and adequate healthcare, the national framework should advocate policies for better social care of people living with rare disease. The framework should implement specific mechanisms to ensure continued coordination between different ministries through a multidisciplinary approach, engaging health, social, work, education and research.

As outlined in this article, to create holistic policies and strategies for rare disease in Malaysia, all relevant ministries need to be involved. Inter-ministerial working groups for rare disease should be continuously supported within this national framework.

The Ministry of Health’s recent steps to improve prevention and treatment strategies are commendable. However, the sustainable well-being of those affected by rare disease needs to be tackled through a holistic inter-ministerial approach, and Malaysia’s first National Framework for Rare Disease has to reflect this.

Vaisnavi Mogan Rao is project coordinator, social policy unit, at the Institute for Democracy and Economic Affairs

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