Rare Diseases – Facing Challenges with Limited Access to Medication

By:Retlaw Matatu Matorwa

Zimbabwe, like many Sub-Saharan African countries has been grappling with the challenge of rare diseases. Rare diseases are classified as any disease that affects fewer than 5 in 10,000 people. According to the World Health Organization (WHO), there are over 7,000 rare diseases, and they are often chronic, progressive, and life-threatening. There are many challenges in diagnosing and managing rare diseases, with limited access to medication and lack of knowledge by medical professionals on how to manage them, being the main obstacles.

Zimbabwe has a high burden of rare diseases, with more than 400,000 people suffering from these illnesses. Some of the rare diseases affecting Zimbabweans include sickle cell anemia, albinism, and several forms of muscular dystrophy. Additionally, there are several inherited metabolic disorders such as cystic fibrosis and phenylketonuria that are common among Zimbabweans.

The prevalence of rare diseases varies across the country, with more cases being reported in urban areas than in rural communities. According to studies, sickle cell anemia affects around 4% of the population, with a higher incidence in the southern region compared to other parts of the country. Meanwhile, albinism affects approximately 1 in 1,400 people.

Apart from the lack of access to medication, patients living with rare diseases in Zimbabwe also face financial challenges due to the high costs of medication. Many of these medications are not available locally and have to be imported, making them prohibitively expensive for many patients.

While experts have acknowledged the need for increased investment in drug development targeting rare diseases, the pharmaceutical industry is yet to respond effectively to this challenge. According to the Global Genes Report 2019, less than 5% of rare diseases have approved drug treatments, partly due to the challenges of small patient populations and the limited financial incentives for pharmaceutical companies to invest in drug development. This leaves many people living with rare diseases with no option but to manage their symptoms with alternative therapies.

In addition to the lack of medication, rare diseases often go undiagnosed or are misdiagnosed due to limited awareness about these diseases among medical professionals. In Zimbabwe, many patients are often treated for other conditions with similar symptoms instead of the underlying rare disease. This delay in diagnosis leads to a decline in the patient’s health, further compounding the burden of rare diseases.

To address these challenges, there is a need for increased public awareness about rare diseases and their symptoms. The government needs to prioritize the production of medication for rare diseases, and the development of clinical guidelines for the management of these illnesses. Additionally, there needs to be increased investment in the training of healthcare professionals on the diagnosis and management of rare diseases.

Best practices in managing rare diseases can be learned from other countries. In Europe, for instance, the Orphan Drugs Regulation incentivizes pharmaceutical companies to research and produce medication for rare diseases. Similarly, the United States has the Orphan Drug Act, which provides incentives to encourage the development of drugs for rare diseases. Such measures can be adopted in Zimbabwe and other African countries.

Rare diseases are a significant public health concern in Zimbabwe, and the challenges that people living with these diseases face cannot be overlooked. Increased investment in drug development, awareness campaigns, and the training of healthcare professionals is required to address this issue effectively. The pharmaceutical industry can play a key role in this, but it will require concerted efforts by governments, healthcare providers, and other stakeholders to ensure that people living with rare diseases get the treatment and care they deserve.

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