Challenges of Living with Cystic Fibrosis in the Middle East, North Africa, and Central Asia

Orphan drug legislation in the USA and Europe has led to the development and rapid approval of new treatments for rare diseases, including Cystic Fibrosis (CF). However, the high costs of these effective treatments make obtaining them a major challenge for CF patients, especially when coupled with the challenge’s manufacturers perceive to distribute in developing countries.

In Africa, the Middle East, and Central Asia, drugs are mostly financed, procured, and distributed by health ministries. The funds spent on drugs are constrained by government health budgets, and these issues lead to insufficient incentives for the distributors to enter their markets. Despite some countries having sizable populations with CF (Turkey and Iran have an estimated >3000 CF patients, each), CF treatment is simply not a priority in developing nations where healthcare budgets are struggling to meet the basic needs of the masses.

This inequity has led to drastic disparities in outcomes. Due to advancements and improvements in the standard of care, including the availability and access to disease-modifying treatments, the average life expectancy of a patient with CF in the USA is 45 years. Many CF patients in the West live well beyond life expectancy and there is now research on the needs of geriatric patients. In MENA countries without access to these essential medicines, including a lack of capacity for basic symptom management that diminishes quality of life, CF patients are not expected to reach the age of 12 years.

The costs of CF drugs in the US and Europe are high and many MECFA member countries are unable to afford the multiple essential medicines patients need to sustain healthy lives. Pharmaceutical companies have been historically unwilling to invest in registering and lobbying for their drugs to be made available due to perceived low or non-confirmatory patient counts, unstable health ministries and other issues including political instability. Where drugs are available, relatively small patient populations make it harder for ministries to negotiate for lower prices. Drug distribution companies in the Middle East are not controlled by the same rules that apply to US or European distributors; increased differential pricing and disparities in prescribing essential medicines are also key factors that impact organizations like the Middle East Cystic Fibrosis Association (MECFA)’s ability to successfully convince health ministers to provide essential CF therapies.

MECFA is well-positioned to lead a coordinated effort to expand access to CF care and essential drugs in the Middle East & North Africa (MENA), and Central Asia. As MECFA continues to invest in developing CF Centers and spreading diagnostics, we must also continue to ensure CF patients have access to basic care and essential therapies.

Currently, there are multiple countries in the region that have small numbers of CF patients and little to no resources available for patient care. Health Ministries in the MENA and Central Asia regions do not consider CF in annual budgets and consequently do not support patient care beyond treatment for lung infections or other CF related problems that cause a patient to be hospitalized. This situation leads to increased early morbidity and suffering for patients. MECFA has been working to increase CF clinical care in the region through a series of educational offerings in collaboration with the international CF community and in particular, the CF Foundation. In addition, MECFA has placed diagnostic equipment in several hospitals in the region and is providing CF diagnostic education and advocacy support to physicians and patients.