The Hospitalization of 'Ambassador of SMA Patients' in Iran; Authorities' 'Indifference' to Drug Supply Continues

Sina Ali Khani, a teenager suffering from Spinal Muscular Atrophy (SMA) in Iran, has been hospitalized due to a lack of medication, and the release of a video showing him in a hospital bed has once again drawn attention to the importance of providing drugs for rare diseases and the indifference of the Islamic Republic's officials to this issue. Sina Ali Khani earned the title 'Ambassador of SMA Patients' about two years ago after a video of him pleading for medication for SMA patients in front of the Iranian Parliament went viral. He informs his followers on Instagram about this disease and efforts to attract the attention of the country's officials to secure medication. Recently, he stated in one of his Instagram posts that after two years and three meetings with the Islamic Republic's President, he is still deprived of the necessary medication. A video of him in a hospital bed, along with his statements about securing funding for the medication needed by these patients during a protest two years ago, is circulating on social media. Morteza Ali Khani, Sina's father, told the IRNA news agency that the condition of some patients is worsening due to the lack of medication, leading to fatalities. In December 2021, when a group of SMA patients' families protested in front of the Iranian Parliament for several days, President Ebrahim Raisi promised to address their issues soon. Last week, a number of SMA patients protested again in front of the Presidential Office, complaining about the lack of medication that was being distributed in limited quantities due to insufficient budget and the 'indifference' of the Islamic Republic's officials. Senior officials of the Islamic Republic, especially President Ebrahim Raisi, have consistently promised to resolve healthcare crises, particularly the 'drug shortage,' promises that have not only gone unfulfilled but have reportedly worsened over time according to domestic sources. Meanwhile, the Islamic Republic government and official media in Iran have repeatedly attributed healthcare and treatment issues to sanctions. Contrary to this narrative, medications and medical supplies have not always been included in the sanctions imposed against Iran and have had 'exceptions' for procurement and import. The newspaper 'Shargh' reported on this protest, mentioning the Health Ministry's claim of 'localizing' medication in Iran. Saeed Azamian, the CEO of the SMA Patients Association, stated: 'The Health Ministry has no plan for providing medication for patients other than the vague and unclear rumor of localizing drugs.' Details about Spinal Muscular Atrophy (SMA): SMA is a rare neuromuscular disorder characterized by the progressive loss of motor neurons and loss of motor and respiratory function, often leading to early death. This disease is primarily observed in young children, with approximately 60% of those born with it experiencing a severe form. Many will not experience independent sitting or walking, and their physical condition will deteriorate rapidly, requiring 24-hour respiratory care and intravenous feeding. Treatments for this disease have gradually been provided since 2017, mainly in high-income countries, and can halt the loss of motor neurons. With early diagnosis and treatment, most patients can experience a better quality of life. Medications used to treat some types of SMA include 'Spinraza,' which targets the backup copy of the defective gene and is injected into the spine every few months; 'Evrysdi,' which targets the backup copy of the defective gene and is taken as a liquid once daily; and 'Zolgensma,' which provides a healthy copy of the gene responsible for the disease and requires a single injection. The issue of drug shortages and high treatment costs in Iran is not limited to SMA patients. Hamidreza Edraki reported that the number of rare disease patients in the country is about 1.5 to 2 million, with 6,500 patients registered so far. Experts consider SMA to be the most expensive rare disease in Iran. In addition to the specific medications for this disease, the use of painkillers and physical therapy to improve the quality of life for these patients has also been limited due to high costs. The cost of each physical therapy session for patients in Iran is reported to be between 500,000 to 700,000 tomans, which many families cannot afford, leading them to limit therapy sessions. Furthermore, due to mobility restrictions, patients must use electric wheelchairs, which are estimated to cost over 40 million tomans. The CEO of the Rare Diseases Foundation of Iran has reported an increase in the number of these diseases in the country, stating that the types of rare diseases have risen from 422 to 433. The limitations in drug supply and access in Iran are increasingly affecting a broader range of individuals, and in cases where the government claims to provide and import drugs, reports indicate that patients receive nothing but low-quality medications. The drug shortage has been a contentious issue since last year, and in May of this year, Voice of America reported on the complex dimensions and widespread nature of this shortage, interviewing some activists in this field in Iran. In this context, a pharmacist residing in Tehran told Voice of America that no foreign currency has been allocated for the procurement of raw materials for medications or medical equipment since last December. He emphasized that due to the lack of currency allocation, shortages have manifested in all pharmaceutical sectors, making it difficult to secure even basic medications. Another pharmacist from Tehran mentioned that currently vital medications, including the heart drug Amiodarone, Plavix for preventing blood clots typically used after a heart attack, and inhalers for asthma and bronchitis, are also unavailable. This report utilized sources such as the World Health Organization and the National Health Service of the UK.