CEO of Iran Dystrophy Association: About 10 Children Under Ten Died in One Month Due to Drug Shortages

With the ongoing drug shortage crisis in Iran, especially for individuals suffering from rare diseases, the CEO of the Iran Dystrophy Association stated that contrary to health officials' promises, no Iranian medication has been produced and no vital drugs for SMA patients have been imported. Ramak Heydari told the Faraz website that in the past month, about 10 children under the age of ten died solely due to the discontinuation of their medications. The CEO of the Iran Dystrophy Association also mentioned that Younes Panahi, the deputy for research at the Ministry of Health, sent a 'very confidential' letter to the presidential office, claiming 'without any scientific backing' that the necessary medications were ineffective. Ramak Heydari stated that this claim was made while 'none of the doctors' accept it, and families say the medications had improved their patients' conditions. He added that despite the Ministry of Health emphasizing the production of Iranian medication for this disease, no company has even proposed to manufacture the drug to this ministry. The CEO of the Iran Dystrophy Association considered the mention of producing an Iranian version of the drug as 'an excuse to cut off medications.' On the 8th of Esfand, which is designated as 'Rare Diseases Day,' a group of SMA patients and their families gathered for several consecutive days in the cold and snowy weather of Tehran, demanding 'medication supply' in front of the presidential office building. The Ministry of Health and Medical Education of the Islamic Republic had previously claimed that 'there is not enough budget to provide medication for these patients' in response to these protests and the arising issues. The CEO of the Dystrophy Association stated: 'Considering the embezzlement of the tea fund, we could have supplied medications for all SMA patients for about 40 years.' In Azar, the hospitalization of Sina Alikhani, a teenager suffering from SMA in Iran due to lack of medication and the release of a video of him on a hospital bed once again drew attention to the importance of providing medication for special diseases and the indifference of Islamic Republic officials to this issue. The CEO of the Iran Rare Diseases Foundation stated that the price of each injection for SMA patients is about 10 million tomans. Islamic Republic officials also focused on domestic vaccine and drug production during the COVID-19 pandemic, banning Western vaccines and drugs that proved effective in multiple global studies by order of Ali Khamenei. Meanwhile, health officials of the Islamic Republic made significant efforts to justify domestic vaccines instead of relying on scientific evidence, resorting to what doctors call 'pseudoscience.' The issue of drug shortages in Iran, especially for rare diseases, has long been a topic in the media, but it continues and is met with officials' indifference. The CEO of the Iran Rare Diseases Foundation recently announced a need for 15 to 18 trillion tomans to cover the costs of rare and 'difficult-to-treat' diseases and stated that 442 new rare diseases have been identified in the country. Official statistics show that the total number of patients with these diseases is recorded at 6,500, while the chairman of the board of the Iran Rare Diseases Foundation estimates the number of patients to be between 2 to 3 million. Meanwhile, the fund for difficult-to-treat and special diseases of the Health Insurance Organization currently covers only 107 rare diseases. Yaser Davoudian, chairman of the board of the Rare Diseases Foundation, told Hamshahri Online that the lack of support has caused many rare patients to become isolated due to economic problems, and the rates of suicide, divorce, and organ selling among these patients are on the rise. SMA, spinal muscular atrophy, is a rare neuromuscular disorder characterized by the progressive loss of motor neurons and loss of motor and respiratory function, usually leading to early death. This disease is primarily observed in young children, and nearly 60% of children born with this disease experience an acute phase of it. Many will not experience independent sitting or walking, and their physical condition will deteriorate rapidly to the point where they will require 24-hour respiratory care and intravenous feeding. Treatments for this disease, which have gradually been provided since 2017, mainly in high-income countries, halt the loss of motor neurons. With early diagnosis and treatment initiation, most patients can experience a better quality of life. Medications used to treat some types of SMA include Spinraza, a drug that targets the backup of the defective gene and is injected into the spine every few months; Evrysdi, a drug that targets the backup of the defective gene and is taken as a liquid once a day; and Zolgensma, a drug that provides a healthy copy of the gene responsible for this disease and requires a single injection. This report utilized sources such as data from the World Health Organization and the National Health Service of the UK, as well as discussions reflected in the Iranian media.