TEHRAN –There are no restrictions on the supply of medicine for rare-disease patients, but some foreign companies prevent the sale of medicine to Iran because of sanctions causing temporary shortages of the needed drugs, an official with the Food and Drug Administration has said.

FDA makes every effort to make sure patients have access to needed drugs including importing medicines for those who are allergic to domestic drugs, IRNA quoted Heidar Mohammadi as saying on Tuesday.

He made the remarks on the occasion of the World Thalassemia Day on May 8.

Most of the drugs for rare-disease patients are domestically manufactured. Sometimes shortages refer to foreign medicines unavailability. Naturally, the FDA supports locally manufactured drugs whose efficiency have been proven, Mohammadi added.

If domestic drugs are found to be inefficient, foreign sample options, in case of being proven effective, will be imported, he said.

Regarding the medicine supply for thalassemia patients, he said,  “With the help of the Thalassemia Association and oncology specialists, domestic drugs are mainly used to meet the needs of patients.”

However, Mohammadi added that due to the different needs of patients and the effectiveness of drugs on them, a part of the needed medicine is imported.

With the help of the Central Bank, the amount of imported drugs for thalassemia patients increased significantly last year (ended on March 19), compared to a year earlier. This year, we aim to continue providing the necessary medicine for these patients with the  Central Bank’s assistance.

Health insurance and rare disease funds cover part of the costs to help patients get medications without financial concerns.

Currently, there are about 19,000 patients with thalassemia in the country, with 15,500 diagnosed with thalassemia major, 2,800 with thalassemia intermedia, and 1,000 suffering from sickle cell disease and other hemoglobinopathy disorders.

Thalassaemia is a genetic blood disorder that hinders the body’s ability to produce hemoglobin, resulting in varying degrees of anemia, fatigue, and related complications. The severity of the condition varies depending on the type of thalassemia, making expert care crucial for effective treatment and management.

442 rare diseases identified in Iran

 A total of 442 rare diseases have been identified in the country according to a recent report by the Rare Disease Foundation.

The incidence of rare diseases is very low to the extent that maybe one or two people with a certain rare disease are present in the country. Some rare diseases such as ‘butterfly skin or EB’, ‘Spinal muscular atrophy (SMA)’, ‘Metabolic diseases’, ‘Autism’, and ‘Dystrophies' are among the most common cases in the country. A sum of 50 trillion rials (about $100 million) was allocated in the Iranian calendar year that ended on March 20, 2023, IRNA reported, adding that the figure rose to 70 trillion rials the next year.

Considering the number of patients identified, the needed medicines, and medical tools, a sum of 150 to 180 thousand trillion (about $300 million) is expected to be allocated for the current Iranian calendar year, beginning on March 21.

MT/MG