Chaired by Health Minister Dr. Fahrettin Koca, the SMA Science Board came together at Bilkent Campus. A written statement was made by Minister Koca after the meeting where the results of SMA scans, the treatment processes of the patients, and the latest developments in treatment methods were discussed.
Reminding that SMA is a hereditary, progressive, chronic, neurological disease, Dr. Koca said, “Until 2016, we were losing nearly 90 percent of babies with Type-1 form of the disease before they reached the age of 2. After the drug with the active ingredient 'nusinersen' started to be used around the world in 2016, an opportunity arose to keep these babies alive. Immediately after this development, nusinersen treatment was started to be given free of charge to all our patients in our country, which can set an example in many countries around the world. Currently, 1024 of our patients receive this treatment free of charge.
Emphasizing that over time, scientific data show the necessity of applying this treatment as early as possible for it to be more effective, Dr. Koca pointed out that to achieve this, SMA newborn screenings were started across Turkey in May 2022.
Minister Koca added, “In this context, 753 thousand 350 babies have been screened for SMA so far. We delivered their treatment as soon as possible, and the survival rate in the first 6 months was 100 percent in our babies who were diagnosed in the newborn screening program and whose loading dose of nusinersen treatment was completed.”
“Our country is one of the few countries that can do the premarital screening program”
Stating that the SMA Science Board has also meticulously followed the developments in the last 5 years regarding the prevention of the disease, and that within this framework, the premarital screening program has been implemented, Dr. Koca futher added that “genetic counseling and selective pregnancy practice that will enable them to have healthy babies are provided free of charge to couples who are found to be carriers of SMA in this screening. Our country is one of the few countries in the world that can do this.”
The final stage has been reached for the licensing of the SMA drug in solution form
Minister Koca emphasized that the Science Board also closely monitors the scientific developments regarding other drugs developed for use in SMA.
“In this context, together with nusinersen, all data on the drug with the active ingredient 'risdiplam' and the drugs named Zolgensma are also monitored. All three of these therapies are gene-based therapies. No superiority was demonstrated for any of the three treatments.
Of these, the legal procedures required for the drug with risdiplam active ingredient to enter our country have been completed by the relevant company and an application has been made. The scientific data regarding this drug, which will also allow our patients to be administered orally in the form of a solution, have been evaluated and the final stage has been reached for the licensing of the relevant drug. In today's meeting held by our science board, all the details of our health system, from how the medicine is prepared and how it is delivered to our patients, were meticulously evaluated. We will have finalized the license process for this drug in the coming days. It was decided to offer this drug as an option for patients to whom nursinersen treatment is difficult to apply. As the country data increases, the application principles will be reviewed by the Science Board.”
Thus, Koca emphasized that two drugs will now be included in the treatment guide for SMA patients.
“We will not allow the hope of our families to be used for commercial purposes”
Minister Koca stated that they aim to maintain the health service and care based on realistic expectations for SMA patients and their families at the highest level by following standard care rules, and made the following assessment:
“We would especially like to point out that we have not allowed the abuse of hope until now, and we will not allow it in the future. We will not allow the hope of our families to be misused for commercial purposes. We have previously declared that we will not allow our children to be used as subjects. We want it to be known that we maintain this position. However, we are ready to provide the necessary convenience for any treatment whose effectiveness has been proven with scientific evidence.”
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