It was stated that there are not enough clinical trials related to the effectiveness and long-term side effects of the drug used in gene therapy, which has come to the agenda in Spinal Muscular Atrophy (SMA), a fatal disease.
Prof. Dr. Tayfun Uzbay, Head of the Department of Internal Medical Sciences at Üsküdar Üniversitesi Faculty of Medicine and Pharmacology Specialist, stated in a declaration to an AA reporter that SMA is a genetic muscle disease.
Uzbay stated that especially Type 1 appears from birth and largely results in death within two years, emphasizing that types with later-onset symptoms have a longer lifespan with appropriate care and some supportive treatments.
Uzbay pointed out that while the incidence of the disease is one in ten thousand worldwide, this rate is one in six thousand in Turkey, stating that the high rate of consanguineous marriages contributes to this.
"Data related to the drug is quite limited"
Uzbay, drawing attention to the extremely limited and expensive drug treatment options for SMA, said, "There is a biotechnological drug available in Turkey, and its reimbursement by the Ministry of Health is also possible. The price of this drug is around 70 thousand euros."
Uzbay provided the following information regarding the treatment:
"A newer drug, approved by the American Food and Drug Administration (FDA) and the European Medicines Agency (EMA), is not yet available in Turkey. Recently, a new drug based on comprehensive gene technology received approval from the FDA for use in children up to 2 years of age and was launched onto the market.
The drug works by replacing the missing or mutated gene called SMN1. The Ministry of Health has not yet approved these two drugs. However, by applying to the Ministry of Health, if deemed appropriate, it can be provided as an imported drug without reimbursement. Although the drug is administered as a single dose, its price is over 2 million dollars."
Uzbay stated that families are looking for a way out because SMA is a disease with a poor prognosis, and made the following evaluations:
"It is normal for families to pay attention to a drug reported to be very effective with a single dose and want to use it, but some points should not be overlooked. Data related to the drug is quite limited. As of now, there are only 58 articles published in reliable scientific sources, and more than half of these include reviews and comments on drug-related research articles.
Neither are there sufficient clinical trials concerning the drug's effectiveness and long-term side effects, nor has a long period passed since its use. In other words, time is needed to speak definitively about efficacy and safety. Furthermore, the drug is approved for children up to 2 years of age. Its application at other ages may either not be possible or may be subject to special ethical permission. If approval was given up to two years of age, it is highly probable that the drug would be ineffective in delayed applications.
The Ministry of Health wants to point out that a new-generation drug whose effectiveness has not yet been proven, and whose clear efficacy, long-term benefits, or harms have not been established, could be risky."
Underlining that it has been witnessed in recent days that vaccines are being questioned extensively by society, Uzbay said, "People do not even trust old-type vaccines, fearing long-term side effects or harms. This drug, however, is a directly gene-targeted drug. It is still uncertain whether it will cause a different genetic problem in these children in the long term."
"The drug should be a product offered with a humanitarian approach focused entirely on public health"
Prof. Dr. Uzbay evaluated the Ministry of Health's statement regarding children with SMA, saying, "We are clearly against our children being used as guinea pigs," as follows:
"There is a new gene therapy approach here. A small amount of positive data gives rise to hope from this. This needs to be amplified. But if it is to be amplified, families must be informed about its risks. We cannot present evidence with a few positive data points. The US and EU have approved this drug for use in children under 2 years of age. It is necessary to start this drug early. Its use in older children may be much riskier and unnecessary. Such an application could be unethical or require a very special examination, responsibility, and specific conditions. Unnecessary use can lead to abuse. This is probably what the Ministry wants to convey."
Uzbay stated that while the risk of the drug causing long-term harm to children should not be overlooked, the benefit-risk ratio should also be considered.
Uzbay pointed out that why the company markets the drug at such a high price is another matter for debate, and continued:
"Drugs and products and technologies aimed at human health should not be marketed using methods similar to textiles. Health is the most fundamental right that should be under state protection and guarantee.
Of course, a drug is a product with commercial value, but its marketing should take place with different and very special ethical rules. Otherwise, health would become a right accessible only to those with money. To prevent this, the state should not leave the research, development, and marketing of particularly vital health products entirely to the discretion of the private sector. The drug should be a product offered with a humanitarian approach focused entirely on public health."
Source: Anadolu Agency