Rights granted to patients with rare diseases in Türkiye
THE GENERAL Health Insurance (GSS) system provided by the Social Security Institution (SGK) has been implemented in Türkiye since 2012. The GSS system refers to the implementation of compulsory public health care insurance covering the whole population. The GSS is a much more comprehensive version of the system that the United States tried to create with the Affordable Care Act (also known as “Obamacare”), partly using Türkiye’s policy as a model during Barack Obama’s tenure as U.S. president. Considering that health and access to treatment is a human right, the GSS, which currently covers more than 99% of our citizens and foreign residents, makes quality health services accessible regardless of the income of individuals.
Although the GSS system establishes a modern health care insurance system similar to its global examples, its distinctive aspect is its wide scope. This is true both in terms of the population covered and the health care services provided.
The General Health Insurance (GSS) system provided by the Social Security Institution (SGK) has been implemented in Türkiye since 2012. The GSS system refers to the implementation of compulsory public health care insurance covering the whole population. The GSS is a much more comprehensive version of the system that the United States tried to create with the Affordable Care Act (also known as “Obamacare”), partly using Türkiye’s policy as a model during Barack Obama’s tenure as U.S. president. Considering that health and access to treatment is a human right, the GSS, which currently covers more than 99% of our citizens and foreign residents, makes quality health services accessible regardless of the income of individuals.
Although the GSS system establishes a modern health care insurance system similar to its global examples, its distinctive aspect is its wide scope. This is true both in terms of the population covered and the health care services provided. Although in many countries, chronic diseases are not covered within the public health care schemes or are covered conditionally with astronomical out-of-pocket payments, in Türkiye, cancer, diabetes, cardiovascular diseases, chronic kidney failure, stroke, nervous system diseases and many genetic and rare diseases are covered without paying a penny extra out of an individual’s pocket. The system also has a qualified infrastructure. Quite often, Europeans who visit Türkiye say, “Your country has already transitioned to a social state.” This is the truth, even if some social policy academics knowingly and willingly and, of course, unethically ignore the fact.
SMA: CHALLENGES, IMPLICATIONS
Spinal muscular atrophy (SMA), covered by Türkiye’s GSS system, is a genetic muscle disorder that affects mobility by targeting the motor nerve cells in the spinal cord. It poses significant challenges for both patients and caregivers, particularly in its most severe form, SMA Type 1, which manifests in childhood and leads to difficulties in swallowing, breathing and sitting without support. Due to severe motor nerve damage, individuals with SMA become reliant on lifelong care, with a life expectancy of only a few years.
Although this genetic disease, occurring in one out of every 10,000 people and considered rare, is difficult to treat, its risk is easily detected even before birth by having parents undergo genetic screening before marriage.
The public health care carrier also provides free treatment for SMA patients. Widely used medications to treat the disease are included in the list of medicines that can be brought from abroad by the Health Ministry and are delivered directly to patients free of charge.
Contrary to occasional misrepresentation to the public, it is important to clarify that all individuals with public health insurance in Türkiye can easily access SMA medication, which is normally quite expensive. The state provides free access to various examinations, treatments and medications for SMA patients and their families. Due to the chronic nature of the disease, patients are not required to pay any additional fees or co-payments, as Türkiye’s system does not collect out-of-pocket payments for any type of chronic disease.
EVOLVING HEALTH CARE COVERAGE
In fact, in the country’s old health care system in the early 2000s, even simple and basic health services such as eye treatment, dental treatment and dialysis were not covered, let alone rare and genetic diseases such as SMA. The patient, at the time, unavoidably would have to turn to the private sector. The Social Security Reform carried out in 2008 and the GSS system of 2012 expanded health insurance coverage to almost the whole population and offered one of the broadest coverages globally.
The most widely used drug in the world in the treatment of SMA, with the active ingredient Nusinersen Sodium, has been covered free-ofcharge by the SGK since July 5, 2017, for SMA Type 1 patients, and since Feb. 1, 2019, for SMA Type 2 and Type 3 patients, within the scope of the Health Practice Communique. Moreover, although a box of this medicine costs 73,000 euros (over $79,000) (previously 90,000 euros), this medicine must be used every month and annual treatment can reach millions of euros. This treatment is provided by SGK.
It is estimated that the amount paid by SGK within the scope of SMA medicine since 2017 has reached $3 billion. Around 1,500 patients in our country can benefit from this treatment. The medication used in the treatment of SMA is not covered even in many advanced health systems globally, yet Türkiye provides this very expensive medicine. Likewise, the effectiveness of new drugs, examinations and treatment methods for both SMA and all diseases continue to be followed and observed closely by the relevant bodies.
AN INCURABLE DISEASE
So, what is the problem regarding SMA? The problem is that neither the SMA drug available in Türkiye nor any drug discovered so far in the world can cure this disease. Unfortunately, with current modern medicine, it is not possible to expect a child with SMA to fully recover and reach adulthood. Only, the quality of life of patients can be increased by providing treatment for the symptoms.
Essentially, the drug containing the active ingredient Nusinersen Sodium only prevents the disease from progressing and turning into a worse condition; it does not cure the patient completely. However, two more drugs are in the trial phase globally. Yet, the Health Ministery and SGK argue that, although these drugs are approved by the American Food and Drug Administration (FDA) and the European Medicines Agency (EMA), the effectiveness of these drugs, which cost up to $2 million for a single course, in completely healing the patient has not yet been widely proven.
It should be noted that two types of studies are currently being conducted to find a definitive treatment for SMA. One of them is gene therapy. This therapy attempts to eliminate the root causes of the disease. The other form of treatment is cellular replacement therapy, where dead cells or cells that begin to die are replaced. Motor neurons are more suitable for regeneration in young people with SMA Type 1. However, it has not yet been determined effective in SMA disorders caused by spinal cord injury. Injecting stem cells into the spinal cord can be beneficial in babies with SMA Type 1. However, according to medical authorities, all these treatments are still regarded as experimental and palliative, rather than being game-changers.
Moreover, as the relevant authorities have stated many times, if the effectiveness in treating SMA is proven or if it is discovered that another drug can treat SMA in the world, the SGK is ready to cover it, regardless of the price. It was also seen during the COVID-19 pandemic that we can say with confidence that Türkiye has one of the most advanced social security and health systems in the world. Thus, pharmaceutical providers are required to prove the effectiveness of their novel medicines to both the SGK and the Health Ministry and show the potential to positively impact the lives of the patients and families devastated by rare and life-threatening neurological genetic diseases. Despite current discussions regarding the pharma reimbursement system of the country, price is not an issue for the Turkish public insurance system.
GENETIC SCREENING
What is the solution? Of course, the solution for a mother who has a child with SMA is to save her child. Let’s hope that modern medicine will soon develop a permanent solution. Rest assured, if such a treatment is developed, the Republic of Türkiye will be one of the first countries to provide it to its citizens.
However, what is more important is to prevent SMA, that is, to ensure that children are not born with the disease. Considering that an average of one in 50 individuals in society can be a carrier of the gene that causes SMA, genetic testing is key. The child can only have SMA if the defective gene is passed on from both parents. Faulty gene transfer only from the mother or only from the father does not cause disease in the child, but it can cause carrier status. It is possible to require genetic screening for every couple getting married and to detect genetic disease risks, especially SMA, from the beginning. This is the definitive solution.
Türkiye has added SMA to the mandatory screening tests before every new marriage since 2021. Since 2023, the Health Ministry has been implementing the SMA Carrier Screening Program throughout the country, focusing on detecting carriers. In summary, a preventive strategy has been adopted in the fight against the disease.
Türkiye’s inclusion of SMA treatment in its health care network symbolizes the system’s comprehensiveness
STRENGTH OF TURKISH SYSTEM
The fact that a rare and costly disease such as SMA is covered by the Turkish health system is a symbol of how comprehensive Türkiye’s social health system is. It demonstrates that the coverage of Türkiye’s health system competes with the developed world. The treatment of not only SMA, which we have exemplified here, but also diseases such as Amyotrophic Lateral Sclerosis (ALS), Duchenne Muscular Dystrophy (DMD), Multiple Sclerosis (MS) and other genetic and chronic diseases for which the definitive cure is unknown, are covered in Türkiye.
Likewise, in the Turkish public health care system, not only the drugs used in the treatment of SMA but also quite expensive drugs used in the treatment of chronic and rare diseases containing active ingredients such as Chenodeoxycholic Acid, Dupilumab, Asparaginase, Ponatinib, Alemtuzumab, Gemtuzumab, Ponatinib, Dinutuximab Beta, Elapegademase, Ataluren, Tafamidis and Vorinostat are also covered. The system has a very wide scope and when a drug is included in the scope of reimbursement, action is taken with concern for the health of the citizens rather than the concern about the expense. In sum, the Turkish health care system includes a system that offers the most advanced examinations and treatments for all citizens, offering a network in which the private and public sectors operate together within the rapidly growing pharmerging market.