Nadir Hastalıklarda Tedaviye Erişim: Mevcut Durum, Engeller ve Çözüm Önerileri

Yıl 2025, Cilt: 15 Sayı: 3, 509 - 514, 24.09.2025

Münire Yıldırım , Hülya Keskin

https://doi.org/10.33631/sabd.1507046

Öz

Nadir hastalıkların çoğunda bilimsel ve tıbbi literatürdeki bilgi eksikliği nedeniyle mevcut tedavi seçenekleri yetersiz kalmaktadır. Bu derleme makalesi, nadir hastalıklarda tedaviye erişim konusundaki mevcut durumu, karşılaşılan engelleri ve çözüm önerilerini inceleyerek, sağlık politikası yapıcıları, sağlık profesyonelleri ve ilgili paydaşlar için kaynak sunmayı amaçlamaktadır. Mevcut durum incelendiğinde, nadir hastalıklarla ilgili tanı ve tedavi süreçlerinin uzun ve karmaşık olduğu görülmektedir. Bu süreçler, hastaların doğru tanıya ulaşması ve etkili tedavi alması açısından önemli zorluklar doğurabilir. Ayrıca, nadir hastalıklarda bilimsel ve tıbbi literatürdeki bilgi eksikliği, mevcut tedavi seçeneklerinin sınırlı olmasına yol açarak tedaviye erişimi daha da zorlaştırmaktadır. Engeller arasında, yüksek maliyetli ilaçlar, klinik denemelerin azlığı ve sağlık sistemlerindeki yapısal zorluklar bulunmaktadır. Çözüm önerileri kapsamında ise, nadir hastalıkların tanı ve tedavi süreçlerinin iyileştirilmesi, araştırma ve geliştirme faaliyetlerinin desteklenmesi, sağlık politikalarının güçlendirilmesi ve uluslararası iş birliğinin artırılması gibi stratejiler ön plana çıkmaktadır. Sonuç olarak, nadir hastalıklarda tedaviye erişimdeki mevcut durumun anlaşılması ve çözüm odaklı yaklaşımların benimsenmesi, sağlık politikası yapıcıları, sağlık profesyonelleri ve ilgili paydaşlar için önemli bir gerekliliktir.

Anahtar Kelimeler

nadir hastalık , tedaviye erişim , eşitsizlik

Access to Treatment for Rare Diseases: Current Situation, Barriers and Solutions

Öz

For most rare diseases, current treatment options are inadequate due to the lack of information in the scientific and medical literature. This review article aims to provide a resource for health policymakers, healthcare professionals and relevant stakeholders by analysing the current situation, barriers, and solutions for access to rare disease treatment. This study is a review of the existing literature on the current situation in access to treatment for rare diseases, barriers, and solutions, and presents a comprehensive assessment of the current situation in access to treatment for rare diseases. These processes can pose significant challenges for patients to reach the correct diagnosis, and receive effective treatment. Furthermore, the lack of information in the scientific and medical literature on rare diseases leads to limited treatment options available, making access to treatment even more difficult. Barriers include high-cost medicines, lack of clinical trials, and structural challenges in health systems. As for solutions, strategies such as improving the diagnosis, and treatment of rare diseases, supporting research, and development activities, strengthening health policies, and increasing international cooperation come to the fore: In conclusion, understanding the current situation in access to treatment for rare diseases and adopting solution-oriented approaches is an important requirement for health policymakers, health professionals, and relevant stakeholders.

Anahtar Kelimeler

rare disease , access to treatment , inequality

Kaynakça

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