محاضرة حول تطوير الأدوية للأمراض النادرة

استضافت كلية الصيدلة يوم الخميس الموافق ٢٠١٦/١٢/١، الدكتور بلال أبو العسل من مؤسسة الغذاء والدواء الأمريكية  (FDA)، والذي ألقى محاضرة بعنوان:

Clinical Drug Development for Rare Diseases 

واستعرض الدكتور أبو العسل التحديات التي تواجه الصناعات الدوائية لتطوير أدوية لعلاج الأمراض العصبية النادرة، وأثر تفعيل Orphan Drug Act ، في تشجيع وتعزيز تطوير أدوية جديدة للأمراض النادرة. كما تطرق الدكتور أبو العسل لأنواع جديدة من تصاميم الدراسات السريرية والتي تم تطويرها للتغلب على مشاكل تحليل نتائج الدراسات السريرية المحدودة من حيث عدد المرضى المشاركين فيها.

جاءت هذه المحاضرة ضمن الفعاليات التي تقوم بها اللجنة العلمية والثقافية في كلية الصيدلة، وقد حضر المحاضرة عدد من أعضاء الهيئة التدريسية والطلبة من كلية الصيدلة في جامعة العلوم والتكنولوجيا الأردنية.

للاطلاع لى ملخص المحاضرة:

Drug development for rare neurological diseases can be very challenging due to small patient populations available for clinical trial, poor understanding of the natural history of the disease and reduce investments due to low profitability of these products. The implementation of the Orphan Drug Act in 1983 stimulated the development of orphan drug products; however, it remains essential that the FDA recognizes the need for flexible regulatory policies for establishing drug safety and efficacy given the consideration of the inherent limitation in rare diseases. 

The goal of this project is to screen the approved orphan drug products in neurology and characterize the contents of the clinical pharmacology packages submitted to the drug applications. In addition, it was intended to identify the characteristics of the efficacy studies that supported the approval of these drug products and identify instances where FDA provided flexibility in reviewing these applications.

About 20% of neurological products approved through 2015 have orphan indications and more than 50% of these products were granted a priority review. About 53% of these products had other non-orphan indication and the majority of these products were developed to treat seizures and movement disorders with limited number of drugs developed for other neurological disorders such as head and spinal trauma. 34% of neurological products were approved based on a single, well-controlled efficacy trial. Most of the orphan applications used parallel designs and tested only one dose or used titrate to effect. No drugs approved based on surrogate end-point or with accelerated approval in neurology. It is also noted that the standard requirements for conducting clinical pharmacology studies for orphan drug products are similar to non-orphan drug products; however, there are flexibility with the time to conduct the clinical pharmacology studies.

The implementation of innovative trial designs and flexible methods of analysis for small clinical trials can improve the drug development and approval for rare disease. In addition, clinical pharmacology can enhance the quantity and quality of data needed to satisfy the regulatory standards for approval by improving the understanding of biomarkers, dose-response and pharmacokinetic-pharmacodynamic (PK-PD) relationships.