•  
  •  
 

Ahmad Nader Fasseeh, Syreon Middle East, Alexandria, EgyptFollow
Baher Elezbawy, Syreon Middle East, Alexandria, Egypt
Nada Korra, Syreon Middle East, Alexandria, Egypt
Mary GamaI, Egyptian Authority for Unified Procurement, Medical Supply and Technology Management, Cairo, Egypt
Ahmad Seyam, Universal Health Insurance Authority, Cairo, Egypt
Noha Hayek, Theodor Bilharz Research Institute, Giza, Egypt
Nagwan Abdel Rahman, Health Insurance Organization, Cairo, Egypt
Sohir Abdelhamid, Egyptian Parliament Health Sector, Cairo, Egypt
Nader Fasseeh, Faculty of Medicine Alexandria University, Alexandria, Egypt
Amr Shafik, Faculty of Medicine Ain Shams University, Cairo, Egypt
Ahmed Elagamy, Egyptian Authority for Unified Procurement, Medical Supply and Technology Management, Cairo, Egypt
Akram Mahmoud, Health Insurance Organization, Cairo, Egypt
Amal Samir Sedrak, Egyptian Authority for Unified Procurement, Medical Supply and Technology Management, Cairo, Egypt
Khaled Elshazly, Presidential Program for Women's Health, Cairo, Egypt
Mariam Eldebeiky, Egyptian Authority for Unified Procurement, Medical Supply and Technology Management, Cairo, Egypt
Monica Talaat, Egyptian Drug Authority, Cairo, Egypt
Noura Maher Mohamed, Universal Health Insurance Authority, Cairo, Egypt
Rania Ahmed Abdelaziz, Universal Health Insurance Authority, Cairo, Egypt
Remonda Refaat, Egyptian Authority for Unified Procurement, Medical Supply and Technology Management, Cairo, Egypt
Shaimaa Akeel, Egyptian Authority for Unified Procurement, Medical Supply and Technology Management, Cairo, Egypt
Randa Adel, Takeda Pharmaceuticals, Cairo, Egypt
Samy Khalil, Takeda Pharmaceuticals, Cairo, Egypt
Sherif Abaza, Syreon Middle East, Alexandria, Egypt
Zoltán Kaló, Center for Health Technology Assessment, Semmelweis University, Budapest, Hungary

Subject Area

Pharmacology and Clinical Pharmacy

Abstract

Introduction High pricing of orphan drugs often restricts patient access. As orphan medicines are usually not cost-effective according to traditional thresholds, countries may choose not to consider the cost-effectiveness criterion in pricing and reimbursement decisions or may adopt higher thresholds for orphan drugs. The objective of this research was to develop a multi-criteria decision analysis (MCDA) framework to serve as a multiplier for determining a differential cost-effectiveness threshold (CET) for orphan drugs in Egypt. Methods A scoping review was conducted to identify criteria used for the evaluation of orphan drugs. This was followed by a policy workshop with the representation of the relevant governmental bodies to develop the MCDA tool by ranking and weighing the final list of criteria. The MCDA score is then converted to a CET multiplier for each orphan drug. Results A broad list of criteria was identified through the literature and was used as a starting point to create the tool. Based on participants’ voting, six criteria were included in the final MCDA tool: “disease severity” criterion was on top of the list with 38.6% weight, followed by “rarity” (24.2%), “budget impact” (16.1%), “robustness of the data” (10.2%), age of patients” (6.4%) and “societal impact” (4.6%). Conclusion Using multiple cost-effectiveness thresholds for common technologies and orphan drugs is increasingly applied in many countries based on different criteria. An MCDA tool was created to calculate the multiplier for orphan drugs for a higher threshold. The tool will improve the evidence, transparency and reproducibility of pricing and reimbursement decisions for orphan drugs.

Additional Files
Orphan designation supplement_V2.docx (19 kB)
Download

Share

COinS