OBJECTIVES: Compare the different evaluation processes and assess their impact on patient access to orphan drugs

METHODS: An extensive review of literature was carried out to collect information on the evaluation models of different orphan drugs in the four countries. The literature search was carried out on the official websites of the agencies and organizations responsible for the regulation of medicines in each country. The analysis was undertaken through several aspects including: the existence of specific legislation for the evaluation of orphan drugs, pricing, reimbursement, and application of health technology assessment (HTA). Finally, a semi-structured interview was conducted at the National Agency for Pharmaceutical Products in order to explore the evaluation process of orphan drugs in Algeria

RESULTS: Among the countries included in our study, three of them have national regulations for orphan drugs except Turkey. Although none of them has an evaluation system dedicated to orphan drugs, specific mechanisms allowing access to these treatments are applied at different levels of the drug's life cycle. The four countries use HTA in the assessment process. In Algeria, there are also no specific procedures for the evaluation of orphan drugs. Abbreviated procedures for the documentary evaluation [scientific expertise documentation relating to the regulatory situation, to product quality, safety and efficacy] during the registration of these drugs have been introduced recently but absence of pharmacoeconomic evaluation procedure or specific incentive measures is an obstacle to the access of Algerian patients to these treatments

CONCLUSIONS: Countries around the world are constantly reviewing their evaluation systems in order to optimize access to medicines, particularly orphan drugs. Algeria started to put in place specific measures applicable to the registration process for orphans like the “accelerated documentary evaluation” which accelerate drugs which could be strengthened with a more comprehensive drug evaluation process by integrating an HTA-based approach