OBJECTIVES: Orphan drugs are medicines designed for the prevention, diagnosis and treatment of rare diseases. Due to the low prevalence of these conditions, the lack of financial viability and the high cost of developing their treatments, patients have difficulty gaining access to them. The aim of this work is to compare access to orphan drugs in Algeria and six other countries: France, Belgium, UK, Turkey, Saudi Arabia and Tunisia.

METHODS: We analyzed regulations and policies regarding orphan drugs in our sample countries, then we conducted a comparison of the registration, the reimbursement and the marketing status of EMA-approved orphan drugs from January 2013 to December 2022 at country level. Data were gathered through a literature review, a semi-structured questionnaire and interviews with local experts from each country, and the use of official health authority websites and database from a pharmaceutical company. The data, percentages and timeframes obtained were compared.

RESULTS: Countries with specific orphan drug policies and regulations and high national revenues had percentages of 40% to 100% of drugs registered and/or reimbursed, with timelines of 3 months to 2 years. In contrast, emerging countries with no specific orphan drug policy had registration and reimbursement rates ranging from 0% to 27%, and delays up to 5 years. Marketing rates and periods fluctuate considerably between countries, ranging from 0% to 79%, with an average delay of 1 year after registration.

CONCLUSIONS: disparities in income levels, identified policy gaps in designation, national plans, regulations, incentives encouraging MO development as well as price and reimbursement regulation need to be addressed to improve access to available orphan drugs. Consensus on well-defined criteria and an assessment adapted to the complexity of rare diseases without omitting alternative financing models, should also be put in place to ensure optimal and sustainable access for patients to these drugs.