Facilitating Access to Medicines in Low- and Middle-Income Countries: Role of EMA?

Background: As part of its contribution to promoting global health and to ensure that public health needs are met as a priority, the European Medicines Agency (EMA) can assess medicines for use outside the European Union (EU) and issue scientific opinions built on collaboration with the World Health Organization (WHO) and national regulatory authorities (NRAs) of non-EU countries likely to use the medicines (EU-M4all). Ten positive scientific opinions have been adopted by the EMA Committee for Medicinal Products for Human Use (CHMP) under this procedure. To date there has not been any measure of the public health impact of this procedure. We have therefore investigated the impact by looking at national approvals relying on these opinions. Method: We included all procedures with a positive scientific opinion (n=10), contacted the sponsors of the medicines (n=8 for the ten opinions), and obtained the lists of countries where approval had been granted based on these opinions. The number of regulatory approvals was then analysed by region, country of approval and therapeutic area. Findings: A total of 138 regulatory approvals have been granted in 90 countries worldwide. Of those, 82 countries are classified as Low or Middle-Income Countries. In particular, 64 approvals have been granted by NRAs in Africa, with the remainder in Latin and South America, Middle East and South-East Asia, and non-EU Europe and Central Asia. Interpretation: While the number of EU-M4all opinions is limited, these have had an impact and contribute to availability of innovative medicines for patients in target countries in many parts of the world. The scientific opinions follow the same review process as for marketing authorisations at EMA, with the same exacting standards. The main difference and value is that the medicine's benefit-risk is evaluated in the conditions of use in the target countries. According to the principle of regulatory reliance, while the benefit-risk assessment is a shared exercise, the approval decision remains with each of the target countries. Funding Statement: The authors state: "No external sources of funding." Declaration of Interests: The authors declare no competing interests. Ethics Approval Statement: The authors declare: "No ethical approval was required."