Disease foci of pharmaceutical research and development as reflected in applications for International Nonproprietary Names, 1953-2022.

Objective: To evaluate trends in pharmaceutical research and development, and to correlate these trends with global medical need.

Methods: We obtained details of proposed pharmaceutical substances from 1953 to 2022 from the International Nonproprietary Names (INN) database. We used the DrugBank and Cortellis databases to obtain the INN included in approved medicines over the same period. To evaluate trends, we categorized INN into 12 therapeutic classes according to their stem classification, and compared these trends with actual global medical need by extracting the INN in medicines included in essential medicines lists.

Findings: Out of a total of 10 611 proposed INN within our 12 therapeutic groups, 2280 were included in approved or registered medicines. We observed a considerable decrease in the number of new INN for anti-infective and antiparasitic, central nervous system and cardiovascular system medicines over the study period. In contrast, the number of new substances in the fields of antineoplastic, immunomodulatory, blood and haemopoietic system, and cell and gene therapy medicines has been increasing. In terms of public health impact, only 17.3% (441/11 453) of all INN in approved medicines are included in the World Health Organization Model list of essential medicines, the highest proportion of which are anti-infective and antiparasitic medicines.

Conclusion: Despite a high demand from global health systems, medicine development for neglected tropical and other infectious diseases remains largely dependent on national policy, governmental and philanthropic funding, and partnerships. Better alignment of research and development strategy and investment in global medical needs is required.