Inter-Relational Dynamics of Factors Affecting the Emergence of Orphan Drugs

Ann Pharm Fr. 2024 Dec 13:S0003-4509(24)00176-7. doi: 10.1016/j.pharma.2024.12.005. Online ahead of print.

Abstract

Orphan drugs are medications that are produced for the treatment of rare diseases. As there are less number of patients, the drug manufacturing companies are not keen in producing these drugs. Due to high costs of research and development and low profitability, companies do not want to invest in manufacturing of orphan drugs. Several laws have been passed by Governments of different nations to encourage the development of orphan drugs and make it available to patients. This study explores the interrelation dynamics of factors that has resulted in the greater availability of orphan drugs in recent times. 10 factors: internet technology, legislation, online patient support groups, government subsidiary, biotechnological advancements, corporate social responsibility, awareness and diagnosis of rare diseases and exclusive budgeting by pharmaceutical industries for orphan drugs related research and development and production were taken for the study. With a sample size of 38 experts, the technique of decision-making trial and evaluation laboratory (DEMATEL) was used for the study. It was found that information technology, legislation, support groups, and budget were the causes and the factors awareness, diagnosis, medicine availability, subsidiary, CSR and Biotechnology emerged to be the effect.

Keywords: DEMATEL; Legislation; Orphan Drugs; Policy; Subsidiary; Support Groups.