Differences in clinical trials for orphan and non-orphan drugs in infectious diseases
A recent study examining drug approvals by the US Food and Drug Administration (FDA) between January 2010 and December 2020 highlights significant disparities between clinical trials leading to the approval of orphan and non-orphan drugs, particularly in the field of infectious diseases.
Out of the total 1122 drugs approved during the study period, 84 were specifically developed for the treatment of infectious diseases. Among these, 18 were classified as orphan drugs, while 66 fell under the non-orphan category.
The study revealed that 57% of the orphan drugs obtained approval in Phase 2 of clinical trials, while only 6% of non-orphan drugs received approval at this stage.
The study found that the clinical trials supporting the approval of orphan drugs had a median enrollment of 89 participants, significantly smaller than the median enrollment of 452 participants for non-orphan drugs. In addition, 37% of orphan drug trials employed blinding techniques, compared to 60% of non-orphan drug trials. Furthermore, randomization was utilized in 42% of orphan drug trials but in a much higher percentage, 87%, of non-orphan drug trials.
Kudyar P, Konwar M, Khatri Z, et al. Evaluation of clinical trials done for orphan drugs versus nonorphan drugs in infectious diseasesan eleven year analysis [2010-2020]. Perspect Clin Res. 2023;14(2):56-60. doi: 10.4103/picr.picr_137_21. Epub 2022 Jul 23. PMID: 37325577; PMCID: PMC10267988.