Rare Disease Treatment Market Segmentation:
Drug Type Segment Analysis
The biologics segment is expected to account for the largest share of 53.8% in the market during the analyzed period. The leadership is primarily attributed to the growing popularity of precision medicine in cases of hard-to-treat conditions. Besides, as a part of this category, biosimilars are becoming eligible candidates for government-led programs promoting affordability, making them widely accepted and commercialized assets for this sector. Moreover, the segment’s reputation in being the gold standard for compliance can be exemplified by the FDA’s approval of Bkemv and Soliris as interchangeable biosimilars for rare conditions in May 2024.
Diseases Segment Analysis
The hematologic disorder segment in the market is poised to hold the highest revenue share of 45.4% between 2025 and 2037. High prevalence and mortality rates of these ailments, such as sickle cell, hemophilia, and paroxysmal nocturnal hemoglobinuria, particularly among children, are the major growth factors behind the dominance. According to a Global Burden of Disease (GBD) study, the number of total births of babies and people living with sickle cell disease increased by 13·7% and 41·4% in the world from 2000 to 2021. On the other hand, the clinically proven efficacy of gene and cell-based therapies attracts greater capital allocation to the development of novel drugs for this medical discipline.
Patient Segment Analysis
The pediatric age group is predicted to be the leading patient segment in the market by the end of 2037, while acquiring a share of 67.9%. As per a 2023 publication from the Journal of Pediatric Nursing, 50-75% of all RDs occur among children. Besides, another NLM finding from the same year revealed that age-specific deaths from sickle cell disease accounted for 81,100 among children younger than 5 years from 2000 to 2021, which was higher in comparison to cause-specific mortality. These figures highlight the reason behind the sector’s major activity being concentrated in this segment, solidifying its forefront position in the sector.
Our in-depth analysis of the market includes the following segments:
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