Rare Disease Treatment Market Analysis

Therapeutic Area (Cancer, Blood-related disorders, Central nervous system, Respiratory disorders)

Based on the therapeutic area, the cancer segment is expected to register a considerable share in 2037 in the rare disease treatment market. A wide number of cancers are classified as rare owing to their low incidence rates and specific genetic mutations. Significant research investments, targeted treatments, immunotherapies, and an urgent need for effective solutions in rare cancers drive growth in this segment. Additionally, regulatory incentives and patient advocacy have spurred pharmaceutical companies to prioritize rare cancer treatments, further reinforcing cancer’s leading position in the rare disease therapeutic area. For instance, in April 2024, the President of India launched CAR-T cell therapy, India’s first home-grown gene therapy, aiming to fight cancer.

Route of Administration (Oral, Injectable)

Based on the route of administration, the oral segment is expected to capture around 55.7% rare disease treatment market share by the end of 2037. The growth is majorly attributed to their ease of use, convenience, and patient-friendly profile, especially for chronic conditions that require long-term management. Patients and healthcare providers often favor oral medications over injectables as they are easier to self-administer and typically involve fewer hospital visits. Recent advancements in oral drug delivery technology have also enabled more effective treatment of complex rare diseases. For instance, in September 2024, the U.S. FDA announced the approval of Miplyffa (arimoclomol), an oral medication for Niemann-Pick disease, type C.

Our in-depth analysis of the global market includes the following segments: