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Rare Diseases Treatment Market Size, Share & Trends Analysis Report By Therapeutic Area (Cancer, Musculoskeletal Conditions), By Route Of Administration, By Drug Type, By Distribution Channel, By Region, And Segment Forecasts, 2025 - 2030
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Rare Diseases Treatment Market Growth & Trends:

The global rare disease treatment market size is estimated to reach USD 374.39 billion by 2030, expanding at a CAGR of 11.6% from 2025 to 2030, according to a new report by Grand View Research, Inc. Increasing prevalence of rare diseases contributes to industry growth. According to the National Center for Advancing Translational Sciences (NCATS), over 30 million of the U.S. population is estimated to be affected by rare diseases in 2022. Increasing prevalence of people living with orphan diseases is anticipated to increase the sales and demand for orphan medicines over the forecast period.

The presence of supportive regulations by government authorities, such as the Orphan Drug Act that support product development by offering the orphan drug designation to potential drug candidates developed by pharmaceutical companies, is expected to support industry growth. This initiative boosts the research and development in the field of orphan disease treatment.

In April 2020, the National Organization for Rare Disorders (NORD) launched the "COVID-19 critical relief" program for patients with rare diseases affected by the COVID-19 pandemic. Under this program, the NORD provided monetary assistance of up to USD 1,000 annually to orphan disease patients and their caregivers for fulfilling their medical and non-medical needs. Thus, the availability of such programs is anticipated to boost the adoption of rare disease treatment over the forecast period.

Moreover, companies have undertaken initiatives such as collaborations and partnerships for the development, manufacturing, and commercialization of products in the international market. For instance, in August 2020, Sarepta Therapeutics, Inc. entered into a collaboration with the University of Florida for the development of novel genetic medicines for the treatment of patients with various orphan diseases, including Duchenne Muscular Dystrophy (DMD).

However, a lack of accurate and early diagnosis of rare diseases can often present a challenge for orphan disease patients, and in some cases, an accurate diagnosis is not obtained for as long as five years. In addition, patient eligibility and recruitment of eligible patients for clinical trials for rare disease treatment may restrain the industry growth. Not only finding the target population is difficult but finding physicians who treat these patients is also complex, thus leading to delays in the new product approval and launch in the market.

Rare Diseases Treatment Market Report Highlights:

Table of Contents

Chapter 1. Methodology and Scope

Chapter 2. Executive Summary

Chapter 3. Rare Disease Treatment Market Variables, Trends & Scope

Chapter 4. Rare Disease Treatment Market: Therapeutic Area Estimates & Trend Analysis

Chapter 5. Rare Disease Treatment Market: Drug Estimates & Trend Analysis

Chapter 6. Rare Disease Treatment Market: Route of Administration Estimates & Trend Analysis

Chapter 7. Rare Disease Treatment Market: Distribution Channel Estimates & Trend Analysis

Chapter 8. Rare Disease Treatment Market: Regional Estimates & Trend Analysis

Chapter 9. Competitive Landscape

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