Rare Diseases Treatment Market Size, Share, Growth, Trends | Report 2022-2030

According to Vision Research Reports, the rare diseases treatment market was surpassed at USD 119.71 billion in 2021 and is expected to hit around USD 335.99 billion by 2030, growing at a CAGR of 12.15% from 2022 to 2030

Report Highlights

• Cancer dominated the market in 2021 with a revenue share of over 25.06%.
• The injectable segment dominated the market with a revenue share of over 50.11% in 2021 and is expected to witness significant growth during the forecast period.
• Biologics dominated the market with a revenue share of over 55.21% in 2021.
• The specialty pharmacy segment dominated the market with a revenue share of over 70.15% in 2021.
• Hospital pharmacy is expected to be the fastest-growing segment during the assessment period.
• North America dominated the market in 2021 with a revenue share of over 60.02%.
• The Asia Pacific is expected to be the fastest-growing region during the forecast period.

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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.

Scope of The Report

Therapeutic Area Insights

Cancer dominated the market in 2021 with a revenue share of over 25.06%. This dominance can be attributed to the high prevalence and recurrence rate of rare cancer indications. According to the American Cancer Society, the estimated incidence of esophageal cancer, chronic myeloid leukemia, and anal cancer was found to be 19,260, 9,110, and 9,090 respectively in the U.S. in 2021. Based on the therapeutic area, the global market is segmented into cancer, neurological conditions, cardiovascular conditions, musculoskeletal conditions, hematologic disorders, infectious diseases, metabolic disorders, endocrine disorders, and others.

Musculoskeletal condition is expected to be the fastest-growing segment during the projection period due to the increasing prevalence of this condition and new product approvals for its treatment. For instance, in March 2020, NS Pharma’s VILTEPSO candidate was approved in Japan for the treatment of patients with Duchenne muscular dystrophy (DMD). It was granted the SAKIGAKE designation by the healthcare body of Japan. This approval is also expected to boost segment growth. Osteogenesis imperfecta, achondroplasia, and fibrous dysplasia are some of the rare musculoskeletal conditions found in patients.

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Route Of Administration Insights

The injectable segment dominated the market with a revenue share of over 50.11% in 2021 and is expected to witness significant growth during the forecast period. This can be attributed to the launch of novel injectables for the treatment of rare diseases. For instance, in February 2021, the U.S. FDA approved Sarepta Therapeutics’ AMONDYS 45 (casimersen injection) for the treatment of DMD. Based on route of administration, the global market is segmented into oral, injectable, and others.

Recently, in July 2022, Pfizer Inc. received approval for Xalkori (crizotinib) by the U.S. FDA for the treatment of pediatric and adult patients with ALK-positive inflammatory myofibroblastic tumor (IMT). The recommended dose in adult patients is 250 mg and is to be administrated orally twice daily until disease progression stops. This approval is also expected to fuel the oral segment growth.

Drug Type Insights

Biologics dominated the market with a revenue share of over 55.21% in 2021. The advancement in biotechnology and research techniques has facilitated the development of novel biologics. High target specificity and potential of biological drugs revolutionizing the treatments of several rare diseases are expected to fuel the segment growth. For instance, in 2019, the U.S. FDA approved Zolgensma, a gene therapy, for the treatment of spinal muscular atrophy. Based on drug type, the global market is segmented into biologics, biosimilars, and small molecules.

The emergence of biosimilars for the treatment of rare diseases proved to be a breakthrough for patients. The patent expiration of orphan biologics and supportive regulatory policies will pave the way for new players to enter the market and drive competition, leading to a reduction in drug price that is used for the treatment of various rare diseases, thereby fueling the segment growth. For instance, according to the Center for Biosimilars, approximately 11% of orphan biologics providers have biosimilar candidates in the product pipeline. However, high costs associated with R&D and a small consumer base may hamper the segment growth.

Distribution Channel Insights

The specialty pharmacy segment dominated the market with a revenue share of over 70.15% in 2021. The dominance can be attributed to strategic initiatives undertaken by key players, such as the acquisition of specialty pharmacies for the distribution of their products. For instance, in December 2020, Centene acquired PANTHERx, the largest specialty pharmacy in the U.S., which engages in distributing high-cost orphan drugs that are used for the treatment of various types of rare diseases. Centene serves as an intermediary for government and privately sponsored health insurance programs. Such acquisitions are expected to boost segment growth during the forecast period. Based on distribution channels, the global market is further segmented into hospital pharmacy, specialty pharmacy, and online pharmacy.

Hospital pharmacy is expected to be the fastest-growing segment during the assessment period. This can be attributed to the high hospitalization rate of SMA. The majority of patients are treated in hospitals due to the high risk of respiratory issues and scoliosis. According to the Orphanet Journal of Rare Diseases (2020), the inpatient ratio was the highest in pediatric patients aged 0 to 4 years.

Regional Insights

North America dominated the market in 2021 with a revenue share of over 60.02% due to the high burden of diseases, favorable healthcare infrastructure, and new product approvals for treatment. In October 2021, AstraZeneca received orphan drug designation for Tezepelumab from the U.S. FDA for the treatment of eosinophilic esophagitis (EoE). The accessibility to products may increase patient compliance, consequently expanding the consumer base and increasing the revenue for the market.

The Asia Pacific is expected to be the fastest-growing region during the forecast period. The growth of the region can be attributed to the initiatives undertaken by governments to support orphan disease patients. For instance, in July 2022, the Indian government directed national and state governments to ensure the effective implantation of health policies developed for the treatment of patients suffering from orphan diseases. This measure creates an opportunity for manufacturers to supply high-quality orphan medicines to the government and generate revenue.

Key Players

• F. Hoffmann-La Roche Ltd.
• Pfizer, Inc.
• PTC Therapeutics
• AstraZeneca
• Novartis AG
• Takeda Pharmaceutical Company
• Bayer AG
• AbbVie Inc.
• Merck & Co. Inc.
• Bristol Myers Squibb

Market Segmentation

• By Therapeutic Area Outlook
  • Cancer
  • Neurological Conditions
  • Cardiovascular Conditions
  • Musculoskeletal Conditions
  • Hematologic Disorders
  • Infectious Diseases
  • Metabolic disorders
  • Endocrine disorders
  • Others
• By Route of Administration Outlook
  • Oral
  • Injectable
  • Others
• By Drug Type Outlook
  • Biologics
  • Biosimilar
  • Small Molecule
• By Distribution Channel Outlook
  • Hospital Pharmacy
  • Specialty Pharmacy
  • Online Pharmacy
• By Regional Outlook
  • North America

• • Europe
  • Asia Pacific
  • Latin America
  • Middle East and Africa (MEA)

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