[Research Report] The rare disease treatment market size was US$ 193.52 million in 2022 and is expected to reach US$ 496.06 million by 2031; it is estimated to register a CAGR of 11.70% from 2022 to 2031.

Insights and Analyst View:

Around 7000 rare diseases affect more than 30 million people in the United States, which are life-threatening. Treatment such as drug, biologic, and device development in rare diseases is challenging, such as complex biology and the lack of understanding of the natural history of many rare diseases-the increasing prevalence of rare disease disorders and an inherently small population of patients with a rare disease. The US FDA has approved hundreds of drugs for rare diseases, but most rare diseases do not have FDA-approved treatments.

Growth Drivers and Restraints:

Increasing Prevalence of Rare Diseases

According to the United States Food and Drug Administration, a rare disease affects less than 200,000 people in the United States and according to the Cheisi Global Rare Disease article in 2022, the burden of rare diseases is approximately 10x higher than mass market diseases per patient per year (PPPY) basis. Additionally, the European Commission Public Health states that the disease affected no more than one person in 2000 in the European Union. Rare disease also affects less than five persons in 10,000 in Canada. Similarly, as per the February 2022 update of rarediseaseday.org, about 300 million people are living with rare diseases, which is a high figure. Thus, the demand for effective and advanced treatment solutions for rare diseases is anticipated to increase due to the high burden and growing awareness. This is expected to fuel growth in the market studied over the forecast period. Increasing demand for rare disease treatment has spiked owing to various distribution channels being used for rare disease treatment, such as online pharmacies, specialty pharmacies, and hospital pharmacies.

Additionally, factors such as the increase in the number of rare disease cases and initiatives favoring vaccinations, coupled with novel therapeutics and drugs, and an increase in the number of new drug launches and favorable government policies are boosting the overall market growth.

Rising Number of Drug Approvals to Drive Rare Disease Treatment Market Growth

According to Pharmaceutical Research and Manufacturers of America, in 2021, around 791 potential orphan rare disease drug candidates were under clinical trials. Among all clinical orphan drugs, 168 were for rare cancers, 120 for rare blood cancers, 56 for neurological disorders, 192 for genetic disorders, 54 for blood disorders, 51 for autoimmune diseases, and 36 for infectious diseases. Around 26 novel orphan drugs were approved for the treatment of the rare disease by the Center for Drug Evaluation and Research (CDER) in 2021. Some of the approved products were Amondys 4, Bylvay, Welireg, Lumakras, Scemblix, Welireg, Cytalux, Besremi, Empaveli, Evkeeza, Exkivity, fexinidazole, and Zynlonta, among others. Therefore, increasing rare disease drug approval and the launch of new orphan drugs are anticipated to boost industry growth.

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Rare Disease Treatment Market: Strategic Insights

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Report Segmentation and Scope:

The rare disease treatment market, by method, is segmented into routes of administration (ROA), i.e., oral, injectables, and others. The injectables segment dominated the market in 2022. The market is also segmented by drug type, i.e., biologics, small molecules, and biosimilars. The biologics segment held the largest market share in 2022. The segment is anticipated to register the highest CAGR from 2022 to 2031 as the segment has gained popularity based on its efficacy as compared to other products. The rising advancement in biotechnology and research and development has facilitated the development of novel biologics. The rare disease treatment market, by therapeutic area, is segmented into oncology, neurological conditions, cardiovascular conditions, musculoskeletal conditions, infectious diseases, and others. The oncology segment held the largest market share in 2022 and is anticipated to register the highest CAGR during 2022-2031. The dominance of this segment can be attributed to the high prevalence and recurrence rate of rare cancer indications. The rare disease treatment market, by distribution channel, is segmented into hospital pharmacies, online pharmacies, and specialty pharmacies. The specialty pharmacy segment held the largest market share in 2022; however, online pharmacy is anticipated to register the highest CAGR during 2022-2031. An increasing number of hospital pharmacies globally and the advent of online pharmacies due to their ease of usage is boosting the segment growth.

The rare disease treatment market, based on geography, is segmented into North America (the US, Canada, and Mexico), Europe (Germany, France, Italy, the UK, Russia, and the Rest of Europe), Asia Pacific (Australia, China, Japan, India, South Korea, and the Rest of Asia Pacific), Middle East & Africa (South Africa, Saudi Arabia, the UAE, and the Rest of Middle East & Africa), and South and Central America (Brazil, Mexico, and the Rest of SCAM).

Segmental Analysis:

The rare disease treatment market, by drug type, is segmented into biologics, small molecules, and biosimilars. The biologics segment held the largest market share in 2022. The segment is anticipated to register the highest CAGR from 2022 to 2031. The increasing advancement in biotechnological companies has facilitated the development of novel biologics: high target specificity and potential of biological drugs transforming the rare disease treatment market. For instance, in 2021, the U.S. FDA approved Zolgensma, which is a gene therapy used for the treatment of spinal muscular atrophy. Additionally, the increasing number of patent expiration of orphan biologics is anticipated to pave the way for new companies, leading to a reduction in drug price that is used for the treatment of various rare diseases, thereby propelling the overall segment growth.

The rare disease treatment market, by route of administration (ROA), is segmented into oral, injectable, and others. The injectable segment held the largest market share in 2022 and is anticipated to register the highest CAGR during 2022-2031. This can be attributed to the rising number of companies involved in the new 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 Duchenne muscular dystrophy (DMD). The oral segment followed the injectable segment. Major juggernauts in the market are also involved in new product innovation. For instance, in July 2022, Pfizer Inc. received U.S. FDA approval for Xalkori (crizotinib) for the treatment of pediatric and adult patients with ALK-positive inflammatory myofibroblastic tumor (IMT) drug is provided orally with a dose of 250 mg until disease progression stops.

Regional Analysis:

Based on geography, the global Rare Disease Treatment market is segmented into five key regions: North America, Europe, Asia Pacific, South & Central America, and Middle East & Africa. In 2022, North America held the largest share of the global rare disease treatment market size. Asia Pacific is estimated to register the highest CAGR from 2022 to 2031. North America's Rare Disease Treatment market has experienced substantial growth in the past few years due to the rising population of the obese population undergoing rare disease treatment and the prevalence of rare diseases in North America and advancements in rare disease treatment. North America accounted for the largest share of the global rare disease treatment market in 2022. The North American rare disease treatment market has experienced substantial growth in the past few years due to the rising burden of rare diseases. Asia Pacific accounted for the fastest-growing region of the global rare disease treatment market. The growth is attributed to several factors, driven by the high potential of rare disease treatment in untapped and emerging markets due to the availability of increase in unmet healthcare needs, increasing prevalence of rare diseases, and demand for rare disease treatment.

Furthermore, emerging economies in the rare disease market are developing at a significant rate due to a rise in demand for enhanced healthcare services, significant investments in improving healthcare infrastructure, and the development of innovations in the healthcare industry. The demand for rare disease treatment is not only limited to emerging countries but is also observed in developing countries. Factors such as the contribution of companies in the rare disease treatment market further drive the growth of the rare disease treatment market. For instance, In October 2021, AstraZeneca received orphan drug designation for Tezepelumab from the U.S. FDA for the treatment of eosinophilic esophagitis (EoE). The products may increase patient compliance, leading to expanding the consumer base and increasing the revenue for the market.

Industry Developments and Future Opportunities:

Various initiatives taken by key players operating in the global rare disease treatment market are listed below:

1. In August 2021, Zevra Therapeutics purchased Acer to catapult itself into a commercial rare disease ecosystem for USD 91 million.
2. In 2021, Orphan Technologies acquired American biotech company Retrophin Inc. for $90 million. This arrangement includes OT-58, a brand-new enzyme replacement treatment. The company is undergoing Phase 1/2 trials to treat classic homocystinuria.
3. In October 2021, UCB and Ra Pharmaceuticals Inc. entered into a merger agreement pursuant to which UCB acquired Ra Pharma to improve treatment options for people living with myasthenia gravis and other rare diseases.
4. In July 2023, Alexion, AstraZeneca Rare Disease entered a definitive purchase and license agreement for a portfolio of preclinical gene therapy programs and enabling technologies from Pfizer Inc. The agreement was carried out to advance next-generation genomic medicines with the addition of complementary pipeline assets and innovative technologies. The acquisition was worth USD 13.3 billion and also strengthened the company’s rare disease product portfolio by offering a high growth opportunity for the market.

Competitive Landscape and Key Companies:

F. Hoffmann-La Roche Ltd., Pfizer, Inc., PTC Therapeutics, Novartis AG, Takeda Pharmaceutical Company, Bayer AG, AbbVie Inc., Merck & Co. Inc., and Bristol Myers Squibb are provided in this report. There are some important players in the market, such as F-Hoffmann La Roche Ltd., Novartis AG, and Bayer AG, which are a few key companies operating in the rare disease treatment market. These companies adopt product innovation strategies in the rare disease treatment market to meet evolving customer demands, which allows them to maintain their brand name.