Market Trends of Rare Disease Treatment Industry
This section covers the major market trends shaping the Rare Disease Treatment Market according to our research experts:
The Biologics Segment is Expected to Hold a Major Market Share in the Rare Disease Treatment Market
Biologics are the drugs that target specific parts of the immune system to treat disease. Biologics are proteins, antibodies, peptides, and some vaccines that are usually injected or infused because they cannot be absorbed orally.
According to the Regulatory Affairs Professionals Society (RAPS), 26 of 50 drugs (52%) in 2021 were approved as orphan drugs for rare diseases. In previous years, the proportion of orphan drugs approved has been inconsistent, with FDA identifying 31 of 53 drugs (58.4%) in 2020 as having been approved by the Center for Drug Evaluation and Research (CDER) as orphan drugs.
An article published in the MDPI Journal of Biologics in August 2021 studied the main biological treatments currently available for inflammatory bowel disease treatment due to the extremely important current role of biologics. The study showed that various biologic therapies, such as anti-TNF-α therapy, anti-integrin therapy, anti-cytokine therapy, and other new biologics, are widely used to treat rare inflammatory bowel disease. The study indicated that further studies are needed to better understand the action mechanism of the biological drugs, which will help understand how to improve the efficacy and safety of the treatment methods.
The strategic initiatives adopted by key players, such as product launches and approvals, partnerships, and acquisitions, are also fueling the segment's growth. For instance, in November 2022, CanSino Biologics Inc. entered into a strategic collaboration agreement with AstraZeneca to improve Chinese patients' access to the diagnosis, prevention, and treatment of rare diseases. The collaboration helps AstraZeneca develop the biopharmaceutical ecosystem and diagnose and treat rare diseases. Such collaborations among market players regarding innovations in biologics are expected to propel the segment's growth during the forecast period.
North America is Expected to Hold a Significant Share in the Rare Disease Treatment Market
The North American region is expected to occupy a major share in the rare disease treatment market owing to the high prevalence of rare diseases in the region, such as Huntington's disease, spina bifida, fragile X syndrome, Guillain-Barré syndrome, Crohn's disease, cystic fibrosis, and Duchenne muscular dystrophy, high awareness about these diseases, and the presence of a robust healthcare system for the diagnosis and treatment of rare diseases.
According to the Genetic and Rare Diseases (GARD) Information Center in May 2022, 1 in 10 Americans (or 30 million people) have a rare disease, and there are approximately 7,000 known rare diseases. In addition, according to the Canadian Organization for Rare Disorders (CORD) Report 2021, every year, about 1 in 12 people in Canada are affected by rare diseases, and about 80% of these diseases are caused due to genetic changes. Hence, owing to the high burden of rare diseases in the region, the market is expected to have significant growth over the forecast period.
Furthermore, in September 2022, the Critical Path for Rare Neurodegenerative Diseases (CP-RND), a public-private partnership aimed at fostering the development of treatments for amyotrophic lateral sclerosis (ALS) and other rare neurodegenerative diseases, was launched by the US Food and Drug Administration and the National Institutes of Health (NIH). Additionally, in October 2022, Takeda Canada Inc. released its report that highlighted the need to develop a single, pan-Canadian definition of a rare disease to help accelerate and support the launch of Canada's Rare Disease strategy. With this, Canada is taking a meaningful step ahead in developing a national strategy for rare diseases, which is expected to increase people's awareness about rare disease treatment and contribute to regional market growth.
