According to the Market Statsville Group (MSG), the global ultra rare disease market size is expected to grow from USD 134.19 million in 2022 to USD 476.04 million by 2033, growing at a CAGR of 12.20% from 2023 to 2033. The presence of a strong product pipeline and the imminent launches are expected to fuel market growth. A multidisciplinary approach with innovative proposals for the development of novel SMA drugs to fulfill the unmet needs in rare disease treatment and unhealthy lifestyle is projected to fuel the market growth over the forecasting period. In addition, around 26 novel orphan drugs were approved for the treatment of rare diseases by the Center for Drug Evaluation and Research (CDER). However, the market expansion may be hindered by the high costs related with the research and development of pipeline candidates used for the treatment of rare illnesses, which adds to increase in medication price in all around the world.
A disease is classified as ultra-rare if it affects one patient per 50,000 individuals (or fewer than 20 patients per million of population), while the majority of ultra-rare diseases impact significantly less than one per million or less. Despite the small number of individuals affected, ultra-rare diseases have a significant impact on patients, families, and their society since many are severe, chronic, and progressive. Individuals suffering from severe and life-threatening ultra-rare illnesses are frequently without hope since there are no viable therapeutic alternatives. The European definition of a ultra rare disease is one that affects fewer than five people per 10,000 of the population (or, approximately 500 patients per million of population).
While the epidemic has caused supply chain disruptions and decreased access to healthcare services in all the locations around the world. The pandemic has restricted availability to healthcare services in some locations, notably in rural areas where medical facilities may be overburdened or unavailable as a result of the epidemic. This can make it more difficult for individuals to receive early treatment for ultra-rare diseases, increasing the risk of complications and death. Several patients with ultra rare diseases were forced to postpone their therapeutic treatments due to the COVID-19-induced economic disruptions and lockdowns, indicating a negative impact on the ultra rare disease treatment sector outlook. Besides these setbacks, significant advances in medical technology, medication approvals, and patent expirations have increased the availability of both generic and branded pharmaceuticals in recent years. Furthermore, the increasing usage of telemedicine services in the post-pandemic period will encourage the rise of ultra rare disease treatment service providers in the near future.
More than 7000 uncommon disorders have been recognised in the European Union (EU), affecting 30-40 million individuals, and around 250 new ultra rare diseases are diagnosed each year. Arguably, in the past few years interest in ultra rare diseases has grown, as demonstrated by the agendas of politicians and health authorities. Most of the European countries and American countries has seen a up serge in these diseases due to unhealthy lifestyle, lack of nutrition, genetics problems and many other reasons. Due to the increase in the number count of patient of ultra rare disease has boosted the market growth over the forecasting period.
Ultra rare diseases have few or no therapies available. While therapies are available, they are typically prohibitively costly, ranging from $100,000 to more than $2 million per patient each year. In 2019, more than half (56%) of all high-priced medications available in Canada, for rare illnesses cost more than $200,000 per patient per year. The small market size, high research and development costs, and limited treatment options made these treatments expensive and unaffordable for middle class people. Moreover, the chance of these diseases is very small. less than 5 in 10,000 of the general population and an ultra-rare disease affects less than one patient per 50,000 of the population. These reasons have hindered market growth over the forecasting period around worldwide.
Increasing government initiative and investing huge investment towards orphan drug will create opportunities for new and existing market player to dominate the market over the forecast period. For instance, European law was adopted to encourage the development of orphan medications far later than the United States Orphan Drug Act of 1983. This legislation requires that the pharmaceutical industry has a right to obtain protocol assistance at a reduced rate, access the centralised authorisation procedure, enjoy lower registration fees; and benefit from 10?years of market exclusivity after registration. This act was established in 1983, however the government is focusing on this act because of the increasing number of patients. This has led to the authorisation by the European Medicines Agency of 124 new orphan drugs in the EU between 2000 and 2015, of which about one-third were for ultra-rare diseases.
The study categorizes the ultra rare disease market based on type and level of disease area at the regional and global levels.
Based on the type, the market is divided into lymphangiomatosis, musculoskeletal conditions, pleuro-parenchymal fibroelasytosis, pulmonary alveolar microlithiasis, ataxia telangiectasia, pulmonary alveolar proteinosis, lysosomal storage diseases, pulmonary dendriform ossification and others. The musculoskeletal conditions segment is expected to dominate the market share in 2022 in the global ultra rare disease market due to the rising incidence of this ailment and new pharmaceutical approvals for this treatment has boosted the segment growth over the forecasting period. For instance, NS Pharma's VILTEPSO candidate was authorized in Japan in March 2020 for the treatment of Duchenne Muscular Dystrophy Patients (DMD). It was granted the SAKIGAKE designation by the healthcare body of Japan. This permission should also help the category flourish. Some of the uncommon musculoskeletal diseases identified in patients include osteogenesis imperfecta, achondroplasia, and fibrous dysplasia.
Based on the regions, the global ultra rare disease market has been segmented across Europe, North America, the Middle East & Africa, Asia-Pacific, and South America. North America is projected to account for the highest market share in 2022 due to the high diseases load, attractive healthcare infrastructure, and new pharmaceutical approvals for therapy of ultra rare disease. For instance, Tezepelumab got orphan drug designation from the US FDA in October 2021 for the treatment of eosinophilic esophagitis (EoE). Access to products may promote patient compliance, hence growing the consumer base and increasing market income. Moreover, The growth of the region can be attributed to the initiatives undertaken by governments to support orphan disease patients. These reasons has boosted the market growth in this region.
The ultra rare disease market is a significant competitor, and extremely cutthroat in the sector are using strategies including product launches, partnerships, acquisitions, agreements, and growth to enhance their market positions. Most sector businesses focus on increasing their operations worldwide and cultivating long-lasting partnerships.
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