Rare Diseases Treatment Global Market Report 2024

The global rare diseases treatment market has grown rapidly in recent years. It will grow from $169.56 billion in 2023 to $192.22 billion in 2024 at a compound annual growth rate (CAGR) of 13.4%. During the historic period, several factors contributed to the growth observed in the rare disease treatment landscape. These include the introduction of orphan drug legislation, which incentivizes the development of medications for rare diseases, fostering increased disease awareness among healthcare professionals and the general population. Furthermore, advancements in biotechnological innovations, such as gene therapies and novel treatment approaches, significantly impacted the landscape. Global health initiatives and a heightened focus on personalized medicine, tailoring treatments to individual patients, also played pivotal roles in fostering growth within the rare disease treatment sector.

The global rare diseases treatment market is expected to see rapid growth in the next few years. It will grow to $312.46 billion in 2028 at a compound annual growth rate (CAGR) of 12.9%. In the forecasted period, the anticipated growth in rare disease treatment can be attributed to several key factors. Firstly, the emergence of innovative therapeutic modalities, including advancements in gene therapy, is expected to significantly impact the landscape. Additionally, evolving healthcare reimbursement policies that better accommodate rare disease treatments are likely to facilitate market growth. The integration of artificial intelligence and data analytics into healthcare practices will further contribute to advancements in treatment approaches. Expansion of patient registries for rare diseases and regulatory support aimed at expediting approvals for these treatments will also play significant roles in driving growth.

The rare disease treatment market is witnessing substantial growth due to the escalating burden posed by these conditions. Unlike more prevalent diseases, rare diseases affect a significantly smaller population. This surge in rare disease incidences and related fatalities is fostering a pressing need for effective treatments. As evidenced by a report from the Orphanet Journal of Rare Diseases, the United States alone accounted for about 15.5 million children with at least one rare disease. Such escalating prevalence is a key driving force behind the advancement of the rare disease treatment market.

Anticipated growth in the rare disease treatment market stems from an increasing demand for personalized medicine. This approach tailors medical interventions based on an individual's unique genetic composition, offering more precise and efficient healthcare solutions. In the realm of rare disease treatment, personalized medicine utilizes genetic, molecular, and clinical profiles to customize interventions, leading to more targeted therapeutic approaches. In 2022, the Food and Drug Administration’s Center for Drug Evaluation and Research (CDER) approved 37 new molecular entities (NMEs), with 34% - 12 of them - classified as personalized medicines by the Personalized Medicine Coalition (PMC). This surge in demand underscores the pivotal role personalized medicine plays in propelling the rare disease treatment market forward.

A prominent trend shaping the rare disease treatment sector revolves around technological advancements. Key market players are incorporating artificial intelligence (AI) into rare disease treatments to innovate and solidify their market positions. For instance, in August 2021, Healx, an AI-driven innovation company based in the UK, introduced HealNet - an AI-powered platform for drug discovery in rare diseases. Leveraging machine learning and AI technology, HealNet streamlines and accelerates the drug discovery process by aggregating data from diverse sources, forming a comprehensive biomedical knowledge graph analyzed using cutting-edge AI methodologies.

Companies operating within the rare disease treatment sphere are intensifying their focus on launching rare disease initiatives to gain competitive advantages. These initiatives encompass coordinated efforts undertaken by various stakeholders - governments, research institutions, and pharmaceutical companies - to address the distinct challenges associated with rare diseases. Notably, in October 2023, the Oxford-Harrington Rare Disease Centre in the UK initiated the Therapeutics Accelerator - a transatlantic endeavor aimed at advancing innovative treatments for rare diseases. With a vision to introduce 40 potentially transformative therapies into clinical trials over the next decade, this non-profit/for-profit model-driven Accelerator plans to allocate funding of up to £200 million. The objective is to support new projects targeting regulatory approvals in key markets such as the United States, the United Kingdom, and Europe.

In July 2021, AstraZeneca, a renowned UK-based pharmaceuticals and biotechnology firm, completed the acquisition of Alexion Pharmaceuticals for a total of $13.3 billion. This strategic acquisition was aimed at bolstering AstraZeneca's footprint within the field of immunology while simultaneously amplifying Alexion's groundbreaking research endeavors focused on drugs for rare diseases. Notably, Alexion Pharmaceuticals, a US-based pharmaceutical entity, has been at the forefront of pioneering research dedicated to developing treatments and medications tailored specifically for individuals affected by rare diseases.

Major companies operating in the rare diseases treatment market report are AbbVie Inc., Actelion Pharmaceuticals Ltd., Alexion Pharmaceuticals Inc., Celgene Corporation, Bayer AG, Johnson & Johnson, Novartis AG, Pfizer Inc., Sanofi S.A., Eisai Co. Ltd., Merck & Co. Inc., Vertex Pharmaceuticals, Takeda Pharmaceutical Company, Eli Lilly and Company, AstraZeneca PLC, Abeona Therapeutics Inc., Akcea Therapeutics Inc., Alnylam Pharmaceuticals Inc., Amicus Therapeutics Inc., BioMarin Pharmaceutical Inc., bluebird bio Inc., Catalyst Pharmaceuticals Inc., Cerecor Inc., Chiesi Farmaceutici S.p.A., CSL Behring Ltd., Dicerna Pharmaceuticals Inc., Diplomat Pharmacy Inc., Esperion Therapeutics Inc., Genzyme Corporation, Horizon Therapeutics plc., Intercept Pharmaceuticals Inc., Ionis Pharmaceuticals Inc.

North America was the largest region in the rare disease treatment market in 2023. The regions covered in the rare diseases treatment market report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East, Africa. The countries covered in the rare diseases treatment market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.

Rare disease treatments encompass two main categories of drugs such as originator drugs (patented molecules) and generic drugs, falling under biologics and organic compounds. Originator drugs are tailored for rare diseases lacking prior treatment options. As the initial versions of a drug patented by the originating company, these treatments tend to be relatively expensive. These treatments for rare diseases are administered through various modes including injectable, oral, and other means. They find applications across diverse medical areas such as cancer, neurological conditions, cardiovascular issues, musculoskeletal disorders, hematologic conditions, infectious diseases, metabolic and endocrine disorders, among other therapeutic fields. The distribution channels for these treatments involve hospital pharmacies, specialty pharmacies, and retail pharmacies, ensuring accessibility to patients in need.

This report provides rare disease treatment market statistics, including rare disease treatment industry global market size, regional shares, competitors with a rare disease treatment market share, detailed rare disease treatment market segments, market trends and opportunities, and any further data you may need to thrive in the rare disease treatment industry. This rare disease treatment market research report delivers a complete perspective of everything you need, with an in-depth analysis of the current and future scenario of the industry.

The rare disease treatment market includes revenues earned by entities by hematology, genetic diseases, neurological diseases and oncology. The market value includes the value of related goods sold by the service provider or included within the service offering. Only goods and services traded between entities or sold to end consumers are included.

The market value is defined as the revenues that enterprises gain from the sale of goods and/or services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD, unless otherwise specified).

The revenues for a specified geography are consumption values that are revenues generated by organizations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the supply chain or as part of other products.


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