Orphan Drugs

Orphan Drugs

Global Orphan Drugs Market to Reach US$959.0 Billion by 2030

The global market for Orphan Drugs estimated at US$361.7 Billion in the year 2023, is expected to reach US$959.0 Billion by 2030, growing at a CAGR of 14.9% over the analysis period 2023-2030. Biologics Drug Type, one of the segments analyzed in the report, is expected to record a 15.5% CAGR and reach US$612.3 Billion by the end of the analysis period. Growth in the Non-Biologics Drug Type segment is estimated at 14.0% CAGR over the analysis period.

The U.S. Market is Estimated at US$99.2 Billion While China is Forecast to Grow at 14.1% CAGR

The Orphan Drugs market in the U.S. is estimated at US$99.2 Billion in the year 2023. China, the world`s second largest economy, is forecast to reach a projected market size of US$145.5 Billion by the year 2030 trailing a CAGR of 14.1% over the analysis period 2023-2030. Among the other noteworthy geographic markets are Japan and Canada, each forecast to grow at a CAGR of 13.2% and 12.4% respectively over the analysis period. Within Europe, Germany is forecast to grow at approximately 10.7% CAGR.

Global Orphan Drugs Market - Key Trends and Drivers Summarized

Orphan drugs are vital in addressing unmet medical needs, specifically targeting rare diseases that traditional market incentives fail to cover due to high development costs and limited patient populations. These drugs are designed for conditions so rare that the potential revenue does not typically justify the enormous investment required, which can reach tens of millions and involve a decade-long journey from molecule discovery to market approval. Despite these challenges, orphan drugs play a critical role in public health, offering the only hope for treatment in many cases. These drugs may also be repurposed versions of existing treatments for common diseases, broadened to cover rarer indications, thus enhancing their therapeutic scope and utility.

The development and support for orphan drugs have been significantly shaped by legislative frameworks, notably the Orphan Drug Act introduced in the United States in 1983. This act, along with similar policies globally, provides various incentives such as tax credits, reduced fees, and extended market exclusivity to stimulate the development of treatments for rare diseases. These diseases affect a tiny fraction of the population, typically fewer than one person per 2000, representing a vast array of conditions that impact millions worldwide. The legislative support aims to make the development of orphan drugs economically viable and encourages pharmaceutical companies to invest in research areas they would likely avoid due to poor ROI prospects under normal circumstances.

Looking to the future, the realm of orphan drugs is set to expand with the growing integration of advanced technologies and shifting regulatory landscapes. Advances in genetic research and biotechnology have dramatically enhanced the identification and understanding of rare diseases at a molecular level, facilitating the development of targeted treatments. The movement towards personalized medicine continues to push the boundaries of precision therapies, tailored to individual genetic profiles, significantly improving patient outcomes. Furthermore, regulatory incentives and fast-track approval processes provided by entities such as the FDA and EMA are crucial in encouraging the development of orphan drugs. Collaborative research initiatives and innovative funding models are also pivotal, reducing the financial burden and fostering faster drug development. As awareness and advocacy grow, so does the support for addressing the unique challenges posed by rare diseases, thereby driving the demand for orphan drugs and underlining the importance of continued innovation and support in this crucial healthcare sector.

Select Competitors (Total 18 Featured) -

• AbbVie Inc.
• Actelion Pharmaceuticals Ltd.
• Alexion Pharmaceuticals Inc.
• Amgen Inc.
• AstraZeneca Plc.
• Bayer AG
• Biogen Inc.
• BioMarin Pharmaceutical Inc.
• Boehringer Ingelheim GmbH
• Bristol-Myers Squibb Company
• Celgene Corp.
• CEL-SCI Corporation
• Concordia International Corp.
• Eli Lilly and Company
• F. Hoffmann-La Roche Ltd.
• Genzyme Corp.,
• Johnson & Johnson
• Merck & Co. Inc.
• Merck Serono SA
• Novartis AG
• Pfizer Inc.
• Recordati S.p.A.
• Sanofi SA
• Shire Plc.
• Vertex Pharmaceuticals

Please note: Reports are sold as single-site single-user licenses. Electronic versions require 24-48 hours as each copy is customized to the client with digital controls and custom watermarks. The Publisher uses digital controls protecting against copying and printing is restricted to one full copy to be used at the same location.

The latest version of Adobe Acrobat Reader is required to view the report. Upon ordering an electronic version, the Publisher will provide a link to download the purchased report.

Prior to fulfillment of an order, the client will be required to sign a document detailing the purchase terms for a publication from this publisher.