"Designing Growth Strategies is in our DNA"
The U.S. orphan drugs market size was worth USD 77.82 billion in 2022 and is projected to grow at a CAGR of 11.9% during the forecast period.
Orphan drugs are intended to treat rare diseases. Based on published articles and national databases, the U.S. Food and Drug Administration (FDA) has approved more orphan drugs than non-orphan drugs in the last five years. The increasing number of patients suffering from rare diseases is propelling the demand for the introduction of effective therapeutics in the country.
Such a huge population of patients with unmet needs encourages manufacturers to actively invest in research and development activities to expand treatment options for rare diseases. The outbreak of COVID-19 impacted the market growth positively. This positive growth was due to increased regulatory approvals for the product.
Robust Pipeline Candidates for Rare Diseases is a Major Market Trend
The growing patient population seeking medical treatment for rare diseases such as spinal muscular atrophy, myelofibrosis, multiple myeloma, B-cell lymphoma, and many others is escalating the need for more effective therapeutic solutions. Market players are focusing on R&D to establish the efficiency of potential candidates in treating various conditions. According to various published articles and press releases, Eisai and Biogen’s lecanemab drug is expected to receive approval from the U.S. FDA at the end of 2023 to treat Alzheimer’s disease.
Various drugs have received FDA approvals in the past few years. In 2021, 40 specialty drugs were approved by the FDA, accounting for 75% of all the drugs approved. In 2022, 22 novel medications were approved by the FDA and 16 more were expected to get approved before the end of 2022.
Favorable Government Policies for Orphan Drugs to Impel the Market Growth
The orphan drug act was passed in 1983, providing incentives to pharmaceutical companies for drug development to treat rare diseases. The U.S. FDA grants orphan drug designation to drugs that qualify certain specific criteria. They have also established the orphan product grant program to fund developing products for rare diseases or conditions. In 2022, 20 of 37, or 54% of novel drugs were approved for rare diseases.
The growing number of approved orphan drugs is driven by technological progress and the introduction of newer modalities such as cell and gene therapies, antibody-drug conjugates, and bispecific antibodies. These modalities are often tested in niche, well-defined conditions that make them more suitable for orphan drug designation. These new modalities approved by the Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER) captured one-third of all products approved in 2022.
Request a Free sample to learn more about this report.
In U.S., over 1.9 million new cancer cases (excluding basal cell, squamous cell skin cancers, and carcinoma in situ (noninvasive cancer), are expected to be diagnosed as per the American Cancer Society 2023 report.
Limited Product Adoption Owing to High Cost May Hinder the Market Growth
One critical factor that is limiting the adoption rate of orphan drugs, especially cell and gene therapies, is their high cost. For instance, Sarpeta Therapeutics’s gene therapy Elevidys was approved for Duchenne muscular dystrophy in June 2023 and is expected to cost USD 3.2 million per patient.
Similarly, three of the gene therapies approved by the FDA in 2022 cost USD 2.8 million per patient. These include Bluebird’s Zynteglo for beta-thalassemia for USD 2.8 million per patient; Skysona for Cerebral adrenoleukodystrophy (CALD), a rare neurological disorder for USD 3 million per patient; and CSL Behring and UniQure’s hemophilia B therapy Hemgenix for USD 3.5 million per patient.
These therapies challenges payers and devoid potential patient population in availing treatment for orphan diseases. This may decline the treatment rate of rare diseases, limiting the U.S. orphan drugs market growth during the forecast period.
Based on therapy area, the market is segmented into hematology, cardiovascular, oncology, respiratory, endocrinology, neurology, immunotherapy, and others.
The oncology segment accounted for the largest U.S. orphan drugs market share in 2022 due to increasing cancer prevalence across the country. A large number of manufacturers offer drugs for the treatment of these cancers, which are designated as orphan drugs.
By drug type, the market is segmented into biologics and non-biologics.
The biologics segment dominated the U.S. market in 2022. The segment’s dominance is attributed to the large number of biologics being approved for multiple indications across the country.
In terms of distribution channel, the U.S. market is segmented into hospital pharmacies, and retail & online pharmacies.
Hospital pharmacies segment held the major market share in 2022. The dominance of this segment is attributed to the fact that most medications are for orphan diseases and for which consulting to a trained medical professional at the hospitals is needed. After a thorough examination, these drugs can be procured through a prescription. Most drugs must be administered intravenously by a trained healthcare professional. These factors are expected to subsequently drive the segment growth.
The market is highly competitive, with market players such as Bristol Myers Squibb and Johnson & Johnson holding the leading positions in 2022. Bristol Myers Squibb's apex position can be attributed to the strong sales performance of its approved products; Breyanzi, Abecma, and Revlimid, approved for Large B-Cell Lymphoma (LBCL) and multiple myeloma. The company is focused on expanding its product offerings in the oncology therapy area. Johnson & Johnson has also established its presence in the market with its star orphan Darzalex and Imbruvica, and Carvykti, which largely serve oncology therapy.
Some other key players with a considerable presence in the market include AstraZeneca, F. Hoffmann-La Roche Ltd., Novartis AG, and others. AstraZeneca leads in the blood category with Ultomiris, which is approved for rare blood conditions such as paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome. Some of the emerging players in this market are Biogen, Vertex Pharmaceuticals, and others. These companies are expanding their footprints in the neurology and respiratory therapeutic category.
To gain extensive insights into the market, Request for Customization
The market report provides a detailed analysis of the market. It focuses on key aspects such as the prevalence of rare diseases in the U.S. and pipeline analysis. In addition, it includes an overview of technological developments, key industry developments, and the impact of COVID-19 on the market. Besides this, the report also offers insights into the market trends and highlights key industry dynamics. It further encompasses several factors that have contributed to the growth of the market over recent years.
CAGR of 11.9% from 2023 to 2030
Value (USD Billion)
By Therapy Area
By Drug Type
By Distribution Channel
Fortune Business Insights says that the U.S. market was worth USD 77.82 billion in 2022.
The market is expected to exhibit a CAGR of 11.9% during the forecast period (2023-2030).
By drug type, the biologics segment leads the market.
Bristol Myers Squibb Company and Johnson & Johnson Services Inc. are the top players in the market.
“This report is really well done and we really appreciate it! Again, I may have questions as we dig in deeper. Thanks again for some really good work.”- U.S.-based biotechnology company focussing on treatment of chronic pain.
“Kudos to your team. Thank you very much for your support and agility to answer our questions.”- Europe-based provider of solutions to automate data centre operations.
“We appreciate you and your team taking out time to share the report and data file with us, and we are grateful for the flexibility provided to modify the document as per request. This does help us in our business decision making. We would be pleased to work with you again, and hope to continue our business relationship long into the future.”- India-based manufacturer of industrial and specialty intermediates with a strong global presence.
“I want to first congratulate you on the great work done on the Medical Platforms project. Thank you so much for all your efforts.”- One of the largest cosmetics company in the world.
“Thank you very much. I really appreciate the work your team has done. I feel very comfortable recommending your services to some of the other startups that I’m working with, and will likely establish a good long partnership with you.”- U.S. based startup operating in the cultivated meat market.
“We received the below report on the U.S. market from you. We were very satisfied with the report.”- UGlobal hearing aids manufacturer.
“I just finished my first pass-through of the report. Great work! Thank you!”- U.S. based solar racking solutions provider.
“Thanks again for the great work on our last partnership. We are ramping up a new project to understand the imaging and imaging service and distribution market in the U.S.”- World’s leading advisory firm.
“We feel positive about the results. Based on the presented results, we will do strategic review of this new information and might commission a detailed study on some of the modules included in the report after end of the year. Overall we are very satisfied and please pass on the praise to the team. Thank you for the co-operation!”- Germany based machine construction company.
“Thank you very much for the very good report. I have another requirement on cutting tools, paper crafts and decorative items.”- Japanese manufacturing company of stationery products.
“We are happy with the professionalism of your in-house research team as well as the quality of your research reports. Looking forward to work together on similar projects”- One of the Leading Food Companies in Germany
“We appreciate the teamwork and efficiency for such an exhaustive and comprehensive report. The data offered to us was exactly what we were looking for. Thank you!”- Intuitive Surgical
“I recommend Fortune Business Insights for their honesty and flexibility. Not only that they were very responsive and dealt with all my questions very quickly but they also responded honestly and flexibly to the detailed requests from us in preparing the research report. We value them as a research company worthy of building long-term relationships.”- Major Food Company in Japan
“Well done Fortune Business Insights! The report covered all the points and was very detailed. Looking forward to work together in the future”- Ziering Medical
“It has been a delightful experience working with you guys. Thank you Fortune Business Insights for your efforts and prompt response”- Major Manufacturer of Precision Machine Parts in India
“I had a great experience working with Fortune Business Insights. The report was very accurate and as per my requirements. Very satisfied with the overall report as it has helped me to build strategies for my business”- Hewlett-Packard
“This is regarding the recent report I bought from Fortune Business insights. Remarkable job and great efforts by your research team. I would also like to thank the back end team for offering a continuous support and stitching together a report that is so comprehensive and exhaustive”- Global Management Consulting Firm
“Please pass on our sincere thanks to the whole team at Fortune Business Insights. This is a very good piece of work and will be very helpful to us going forward. We know where we will be getting business intelligence from in the future.”- UK-based Start-up in the Medical Devices Sector
“Thank you for sending the market report and data. It looks quite comprehensive and the data is exactly what I was looking for. I appreciate the timeliness and responsiveness of you and your team.”- One of the Largest Companies in the Defence Industry