U.S. Orphan Drugs Market Size, Share, Analysis, By Therapy Area (Oncology, Hematology, Neurology, Endocrinology, Cardiovascular, Respiratory, Immunotherapy, and Others), By Drug Type (Biologics and Non-Biologics), By Distribution Channel (Hospital Pharmacies, Retail Pharmacies, and Online Pharmacies), and Country Forecast, 2024-2032

The U.S. orphan drugs market size was worth USD 84.70 billion in 2023 and is projected to grow at a CAGR of 12.3% 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.

• According to the FDA data published in 2022, over 7,000 rare diseases affect more than 30 million people in the U.S.

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.

LATEST TRENDS

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.

DRIVING FACTORS

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.

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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.

RESTRAINING FACTORS

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.

SEGMENTATION

By Therapy Area Analysis

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.

• According to the American Cancer Society 2023 report, an estimated 89,380 new lymphoma cases will be diagnosed in the U.S. in 2023 and 21,080 people will die from the disease. Lymphoma is broadly grouped as Hodgkin lymphoma (8,830 new cases and 900 deaths in 2023) and non-Hodgkin lymphoma (80,550 cases and 20,180 deaths in 2023).

By Drug Type Analysis

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.

• According to an article published by the Academy of Managed Care Pharmacy Nexcus in October 2022, 39 biosimilars have been approved by the FDA for nine different reference biologics, of which 23 are currently available in the market. Furthermore, ten biosimilars are expected to enter the market in 2023.

By Distribution Channel Analysis

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.

• For instance, Soliris and Upliza are two FDA-approved drugs that were indicated to treat symptoms of neuromyelitis optica soectrum disorder (NMOSD) in 2019 and 2020, respectively. Both these drugs need to be injected intravenously in a clinical setting.

KEY INDUSTRY PLAYERS

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.

LIST OF KEY COMPANIES PROFILED:

• Amgen Inc. (U.S.)
• Bayer AG (Germany)
• F. Hoffmann-La Roche Ltd (Switzerland)
• Alexion Pharmaceuticals Inc. (U.S.)
• Novo Nordisk A/S (Denmark)
• Novartis AG (Switzerland)
• Bristol-Myers Squibb Company (U.S.)
• AstraZeneca (U.K.)
• DAIICHI SANKYO COMPANY, LIMITED (Japan)
• GlaxoSmithKline plc (U.K.)

KEY INDUSTRY DEVELOPMENTS: 

• April 2023 – Biogen received FDA approval for QALSODY (tofersen) for treating amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene.
  
  
• March 2023 – Pharming Group N.V. received FDA approval for Joenja (leniolisib) for the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in patients 12 years of age and older.
  
  
• February 2023 – Travere Therapeutics, Inc. received accelerated approval from the U.S. FDA for FILSPARI (sparsentan), the non-immunosuppressive therapy that is indicated to reduce proteinuria in adults with immunoglobulin A nephropathy (IgAN).

REPORT COVERAGE

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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.

Report Scope & Segmentation