North America Orphan Drugs Market

North America Orphan Drugs Market Size (2024 to 2029)

The size of the North American Orphan Drugs Market is estimated to be worth USD 70.77 billion by 2029. The market was valued at USD 49.27 billion in 2024 and is growing at a CAGR of 7.51% during the forecast period.

Orphan drugs are medical products utilized to treat ailments or conditions that concern a tiny percentage of the population. Diseases with low occurrence rates are no less important than those with a higher frequency.

Orphan drugs are a comparative niche segment because they are utilized to treat rare diseases. Finding the right equilibrium between cost and profit can be a tightrope walk for an ailment with a low frequency. The number of illnesses considered orphans stands at about 7,000 currently, with just about 250 new diseases being added to this list yearly.

Factors such as increasing health disorders among people due to the adoption of sedentary lifestyles are major factors driving the market demand. Rare diseases are increasing daily, and manufacturers have to focus on developing new drugs or vaccines, which is accelerating the market. Changes in the schemes and policies by the government favoring the end-users are lavishing the market's growth. Increasing investments in the healthcare centers and pharmaceutical industries to ensure patients' safety are expanding the size of the market.

However, the absence of skilled people manufacturing drugs with the latest formula is slowly hindering the market's growth. Also, fluctuations in the prices of final products due to decreased availability of raw materials have remained a challenging factor for the market developers. In addition, stringent rules and regulations by the government in approving new products are inhibiting the market's demand. Moreover, increasing side effects with long-term use of these drugs, especially in cancer patients, limit the market's growth rate.

This research report on the North American orphan drugs market is segmented and sub-segmented into the following categories:

North America Orphan Drugs Market Analysis By Type

• Biological

• Non-biological

North America Orphan Drugs Market Analysis By Therapeutic

• Hematology

• Neurology

• Oncology

• Others

North America Orphan Drugs Market Analysis By Country

• The United States

• Canada

• Rest of North America

Regional Analysis

Regionally, the United States orphan drugs market is predicted to lead the North American orphan drugs market, followed by Canada during the forecast period. Although, benchmarks for the designation of orphan disease differ from country to country. In the United States, if the disease is prevalent in fewer than 200,000 people, then it's termed a rare disease. The market's growth in this region is powered by factors such as prolonged market exclusivity and government enticements for orphan medicine.

The U.S. has the largest market share of orphan drugs. It dominates the development of orphan drugs with more than 300 clinical trials. There is a lot of research and development in orphan drugs. Around 200000 patients in the United States need orphan drugs for rare diseases. The US FDA approved 80 new drugs in 2017. The orphan drug act in 1983 encouraged the pharmaceutical company to invest in such a small market. In the U.S., orphan drugs cover pharmaceutical, biological, medical devices, and dietary products. The office of orphan product development (OOPD) with the U.S. Food and Drug Administration promotes the availability of safe and efficient products to treat rare diseases. The government gives tax credits for clinical research of orphan drugs.

The Canadian orphan drugs market is estimated to showcase a healthy CAGR from 2024 to 2029. There is no regulatory framework for orphan drugs in Canada. The rare diseases are collectively estimated to affect 1 in 12 Canadians. The Canadian government is collaborating with U.S. government organizations for research on new drugs. An increasing number of COVID-19 cases is leveling up the market's growth rate. 

KEY MARKET PARTICIPANTS

The major companies dominating the market in this region are Novartis, GlaxoSmithKline, Roche, Alexion, Sanofi, Bristol Myers Squibb, Pfizer, Vertex, Celgene, and Merck.