Orphan Drugs Market Size, Share, Growth Analysis, By Drug Type (Biological, Non-biological), By Disease Type (Oncology, Hematology), By Distribution Channel (Pharmacy, Hospital), By Region - Industry Forecast 2024-2031

Global Orphan Drugs Market size was valued at USD 175.06 billion in 2022 and is poised to grow from USD 195.76 billion in 2023 to USD 478.49 billion by 2031, growing at a CAGR of 11.82% during the forecast period (2024-2031).

Orphan drugs are designed to treat illnesses that are so uncommon that their sponsors are reluctant to develop them under standard marketing guidelines.

According to the World Health Organization (WHO), this condition occurs less frequently than 6.5 to 10 times per 10,000 people.

The journey from a new molecule's discovery to its commercialization often takes ten years, costs several tens of millions of euros, and is fraught with uncertainty (among ten molecules tested, only one may have a therapeutic effect). The money spent on its research cannot be recouped when a medicine is developed to treat a rare condition.

Since the introduction of the Orphan Drug Act in 2019, intended to promote development of treatments for rare diseases, at least 378 orphan drugs have been approved. Kalydeco may be the orphan drug of all orphan drugs.

The U.S. dominated development of orphan drugs, with more than 300 in clinical trials, followed by Europe. Cancer treatment was the indication in more than 30% of orphan drug trials.

There are currently approximately 7000 rare diseases listed on the National Institute of Health’s (NIH) Office of Rare Diseases website, affecting an estimated 25–30 million people within the United States³, most of these rare diseases are genetic and many are neurological. Approximately 250 new rare diseases are described each year⁴, and with advances in genetics, many more common disorders will be subdivided into genetically distinct “rare” diseases.

US Orphan Drug Market is poised to grow at a sustainable CAGR for the next forecast year.