The Future Of Orphan Drugs: Insights On The Current Market, Trends & Strategic Partnerships Driving Innovation

With around 6,000–7,000 rare diseases affecting approximately 300 million people worldwide, there is a substantial unmet need for effective treatments. The global market for orphan drugs is set to experience significant growth, with a projected compound annual growth rate of 12% from 2019 to 2024. The market, currently valued at $150 billion, is expected to reach $242 billion by 2024, capturing about 20% of the overall prescription-drug sales globally. Major pharmaceutical companies like Celgene, Roche, and Novartis have generated substantial revenues from orphan drugs, with acquisitions of biotech firms being a common strategy. Orphan-drug status in the US offers benefits such as tax credits, exclusive marketing rights, and FDA assistance, while expedited development pathways accelerate drug availability. Repurposing approved drugs through regulatory pathways helps save time and costs. However, the fast-paced nature of orphan-drug development presents challenges, leaving little room for error or unforeseen issues. The report will delve into these trends, focusing on expedited development, and provide insights into overcoming development and manufacturing obstacles with the support of contract development and manufacturing organizations (CDMOs).