The Future Of Orphan Drugs Insights On The Current Market Trends Strategic Partnerships Driving Innovation – Life Science Leader Magazine

Orphan drugs are pharmaceuticals that treat rare conditions or diseases. In the United States, “rare” translates into a disease that affects fewer than 200,000 patients; in Europe, it’s a disease that affects fewer than five in 10,000 people. Globally, approximately 6,000–7,000 rare diseases affect approximately 300 million people, but there are treatments for only about 5% of these diseases.1,2 Many pharma and biotech companies use incentives offered by worldwide regulatory bodies to develop orphan drugs and fill these patients’ vast unmet needs, and with advancing technologies and manufacturing capabilities, the potential to deliver life-changing therapies is becoming a reality.
Forecasts predict that the global market for orphan drugs will grow at a compound annual growth rate (CAGR) of about 12% from 2019 to 2024, which is about double the rate forecasted for the market for non-orphan drugs.3 Currently the global market for orphan drugs is valued at about $150 billion, and by 2024, orphan drugs are expected to have a market size of $242 billion and capture about 20% of all worldwide prescription-drug sales.3 Therapies for rare diseases come in all shapes and sizes, from small to large molecules (biologics) to cell and gene therapies. The three companies earning the highest revenues in 2018 from orphan drugs were Celgene, Roche and Novartis, each with more than $10 billion in worldwide sales. Small-molecule drugs Revlimid® (lenalidomide) and Tafinlar® (dabrafenib) were the leading orphan drugs for Celgene and Novartis, respectively, while Hemlibra® (emicizumab-kxwh), a mAb from Roche, led its orphan-drug portfolio.3 With these types of revenues, it is no surprise that big pharma is actively seeking to acquire biotech companies with orphan-drug assets. Just in 2019, Bristol-Myers Squibb completed its acquisition of Celgene for $74 billion10, and Roche acquired Spark Therapeutics for $4.8 billion.3
U.S. orphan-drug status comes with several advantages, including tax credits, a provision for seven-year exclusive marketing rights after approval, waiver of prescription-drug user fees, annual grants to pay for qualified clinical testing and design assistance from the Food and Drug Administration (FDA) for clinical research. Accelerated development pathways that companies may apply for to get their orphan drugs to the clinic faster include Fast Track, Priority Review, Accelerated Approval, Breakthrough Therapy, and, the most recent, Regenerative Medicine Advanced Therapy (RMAT).
A popular trend is repurposing drugs already approved for a different indication by using the 505(b)(2) regulatory pathway. This has several advantages, enabling companies to save time and money in developing drugs and speeding the delivery of much-needed treatments to patients. Similar legislation has been passed in Europe by the European Medicines Agency (EMA), beginning with the Orphan Regulation of 2000, and more recently the Priority Medicines (PRIME) scheme launched in 2016 to significantly address patients’ unmet medical needs.
Similarly, these expedited development pathways provide certain benefits for companies developing orphan drugs, such as extended market exclusivity, free scientific advice (protocol assistance), fee reductions and grants.
However, there is a caveat to the accelerated pace of orphan-drug development: Limited time means companies are under pressure to get it right the first time. In addition, these organizations have less time to react to unexpected problems that may arise during drug development and face restricted opportunities to rectify these issues.
In this report, we will highlight the trends shaping the orphan-drug market, with a particular focus on expedited drug development. Expert insights on navigating development and manufacturing challenges with orphan drugs will be included, with insights on how drug sponsors of all sizes work with contract development and manufacturing organizations (CDMOs) to achieve their goals.
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