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About 30 million people in the European Union are living with a rare disease, and there is an estimated 25 to 30 million people in the US who have been diagnosed with a rare disease.
However, the definitions of what constitutes “rare” are different globally. There may be as many as 7,000 individual rare diseases. In the US, this includes conditions affecting fewer than 200,000 in the country. In the EU, a rare disease affects fewer than 1 in 2,000 people. The number of patients suffering from one of these diseases is often very small, but rare diseases affect a significant portion of the global population. Orphan designated diseases are an increasing part of the pharmaceutical industry, providing 18% of revenue, and estimated to rise to 22% by 2022.
Therapeutic candidates with Orphan Drug Designation in the US benefit from development incentives, including tax credits for qualified clinical testing. Also, a marketing application for a prescription drug product that has received orphan designation is not subject to a prescription drug user fee in most cases. Orphan products are often expensive for payers and patients. Looking at 2017 data, reports have placed the median annual cost for an orphan drug in the US at approximately $46,800. Though costs for drugs treating the top 10 rare diseases are lower.
This e-book includes features on market access strategies in the US, Europe and further afield, with a specific focus on prescription drugs for rare diseases. For example, one article explores the use of orphan drugs in emerging markets and the different strategies required. Emerging markets and orphan drugs are two of the most rapidly growing sectors for the pharmaceutical industry. Just one issue highlighted here, though, is that developing countries often have limited health care budgets and may not have accurate data on the local prevalence of a disease. These regions require a different access approach compared
with markets that have established and sophisticated health care systems.
Lucie Ellis
Managing Editor, In Vivo
Informa Pharma Intelligence
