Rare diseases are known to bring significant impact on the quality of life of thousands to millions of people across the globe. Rare diseases are also referred to as orphan diseases because pharmaceutical companies refuse to adopt them to develop treatments. In fact, according to the National Center for Advancing Translational Sciences, there are approximately 7,000 rare diseases affecting around 25 to 30 million Americans. Unfortunately, there is still a long way to go to find solutions to end the suffering because treatment exists for only 200 to 300 orphan diseases and little is known about the cause of most rare diseases.
Yet, pharmaceutical companies oftentimes look at large disease populations as the main focus of their investment and show little interest in developing orphan drugs due to a number of reasons. One of these is that rare diseases only affect a small population, which equates to very low profitability. Furthermore, recruitment of patients for research and development (R&D) is also another challenge because it is difficult to locate patients and may eventually incur additional R&D costs. Fortunately, the industry has shifted its perception. In fact, some pharmaceutical companies are willing to take the risk of developing orphan drugs, which they now consider as potential money makers that can bring big revenue and a promising return on investment.
Aside from this, to address the unmet treatment needs of various rare diseases, laws such as the Orphan Drugs Act of 1983 in the United States that gives financial incentives, were promulgated in order to encourage companies to venture on developing orphan drugs. Moreover, according to Thomson Reuters, other benefits given to drug companies for the R&D of orphan drugs are tax credits, favorable reimbursement, R&D grants, fewer hurdles to approval, waived FDA fees, longer exclusivity, shorter development timelines, lower marketing costs, greater regulatory success, faster uptake and premium pricing.
Though orphan drugs cater to a small patient pool, the high price of the drugs offsets the aforementioned challenge. In fact, in the year 2010, Soliris, which is known to treat paroxysmal nocturnal hemoglobinuria (PNH) that affects 1 out of 500,000, was actually considered as the industry’s most expensive drug amounting to $409,000 per year of treatment, which generated a total of $541 million revenue for Alexion Pharmaceuticals. In addition, in the year 2014, the top selling orphan drug in the USA in the year 2014 was Rituxan by Roche, a chronic lymphocytic leukemia drug, which yielded to $ 3.646 billion in total sales.
Development of orphan drugs can be one of the biggest breakthroughs in the pharmaceutical industry. It is projected that by the year 2018, the market will reach $127 billion or approximately 16% of the total prescription sales. Aside from the profitability associated with the commercialization of orphan drugs, patients with rare diseases will be given greater chances of surviving and hope as they face the battle against their illness. The figure below will give us an overview of the top 20 orphan drugs that will have the projected highest revenue in 2018.