Orphan drugs grow up
By: Judy Walker, MD, FRCP | February 26, 2016
In advance of Rare Disease Day on February 29, Judy Walker discusses five benefits of orphan drug research.
February 29 is Rare Disease Day, a day we set aside to raise awareness about rare diseases and their impact on patients' lives. In honor of this noteworthy event, I think it’s important to explore the myths and realities of rare/orphan drug research.
In the past, rare and orphan disease research was eschewed by biopharma as too complicated and offering too little return on investment to pursue outside of philanthropic efforts. And they weren’t entirely wrong. These trials can be difficult to recruit for, face innumerable unknown risks, and have a much smaller consumer population, making them appear less attractive from a business standpoint. However, changes in the commercial, economic and regulatory landscape have made rare disease research more appealing for biopharma companies in recent years. These development efforts now are encouraged by fast tracking initiatives, tax breaks, longer exclusivity, and the opportunity to be the first to market to solve an unmet medical need. Thanks to these incentives, rare disease research is coming to the fore, with dozens of orphan drugs gaining approval and seeing commercial success every year.
In each of the past two years, the US Food and Drug Administration approved more orphan drugs for rare diseases than in any previous year in its history. In 2014, 41% of all novel new drugs approved in the US were for the treatment of rare diseases, including Sylvant™ to treat Castleman’s disease, and Impavido™ to treat forms of the tropical disease, leishmaniasis. In 2015, that rate rose to 47%, with 21 of 45 novel drugs addressing rare diseases, including Orkambia™, a therapy for cystic fibrosis, and Unituxin™ to treat pediatric patients with high-risk neuroblastoma.
Although these drugs may have smaller target patient populations, their commercial potential can still be significant. The orphan oncology drug Rituxan™, for example, is second only to Lipitor in profitability, and is expected to garner more than $150 billion in revenue over its lifetime, the majority of which is for orphan indications. And it’s not alone. Worldwide, orphan drug sales are forecast to total $178 billion by 2020, with many of these drugs achieving blockbuster status.
And there are many un-explored development paths yet to be taken. According to the National Institutes of Health (NIH), there are 7,000 rare diseases – defined in the US as those that affect fewer than 200,000 people – affecting a total of 350 million people worldwide. That means the dozens of drugs that win approval each year are barely scratching the surface of what still needs to be done.
Five benefits of orphan drug research
Meeting an unmet medical need while achieving bottom-line results is the Holy Grail for most biopharma research teams, though it is important to acknowledge that these projects won’t be easy. Most rare diseases have few if any treatments developed for the condition, which means there will be no well-defined development pathway and there may be no or few validated outcome measures. Recruiting is also a challenge as these patient populations are small and widely dispersed. These diseases also often impact pediatric populations, adding additional emotional, logistical, and regulatory challenges, including requirements for extra toxicology studies to prove the safety of the treatments before patients can enroll. Finally, biomarkers to identify patients for trials and to guide development may not be as widely available in rare diseases as they are in more common diseases due to decades of research underfunding in this space.
But there are several upsides to this research that you won’t find in mainstream drug development projects.
Rare disease research is difficult but rewarding, and with the right partners and a clear understanding of the science and regulatory environment, it can also be a lucrative path to follow. In an era where blockbuster drugs are a rarity, this research path should be hard to pass up.