The Value of Repurposing Old Drugs to Treat Rare Disease

According to a story from, medical researchers are beginning to more closely investigate that possibilities that drug repurposing, or using old drugs for treating different illnesses, could offer. While it is true that the development of new drugs is moving apace, it is often a long and costly process. On average, the development of a new therapy costs around a billion dollars and can take nearly a decade to complete.

Drug repurposing offers a potentially cheaper and quicker alternative, and has the potential to make significant impact in treating rare disease. After all, developing rare disease drugs is often a more timely and costly process than making treatments for the most common illnesses. Many drug developers don’t bother because of the limited pool of patients that would benefit and therefore spend money on the drug; even with federal incentives like orphan drug designation, it simply isn’t worth it to many pharma companies.

The problems of time and cost are also getting progressively worse, indicating a significant loss in research and development efficiency in the pharmaceutical industry. Of all drug candidates submitted for trials and FDA approval, only about ten percent ever make it to the market. However, about 63 percent of drugs to clear the first Phase I trial hurdle. This could be a potential resource of drugs that could be repurposed, and if they have made it through Phase I, then they have at least been established as safe to use.

Research with these drugs in order to find new purposes for them could save a lot of time and money, and when a drug is used for a different rare disease, it gets a new patent that creates another monetary incentive for drug companies. Drug repurposing is not just a theoretical phenomenon either; Viagra, for example, widely used as a treatment for erectile dysfunction, was originally developed for treating cardiovascular disease.

It is not uncommon for conditions that may appear unrelated to share some of the same symptoms and underlying causative mechanisms. Clearly, repurposing is simply not being exploited effectively. However, before repurposing can take place on an industry scale and start helping rare disease patients, the norms around drug research need to change. Because research fields are often highly specialized, a compound being developed for one rare disease is often totally unknown outside of the small circle of researchers that are focused on it.

Share this post

We believe rare disease patients are people, not a diagnosis. Through education, awareness and some humor, we help patients, caregivers and support persons by providing relevant and often inspirational news and stories.
Our goals are to share stories, cultivate strong community, provide the latest medical findings, connect people and pioneer production of patient worthy information. Help us attain these goals by telling us a little bit about yourself!

© Copyright Patient Worthy

Close Menu