• Briana Barnett

There is Value in Rarity: Rare Diseases and Drug Development


What is a Rare Disease?


Over 7,000 known rare diseases have been discovered, and it is certain that many more remain unidentified. For a disease to be referred to as “rare” it must affect less than 200,000 people in the United States, and there are roughly 358.24 million people in the US total. While the population of these rare diseases may seem small in reference to the population of the country, that does not mean they are less important. The goal is to be able to treat all diseases and conditions. While we are not quite there yet, we are working to get as close as possible. Amid this effort, many scientists and researchers are faced with obstacles that hinder further innovation and development of rare disease treatments.


Challenges of Rare Disease Treatment


Because rare diseases affect so few people, it makes it difficult to do research on such a small population. Clinical trials are where researchers gather information that helps determine how to treat these diseases, but for the clinical trials to be successful, willing individuals are needed. Some of the biggest problems researchers face are patient accessibility, recruitment, and retention.


Let us just pretend all the affected individuals of a particular disease are willing to participate in a clinical trial, however, almost 50% of the affected are not able to travel to the nearest site, which could be hundreds of miles away. Whether money to fund transportation or childcare is the issue, these obstacles discourage patients from enrolling in a clinical trial and for those who have already enrolled, it discourages continuous participation. If the patient feels like it would cause extra stress on their lives, they determine that their participation is not worth the hassle. In this example, although the patients want to participate, patient accessibility can hinder the enrollment and retention rates of the clinical trial.


What Reveles is Doing to Help


It is simple, Reveles is Bringing Hope Closer to Home. All of that includes solutions to the challenges stated above: accessibility, recruitment, and retention. Reveles is a decentralized clinical trial pharmacy, providing support and services for patients while in the comfort of their home. This allows patients who are unable to travel, for whatever reasons, to receive care and treatment. So, in reference to the 50% mentioned earlier who were not able to travel to the closest site, these patients now have an opportunity to receive treatment without worrying about the commute which increases the 50% participation to 100% participation. In-home care increases both enrollment and retention rates by creating an alternative for patients who would otherwise have to travel far distances to receive treatment.


Reveles also focuses an interest on rare disease and is advocating on its behalf. Because we are People-Focused, we make sure to educate potential patients about clinical studies and how drug development benefits from their participation. In a world where social media dominates, we utilize social media to reach more people. This is key in recruitment. Most patients decline participation simply because they do not understand the process or even how important their contribution can be to research, and ultimately themselves. Reveles strives to provide resources to increase visibility, efficiency, and viability.


What YOU Can Do to Help


It may seem like there is nothing you can do to help rare disease drug development, but you can help in more ways than one. First, educate yourself. Awareness is the first step and as G.I. Joe put it, “Knowing is half the battle.” Getting familiar with rare diseases will help you better understand the process and also give you a better idea of what role you can play to help. Second, if you or anyone you know suffers from a rare disease and does not have a treatment quite yet, we encourage you or your loved one to speak with a physician to see if a clinical trial is available. Ultimately, advocating for clinical trials contribute to the research and development of new drugs and treatments.


Please visit the link below for more information:

From our perspective: Encouraging drug development for rare disease

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