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Precision medicine: the next step for rare disease?

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precision medicine

Unlike traditional methods of diagnosis and treatment, which generally rely upon a trial-and-error technique, precision medicine will provide an answer based on an individual’s specific DNA.

Precision medicine, sometimes referred to as personalized medicine, uses a person’s genetic information to identify the best treatment for them. Approximately 80% of all rare diseases are caused by genetic mutations, so precision medicine could potentially be used to improve research and find a better treatment for someone with a rare condition.

What are the benefits?

Unlike traditional methods of diagnosis and treatment, which generally rely upon a trial-and-error technique, precision medicine will provide an answer based on an individual’s specific DNA. Instead of trying multiple different treatments to find the best one, a patient will be given the appropriate treatment by comparing their genetic information to a huge database of DNA. This may also mean a reduction in the cost of healthcare overall, as money won’t be spent on ineffective treatments.

Are there any drawbacks?

The combining of large amounts of genetic data is a big challenge. The process will require collaboration and sharing of data across many organisations, and there will be a number of hurdles when it comes to managing the data and keeping it safe.

What will it mean for rare disease?

In cystic fibrosis, precision medicine research has already identified markers in DNA which may help predict how the condition will progress, and help to understand the best treatments for individual patients. 

Although precision medicine is promising, the amount of data available for rare diseases is more limited than in other areas. As a result, it may take time for enough data to be available for precision medicine to be an option for many rare conditions. 

What does the future look like?

Following the implementation of The Precision Medicine Initiative by President Obama, the transition to precision medicine in the US has gained momentum. Healthcare organizations all over the world are starting to consider this new technique. Personalized care may be closer than we think.

Check out our article about a new database that is helping scientists and doctors identify the genetic causes of rare diseases, and give rare disease patients a diagnosis sooner.

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