Study Assesses Rare Disease Treatments for Children From 2010-2018

According to a story from Charcot-Marie-Tooth News, a new study highlights the advances that have been made in the last decade for treating rare diseases that affect children. However, the vast majority of rare diseases lack treatment despite the progress. Additionally, a significant portion of advances have involved the repurposing of previously approved drugs instead of developing new treatments from the ground up.

The Struggle to Treat Rare Disease

Around half of all rare disease patients in the world are children. There is a dire need for more treatments to be developed so that these patients can enjoy a good quality of life, but the inherently small pool of patients means that most drug companies aren’t interested in working on rare disease drugs. Several incentive schemes have been developed in the US in recent years in order to encourage drug companies to begin working on rare disease drugs, such as Orphan Drug designation.

Rare Disease Treatment: Progress and Problems

The researchers determined that a total of 402 orphan drugs have been approved by the US Food and Drug Administration (FDA) in the period from 2010-2018. About a third of these drugs were developed specifically for pediatric patients. These drugs targeted 87 rare diseases in total; there were also 21 diseases that had more than one approved treatment. Acute lymphoblastic leukemia and cystic fibrosis were the most common targeted diseases, with both having a total of 12 different treatments. Meanwhile, hereditary angioedema had a total of six approved treatments.

Remarkably, most of the drugs approved for pediatric rare disease patients were for previously approved drugs, including 45 that had previously been approved for a more common disease and 31 that had previously been approved for a rare disease. 

While the study authors commended the progress that had been made, they also said that their analysis also raised some concerns. They claim that many of the approvals hold more value and impact than others. There were also noticeable deficits, where certain diseases were having multiple therapies gain approval whereas countless others still don’t have any approved treatment. The authors say that the top priority should be encouraging the development of medicines for rare diseases that don’t have any treatment options.

Check out the original study here.

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