Why 2024 Is Already an Encouraging Year for the Rare Disease Community
Recent headlines in research and advocacy show promising news for the treatment of rare diseases.
By Jennifer Bagnall
We’re approaching the end of 2024’s first quarter, and we're thrilled to be able to say the year is off to a great start for the future of rare diseases. In various areas, from new drug approvals to research on emerging treatments to healthcare advocacy, there are several areas of progress that have caught Know Rare’s attention. Read on to discover some of the year’s early highlights in rare disease news.
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NEW DRUG APPROVALS
The FDA has already approved 15 new drugs that affect rare diseases, including some that are intended to treat rare cancers. One of these drugs, Dupixent (dupilimab) was approved to treat eosinophilic esophagitis, illustrating how some drugs that are previously approved to treat a common disease can have additional impact for people with unrelated rare diseases.
ORPHAN DRUG RESEARCH
In the EU, the European Medicines Agency granted orphan drug designation to 22 drugs affecting 25 different rare diseases. Orphan drug designation encourages pharmaceutical companies to conduct molecular research that has the potential to lead to benefits for specific patient groups. (In 2023, almost half of all new medications approved by the FDA were classified as orphan drugs.)
STATE HEALTHCARE REPORT CARDS
The National Organization for Rare Disorders (NORD) recently released their annual state report card, which assesses the performance of states across the US in the areas of access, affordability, and overall effectiveness with relation to health coverage. Key assessment areas included findings on state-regulated insurance, the costs of out-of-pocket prescription drugs, access to medicaid, access to telehealth services, and the impact of rare disease advisory councils. you can find out how your state performed on NORD’s website.
For more rare disease news, visit the Know Rare blog.
