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A Tender Age: Pediatric Patients with Rare Diseases

Amanda Stevenson-Grund - Friday, October 09, 2015

Approximately 50% of the people affected by rare diseases are children. 30% of children with rare disease will not live to see their 5th birthday.”
 – Global Genes. RARE Diseases: Facts and Statistics

 A zebra-print ribbon represents pediatric rare diseases

Many people hope to grow older and wiser. Some just hope to grow older. Pediatric patients, especially those with rare diseases, face life-threatening challenges. Three problems pediatric patients with rare diseases face are late diagnosis, few approved treatment options and lack of age-appropriate formats.

Rare diseases can be difficult to diagnose in children, and many physicians do not have the proper training and knowledge to diagnose such diseases. According to a 2014 Medscape Multispecialty article, the average patient with a rare disease sees 7.3 physicians before diagnosis, and 70% of physicians believe additional training in rare diseases would be helpful. A rare disease can spread and worsen during the lengthy time before diagnosis and the start of the proper treatment.

Pharmaceutical companies are unlikely to work on drugs if there is no financial profit to be earned. By definition, rare diseases don’t affect much of the population, so there is little financial gain for pharmaceutical companies. The U.S. government has created the Orphan Drug Act and the rare pediatric disease priority review voucher programs to add financial incentives, and the development of drugs for rare diseases is becoming more common. However, many of the drugs for rare diseases are approved for adults. Physicians sometimes have no choice but to prescribe off-label use of such drugs for children.

Drugs for rare diseases, whether approved for pediatric patients or being used off label, often come in formats that are inappropriate for children. Most young children have trouble swallowing tablets or capsules, and many children are sensitive to bitter-tasting drugs. Sometimes, however, such formats are all that is available, which can lead to compliance issues.

Pediatric patients with rare diseases face obstacles, such as late diagnosis, few approved treatment options and lack of age-appropriate formats. Orbis Biosciences solves some of the problems that face pediatric patients. Through our Precision Particle FabricationTM technology, we develop format-flexible and taste-masked medications, which are suitable for children. As our experience using our technology to meet the needs of the pediatric population continues to grow, our treatments will become even better. We continue our work so that more children can grow older.

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