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The President signing off on “Advancing Hope Act of 2016,” which modifies the FDA’s priority review voucher program for rare pediatric diseases means an extension of the program through the end of the year, for now. Even more importantly, this means that between now and December 31st, Congress will examine and deliberate ways to amend the program and extend it past 2016. The sign off also clarifies one of the more controversial aspects of the bill - the definition of “rare pediatric diseases."

PAST: For over 20 years scientists and researchers have been investigating ways gene therapy can both treat or prevent genetic diseases; attempting to treat the disease at the molecular level by correcting mistakes at the gene level. The 80’s kicked off the Human Genome Project. Completed in it's entirety in 2003 the project identified and mapped all genes from the human genome. With this, researchers are more easily able to identify target genes. By the early 90’s the first gene therapy was being delivered to newborn patients with ADA deficiency (adenosine deaminase deficiency, ADA-SCID) a rare metabolic disease.

In many cases, developing a product for a rare disease means developing a product for fewer than 1000 people worldwide and in some cases, less than 100. These products are clearly not being developed for the masses, they do not expect to be “blockbuster” drugs like Humira (with sales of just over $14B in 2015) and the development is certainly not expected to be (nor will it be) a walk in the park. Getting an orphan product to the market may be done in a shorter timeframe, but the challenges of developing such therapies are often abundant and costly. Under normal market conditions, developing products intended to treat such a small number of patients would prove little interest to the industry and quite certainly would rarely happen due to low ROI.