By Peter Saltonstall
More than 15 million sick children need Congress to act before Sept. 30. Unless Congress acts before it goes on recess, it will be neglecting a vital resource in the nation’s ongoing research into drugs for rare pediatric diseases.
Eliza, a precocious six-year old from South Carolina, is battling Sanfilippo Syndrome, a rare genetic disease nicknamed “childhood Alzheimer’s” for its degenerative progression. Without treatment, Sanfilippo will rob children of their speech, their ability to walk, their ability to feed themselves, their memory, and in their teens, their life.
Hanna, a thirteen-year old from New York, is fighting Spinocerebellar ataxia, autosomal recessive 9 (SCAR9), an incredibly rare genetic disorder characterized by a laundry list of symptoms.
There’s Arturito, a four-year old from Baltimore with TK2 Mitochondrial DNA Depletion Syndrome, a disease that wastes muscles similar to Parkinson’s.
And then there’s Sam, the inspiring 17-year old from Massachusetts who passed away from Progeria, who moved us all in the 2013 documentary, “Life According to Sam.”
We spend every day at the National Organization for Rare Disorders (NORD) fighting for diagnoses, treatments, and cures for the 30 million Americans with a rare disease, more than half of whom are kids. We look for every opportunity to serve Eliza, Hanna, Arturito, Sam, and the millions of children like them, by spreading awareness, educating physicians, changing laws and regulations, and supporting their families and friends.
This month, we need Congress to join our fight by reauthorizing the Rare Pediatric Disease Priority Review Voucher (PRV) Program.
Developing therapies for people with rare diseases is incredibly difficult. Rare diseases are scientifically and medically complex, often locked away in the genetic code. Patient populations are small and dispersed, making clinical trials difficult. These obstacles are only more pronounced in kids.
In 1983, Congress passed the Orphan Drug Act to incentivize therapeutic development in rare diseases. In the resulting 33 years, over 500 orphan therapies have been developed for rare diseases compared to the only 34 orphan therapies in existence prior to 1983.
Thirty years later, in 2012, Congress recognized that more needed to be done for kids and created the Rare Pediatric Disease Priority Review Voucher Program. Since 2012, the program has spurred drug development for rare pediatric diseases, resulting in six new therapies for seven rare diseases that previously lacked a Food and Drug Administration (“FDA”)-approved therapy.
The voucher program works by awarding companies with a priority review voucher if they develop a therapy for a rare pediatric disease. The company can then use that voucher for one of their subsequent products, or sell it to another company. These vouchers allow companies to move through the FDA review process more quickly and are incredibly valuable; the latest voucher sold for $350 million.
These vouchers encourage the development of therapies for kids like Eliza. Abeona Therapeutics is taking gene therapy technology developed by researchers at Nationwide Children’s Hospital in Columbus, Ohio to treat Sanfilippo. The promise of this voucher has allowed Abeona Therapeutics to continue to raise money from investors, including George Soros, to begin the clinical trial. The reauthorization of the Rare Pediatric Disease Priority Review Voucher Program will ensure investors remain committed to providing resources as the clinical trial continues to provide hope to children like Eliza.
Congress can reauthorize this program by passing the Advancing Hope Act of 2016 (S.1878). This bill reauthorizes the program through 2022 while making additional improvements to the program.
But if Congress fails to act by Sept. 30, this valuable incentive is gone, and treatments for our nation’s most vulnerable and neglected children may go undeveloped.
Congress must put our children first and pass S. 1878, the Advancing Hope Act of 2016, before the program expires. First-ever therapies or potential cures are on the cusp for children with not only Sanfilippo syndrome, but also Sly syndrome, Krabbe disease, Batten disease, metachromatic leukodystrophy, the childhood cerebral form of x-linked adrenoleukodystrophy, epidermolysis bullosa, spinal muscular atrophy, and Duchenne muscular dystrophy.
These disorders are fatal. These disorders are debilitating. These disorders are heartbreaking. At times it can feel hopeless; with so many children with such cruel diseases, how can we make a difference?
Let’s all learn from Sam. Before he passed away, he imparted his philosophy for a happy life, saying, “be okay with what you ultimately can’t do because there is so much you can do.”
We can make a difference in these children’s lives. Providing a meaningful extension to the Rare Pediatric Disease Priority Review Voucher Program prior to September 30 will give children with these conditions and their families hope that they will be treated and possibly cured. Eliza, Hanna and Arturito deserve it.