Spurring Drug Development for Life-Threatening, Rare Pediatric Conditions

Hope is a necessity, especially when you have a rare disease in your family. My family lives with one of the 7,000 rare diseases without a cure, so our one true hope is for a treatment that increases the quality of life of our children. That hope relies on a strong government/industry partnership. This partnership began in earnest in 1984 with the passage of the Orphan Drug Act, and must be strengthened by the Rare Pediatric Disease Priority Review Voucher Program, an incentive for drug development that is set to expire on Sept. 30.

My 9-year-old daughter Rafi is one of the 25,000 people in the U.S. who suffer from a severe form of epidermolysis bullosa. EB is a painful, fatal condition characterized by extremely fragile skin that blisters and tears from friction. Friction from wearing shoes, brushing teeth and scratching an itch can cause large, painful blisters or erosions that are prone to life threatening infections from bacteria like MRSA. Internal organs and bodily systems are affected by the more severe forms of EB, causing secondary illnesses and complications that require interventions from a range of medical specialists.

EB is a rare disease that benefits from incentives like the Rare Pediatric Disease Priority Review Voucher Program. There is no Food and Drug Administration-approved therapy for EB, yet companies researching promising new treatments plan to apply for the voucher to bring their respective therapies to market faster or to offset some of their development costs. When Rafi was born in 2007, there were no companies developing treatments. Today, incentives like the voucher program have encouraged seven companies, including Amicus Therapeutics and Abeona Therapeutics, to pursue therapies for EB. Incentives have proven their value, and the Rare Pediatric Disease Priority Review Voucher will undoubtedly prove to be just as powerful as earlier incentives that drove innovation.

Since the Orphan Drug Act and the Rare Pediatric Disease Priority Review Voucher Program were enacted, there has been a substantial uptick in the number of companies developing therapies for rare diseases. According to the FDA, in 1983 and 1984 there were five drugs approved for rare diseases. In 2014, 49 so-called “orphan” drugs were approved. And from 1985 to 2014, the FDA approved 511 orphan drugs, granted orphan drug designation to 3,280 drugs, and received 4,738 requests for designation. Furthermore, the FDA’s Director for the Office of Orphan Products Development, Gayatri Raom, has stated “the number of requests for orphan drug designation received … has grown dramatically in recent years. … In 2014 we saw a 30% increase over the prior year’s number … that record was broken the very next year when we received close to 470 requests. And the pace does not seem to be slowing. In fact, comparing the number of new requests received in 2016 with the corresponding date in 2015, there appears to be yet another 30% increase.”

The Rare Pediatric Disease Priority Review Voucher Program provides a choice to a sponsor of a drug or biologic intended to treat a rare disease that affects pediatric populations. Those developing a drug for a rare disease who qualify for a voucher can choose one of two paths; to have their clinical trial data reviewed in an expedited manner, or to sell the voucher. Option one means that patients, if the drug is approved, would have access to the therapy faster. The second option provides an opportunity for the industry partner to recoup development costs that are extremely expensive. Out of the six vouchers that have been awarded to date, one sold for $350 million. Either choice provides an incentive for development — at no cost to the taxpayer. Allowing this program to expire would potentially decrease the number of companies looking to develop drugs for rare pediatric diseases.  Without incentives it is foreseeable that rare diseases with high unmet medical needs that do not promise significant financial returns will once again lose industry interest.

When you live with a rare disease, and in particular when you are the parent of a child with a life threatening and painful rare disease, it is incredibly easy to let go of hope. When every day something as routine as bathing your child causes excruciating physical and emotional pain, when you hear your child say, “stop Dad, you are hurting me,” and when you wake up at 4:30 am to start the rituals of giving medicines and preparing bandages for the day, you need that small sliver of hope to go on. A continuing strong partnership between industry and our government provides hope of more development.

I am lucky that EB has gotten the industry attention lately. I often say that this is the first year where I can see light at the end of the tunnel and know it is not an oncoming train. This feeling of hope and expectation of a better life for my child and the other 200 children a year born with EB is a direct result of the strong partnership between government and the private sector and of the proven incentives like the Rare Pediatric Disease Priority Review Voucher Program.

Now that Congress is working to wrap up its work before recess, it is our hope that they heed the words of Representative Joe Crowley (D-N.Y.).  In July, Crowley, who serves as co-chair of the Congressional Rare Disease Caucus, created a sense of urgency among his colleagues to immediately reauthorize the Rare Pediatric Disease Priority Review Voucher Program for the benefit of children with EB. His message was simple — Congress “must not let this deadline slip and this important program expire.” Our families depend on it.


Brett Kopelan is executive director of debra of America (The Dystrophic Epidermolysis Bullosa Research Association of America), the only U.S. nonprofit providing all-inclusive support to the EB Community, through funding research for a cure and by providing free programs and services for those with Epidermolysis Bullosa — “The Worst Disease You’ve Never Heard Of.”

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