Brown: We are Falling Woefully Short in our Efforts to Treat Rare Pediatric Diseases

At Committee Hearing, Brown Calls for Innovative Measures to Expand and Encourage Research into Rare and Neglected Pediatric Diseases

WASHINGTON D.C. - U.S. Sen. Sherrod Brown (D-OH) today attended a hearing-called at his request-of the Health, Education, Labor, and Pensions (HELP) Committee that examined rare and neglected pediatric diseases.

At the hearing, "Treating Rare and Neglected Pediatric Diseases: Promoting the Development of New Treatments and Cures," Brown noted that while children make up more than 20 percent of the American population, "pediatric research by the National Institutes of Health (NIH) accounts for a mere 5 to 10 percent of the total NIH budget."

Furthermore, Brown continued, "If we are falling short in our efforts to cure and treat pediatric diseases and conditions - we are falling woefully and inadequately short in our efforts to cure and treat rare and neglected pediatric diseases and conditions. Seven thousand known rare or orphan diseases afflict nearly 30 million Americans - approximately 50 percent of whom are children."

Brown has been a leader in Senate efforts to increase research and development into rare and neglected pediatric diseases. In addition to requesting today's hearing, Brown worked with his Republican colleague, Senator Sam Brownback (R-KS), to successfully offer an amendment to the FDA Amendments Act of 2007 that awards a priority review voucher to any company that obtains approval for a treatment for a neglected tropical disease, thereby encouraging pharmaceutical companies to develop treatments for otherwise neglected diseases. Brown also joined Senator Brownback to successfully offer an amendment to the Agriculture, Rural Development, Food, and Drug Administration, and Related Agencies Appropriations Act of 2010 directing the FDA to establish two new review groups to recommend solutions for the prevention, diagnosis, and treatment of rare diseases and neglected diseases of the developing world. 

Brown also introduced bipartisan legislation with Senator Christopher Bond (R-MO) that would create a nationally-coordinated research network to pursue new treatments and cures for childhood diseases, the Pediatric Research Consortia Establishment Act.  Modeled after the highly successful National Cancer Institute Centers, these consortia would conduct both basic and translational research.  This legislation would address existing gaps in pediatric research in order to facilitate the development of new treatments for our nation's children.

Brown has also been fighting to secure $15 million in appropriations for Epidermolysis Bullosa (EB) -a rare but debilitating disease affecting thousands of American children- through the fiscal year 2011 appropriations process.

Brown's full remarks, as prepared for delivery, are below.

The unfortunate reality is that – whether we’re talking education, poverty, violence, or health – the well-being of our nation’s children is not made a high enough priority.

While great strides are being made with respect to children’s health – thanks to Medicaid, the Children’s Health Insurance Program, and the recently passed health reform legislation – there are still areas where our efforts fall short.

One such area is research and development for pediatric diseases and conditions.   

Despite the fact that children and adolescents account for more than 20 percent of our nation’s population, pediatric research by the National Institutes of Health (NIH) accounts for a mere 5 to 10 percent of the total NIH budget.

Despite the fact that children account for more than 20 percent of our nation’s population, most drugs on the market today have never been tested in children. 

If we are falling short in our efforts to cure and treat pediatric diseases and conditions – we are falling woefully and inadequately short in our efforts to cure and treat rare and neglected pediatric diseases and conditions.

Seven thousand known rare or orphan diseases afflict nearly 30 million Americans – approximately 50 percent of whom are children. 

Even with millions affected by rare diseases, research opportunities remain scarce, approved therapies even scarcer.

One of these rare diseases is Epidermolysis Bullosa, otherwise known as “EB.”

EB is a debilitating, disfiguring, and potentially deadly skin disease directly affecting approximately 12,000 people in the U.S., most of them children. 

Because these children are born without the typical anchors that hold the epidermal and dermal skin layers together, any form of pressure creates unbearable friction and can make the simplest of activities – like hugging, playing, or even sleeping – calamitous.

The most common characteristics of EB are chronic blistering, ulcers, and scarring.

Though the genes causing all types of EB have been identified, a cure continues to elude the medical community – primarily because funding is insufficient to support full clinical trials and because market incentives simply do not exist for this rare pediatric disease. 

With no cure and limited treatment options, children and families struggling with EB continue to suffer as they wait – and hope – for cures or treatments that have yet to be developed.

Waiting and hoping for a cure that may or may not materialize is, unfortunately, all too often the reality for families affected by rare or neglected diseases.

Because rare diseases each affect fewer than 200,000 people in the U.S. – and because neglected diseases often affect impoverished or disenfranchised populations in developing countries – there exist significant barriers and limited market incentives for research and development.

Because of the unique set of circumstances that discourage innovation in this field, thinking outside the box becomes a necessity.

I would like to commend Senator Brownback – who has been a leader in developing innovative ways to encourage research on diseases that lack a large market incentive.

Senator Brownback and I were successful in authorizing a “priority review voucher program” that awards a voucher for expedited FDA review to any company that obtains approval for a treatment for a neglected tropical disease. 

We are working now to expand that program to include rare childhood diseases – like EB – in that program.

Another innovative strategy – this one spearheaded by Senator Specter – is the Cures Action Network. 

This grant program authorized under health reform and housed within the Office of Director at NIH, was established for the express purpose of helping ensure that treatments and cures for rare diseases make it over the finish line when funding for later stage research is holding them back.  

The next step is to fund this tremendously promising program so we can realize its critical goals.

Examining solutions like these, as well as barriers to research and development, is why we are here today.

Witnesses on our first panel will be able to talk about current research and development initiatives taking place with respect to rare and neglected pediatric diseases.

Dr. Jesse Goodman and Dr. Alan Guttmacher will discuss ongoing efforts at FDA and NIH to enhance research and development for rare and neglected pediatric diseases.

And witnesses on our second panel will discuss barriers that hamper our efforts to cure and treat rare these diseases and will provide suggestions about how we can move forward.

For instance, I know the National Organization for Rare Disorders (NORD) has long advocated for more transparency in the regulatory system so that investigators and drug and device manufacturers have a better handle on expectations for the approval of new products.

I know that the American Academy of Pediatrics (AAP) has long fought – and continues to fight – to ensure that drugs and devices are studied, and labeled, for pediatric populations. 

I know that the biotech industry is developing some of the most effective and innovative treatments for rare pediatric diseases, but that attracting sufficient capital for these efforts is an ongoing struggle.

And, finally, I am so pleased that Mr. Alex Silver is here today.

As the father of a son with EB, Mr. Silver has fought tirelessly to raise awareness about this rare pediatric disease and he will be offering a number of suggestions to improve private and public efforts.

I’d like to thank Chairman Harkin and Senator Enzi again for their commitment to rare and neglected pediatric diseases and I’d like to thank all of our witnesses for taking the time today to discuss these issues, which are so close to all of our hearts.

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