Last week, the House passed H.R. 6, the 21st Century Cures Act, with a bipartisan vote of 344-77. The 21st Century Cures Act contains some important provisions for the pediatric and rare disease community.  This bipartisan effort was led by Energy and Commerce Committee Chairman Fred Upton (R-Mich.) and Ranking Member Frank Pallone, Jr. (D-N.J.), Health Subcommittee Chairman Joe Pitts (R-Pa.) and Ranking Member Gene GreenRaymond (Gene) Eugene GreenLatina Leaders to Watch 2018 Overnight Health Care: Big win at Supreme Court for anti-abortion centers | HHS chief grilled on migrant children | Boom time for ObamaCare insurers? The Hill's Morning Report — Sponsored by Better Medicare Alliance — Washington grapples with civility, protests in charged political times MORE (D-Texas), and Oversight and Investigations Subcommittee Ranking Member Diana DeGette (D-Colo.).

Proponents of this legislation will now turn all of their attention to Senate Health, Education, Labor, and Pensions Chairman Lamar AlexanderAndrew (Lamar) Lamar AlexanderOvernight Health Care — Presented by the Coalition for Affordable Prescription Drugs — Senate blocks Dem measure on short-term health plans | Trump signs bill banning drug price 'gag clauses' | DOJ approves Aetna-CVS merger | Juul ramps up lobbying Trump signs bills banning drug pricing 'gag clauses' Senate defeats measure to overturn Trump expansion of non-ObamaCare plans MORE (R-Tenn.) who plans to advance his own version of the same legislation in the fall of 2015 or early 2016.

Ten-year-old Gabe Griffin of Shelby County, Alabama has a rare and deadly muscle-wasting disorder called Duchenne Muscular Dystrophy.  Through genetic testing doctors have identified the precise location where a piece of Gabe’s “genetic track” is missing, causing a “derailment” that denies Gabe’s muscles an important protein called dystrophin. Duchenne kills children as young as 9 or 10 and very few will ever reach the age of 25. 

Because the cause of Duchenne is known, it is reasonable to expect some breakthrough treatments to reach the market.  Families of Duchenne patients praise efforts in Congress to modernize the law to speed the approval of what they believe are safe and effective treatment options. 

Congress has given the FDA more tools to prioritize rare and pediatric diseases in recent years, and the FDA should be praised for their collaboration with the Duchenne patient community that resulted in a June release of draft guidance for industry to use when developing drugs to treat Duchenne. 

During a recent Associated Press interview Gabe Griffin’s father, Scott, described how the effects of Duchenne on Gabe are almost certainly worse than any potential side effects of drugs that have shown no harmful toxicity and yielded promising results in early clinical testing.
"We talk about side effects often in safety, and I get it, I get it," Scott Griffin said. "But my son is going to have tubes coming in and out all part of his body, with a steel rod shoved in his back, his Achilles heel chords cut, not being able to roll over, not being able to lift his head up. I don't think it gets much worse than that."

Below are a few examples of provisions in the 21st Century Cures Act and how they relate to Gabe’s Duchenne Muscular Dystrophy. 

PATIENT EXPERIENCE

The Griffin family believes strongly that Gabe’s experience as a patient, knowing that all of the nightmarish experiences his dad described are coming, should be a bigger factor in the FDA decision-making process.  Supporters of the 21st Century Cures Act agree, and the legislation requires the FDA to incorporate patient experience during consideration of a drug’s benefits and risks.

PEDIATRIC RESEARCH NETWORK

Our nation is blessed with the best children’s research hospitals in the world.  Children’s of Alabama, St. Jude, Le Bonheur, Monroe Carell Jr., to name a few, are ranked by U.S. News and World Report among the nation’s best.  The 21st Century Cures Act requires the NIH to establish a pediatric research network composed of research institutions that would operate as a consortium in order to pool resources and coordinate activities related to pediatric rare diseases and birth defects.  This collaboration would lead to a better pairing of research and clinical delivery, ultimately saving lives.

PRECISION MEDICINE

Inside one of Gabe Griffin’s genes a few of the tiny information processors, or exons, are missing.  In fact, Gabe is missing exons 44, 45, 46, and 47 so based on this technology skipping exon 43 will work for Gabe.  One drug has been in Phase III trial for over three years but it only helps children who need to skip exon 51.  This method/technology of treatment is called exon-skipping, which tricks the body into “thinking” it has the exon(s) it is missing, thus allowing a needed protein to be produced.  The 21st Century Cures Act would authorize the FDA to rely upon data successes of exon 51 skipping in the approval process for a drug that targets exon 43 for skipping.  This targeted drug delivery, or “precision medicine,” is possible because of genetic sequencing and this legislation would help speed approvals of drugs that use similar approaches to solve the same problem.

MODERN TRIAL DESIGN AND EXPEDITED PATIENT ACCESS

The 21st Century Cures Act requires FDA to issue guidance documents to assist industry in the development of adaptive clinical trial designs and reaffirms the sense of Congress that FDA should continue to expedite the approval of breakthrough treatments as early as possible in the clinical development process when established standards of safety and effectiveness are met.
This legislative effort is an important undertaking that will have long-lasting effects on industry, patients, the FDA, and the physician and research communities.

Staley is senior policy adviser at Waller (wallerlaw.com). He served as chief of staff for former Rep. Spencer BachusSpencer Thomas BachusThe key for EXIM's future lies in accountability Manufacturers support Reed to helm Ex-Im Bank Manufacturers ramp up pressure on Senate to fill Ex-Im Bank board MORE (R-Ala.) from 2007 to 2014.