Energy and Commerce Committee Chair, Cathy McMorris Rodgers shares Peter & Maggie's story at legislative committee meeting in Washington DC May 16, 2024
Energy & Commerce Committee chair, Congresswoman Mcmorris rodgers advocates for lgmd
Washington D.C. — House Energy and Commerce Committee Chair Cathy McMorris Rodgers (R-WA) delivered the following opening remarks at today’s Health Subcommittee markup of 23 legislative proposals to extend Americans’ access to telehealth services, strengthen and preserve Medicaid, and encourage innovation to help children with rare diseases.
ENCOURAGING INNOVATION THROUGH THE FDA
“Lastly, the committee is working on a six-year reauthorization of the rare pediatric disease priority review voucher program ahead of its Sept. 30 expiration.
“We've heard testimony on the importance of this program, and this week the Dion family has been meeting with offices to share their story.
“When Peter and Maggie Dion were born, they were each discharged from the hospital with a clean bill of health and initially seemed to hit their developmental milestones.
“Peter loved to fish for tuna with his dad. Maggie enjoyed dance and running with her mom.
“Everything changed when Peter was almost 10 and was struggling to climb up a set of stairs.
“Peter was diagnosed with a form of muscular dystrophy, limb-girdle type 2C.
“Later that year, his then six-year-old sister Maggie was also diagnosed with the same disease.
“This type of muscular dystrophy is a rapidly progressive muscle wasting disease, and there is currently no cure.
“As their father put it, there are so many genetic variations, doctors ran out of letters in the alphabet to identify the condition.
“The prevalence of the disease is low as is.
“And with over 30 subtypes of the disease, each requiring a different gene replacement therapy, spurring the much-needed research and development proves challenging without additional incentives.
“Without the Rare Pediatric Disease Priority Review Voucher Program, Maggie and Peter’s parents say their chances of getting treatments for their kids becomes almost impossible.
“For anyone who doubts why this program is needed, I urge you to consider the Dion family and their story.
“And, sadly, it is a story known to many families.”
RARE DISEASE RESEARCH
“Just this week, the National Organization of Rare Diseases, which represents patients with rare diseases and their families, released analysis of the importance of the PRV program.
“It is clear from their analysis that the program is successful, but the report also provides important insights on how long it can take to see that success.
“Drugs take years—and hundreds of millions or even billions of dollars—to develop.
“While the rare pediatric PRV program was started in 2012, more than half of the designations, awards, and voucher redemptions came in the last four years.
“I want to thank Mr. Bilirakis, Ms. Eshoo, and many others for all their work on the rare disease legislation being considered today.
“These bills will strengthen the orphan drug pathway, make sure drugs are studied for children battling cancer, and continue the important PRV program.
“I am proud of all the work and patient voices reflected in the legislation before us today.
“I also know that more can and should be done.
“This subcommittee has heard testimony on many other proposals, such as my legislation to improve access to home and community-based services in Medicaid.
“I look forward to continuing to work with members to move those forward in the future.”
Congress: Reauthorize the Rare Pediatric Disease Priority Review Voucher Program
Legislation has been introduced in the House and Senate to reauthorize this important program that has helped spur a safe and effective treatment option for almost 40 rare disease patient communities. To show the rare disease patient community’s strong support for the program, NORD led a patient organization sign-on letter to House and Senate leaders, urging their swift reauthorization of the program for at least five years through H.R. 7384 / S. 4583, the Creating Hope Reauthorization Act. Nearly 200 patient organizations signed on to the letter, which you can view here.
