[Congressional Record (Bound Edition), Volume 162 (2016), Part 4]
[Senate]
[Pages 5327-5328]
[From the U.S. Government Publishing Office, www.gpo.gov]




                      DUCHENNE MUSCULAR DYSTROPHY

  Mr. WICKER. Mr. President, the fight against muscular dystrophy is a 
cause I have championed since my days in the House of Representatives. 
My fight against Duchenne muscular dystrophy began when a parent told 
me about his son's diagnosis with the disease.
  This parent refused to accept that there was no hope. The House and 
Senate agreed with the MD-CARE Act and, since that time, the life 
expectancy of the average Duchenne muscular dystrophy patient has 
increased by a full decade. This is progress we have made on behalf of 
sick people whose lives were threatened, and this is an example of 
government at its best.
  On Monday of this week, I saw the same devotion in the hundreds of 
Duchenne families who attended a meeting of the advisers of the Food 
and Drug Administration. The meeting's attendance broke records. I 
thank the FDA for making the appropriate accommodations to handle a 
crowd of this size. Some 11,000 people also tuned in remotely, watching 
the meeting via live stream.
  Monday's gathering was about what could be the first disease-
modifying therapy for Duchenne muscular dystrophy. For more than 3 
hours, the advisory committee heard from parents, doctors, and patients 
about the drug's impact on their lives. The stories were heartfelt and 
hopeful, reinforcing the importance of patient engagement in the drug 
approval process. The dedication of the Duchenne community continues to 
set an example for advocates of other rare diseases.
  Patient voices should be part of the drug review process, and I am 
glad to see the FDA is implementing greater stakeholder involvement in 
this process. This was one of the goals of the

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Food and Drug Administration Safety and Innovation Act, which Congress 
passed in 2012. It continues to be a goal of my Patient-Focused Impact 
Assessment Act, introduced last year, which would require FDA to share 
how they use feedback from patients and advocates in the drug approval 
process.
  Unfortunately, the advisory committee decided this week not to 
recommend the approval of the first Duchenne drug. This is 
disappointing news for me and for thousands of Duchenne families, even 
those who might not benefit directly from this drug but from other 
advancements that could stem from it.
  Before a final decision is made next month, I hope the FDA will take 
into consideration the perspectives of Duchenne patients and parents. 
The individuals fighting the good fight every day are ``the real 
experts,'' to quote Austin Leclaire, who suffers from Duchenne and has 
experienced increased mobility because of the drug. People like Austin 
have a life-threatening disease now. They don't have much time.
  No matter the outcome of the FDA's decision next month, I will 
continue to fight the good fight on behalf of those with Duchenne 
muscular dystrophy. In the 15 years since I introduced the MD-CARE, I 
have learned that small wins can lead to big victories.
  MD-CARE was the first Federal law to focus on muscular dystrophy. It 
helped set in motion the research and trials that have produced 
groundbreaking therapies. The life of muscular dystrophy patients now 
is an average of 12 years longer--I think I earlier said a decade; it 
is actually 12 years longer than it was in 2001--a wonderful 
achievement. There are more trial participants needed today than there 
are Duchenne patients.
  Young adults with Duchenne were a population that did not exist when 
we first funded research for the disease. They never got to adulthood. 
Today they are getting to adulthood because Congress acted. Because of 
the MD-CARE amendments that became law last Congress, research at the 
National Institutes of Health has been updated in ways that could help 
patients lead even longer, healthier lives. We want this research to 
continue. We want companies to continue to invest in drugs and 
therapies that could change the lives of those with rare diseases.
  Duchenne is still a fatal disease, affecting 1 out of every 3,500 
boys--mostly boys. Most young men with Duchenne live only to their mid 
to late twenties. We should take every opportunity to find a 
breakthrough. We should take every opportunity to improve quality of 
life. This is about the futures of young people who face this disease 
every day and the families who refuse to give up hope.
  I look forward to the FDA's full and final decision on this matter 
next month, and I certainly am hoping for a positive answer from the 
FDA.
  Thank you.
  I yield the floor.
  The PRESIDING OFFICER. The Senator from South Dakota.


  

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