U.S. Senator Kay R. Hagan (NC) today visited North Carolina Children's Hospital to discuss a bill that she has introduced that will accelerate patients' access to life-saving treatments. Hagan toured the hospital and visited with patients and families that would be aided by her bill, the Transforming the Regulatory Environment to Accelerate Access to Treatments (TREAT) Act.
"One of my top priorities is to ensure a smoother and more successful transition for innovative research and treatments from the laboratory to the patients that need them," said Hagan. "For the children I met today - who are suffering from devastating rare diseases for which current treatments are not adequate or don't exist at all - medical advances can't come fast enough. The TREAT Act transforms the regulatory environment to get innovative treatments to targeted patients faster, without lowering the FDA's high standards for safety and effectiveness, and brings FDA's focus back to patients' needs."
Today Hagan met with a number of patients and families, including Meagan and Jarrod Hendren of Lumberton, whose identical 13-month-old twins Lucas and Logan are being treated at the Children's Hospital. Lucas and Logan were diagnosed with Type II and Type III Gaucher disease, a metabolic disorder for which there is currently no cure.
"We offer diagnostic evaluations, genetic counseling and ongoing care for the children," Dr. Wesley Burks, physician in chief at the N.C. Children's Hospital said today. "Sometimes though it's difficult when we just have to say we don't really have a treatment. The bill that [Senator Hagan] is pushing through hopefully will help us in five years, in 10 years and certainly in 20 years not have to say that anymore. There are 30 million Americans living with rare diseases and it's important that we are supportive of legislation like this that will help the children here at North Carolina Children's Hospital as well as the children throughout the United States."
In addition to Dr. Burks, Hagan was joined by Kenneth Moch, president and CEO of Chimerix, a Durham biotechnology company, and Diane Dorman, vice president of public policy for the National Organization for Rare Disorders (NORD).
"Through the TREAT Act, Senator Hagan has offered renewed hope to patients desperately in need of cures and cutting-edge new medicines," said Kenneth Moch of Chimerix. "The TREAT Act will help break the institutional barriers to a faster idea-to-market pathway and speed new cures to patients most desperately in need. Further, it will ensure a transparent and more predictable pathway for the development of safe and effective next generation medicines and cures while maintaining U.S. leadership in biomedical innovation. I look forward to supporting efforts to get TREAT Act provisions enacted into law, and in so doing, tap into the enormous scientific potential that resides in the thousands of small, medium and large U.S. biotech companies to develop new medicines that save, extend and improve the quality and length of life for patients worldwide."
Last week, important provisions of Hagan's TREAT Act were incorporated into the Food and Drug Administration Safety and Innovation Act (FDASIA), a bill that will modernize and update the tools the FDA has to review drugs and medical devices for sale to the public. FDASIA, with Hagan's TREAT Act incorporated, passed out of the Health, Education, Labor and Pensions (HELP) Committee and will now move to the Senate floor for a vote. Also last week, the Biotechnology Industry Organization honored Senator Hagan as the Legislator of the Year for her work on the TREAT Act.
Background on the TREAT Act
The TREAT Act is a commonsense bill that expedites development of and access to safe and effective treatments for patients with serious or life-threatening diseases, including rare diseases. The bill ensures the best science and greater innovation in medical treatments, while maintaining the FDA's high safety and effectiveness standards.
The TREAT Act does this by accelerating the review and approval processes for treatments that:
· Treat an unmet medical need,
· Significantly advance the standard of care, or
· Are highly targeted therapies for distinct sub-populations
For additional background on Senator Hagan's TREAT Act, please click here.
Below is a list of 35 patient advocacy groups that have voiced their support for Senator Hagan's TREAT Act:
1. Alliance for Patient Access
2. American Autoimmune Related Diseases Association
3. American Institute for Medical and Biological Engineering
4. American Society of Clinical Psychopharmacology (ASCP)
5. Californians for Cures
6. Celiac Disease Center at Columbia University
7. Colon Cancer Alliance
8. Crohn's and Colitis Foundation of America
9. Digestive Disease National Coalition
10. EveryLife Foundation for Rare Diseases
12. Hope4Bridget Foundation
13. Huntington's Disease Society of America
15. International Cancer Advocacy Network (ICAN)
16. Lung Cancer Alliance
17. Lupus Foundation of America
18. MLD Foundation
19. Muscular Dystrophy Association
20. National Family Caregivers Association
21. National MPS Society
22. National Venture Capital Association
23. NBIA Disorders Association
24. New Jersey Association for Biomedical Research
25. Parkinson's Action Network
26. Polycystic Kidney Disease Foundation
27. RARE Project
28. Sickle Cell Disease Association of America, Inc.
29. Student Society for Stem Cell Research
30. The Reflex Sympathetic Dystrophy Syndrome Association (RSDSA)
31. Tuberous Sclerosis Alliance
32. US Against Alzheimer's
33. Veterans Health Council
34. Vietnam Veterans of America
35. ZERO - The Project to End Prostate Cancer