ACTION ALERT: Accelerated Approval for Rare Diseases

URGENT ACTION is needed from the patient community to ensure rare diseases have access to the Accelerated Approval pathway.

We ask that you contact your Member of Congress and ask them to sign the letter to FDA supporting Accelerated Approval for Rare Diseases.   Click here  to contact your Member of Congress.

Congress is circulating a letter to compel the Food and Drug Administration (FDA) to fulfill the mandate to improve access to the Accelerated Approval pathway for rare diseases required by The Food and Drug Administration Safety and Innovation Act (FDASIA), [P.L. 112-144]. On June 25, FDA issued a draft guidance for Industry on Expedited Programs for Serious Conditions-Drugs and Biologics to fulfill the Section 901 mandate. Unfortunately, the draft guidance fails to address the specific issues with rare disease drug development as clearly required by FDASIA. Outcomes of Access to the Accelerated Approval Pathway for Rare Diseases:

  •  A more science-based and predictable FDA regulatory process
  •  A surge in investment and development activity for even the most rare disorders
  • More patients with rare diseases will get earlier access to safe and effective treatments
  • Increased investment in early-stage biotech companies focused on rare diseases will have a positive economic impact in local communities and bring new high paying biotechnology jobs

Read the FDASIA Implementation One-Sheet for more information on Accelerated Approval for rare diseases

Read the Congressional Letter to FDA

Read the Dear Colleague Letter from Gus Bilirakis and other signers.

      Congressional Letter Signers:

1. Gus Bilirakis (R-FL)
2. GK Butterfield (D-NC)
3. Ileana Ros-Lehtinen (R-FL)
4. Jim Moran (D-VA)
5. Marsha Blackburn (R-TN)
6. Mike McCaul (R-TX)
7. Jared Huffman (D-CA)
8. Matthew Cartwright (D-PA)
9. Lou Barletta (R-PA)
10. John Carter (R-TX)
11. Jared Polis (D-CO)
12. Mike Michaud (D-ME)
13. Wm. Lacy Clay (D-MO)
14. Michael Burgess (R-TX)
15. Kerry Bentivolio (R-MI)
16. Chris Collins (R-NY)
17. Robert Pittenger (R-NC)
18. Andre Carson (D-IN)
19. John Carney (D-DE)
20. Michael Fitzpatrick (R-PA)
21. Tom Petri (R-WI)
22. David Joyce (R-OH)
23. Sheila Jackson-Lee (D-TX)
24. Terri Sewell (D-AL)
25. Kenny Marchant (R-TX)
26. Bill Cassidy (R-LA)
27. Julia Brownley (D-CA)
28. Bill Owens (I-NY)
29. Carolyn Maloney (D-NY)
30. Doris Matsui (D-CA)
31. Jim Langevin (D-RI)
32. Peter Roskam (R-IL)
33. Mario Diaz-Balart (R-FL)
34. Greg Harper (R-MS)
35. Gloria Negrete McLeod (D-CA)
36. K. Michael Conaway (R-TX)
37. Vern Buchanan (R-FL)
38. Andrew Harris (R-MD)
39. Bill Foster (D-IL)
40. Frank Wolf (R-VA)
41. Joe Garcia (D-FL)
42. Danny Davis (D-IL)
43. Bill Posey (R-FL)
44. Donald Payne (D-NJ)
45. Stephen Fincher (R-TN)
46. Colin Peterson (D-MN)
47. Bill Shuster (R-PA)
48. Mick Mulvaney (R-SC)
49. Corrine Brown (D-FL)
50. David Schweikert (R-AZ)
51. Adam Smith (D-WA)
52. Leonard Lance (R-NJ)
53. Juan Vargas (D-CA)
54. Tony Cardenas (D-CA)
55. Madeleine Bordallo (D-Guam)
56. Cathy McMorris Rodgers (R-WA)
57. Ruben Hinojosa (D-TX)
58. Eddie Bernice Johnson (D-TX)
59. Theodore Deutch (D-FL)
60. Lee Terry (R-NE)
61. Blaine Luetkemeyer (R-MO)
62. Brett Guthrie (R-KY)
63. Richard Hanna (R-NY)
64. Alan Grayson (D-FL)
65. Bill Young (R-FL)
66. Susan Brooks (R-IN)
67. Shelley Moore Capito (R-WV)
68. Joyce Beatty (D-OH)
69. Rodney Davis (R-IL)
70. Glenn Thompson (R-PA)
71. Pete Sessions (R-TX)
72. Kathy Castor (D-FL)
73. Peter King (R-NY)
74. Zoe Lofgren (D-CA)
75. Aaron Schock (R-IL)
76. Joseph Crowley (D-NY)
77. Renee Ellmers (R-NC)
78. Matt Salmon (R-AZ)
79. Rick Crawford (R-AR)
80. Bill Johnson (R-OH)
81. Jim Gerlach (R-PA)
82. Dutch Ruppersberger (D-MD)
83. Frederica Wilson (D-FL)
84. Pete Olson (R-TX)
85. Bruce Braley (D-IA)
86. Tom Rooney (R-FL)
87. John Mica (R-FL)
88. Charles Dent (R-PA)
89. Eni Faleomavaega (D-American Samoa)
90. Bob Latta (R-OH)
91. Kevin McCarthy (R-CA)
92. Morgan Griffith (R-VA)
93. Dan Lipinski (D-IL)
94. Phil Gingrey (R-GA)
95. Walter Jones (R-NC)
96. Gerald Connolly (D-VA)
97. Ted Yoho (R-FL)
98. Carol Shea-Porter (D-NH)
99. Jeff Miller (R-FL)
100. Sean Patrick Maloney (D-NY)
101. Tim Murphy (R-PA)
102. Patrick Murphy (D-PA)
103. James Lankford (R-OK)
104. Lynn Jenkins (R-KS)
105. Adam Kinzinger (R-IL)
106. John Culberson (R-TX)
107. Billy Long (R-MO)
108. Rush Holt (D-NJ)
109. Michelle Lujan Grisham (D-NM)

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On July 24th, 2013, posted in: Advocacy, Blog, FDA, Legislation, News, Rare Diseases in the News by

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