NORD Supports FAST Act Introduced by Representatives Stearns and Towns

Two bills have been introduced in Congress which are intended to expedite the drug approval process for diseases with serious unmet medical need such as SMA. On February 15, 2012, the Transforming the Regulatory Environment to Accelerate Access to Treatments (TREAT) Act, S. 2113, was introduced by Senator Kay Hagan (D-NC) and on March 5, 2012 the Faster Access to Specialized Treatments (FAST) Act, H.R. 4132 was introduced by Representatives Cliff Stearns (R-FL) and Ed Towns (D-NY).

Both pieces of legislation are supported by a wide constituency including the National Organization for Rare Disorders (NORD) and the Biotechnology Industry Organization (BIO). Text from NORD’s press release on the acts is attached below.

John Maraganore (a BIO board member and the CEO of Alnylam) provided testimony to the House Committee on Energy & Commerce, Subcommittee on Health in support of the FAST act on March 8, 2012. The full testimony is attached below– on page 6, SMA is discussed as a rare disease that could benefit from this legislation.
Jonathan Leff (a board member of BIO and the SMA Foundation) along with a number of SMA clinicians and researchers provided background information on SMA for the testimony.


NORD Supports FAST Act Introduced by Representatives Stearns and Towns

Bill to Expedite Development of Therapies for Patients with Unmet Medical Needs

WASHINGTON DC—–The National Organization for Rare Disorders (NORD) supports proposed legislation introduced this week by Representatives Cliff Stearns (R-FL) and Edolphus (Ed) Towns (D-NY) to accelerate the development of innovative therapies for people with serious or life-threatening diseases.

H.R. 4132, Faster Access to Specialized Treatments or the FAST Act, is designed to modernize and expand the Food and Drug Administration’s Accelerated Approval pathway to encompass a broader range of diseases and encourage innovative drug development tools and strategies.

“We applaud Representatives Stearns and Towns for their leadership in introducing this much-needed legislation,” said NORD President and CEO Peter L. Saltonstall.  “There are millions of Americans with rare diseases who have no FDA-approved treatment.  Accelerating the development of safe, effective therapies for these patients is a top priority for the rare disease community.”

Saltonstall said Stearns and Towns have listened to the concerns of patients and have responded with legislation that will speed the approval of new therapies for patients with serious and life-threatening diseases, while also maintaining FDA’s high standards for safety and efficacy.  He said the bill also provides further clarity of the FDA approval process by codifying and modernizing the well-established Accelerated Approval program.

NORD also supports legislation introduced by Senator Kay Hagan (D-NC) in the U.S. Senate recently known as the TREAT Act.  The Senate bill also is intended to enhance and expand the Accelerated Approval process, with additional provisions to promote development of treatments for unmet medical needs.

“Accelerated Approval” refers to a pathway at FDA that allows for earlier approval of drugs for serious, unmet medical need based on a surrogate endpoint or marker predicting clinical benefit.  Subsequent testing must be done to verify the anticipated benefit.

Of the approximately 7,000 rare diseases, only about 250 currently have treatments approved by FDA.  In the U.S., a disease is considered rare if it affects fewer than 200,000 Americans.

NORD represents the nearly 30 million Americans who have rare diseases.  More than half of these patients are children, and most rare diseases are serious, life-altering or even life-threatening diseases.