[Congressional Record Volume 158, Number 25 (Wednesday, February 15, 2012)]
[Senate]
[Pages S702-S703]
From the Congressional Record Online through the Government Publishing Office [www.gpo.gov]

      By Mrs. HAGAN:
  S. 2113. A bill to empower the Food and Drug Administration to ensure 
a clear and effective pathway that will encourage innovative products 
to benefit patients and improve public health; to the Committee on 
Health, Education, Labor, and Pensions.
  Mrs. HAGAN. Mr. President, today I am proud to introduce the 
Transforming the Regulatory Environment to Accelerate Access to 
Treatments, TREAT, Act.
  This bill empowers the Food and Drug Administration to ensure 
consistent processes and a clear and effective pathway that will 
encourage the development of innovative treatments to benefit patients, 
particularly subpopulations and those with rare diseases, and improve 
the public health.
  Without question, the FDA plays a critical role in helping to ensure 
that new medicines are safe and effective. At the same time, by 
promoting investment in and development of innovative treatments for 
unmet medical needs, the FDA can positively influence our national 
strategy to identify and treat serious and life-threatening diseases 
and improve the quality of life for millions of Americans.
  In order for FDA to accomplish this goal, however, Congress needs to 
give the agency the tools necessary to transcend existing barriers, 
reform its processes, and provide greater clarity, consistency, and 
transparency to industry.
  The bill accomplishes this in three ways.
  First, it provides the FDA with the authorities and tools that are 
reflective of the agency's responsibilities and that are necessary to 
ensure maximum operational excellence by updating FDA's mission 
statement and creating a management review board.
  Second, it advances regulatory science and innovation within FDA to 
ensure that evaluations of innovative treatments, therapies, and 
diagnostics are conducted by those who have the best available 
knowledge. To do this, the bill creates a chief innovation officer and 
chief medical policy officers, and expands participation on advisory 
committees by those experts most familiar with the disease being 
considered.
  Finally, the bill promotes the utilization of modern scientific tools 
and methodologies to ensure patients have timely access to innovative 
products by creating a clinical informatics coordinator, providing more 
information to drug sponsors when an application has not been approved, 
and enhancing and codifying the accelerated approval process.
  In the nearly 2 decades since the accelerated approval mechanism was 
established by FDA to more expeditiously approve treatments, advances 
in medical sciences, including genomics, molecular biology, and 
bioinformatics, have provided scientists with an unprecedented 
understanding of the underlying biological mechanisms and pathogenesis 
of disease.
  A new generation of modern, targeted, personalized medicines is 
currently under development to treat serious and life-threatening 
diseases. Some apply drug development strategies based on biomarkers or 
pharmacogenomics, predictive toxicology, clinical trial enrichment 
techniques, and novel clinical trial designs, such as adaptive clinical 
trials that can be altered based on observed patient outcomes in the 
interim.
  In order to ensure these scientific advances are translated into 
treatments that benefit patients, Congress should allow FDA to 
implement a more effective process for the expedited development and 
review of innovative new medicines intended to address unmet medical 
needs for serious or life-threatening diseases or conditions.
  FDA is already doing this, to some extent. However, application of 
the accelerated approval process has been somewhat limited, largely to 
HIV and oncology drugs, and inconsistently applied to other disease 
targets. For example, a 2011 report by the National Organization for 
Rare Disorders compared the approval process for 135 non-cancer orphan 
therapies approved by FDA from 1983 through June 2010. The report found 
that 45 went through the conventional approval process; 32 were 
approved with some sort of administrative flexibility; and 58 were 
approved on a case-by-case flexibility process. This report illustrates 
that while FDA does have the authority to approve these treatments with 
some flexibility, there does not appear to be uniformity or consistency 
in employing this flexibility.
  The TREAT Act allows FDA to tap into modern scientific advances by 
using a broad range of surrogate or clinical endpoints and modern 
scientific tools earlier in the drug development cycle, when 
appropriate, to approve treatments for patients. Employing these modern 
scientific tools may result in fewer, smaller, or shorter clinical 
trials for the intended patient population or targeted subpopulation 
without compromising or altering FDA's existing high standards for the 
approval of drugs.
  It is the patients suffering from these serious and life-threatening 
diseases that benefit from expedited access to safe and effective 
innovative therapies. For the 30 million Americans living with rare 
diseases, new advances in science and medicine cannot come fast enough. 
That is why I am proud that this bill has the support of the National 
Organization for Rare Disorders (NORD) and Friends of Cancer Research. 
The TREAT Act provides FDA with the tools needed to modernize its 
processes and encourage the development of innovative products to 
benefit patients, particularly subpopulations and those with rare 
diseases.
  I urge my other colleagues to join us in supporting this important 
bill.

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