[Congressional Record (Bound Edition), Volume 162 (2016), Part 2]
[Senate]
[Pages 2869-2870]
[From the U.S. Government Publishing Office, www.gpo.gov]




                              ORPHAN DRUGS

  Mr. HATCH. Mr. President, in light of recognition of Rare Disease 
Day, I wish to speak about orphan drug exclusivity and trade promotion 
authority.
  Congress enacted the bipartisan Orphan Drug Act, ``ODA'', of 1983, 
Pub. L. 97-414, to address a longstanding unmet need to develop new 
treatments, diagnostics, and cures for rare diseases and disorders. I 
am proud to be one of the lead Senate sponsors of the ODA, which was 
passed with overwhelming bipartisan support. This act and the Rare 
Diseases Act of 2002--which I also championed--created financial 
incentives for the research and production of orphan drugs, including 7 
years of market exclusivity, tax credits, and research grants, and also 
established the Orphan Products Board at FDA and the Office of Rare 
Diseases under the National Institutes of Health.
  The purpose of these acts was to encourage the development of new 
``orphan'' treatments, diagnostics, and cures for the millions of 
Americans with rare disease who lacked access to effective medicines 
because the existing incentives were insufficient to develop and market 
drugs for such small groups of patients.
  The ODA has been enormously successful. Before Congress enacted the 
ODA in 1983, the Food and Drug Administration, FDA, approved only 38 
drugs in the United States specifically to treat orphan diseases. From 
the passage of the ODA in 1983 until May 2010, the FDA approved 353 
orphan drugs and granted orphan designations to 2,116 compounds. As of 
2010, 200 of the roughly 7,000 officially designated orphan diseases 
have become treatable.

[[Page 2870]]

  Yet, despite the benefits of these policies, the incentives and 
access guarantees found in the ODA are not yet part of any free trade 
agreement negotiations.
  The Bipartisan Congressional Trade Priorities and Accountability Act 
of 2015, or TPA, contain a number of negotiating objectives for the 
administration to follow. For example, the TPA law's negotiating 
objectives require that U.S. trade agreements provide a standard of 
intellectual property rights protection that is similar to that found 
in the United States, which includes providing incentives for 
biopharmaceutical innovation that are similar to those in the United 
States. The language in the TPA law is necessarily broad, and although 
it does not explicitly reference critical incentives for orphan drug 
development, I want to make it clear that these incentives, including 
the 7-year market exclusivity at the heart of the ODA, are consistent 
with the TPA law's requirement that U.S. trade agreements provide a 
standard of intellectual property protection that is similar to U.S. 
law.
  This is especially important because vital incentives for orphan drug 
development are lacking in many markets outside the United States, 
hindering the development of treatments, diagnostics, and cures for 
rare diseases--particularly diseases endemic to those markets. A lack 
of incentives for orphan drug development in any one country can have a 
very real impact on the likelihood of investment into a research or 
cure for a given disease. Particularly in the case of ultra-rare 
diseases, those affecting fewer than 1 in 50,000 individuals, there may 
only be a handful of patients around the world who would benefit from a 
particular treatment or cure, and removing a number of them from the 
pool of potential patients may render investments in these therapies 
untenable and could drive up costs for rare disease patients in the 
United States.
  Therefore, I want to make it clear that I believe it is appropriate 
for the administration to negotiate ODA incentives and access 
guarantees, including the 7-year market exclusivity period, in future 
U.S. trade agreements and that the intent of Congress is that TPA's 
negotiating objectives are consistent with that goal.

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