[Congressional Record (Bound Edition), Volume 163 (2017), Part 9]
[Senate]
[Page 12610]
[From the U.S. Government Publishing Office, www.gpo.gov]




                        FDA REAUTHORIZATION BILL

  Mr. HATCH. Mr. President, I wish to speak on the importance of 
maintaining a strong Food and Drug Administration. Today we approved 
the user fee reauthorizations for the FDA. We have done the important 
work of passing these essential user fee agreements out of the 
committee and have now debated and passed them on the Senate floor.
  The HELP Committee is filled with strong personalities. These 
personalities reflect the passion and diversity of opinion of millions 
across our nation today. While we may disagree on certain policies, 
most of us can agree that funding the drug, device, and biologic 
centers of the FDA is essential.
  Our future scientific endeavors require a strong FDA that 
communicates openly with the industry that it regulates, and this 
agreement sets up protocols to achieve that goal. A strong FDA also 
requires clear steps for product review, and only through such 
deliberative actions can we bring more competition and clarity to our 
drugs, devices, and biologic products.
  I have championed multiple provisions in this bill, but there are two 
I would like to highlight today. First, there is the counterfeit and 
diverted drug language. This language makes importation neither harder 
nor easier. In fact, it doesn't change importation laws at all. Rather, 
it protects and strengthens the drug supply chain by simply increasing 
penalties for criminals that choose to divert drugs into the United 
States or sell counterfeit drugs.
  Current penalties for illegally diverting drugs in the United States 
change arbitrarily based on the location where the drugs are 
manufactured. Our bill addresses this disparity by enforcing the same 
penalties for diverting drugs made outside the United States as for 
those made inside the United States. To ensure public health and to 
enhance consumer confidence, it is critical that Congress eliminate 
these differing penalties for certain types of diversion and 
counterfeiting.
  The second provision I wish to call attention to is a bipartisan 
proposal from Senators Bennet, Burr, and Casey. These fine Senators 
have joined together to address how clinical trials are designed early 
on in their development. By offering guidance on how to include the 
intended patient population, especially those with rare diseases, drug 
sponsors can craft trials that generate useful data for health 
professionals and patients to review.
  This bill builds upon the success of other expanded access provisions 
that put the patient at the heart of the healthcare system. FDA does 
consummate work when reviewing products for market, but including a 
wider patient mix, when appropriate, will enable phase I, II, and III 
trials to be more complex. I strongly believe that accurately 
portraying the intended patient population in a clinical trial is key 
to ensuring that drugs are both safe and effective.
  I support this bill, but I also feel compelled to speak for a moment 
on the OPEN ACT. While not included in the package being debated today, 
the provisions of the Orphan Product Extension Now Accelerating Cures 
and Treatments Act--a bill I introduced this Congress with Senator 
Menendez and last Congress with Senator Klobuchar--would promote new 
therapies for rare diseases.
  New therapies are essential to help the nearly 30 million Americans 
suffering from a rare disease or condition. Because complex rare 
diseases with small patient populations have limited market potential, 
there are few economic incentives to develop new drugs targeting those 
diseases. While there are 7,000 rare diseases that impact millions of 
Americans, 95 percent of these diseases have no treatment. All too 
often, misconceptions about the dangers of exclusivities keep 
bipartisan measures from being introduced. We must remain focused, 
however, and remember that, each day we delay in getting treatments to 
the rare disease community, patients and their families suffer.
  Drug companies possess considerable scientific knowledge on drugs 
that have already been approved for common diseases. Some of these 
drugs could be repurposed for the treatment of rare diseases. 
Repurposing drugs is faster, less expensive, and generally less risky 
than traditional drug development.
  The OPEN ACT would encourage such repurposing by providing an 
additional 6 months of market exclusivity to drugs that are repurposed 
and approved by FDA for a rare disease or condition.
  Finding legislative ways help medical innovators treat rare diseases 
has been among my top priorities for over 30 years, since I first 
championed the bipartisan, bicameral Orphan rug Act in 1983. The OPEN 
ACT is a natural next step in expanding that effort to close the gap 
for rare diseases for which we have yet to develop treatments. In 
addition to increasing the number of rare disease therapies, this 
legislation will boost innovation and provide safer options for rare 
disease patients using drugs off-label. My bill enjoys enormous support 
with the backing of over 225 rare disease organizations and patient 
advocacy groups, not to mention overwhelming support from academic 
medical and research centers.
  Although this provision is not in the bill before us, I have had 
assurances from Chairman Alexander that he will continue working with 
me and the cosponsors of this bill to see it become law. I have spoken 
to Ranking Member Murray in the past about it, and I remain optimistic 
that my colleagues share my concern for the rare disease community and 
are willing to advance this legislation in the future. I would like to 
thank the chairman and ranking member for their dedication to children 
and families in need.
  I wish to conclude by reminding my colleagues that many of the 
debates that have led to the bill before us today are the culmination 
of years of experience. When I led the effort to pass what became 
Hatch-Waxman, the true impact of that law dwarfed even our loftiest 
hopes. Hatch-Waxman was a resounding success because Senators and 
Congressmen worked together to improve our country's situation and 
reduce barriers to market entry. This bill is vital to continuing that 
goal, and I am pleased to see where the negotiations have landed.

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