ULTRA

My colleagues, this reauthorization of the FDA's user fees will provide stability for the FDA's new product review as companies submit new and innovative drugs, medical devices, and biologics for approval.

I am especially proud that my bill, the Faster Access to Specialized Treatments, H.R. 4132, FAST, was included in the FDA Reform Act. FAST modernizes the FDA's accelerated approval pathway to reflect scientific developments that have occurred over the past 20 years. This will allow for new drugs for people suffering from rare diseases. There are 30 million Americans suffering from one of over 7,000 rare diseases, but only 250 currently have any treatment. FAST will save lives.

I am pleased also that the bill includes the EXPERRT Act, H.R. 4156. This will help the FDA consult with medical experts when evaluating drugs designed for rare diseases, such as cystic fibrosis. As the cofounder of the Cystic Fibrosis Caucus, I am glad we are finally providing this tool to the FDA.

I obviously support the passage of this bill.

Mr. Speaker, the Food and Drug Administration Safety and Innovation Act (S. 3187) is based on user fee negotiations between FDA and the prescription drug, generic drug, biologic, and medical device industry. This reauthorization of the FDA user fees will provide stability with FDA's new product review as companies submit new and innovative devices and drugs for approval.

This bill is the result of hard work and negotiations between industry and FDA, and the hard work between Republicans and Democrats, and between the House and the Senate. This bill is a true bipartisan, bicameral bill that will serve the American people well.

In codifying the User Fee Agreement, this committee has included additional provisions designed to address some of the defects of the regulatory structure and overreach by the FDA. Under my Chairmanship of the Oversight and Investigation Subcommittee, we held a hearing into FDA's regulatory efforts in the medical device space. During our hearing, many of the witnesses talked about the reluctance of FDA to approve devices and how FDA continually moved the goalposts for approval. I am glad that Title VI of this bill includes a significant number of reform provisions designed to bring certainty to the medical device field.

In addition to reforming approaches to medical devices through Title VI, the FDA's approach to rare diseases must also be modernized.

I want to take this opportunity to thank Dr. Emil Kakkis, Julia Jenkins, Harry Sporidis, Tim Perrin, Steve Stranne, everyone at the EveryLife Foundation for Rare Diseases, Pat Furlong, Nick Manetto, everyone at the Parent Project Muscular Dystrophy, and the other 150 rare disease groups that supported FAST and ULTRA. In 2011, I met with Dr. Kakkis who introduced me to two parents who had children with rare diseases and limited options as most rare diseases do not have treatments. One parent talked about his frustration at not having any treatments, except for a drug trial happening in Europe, not the United States. We talked about how we need FDA to properly address the issue of drug approval for the rare disease community, which led to examining the Accelerated Approval pathway and trying to modernize it. We developed the Unlocking Lifesaving Treatments for Rare-Diseases Act (ULTRA, H.R. 3737), which I introduced with my friend and colleague, Rep. Ed Towns, to nudge the FDA into using Accelerated Approval for rare diseases.

However, after further review of the law, FDA's history of usage of Accelerated Approval and the feedback we received from stakeholders, we realized that amending the law was not sufficient. Instead, we worked with all the stakeholders to rewrite the entirety of the Accelerated Approval statute. In March, Representative Towns and I introduced the Faster Access to Specialized Treatments Act (FAST, H.R. 4132). FAST updates and modernizes Section 506 of the Food, Drug & Cosmetic Act, and updates the Accelerated Approval statute to reflect two decades worth of medical sciences that has occurred since Accelerated Approval was first created. FAST will help FDA implement broadly effective processes for the expedited development and review of innovative new medicines intended to address unmet medical needs for serious or life- threatening diseases by using modern scientific tools.

The use of surrogate endpoints may result in fewer, smaller or shorter clinical trials without compromising FDA's existing high standards for safety or efficacy. Surrogate and clinical endpoints only need to be reasonable predictors of clinical benefit to support accelerated approval. They do not need to be validated or proven first. The changes made to current law permitting the Secretary to require validation of surrogates following accelerated approval is not intended to change FDA's long history of granting accelerated approval based on unvalidated, but predictive, surrogate endpoints.

Additionally, FAST includes explicit language for FDA to think about the challenges of rare diseases when developing their guidance and gives the rare disease community an opportunity to publically comment on FDA's draft guidance. FAST ensures that the voices of the 30 million Americans with a rare disease will be heard by FDA. There are about 7,000 rare diseases and only about 250 have any treatment. FAST will save lives, and give a voice to the voiceless; and I am glad it is in the final bill.

Lastly, the committee included the Expanding and Promoting Expertise in Review of Rare Treatments, (EXPERRT Act, H.R. 4156), a bill my fellow Co-Chairs of the Cystic Fibrosis Caucus and I introduced. EXPERRT will have the FDA consult with experts in rare diseases. This will ensure that FDA has access to the knowledge needed when dealing with drug approvals for diseases where FDA may lack subject matter expertise. As one of the Co-Founders of the Cystic Fibrosis Caucus, I am glad that we are giving this tool to the FDA. I also want to thank Stephanie Krenrich and the Cystic Fibrosis Foundation for all their hard work in developing EXPERRT.

I would like to submit these letters from the EveryLife Foundation for Rare Diseases and the Parent Project Muscular Dystrophy into the Record.

S. 3187 is a good bill that will help new drugs and new medicines get into the market and be available to patients. I support passage of the FDA Safety and Innovation Act.

Parent Project

Muscular Dystrophy,

Hackensack, NJ, June 20, 2012.

Hon. Cliff Stearns,

U.S. Congress, Washington, DC.

Rayburn House Office Building,

Dear Representative Stearns: On behalf of all patients and

families living with Duchenne muscular dystrophy--the most

common form of muscular dystrophy and the most common lethal

genetic condition diagnosed in childhood--Parent Project

Muscular Dystrophy (PPMD) would like to express its deep

gratitude for your efforts to include provisions of deep

interest to the rare disease community in S. 3187, the Food

and Drug Administration Safety and Innovation Act. The final

user fee reconciliation package between the House of

Representatives and Senate includes a number of measures that

will accelerate the Food and Drug Administration (FDA)

process of reviewing potential therapies for serious life-

threatening conditions like Duchenne, will ensure that the

patient voice has a seat at the table when key decisions are

made, and will incent industry to develop treatments for

pediatric rare diseases.

As you know, Duchenne muscular dystrophy exemplifies the

challenges faced by many patients and families afflicted by

rare diseases. It is a fatal condition with most patients not

living past their late 20s, and the only approved therapies

are steroids, which cause significant complications long-

term. With nearly 20 potential therapies in various stages of

clinical trials, our community is hopeful that better times

are ahead, and we recognize that a more efficient FDA attuned

to the needs of the rare disease patient population is

critical to our success. Again, we are most appreciative of

your efforts to ensure that the above mentioned provisions

were included in the final legislation. On behalf of Duchenne

and the broader rare disease community, thank you for your

leadership and support.

Sincerely,

Pat Furlong,

Founding President and CEO.

Mr. Speaker, I move to suspend the rules and pass the bill (S. 3187) to amend the Federal Food, Drug, and Cosmetic Act to revise and extend the user-fee programs for prescription drugs and medical devices, to establish user-fee programs for generic drugs and biosimilars, and for other purposes, as amended.

Mr. Speaker, I ask unanimous consent that all Members may have 5 legislative days in which to revise and extend their remarks and insert extraneous material in the Record.

Mr. Speaker, I yield myself 2 minutes.

Mr. Speaker, I want to thank Mr. Waxman, Chairman Harkin, Senator Enzi, and Members on both sides of the aisle in both the House and the Senate who played a role in this process. S. 3187 is a reflection of the hard work put in by both Members and staff, and of everyone's willingness to put partisanship aside to look at the issues together. Because of that outstanding dedication, we have a bill today that will make a real difference in the lives of so many patients and provide much-needed support for innovators across our great country.

At the outset of this Congress, I set a goal of enacting this bill by the end of June--and here we are, well before the clock expires for this month--in order to provide certainty for American patients and innovators. I never lost confidence that we could deliver the bipartisan reforms we needed, and I am so proud that we will accomplish that goal.

Mr. Speaker, this is a jobs bill, and it's a medical innovation bill. And as we put this package together, our goal was to improve the predictability, consistency, transparency, and efficiency of FDA regulation. These reforms will help get new treatments to patients more quickly. They will help us not only keep jobs in Michigan and all across the country, but also to create new ones. In order to get it right, we turned to patients, innovators, and job creators who provided firsthand experience of how the current system is broken. And we included many of their suggestions in the bill.

This bill includes significant accountability and reform measures designed to hold the FDA responsible for its performance. The measure includes independent assessments of FDA's drug and device review process. It also includes requiring quarterly reporting from the device center so we don't have to wait a year to find out FDA's progress. The bill is about patients, and that's why so many patient advocates have spoken out in support of these reforms. Whether it is steps that we took to support treatments for rare diseases or mitigate drug shortages or speed up the approval of devices that will improve a patient's quality of life, these are steps that will make a real and significant difference.

They're going to keep the U.S. at the forefront of medical innovation where we belong.

This bill is just the first step. This bill provides the resources and the game plans so that FDA can improve its performance.

I yield myself an additional minute.

It is now up to the FDA to execute that game plan. And I give my commitment today that our committee will continue to monitor and hold the FDA accountable for its performance. So, together, the Members of the House and the Senate have produced a bill that is a win for American patients, innovation, and job creation.

Before I conclude, I would like to recognize Warren Burke and Megan Renfrew from the Legislative Counsel's Office for their tireless work. The role of Legislative Counsel often goes unnoticed. I also want to appreciate our staff, starting with our staff director, Gary Andres, for pushing this legislation over the finish line; Clay Alspach, on the majority staff; Rachel Sher, on the minority staff; and in particular, Ryan Long, the chief counsel for the Health Subcommittee.

This bill, when it becomes law, patients will benefit from faster, newer, and better treatments, and American workers will keep us on the cutting edge of medical innovation.

I reserve the balance of my time.

Mr. Speaker, I yield 1 minute to the chairman emeritus of the Energy and Commerce Committee, the gentleman from Texas (Mr. Barton).

(Mr. BARTON of Texas asked and was given permission to revise and extend his remarks.)

Mr. Speaker, I yield 2 minutes to the distinguished chairman of the Health Subcommittee, the gentleman from Pennsylvania (Mr. Pitts).

Mr. Speaker, I yield 2 minutes to the gentleman from Texas (Mr. Burgess), the distinguished vice chair of the Health Subcommittee.

Mr. Speaker, I yield 1 minute to the distinguished gentleman from Florida (Mr. Stearns).

Mr. Speaker, I yield 1 minute to the distinguished gentlewoman from North Carolina, the vice chair of the Energy and Commerce Committee, Mrs. Myrick.

I yield 1 minute to the distinguished gentleman from Pennsylvania, Dr. Murphy.

Mr. Speaker, I yield 1 minute to a member of the committee, the distinguished gentleman from California (Mr. Bilbray).

Mr. Speaker, I yield 2 minutes to the distinguished gentleman from Georgia, Dr. Gingrey, a member of the committee.

Mr. Speaker, I yield 1 minute to the gentleman from New Jersey (Mr. Lance), a member of the committee.

Mr. Speaker, I yield 1 minute to the gentleman from Kentucky (Mr. Guthrie).

Mr. Speaker, I yield 1 minute to the gentleman from New Hampshire (Mr. Bass), a member of the committee.

Mr. Speaker, I yield 1 minute to the gentleman from Minnesota (Mr. Paulsen).

Mr. Speaker, may I ask how much time remains on each side?

Mr. Speaker, I have no further requests for time. So if the gentleman wants to close, then I will close.

Thank you, Mr. Speaker.

Mr. Speaker, I just want to say that with all of the positive comments here, this bill was not a piece of cake. There was a lot of hard work on both sides of the aisle, particularly by the staff on both sides of the aisle. Again, I want to cite Clay and Ryan on our staff.

But let's face it: All of us particularly involved on the health side of the issues, as we meet with different folks afflicted with different diseases, we want to find a cure. And it would be great to find that cure here in America because we have outstanding pharmaceutical industries that have the talent and the staff to work with the different departments, whether it be the NIH, the CDC, certainly the

Mr. Speaker, I yield myself 3 minutes.

Today, the House considers a bill that represents a significant bipartisan and bicameral achievement.

On May 30 of this year, the House passed its user fee legislation by a dramatic vote of 387 5. That bill was a strong one, but through our collaborative process with the Senate, we have made it even better.

It has been a pleasure to work not only with Mr. Upton, Mr. Pitts, Mr. Pallone, and Mr. Dingell, among many involved House colleagues, but also with our Senate colleagues, Senators Harkin and Enzi.

When we began this process, there were divergent views on the various issues contained in this bill. But we worked together and found ways to bridge our differences in a fashion that protects patients and fosters innovation.

This legislation contains many provisions that are critical to the functioning of major parts of the FDA. We reauthorize the FDA's drug and medical device user fee programs which will provide resources to enable the efficient review of applications and give patients rapid access to new therapies. We're also reauthorizing two pediatric programs which foster the development and safe use of prescription drugs in children.

This year, we're establishing two new programs to help the FDA speed up their review of new generics and biosimilars. These provisions illustrate our bipartisan commitment to ensuring a vibrant generic marketplace. All

of us will see the benefits when more low-cost generics are on the market.

One of the most important improvements to the House-passed bill is in the area of antibiotics. We accepted the Senate language that directs incentives for the development of antibiotics toward serious and life- threatening infections.

This bill also includes provisions to modernize FDA's authorities with respect to the drug supply chain. Today, 80 percent of active ingredients and bulk chemicals used in U.S. drugs come from abroad and 40 percent of finished drugs are manufactured abroad. FDA has been trying to keep pace with this increasingly globalized drug supply change using an outdated statute. This legislation will give the FDA critical new tools to police this dramatically different marketplace.

We have also worked to address the area of drug shortages, which is a complex and multifaceted problem, but this legislation takes some sensible first steps.

I want to thank my colleagues on both sides of the aisle and their staffs for the hard work they've put into making this a strong bipartisan bill. I particularly want to thank Mr. Pallone and Mr. Dingell's staff members, Tiffany Guarascio and Kim Trzeciak, as well as Mr. Upton and Mr. Pitt's staff, Ryan Long and Clay Alspach.

I yield myself an additional 30 seconds.

Warren Burke and Megan Renfrew have done tremendous work on this bill. I'd like to express my appreciation for their efforts. I want to thank my own staff: Karen Nelson, Rachel Sher, Eric Flamm, and Arun Patel.

The American public will benefit from the provisions of this bill. The FDA will have the resources to remain the gold standard for the future. This is an important bill, a good one. I urge its support.

I reserve the balance of my time.

Mr. Speaker, at this time, I'd like to yield 3 minutes to the gentleman from New Jersey (Mr. Pallone), the ranking member of the Health Subcommittee, the subcommittee that was responsible for this legislation in its first instance.

I ask unanimous consent that Mr. Pallone be permitted to manage the rest of the time on our side of the aisle.

Mr. Speaker, this is a good bill. I urge my colleagues to support it. I rise in strong support of it, and I urge my colleagues to join.

This legislation enjoys broad bipartisan support on both sides of the Capitol and from industry and patient groups. We should also be proud of the work we have done to get it here today.

I would observe that it has been done because the Members worked together in the finest traditions of this body. And I'm also proud of the work that my colleagues on the committee and the staff have done on this matter. I was pleased to work with them to include strong upstream drug supply chain provisions, something that's been a long priority of mine.

I'm also pleased that, for the first time, commercial importers will be required to register, so we'll know who's bringing what in and whether it's safe or not. There will also be parity between inspections of domestic and foreign drug facilities, something which is a major problem because foreign facilities and foreign manufacturers now import much into this country, much of which is unsafe and improperly inspected.

FDA will be able to maintain a practice in which they will detain and destruct counterfeit drugs and those which are unsafe or intentionally or otherwise adulterated, and they will be able to impose increased penalties on those who adulterate these drugs and pharmaceuticals.

These provisions, which mirror safety provisions in my drug safety bill, will equip FDA with the authorities it needs to better oversee our increasingly globalized drug supply chain and will give American families comfort that the pharmaceuticals that they are taking are safe, and help to deter and to respond to any future heparin-like incidents which killed some 80 Americans and hurt thousands more.

While I am disappointed we were unable to come forward with a consensus on a national track-and-trace standard, it's my hope that we will continue to work on this in coming days. And I want to commend my colleagues, Mr. Matheson and Mr. Bilbray, for the fine work they have done on this matter.

I've also been working on this issue for many years, and we've come closer than ever before to finding a consensus. Given additional time, I think we could have resolved this issue; but because of time pressures, we were not able to.

I also want to thank my friends, Mr. Upton, Mr. Harkin, Ranking Members Waxman and Enzi, and their staff for the hard work they did to send this critical bill to the President before July 4. I also want to thank Kimberly Trzeciak of my staff for her diligence on the supply chain provisions and other matters.

I urge my colleagues to support this bill. It will be something of which we will be proud. It will confer much safety on the American people in areas of very substantial danger; and it will see to it that, to a modest degree at least, the industry-supported provisions, including those which involved the collection of fees, will begin to work for the benefit of the American people.

Mr. Speaker, recently I met Jill Wood from my district in Brooklyn. I was very moved by this meeting as she described the struggles of her son, who is diagnosed with a rare genetic disease known as Sanfilippo syndrome.

Before we met, I was not familiar with this particular disease, but she touched my heart to hear about her child's courage. Every day he has to overcome physical disabilities that make it almost impossible for him to complete a very simple task that we complete with ease. I was inspired by the strength of their family and the bond that they share.

I have long been a strong advocate for rare disease research and development. In fact, this is why I am working with my colleague from Florida, Congressman Stearns, on H.R. 3737, the ULTRA Act. This bill would codify the flexibility the FDA needs to encourage development of treatments for rare diseases like Sanfilippo syndrome.

It is our duty, as Members of the United States Congress, to come together and support measures that aid the rare disease community. Imagine being afflicted with a disease your physician has never heard of and has no idea as to how to treat it. Can you imagine the devastation this would cause to your family?

We must provide the National Institutes of Health with additional funding to support the important research for orphan and rare diseases. We must also give flexibility and support to the FDA to help the agency bring potential cures and treatments to the market much sooner.

How long must we wait and continue to suffer until lifesaving treatments are available?

Nearly 30 million Americans are affected by 7,000 rare diseases. We must do everything in our power to support education, advocacy, research, and patient assistance to bring this number down. Imagine the families out there watching their loved ones suffer because we have not yet provided enough support for this cause.

We are a great Nation of innovation, but that innovation and drive only goes so far without the proper support coming from the government. Our constituents need to know that we hear their needs and that, as their elected officials, we are determined to make available the resources that will support them.

The next time I speak to a family affected by rare diseases, I want to be able to look them in the eyes and tell them that we have helped, that we made available the means necessary to support lifesaving research and development, we care and we will do everything in our power to ensure that everyone has the chance to live full, healthy, and prosperous lives.

Thank you, Jill, for bringing this to my attention.

I urge my colleagues to support the ULTRA Act and increase funding for rare disease research and development. It is so important that we do everything possible to be able to bring the numbers down.