PAUL: "Mr. Chairman, I want to thank you for the opportunity to appear before the Committee today, and I want to thank you for holding this exceptionally important hearing on the pediatric drug testing incentives contained in the Food and Drug Administration Modernization Act (FDAMA). I want to thank Senator Dodd, a member of this Committee, who continues to be one of the hardest-working and committed champions of this issue. I want to say a special word of thanks to Senator DeWine, a former member of the Committee, who is one of the architects of the original law and now a key player in the effort to renew it. And I want to thank the Ranking Member, Senator Kennedy, whose outstanding record on children's issues is well known throughout the country.

I also want to thank the other members of the Committee, including Senator Clinton, who was extremely active in pressing for this provision. For years we could not get results on this crucial issue. It was not until some of the leaders in this room took a stand on behalf of our children that things started to change.

Most people know me as an actor and a filmmaker. I come before you today wearing two other hats: Chairman of the Board of the Elizabeth Glaser Pediatric AIDS Foundation, and that of a parent who, along with my wife Tracy, wants only the best for our three and a half year-old Zoe, and our sixteen year-old son, Jake.

The Foundation, founded by my late wife, Elizabeth, funds pediatric research and the training of researchers, and it fights for the best public policy for our children. Since its inception, the Foundation has raised more than $120 million dollars to help children with HIV/AIDS and other serious and life-threatening illnesses.

For more than a decade, we focused exclusively on pediatric HIV/AIDS in the United States . In recent years, our role has broadened. We are now funding programs in 12 developing nations and at 70 sites to prevent mother-to-child transmission of HIV through simple, affordable preventive interventions. Because of the pediatric drug testing law, I am pleased to report that industry has completed pediatric studies on two HIV/AIDS drugs -- Epivir and Ziagin. There are 12 more HIV/AIDS drugs in the pediatric testing pipeline. The question is how fast they will be tested and made available to children throughout the world, for the ugly truth is that AIDS is a pandemic that is still in its early stages. Its future impact on the world isn't even measurable.

Of more significance to today's hearing, we have established the Glaser Pediatric Research Network. The Network is a collaboration of 5 premier academic medical centers whose aim is to conduct research on the relationship of all serious life-threatening pediatric illnesses to each other, with the understanding that what we learn from one disease can better help us learn about other diseases. This is a concept that will speed up our discovery process and bring faster answers and relief to all children. Several years ago, when the Foundation fought hard for pediatric testing regulations and incentives, we were focused on the importance of testing HIV/AIDS drugs, but we understood the importance of the issue to all children. Now that we are expanding our research to include a range of childhood diseases, we are determined to ensure that all children have the same safety and efficacy information that adults take for granted. This should be standard practice whether the drug is for asthma, allergies, HIV/AIDS, or the next pediatric disease Mother Nature is most assuredly going to send our way.

My interests as a parent are the same as other parents. I am convinced that the policies of the Federal Government should always protect children's health care. In a system such as ours, where those with power and money often get the most attention, there has historically been too weak a voice on behalf of our children. Pharmaceutical companies have regarded children as representing too small a market share. It is far more profitable to create and sell to an adult market of symptoms rather than the long-range investment in preventative research and care. Never mind that a stronger populace means a stronger country and ultimately, a stronger economy.

In the face of an addiction to "self-serving" in our society, it becomes increasingly important that someone needs to be the good parent. Someone needs to state the moral imperative. This issue goes right to the root of what government is all about. I can give you facts and figures and testimonials, as will many of the expert witnesses who will testify today. But this issue comes down to who will take responsibility for the future of our children. This is not an issue about votes or politics or parties or pressure groups. It is an issue about the responsibility of the government to stand up and do something important on behalf of our children. It is about seizing the moral high ground and holding it. And it is about respecting the importance of governing on behalf of the people. I am sure you understand this, or else you wouldn't be here. You have done a wonderful job. I simply believe that this is the core issue, and it can never be said enough.

I am sure each of you knows a personal story involving the senseless tragedies that afflict our children. My story is fairly well known. I lost a daughter, Ariel, and a wife, Elizabeth, to AIDS. I have a 16-year-old son who is infected and lives in a state of vulnerability no person should have to endure. And he was lucky. Because of the dearth in research and testing of pediatric drugs, my daughter Ariel never had that option. What little help came, came too late.

Until tragedy struck my family, it never occurred to me that drugs tested in adults had not been tested for use in children. But I soon found out, as my daughter Ariel struggled to survive, that life-prolonging AIDS drugs had not been tested for children.

Why does it matter so much? Because children are not miniature adults. Children in different age groups from newborns to toddlers to young children to adolescents respond differently to medicines than adults do. Without any information on how they will respond, doctors are forced to rely on anecdotal information or guesswork when prescribing many drugs to kids, or to withhold treatment completely.

Ariel's condition worsened, and she later died, in part because lifesaving AIDS drugs hadn't been studied and were unavailable to children at the same time as adults. Tragically, no one knew the proper dosage, or the possible side effects, or even the right way to administer many drugs for HIV/AIDS to children. In fact, we couldn't even get access to the right drug at first.

Sometimes a totally different formulation is needed; the old method of crushing a pill into applesauce simply won't do. That was the case with Ariel, who did not respond to the pill form of one leading AIDS drug and only showed improvement when she had access to an intravenous form of that drug. Unfortunately, it was too late to make a real difference.

Without testing, the dosage is sometimes too small to be effective. Without testing, the drug may produce unknown adverse side effects in children. And without testing, the results are sometimes tragic.

In the late '50's, many newborns died when they were given a drug for bacterial infections that was tested only in adults. As recently as 1999, seven newborns who were given the widely prescribed drug erythromycin became seriously ill and required surgery because the drug had not been tested in this age group. These tragedies occurred because, until recently, a shocking 80 percent of all drugs had never been tested for use in children. This included drugs for such conditions as seizures, severe pain, Tuberculosis and HIV/AIDS, among others. Even though the federal government and children's organizations called on drug companies to improve their record for decades, the companies were hesitant to conduct studies in children.

Mr. Chairman, isn't it pretty straightforward? If you are a parent, and you find out your child is seriously ill, you want to know that the medications for that illness are safe and effective for your child. Every parent in that situation wants to know that their child will receive the best possible care, and rightly so. And this Committee - this federal government - has the power to ensure that the needed pediatric studies are conducted.

That is why we are all gathered here today. In 1997 you passed a law to help change things - to provide six months of additional exclusive sales for manufacturers that test their drugs in children. The law has now been in place for several years, and it is your job to assess its record.

The most important question you need to answer is: "Has it worked? Did it get the job done?"

Mr. Chairman and Members of the Committee, let me make your job very simple. "Yes, it has worked, and how!"

The answer is all in the numbers. Seven years prior to enactment of this law, the drug manufacturers promised 70 pediatric studies of marketed drugs but conducted only eleven. In less than three years since enactment of the law, more than 400 studies have been requested by the FDA, and the drug industry is already acting on more than 300 of these requests. The study process is already complete for 33 products (with 28 of these already receiving the right to 6 months of additional, exclusive sales), and dozens more will be completed in the near future.

Let me tell you how good that is: it's exceptionally good. This is a uniquely successful law. For me, there is no question as to whether the pediatric testing incentives should be renewed. Is there a choice? Can we really go backwards and have less testing of pediatric drugs? Can we really return to our very recent history when 80% of all drugs had never been tested for use in children? Can we return to a time when drug companies had done a paltry 11 studies over seven years?

True, the advances we have seen have all been accomplished by ensuring an incentive for the drug companies. This is an incentive that has become such a windfall for blockbuster drugs that six months of sales exclusivity has produced increased sales in one instance of close to a billion dollars. It is an incentive that can and most likely will have economic repercussions. It is ironic, for it begs the question of how much more profit the drug companies will require the next time around. However, as I see it, that is all part and parcel of buying into our system that stresses money and power and where there is very little room for mandating a moral imperative. This is the deal we made, and until people are prepared to acknowledge the Federal Government's obligation to protect the common good, this is the deal we will live by. There is no choice. We have to protect our children. That being said, one reasonable way to address the criticism that the incentive is excessive would be to consider a more limited incentive for drugs with extremely high sales.

The good news is that this law works. It has and continues to accomplish a great deal.

The Foundation recently sent a letter to the members of this Committee, urging their strong support of reauthorization of the pediatric drug testing incentives. Several Board Members of the Foundation and I signed the letter. These included: Dr. David Kessler, the former Commissioner of the FDA and current Dean of the Yale School of Medicine; Dr. Philip Pizzo, the Dean of the Stanford School of Medicine and a leading national pediatric researcher; and Ms. Kate Carr, the CEO of the Foundation. A copy of this letter is attached to my testimony, and I ask that it be placed in the permanent record so that the views of Drs. Kessler and Pizzo, who are national experts on pediatric policy, may also be available to the Committee.

In a nutshell, it says this law deserves to be renewed, and it deserves to be renewed this year, before its scheduled expiration date. To allow it to expire, or even to delay the renewal, would threaten the future of one of the most successful federal initiatives ever to improve children's health.

This law is so successful, in fact, that its scheduled renewal should be unanimous by acclaim without question. But as all of us know, politics is not always that simple in the nation's capital. In the press and in other quarters, the law has come in for criticism, accused of being too costly, and ironically, of endangering children's safety. I want to spend a few minutes setting the record straight.

The testing of all drugs poses risks, whether it is done in children or adults. Those risks are minimized when studies are properly designed, when there is careful attention to informed consent, and when there is alert oversight by institutional review boards (IRBs). Now that the Glaser Pediatric Research Network is up and running, we have brought together some of the most skilled and caring physicians in the country, each of whom have extensive experience in conducting clinical trials with children. And if you speak with these researchers, as I have, you will learn that children in clinical studies are actually safer, due to careful monitoring by the researchers, than children taking untested medicines without close supervision.

Of course, vigilance, specified and accounted for by the pharmaceutical companies, is of paramount importance. But the alternative -- an ongoing national experiment with giving untested drugs to millions of children, or the tragedy of denying children access to lifesaving treatments is even riskier than involving children in clinical trials. The fact is that recent newspaper reports on problems represent rare exceptions, rather than the norm, in pediatric testing.

With respect to costs, what I am going to say will probably offend both sides of the issue. First, much of the harsh criticism of the law's costs that is now circulating does not stand up to close scrutiny. The FDA estimates, in its recent report to you, that consumer prices for drugs are increased by one half of one percent each year as a result of the pediatric drug testing provision. Clearly, we have to consider whether the incentive is too large for some of the top-selling blockbuster drugs. But again, the alternative is unacceptable. What parent wouldn't pay a little extra to ensure their child's well being?

And while it's harder to quantify, since no one is keeping detailed statistics, better care of children due to better information on the use of drugs results in lower hospitalization and health care costs for the economy and the taxpayers. In fact, the FDA estimates that, for five childhood diseases alone -- asthma, HIV/AIDS, cancer, pneumonia, and kidney infections -- the savings might be more than $200 million annually. The real savings associated with improved care for the full range of illnesses affecting children, is naturally much higher.

A related criticism suggests that drug companies are only focusing on the highest selling "blockbuster" drugs in carrying out pediatric studies. But a close look at the law's results demonstrates that this is not true. Only two drugs among the 28 that have received six months of additional exclusive sales are in the top 10 in annual sales and qualify as outright "blockbusters". The majority, however, are scattered among the top 200 drugs in annual sales, and 9 don't even make the top 200 list at all.

For example, Epivir, one of the HIV/AIDS drugs I mentioned, is number 102 on the list of the top 200 drugs in annual sales, according to Pharmacy Times data. The HIV/AIDS drug Ziagin is not on the list of the top 200 drugs in annual sales. Even when we give high-selling drugs 6 months of additional sales, it's erroneous to imply, as some have, that studies of these drugs don't help children. Many are of enormous importance to children, who suffer from the full range of illnesses that adults do. For example, Zantac, the well-known drug for acid reflux, is of crucial importance in preventing reflux among infants, who can suffer lifelong damage to their lungs if left untreated. As a result of the pediatric drug testing provision, we now have information on how to use Zantac in newborns, who are most vulnerable to life-threatening damage from this condition.

But there is one aspect of the cost question that deserves careful attention from this Congress. It does seem clear that the profits from some of the top-selling blockbuster drugs are unjustifiably large. At the Foundation, we think that you consider a cap or an "excess profits clause" to limit the extent of the incentive for blockbuster drugs. As to what the cap should be, or the best way to implement it, I don't pretend to have the answer -- we all need to continue discussions on this point. However, as an organization that has worked to make sure that HIV/AIDS care and treatment services are available to all, we are especially sensitive to the fact that drugs must be affordable to those who need them.

The bottom line, however, is that we need to get the pediatric drug testing provision renewed this year. It is clear that it can be improved and made more specific in order to ensure simple and common sense changes that will create added benefits for our children. That is what Senators Dodd and DeWine have done with their Better Pharmaceuticals for Children Act, which we firmly endorse. And we hope that all Members of the Committee will join us in supporting the following important changes that they have included in their bill:

- Study of Generic Drugs: The current law does not provide any mechanism for the study of generic medicines, even though they are widely prescribed for children. However, the Dodd-DeWine bill will ensure that generic drugs of importance to children are also studied to ensure their safety and effectiveness. It does so by creating a public research fund that will result in much-needed safety and dosing information for these medicines.

- Swift Label Changes: Under current law, drug companies are granted six months of additional exclusive sales, but they sometimes fail to follow through with swift label changes that provide complete pediatric safety and dosing information. The Dodd-DeWine bill insists on prompt label changes within a reasonable time period -- one year at the most -- and it gives the FDA strong recourse if the companies do not comply.

- Dissemination of Study Results: The results of pediatric studies are of great use to pediatricians and other health professionals. So the Dodd-DeWine bill ensures that a wide range of information from pediatric studies will be disseminated to pediatricians and can be used in real-life treatment.

- More Attention to Neonatal Studies: Additional studies among neonates are important, but often can't be planned or accomplished safely until studies in older age groups are complete. The Dodd-DeWine bill calls on the FDA to include neonatal studies in their original request to industry whenever it is appropriate.

- Creation of an Office of Pediatric Therapeutics: The Dodd-DeWine bill enhances the importance of pediatrics within the FDA by creating a new Office of Pediatric Therapeutics, responsible for pediatric drug testing and other pediatric issues. We are intent on providing the FDA with additional resources to carry out its pediatric mission.

Further, while I congratulate the brand name drug manufacturers for their role in making this provision a success and for doing the studies that are needed to improve our children's health, it is time to ask them to make a larger contribution to this process. The Dodd-DeWine bill appropriately calls on the companies to pay user fees for the FDA's administration of the pediatric drug testing law.

There are several other ways in which the companies might participate more, and I call on the drug companies and Congress to work with us to search for the best way to make these changes happen. First, we do need to find a way to limit the incentives for the most lucrative drug products, and the drug companies should acknowledge this and work toward a solution that is easy to administer. Also, we need to continue to look at ways in which the brand name companies could contribute to the research fund for the study of generic drugs contained in the Dodd-DeWine bill, so that the research fund would not be as dependent on yearly appropriations.

The Foundation understands that a meaningful incentive must be maintained to ensure that the needed pediatric studies are completed. At the same time, much is asked of those to whom much is given. Drug companies should pitch in more than they are now.

The choice is clear. For those of use who can affect this choice, it is imperative. My daughter, Ariel, and hundreds of thousands of other unfortunate children didn't have that choice. They had to suffer the distinction of being deemed "unprofitable" and of less importance by a system that too often is reactive and can only think in the short term. So I ask you, what's to become of our children, or your children? I am sure you will agree with me when I offer that they are our greatest, most precious resource, and for every reason under the moon and sun they need to be taken care of.

I am very thankful that my son Jake has been fortunate enough to benefit from this law. He's doing well now, and if we can maintain a focus on critical HIV research, he stands to have a full life. And I am very thankful that this committee has convened to study how we can extend and improve this pediatric drug testing provision that has already proved its ability to dramatically improve the amount of information on essential treatments for children.

As the Chairman of the Board of the Elizabeth Glaser Pediatric AIDS Foundation, I call on you to support swift reauthorization of this law, with appropriate changes to make it even more effective for our children.

And as a parent, I call on you to prevent future tragedies -- of children harmed and families ripped apart because we weren't willing to make drugs as safe and effective for our children as we insist on doing for ourselves.

The law you passed in 1997 has been a resounding success, and we now have the opportunity to build on that success. Thank you for letting me, as a father and as a representative of the Foundation, contribute to your effort. "31

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Last updated: June 6, 2010