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Jane E. Henney, M.D. - Pediatric Academic Societies and American Academy of Pediatrics

This text contains Dr. Henney's prepared remarks. It should be used with the understanding that some material may have been added or deleted during actual delivery.

Remarks by:
Jane E. Henney, M.D.
Commissioner of Food and Drugs
U.S. Food and Drug Administration

Pediatric Academic Societies
American Academy of Pediatrics
Joint Meeting
7th Annual Public Policy Plenary Program
Pediatrics in the New Millenium: Compelling Issues in Public Policy
Increasing Pediatric Access to Medical Therapies

Boston, MA
May 15, 2000

Good morning. I'm pleased to be here today to give you an update on FDA's activities in the pediatric area.

The assigned title of my talk is "Increasing Pediatric Access to Medical Therapies." But when you think about it, that title misses the point. Most of the medical therapies that are available to adults have long been made available to children, off-label. The real problem is that for far too long, we haven't had enough scientific data to support their use in the pediatric population...a population made up of distinct subgroups, from infants to teenagers, each with its own biological and physiological characteristics.

This lack of data has led to a paucity of product labeling that traditionally (at least traditionally for those of us who frequently treat adult patients) tells physicians whether these products can be used safely. Physicians have had-literally(little or no information to go on regarding appropriate dose for children of any age or health status, or how to increase the odds of getting the desired results. Without the needed data, pediatricians and family physicians who care for children have been forced to work in the dark to some degree, extrapolating data from adults to children.

However, as you're all well aware, in the past there's been a reluctance to study children, and I think most of us recognize the reasons for this--the difficulty in determining and measuring endpoints in children, increased costs, liability concerns, the relatively small market, and the difficulty in recruiting patients for clinical trials.

The dogma of the day has been that it is not ethical to do drug testing in children. A more compelling position is that it is not ethical to provide drugs to children without testing. FDA has had a role for years in encouraging pharmaceutical companies to do so-to seemingly little avail.

Things are improving. Recent changes in legislation and regulation were designed to address the lack of adequate pediatric information in the labeling of products intended for use in children.

These changes were intended to encourage sponsors to develop the data needed to support the safe and effective use of drugs and biologics in the pediatric population.

And recent actions give us confidence that the future will be different for pediatricians, patients and their caregivers.

We must continue to work together to learn more about how to use drugs safely and effectively in children. To do less would be to jeopardize the health and well-being of this very special population. All of us in this audience must use both our control and our influence to make a greater effort to include children in clinical trials, and develop the data needed for the safe and effective use of medical products in children. This information can then become a part of product labeling, so that physicians can make consistent and science-based decisions on their use.

Let me quickly review the most recent changes.

In 1997, Congress passed the FDA Modernization Act. Section 111 (better known as the Pediatric Exclusivity provision) created an incentive for drug sponsors to conduct pediatric clinical trials. When studies are conducted as requested by FDA, the company will receive an additional 6 months of marketing exclusivity for products that have existing exclusivity or patent protection.

In 1998, FDA published the "Pediatric Rule" which allows the agency to require pediatric clinical trials if the drug is expected to be widely used in children or represents a significant medical advantage over previously approved products.

We've been pleased that the response to these new laws and regulations has been so positive. During the period from 1991 to 1997, before the exclusivity legislation was passed, the industry had made commitments to perform 70 studies in the pediatric population.

By comparison, as of May 1st of this year, we've issued over 145 Written Requests for pediatric clinical trials involving 298 studies. If only half of these are completed, potentially more than 18,000 children will be involved in these clinical trials. The areas of medicine these studies encompass range from cardiology, neurology and anesthesiology, to endocrinology, infectious diseases and oncology.

To find out just how successful this classic "carrot and stick" incentive is, we've published a Federal Register notice requesting comments on the exclusivity provision in the FDA Modernization Act. For those of you here today who are affected by this provision, I hope you will take the time to give us your candid opinions on how well the process is working for you.

As you know, the practice of medicine is being transformed as we acquire new knowledge and develop novel ways to apply it in preventing, diagnosing and treating disease. We talk frequently about leaving the world a better place for our children. As I see it, when new medical products come on the scene, the welfare of our children should be a top priority, not an afterthought.

FDA is working with this in mind. In addition to encouraging more pediatric studies we have begun a number of other activities to assure that medical products are not only more accessible for use in children, but also that they're used safely in this population. Obviously, I can't cover everything but I want to give you an idea of the range of initiatives we're working on to ultimately get more pediatric drug information into the hands of physicians.

In 1999, FDA established the Pediatric Advisory Subcommittee to help address some of the issues that have come up in the process of deciding what clinical trials are necessary to generate pediatric data.

The Subcommittee has focused initially on ethics and public health benefit questions. They've recommended that, in general, pediatric studies should be conducted in subjects that may potentially benefit from participation in the trial. This may mean concentrating on children who have a specific disease or are susceptible to it. "Potential benefit" was defined quite broadly, so that, for example, a pharmacokinetic trial for an antibiotic intended to treat otitis media could include a healthy child because most children have the potential to benefit from a treatment of otitis media.

Second, they recommended that children who are able to give consent or assent should be enrolled in a study before using children who cannot give assent.

Third, the Subcommittee recommended that FDA adopt the principles described in the Department of Health and Human Service's regulations offering additional protections for subjects that are involved in research regulated by FDA.

And fourth, the Subcommittee has addressed the more specific issue of whether the treatment of insomnia, as it is defined in adults, warrants drug treatment in the pediatric population.

This Pediatric Advisory Subcommittee will meet again in September to address placebo-controlled trials, the development of a plan for studying psychotropic drugs for use in children, a pediatric oncology development plan, and lactation research and labeling--all very important issues.

Meanwhile, so that clinical investigators understand clearly how to proceed, several guidances for industry have been or will be published on conducting pediatric clinical trials, qualifying for pediatric exclusivity, and performing pharmacokinetic studies in children.

But providing guidance to investigators is only part of the solution. We're also spreading the word publicly about the importance of including children in clinical trials. To do so, we're using a variety of mechanisms including our pediatric web page, a pediatric speaker program and dozens of public forums to educate the public about the pediatric drug development situation.

There is a particular area of pediatric medicine that probably deserves special consideration...and that's pediatric oncology. The good news is that cancer affects relatively small numbers of children compared to adults. The unfortunate news is that differences in the biology of tumors in children and adults make it difficult to extrapolate clinical drug effects from adults to children. That means it's usually impossible to rely on the pharmacokinetic and safety data for a particular drug that's used in adults, when that drug is used in children.

As a result, most children with cancer receive the majority of their care as participants in clinical research trials. So, participation in oncology trials has long been recommended and has essentially become the standard of care in pediatric oncology.

If we're to continue to make progress in the care of children with cancer, it's critical that we evaluate the effectiveness and safety of cancer drugs in pediatric populations. And so we're working with others to facilitate drug development in children with cancer, and to get the word out about including children in clinical trials.

Medical devices represent another area where we have a number of pediatric-related activities underway. You may know that we approved a cochlear implant for children 18 months of age and older. This is significant in that it was the first cochlear implant approved for children this young.

Along the same lines, we review devices that are used to test the hearing of newborns. Clearly this technology is critical to the normal language and cognitive development of congenitally deaf children.

I would be remiss if I didn't mention the recent approval of a Pneumococcol vaccine for active immunization of infants and toddlers against invasive disease caused by Streptococcus pneumoniae. In addition to preventing this serious disease, there may be a side benefit to using this vaccine. It would eliminate the need for antibiotic treatment of these cases. This means less antibiotic use that might contribute to antibiotic resistance.

In closing, let me point out that we are seeing less resistance by companies to conducting clinical testing of pediatric medical products. In part this is due to the incentive created by the exclusivity provision. Possibly, it is the result of increased awareness about the critical need for pediatric drug information.

In addition, research is providing clinical investigators with new technologies and methods that allow data to be collected where it was not possible before. New techniques for blood sampling, methodologies for PK and PK/PD studies, and improved diagnostic equipment are making it much more feasible to gather the data needed to support the use of drugs in children.

Let me again underscore that we each have an important role to play to see that this testing is done well. For our part, we'll continue to define what pediatric studies are needed, and request sponsors to perform these studies. We're committed to continuing our work with the scientific, medical and consumer communities. I can't think of anything that is more important than promoting and protecting the health and well-being of our nation's children.

I look forward to an interesting discussion.