Remarks by Scott Gottlieb, M.D., Commissioner of Food and Drugs
Public Meeting on Pediatric Medical Device Development
FDA White Oak Campus, Silver Spring, Maryland
 

I’m pleased to join you for this important discussion about pediatric medical devices. And I want to add my welcome to the others you’ve heard from already this morning. 

Today’s topic has special resonance for us today. 

We live in a time of extraordinary scientific and medical innovation. We’re at an inflection point for science, medicine, and policy. This provides us with enormous opportunities to strengthen public health through new innovation.

During the next two days, you’ll be discussing a number of these advances and the opportunities they afford us to develop successful new pediatric devices.

As you do, I hope you’ll take note of the role that the FDA has played in the development of these devices -- and in helping make sure they’re safe and effective.

Consider, for example, the historical exhibit that sits just outside this room.  It documents a traveling exhibit put together by the FDA in 1933, called “The Chamber of Horrors.” It was designed to highlight many of the dangerous products then on the market. 

That exhibit was instrumental in in getting Congress to pass the 1938 Food, Drug and Cosmetic Act, which granted FDA the authority to remove these types of products from the market.

One such product is a device that its manufacturer advertised as an effective treatment for diphtheria and other ailments in children.  The Vapo Cresolene apparatus used kerosene to heat a chemical that produced coal-tar vapors for a child to inhale. 

Not only was there no evidence that the device worked, but it’s almost certain, it caused more harm than help to those using it.

We’ve come a long way since then. And today, more than ever, we recognize the unique needs of children. And we’re working to encourage the development of safe, effective medical devices designed specifically for pediatric patients.

We have much to do when it comes to addressing the specific needs of the pediatric population. There are still far too few devices on the market designed specifically to treat, diagnose, or cure diseases in children.

As you heard this morning, over the past decade, despite legislation from Congress and regulatory process improvements by our Center for Devices and Radiological Health, novel medical devices designed, evaluated, and approved for pediatrics is only about a quarter of those for adults.  And the majority of pediatric approvals are not for children under 18 years of age. 

In 2017, we approved 66 devices through the PMA and HDE pathways. Only 18 of those were indicated for use in a pediatric population. Of the remaining 48 approvals indicated for adults, 42 were determined by internal pediatric experts to have the potential to treat, diagnose or cure a disease which occurs in a pediatric subpopulation. But we know there are challenges surrounding the development of pediatric medical devices.

One issue is the obvious – there are physical differences between children and adults that can affect development. There are also higher costs sometimes associated with development of medical devices for any affected population of a small size, and especially those designed for a pediatric market.

These are just some of the practical roadblocks to overcome.

We’re committed to doing our part. We’re committed to supporting the development and availability of safe and effective pediatric medical devices, and to encourage device innovation for medical conditions that impact young populations. 

We have a number of current initiatives underway to advance these goals, some I know you are familiar with.

For one thing, we’re increasing the number of medical devices with labeling for pediatric patients by incorporating known information about device effects in other populations to support pediatric indications;

We’re also recruiting pediatric experts for FDA advisory panels whenever there is a reasonable likelihood that the device under discussion will be used for children;

We’re collecting data on the unmet needs for pediatric medical devices and the barriers to the development of new pediatric devices; and

And we’re taking new steps to protect children who participate in clinical trials.

In addition, FDA has made a significant investment in pediatric expertise.  The Office of Pediatric Therapeutics has a full-time pediatric ethicist and a neonatologist. And we’ve added the Division of Pediatric and Maternal Health in our Center for Drugs.

Other programs, like the Pediatric Device Consortia Grant Program and the Humanitarian Device Exemption pathway, have helped foster the approval of a number of pediatric-specific medical devices and devices with a pediatric indication. 

And our incentive programs for orphan diseases, which are designed to help meet the needs of smaller medical populations, can also be helpful in advancing pediatric medical devices and other products.

We’re also working hard to integrate NEST, our National Evaluation System for health Technology, and to incorporate Real World Evidence generation strategies in advancing the development of safe, effective new products.

This can provide us with data that promotes the kind of patient-centered, benefit-risk approach that supports pediatric medical device development.

But we can—and need to-- do more. Public meetings like this one are essential to supporting these efforts. We look forward to hearing from those with a direct stake in this process. 

So, thank you again for joining us for this meeting and for your continuing engagement on this issue. 

We look forward to hearing your comments. And we look forward to working closely with you and all stakeholders in the use of technology to help serve the complex needs of children and other special populations and accelerate the development of safe and effective pediatric medical devices.