(AP/Toby Talbot)

When the Food and Drug Administration last month approved the powerful painkiller OxyContin for use in children ages 11 to 16 who suffer from severe pain that can’t be treated adequately with other medications, the move provoked a decidedly mixed reaction.

Some physicians who treat pediatric cancer patients and other children with dire medical conditions welcomed the approval, saying additional information about when and how to administer the potent narcotic would lead to safer use of the drug. But critics insisted the agency had acted recklessly, and that the approval will lead only to more OxyContin prescriptions in children, putting them at risk of addiction.

The Post spoke recently with two senior FDA officials -- Janet Woodcock, director of the FDA's Center for Drug Evaluation and Research, and Sharon Hertz, director of the agency's division of anesthesia, analgesia and addiction products -- about the reasons behind the recent decision and the implications going forward.

Here are excerpts of that conversation, which have been edited for length and clarity.

Washington Post: In a nutshell, tell me why the FDA asked Purdue Pharma to study the use of OxyContin in children. What prompted that?

Janet Woodcock: Since the 1990s, we were aware that drugs were massively being used “off label” in children with no information about how to use them, how to properly dose them, whether the condition was the same, what the side effects in children might be.

At that time, FDA put forward a regulation about studying children. That went into court and was overturned eventually. Then, [beginning in 1997] Congress put in place requirements -- and also incentives to companies -- for studying children. And the reason for that was, it was realized this extensive off-label use was not good. While drugs were being studied in adults and we had directions on how to use them, that wasn’t the case in pediatrics.

It has actually been a triumph of evidence-based medicine over the past 10 to 15 years. Now, we have changed 535 labels to include pediatric indications … This is widely recognized by the pediatric community as really beneficial legislation that was put in place.

In the pain world, it’s the same ... We issue requests to companies, that they study these drugs and they do certain studies in children. In some cases, they get additional exclusivity, at some point, for doing that. Those are obligations a company has to fulfill. We have a very elaborate oversight program here at the agency, some of which was put in [place] by Congress … to make sure these requests are consistent and reasonable, and that the right pediatric groups are covered.

And so, the request for OxyContin was part of a very large and very successful program we have to have on-label use of drugs in children.

WP: Could you talk about why the FDA ultimately decided to approve OxyContin for 11 to 16 year olds? Why that specific age group?

Woodcock: Don’t forget, this drug, like many other drugs, was already being used off label in children with severe pain, without instructions, without underlying data on how to use it in children.

Sharon Hertz: When the studies came in to support the pediatric [approval], we looked at who was enrolled in the studies, and we looked to see where there was adequate information to describe the safe use. Based on review of the studies, we found that the 11-to-16 age group had sufficient evidence to warrant being included in the labeling. So that’s where that came from. It was based directly on the study data.

WP: Does the FDA’s approval require OxyContin be given to children only in a supervised setting, such as a hospital? Or can you have a prescription and take it home, as an adult could?

Hertz: There is no restriction for use only in the hospital ... The way we see this product being most safely used is how it was studied -- in patients who required the opioids, and who already have been shown to tolerate the immediate-release form, and require them for a long period for these major situations. We have now a safe way to transition them to OxyContin for that period of time when the opioids are necessary.

WP: As you both know, the agency has gotten a lot of criticism from people who think this could lead to more, perhaps unnecessary prescriptions for children. And, that it could cause young people to get addicted to opioids. Sen. Joe Manchin (D-W.Va.) called it a “reckless act” and warned that we were “poisoning” children. Do you think those concerns are unfounded or unfair?

Woodcock: We have previously approved another high-potency opioid [for children] a long time ago [known as Duragesic] under this program. Certainly, we think it’s better that there is evidence-based prescribing than off-label prescribing. Because this is now an approved indication, we can put into place -- and we have put in place -- post-marketing requirements on the company, where they are going to keep track of all this. Whereas, before it was approved, we would have to go out ourselves [and gather that information] ... So, in those ways, we think it’s very beneficial, both for patients, and also for our ability to see what’s happening with this use.

Now, we fully understand that there has been a lot of inappropriate prescribing of opioids, particularly in the adult population … And we have made many changes and steps to try and do our part in controlling this epidemic of prescription drug abuse. We are by no means unaware of that. [But] for the pediatricians who would be using this and the pediatric surgeons and so forth, this is a setting in which the use of these opioids is appropriate. Certainly, tolerance to the opioids and some people abusing them is one of the risks of using these types of medicines. But this prescribing community will be very aware … Don’t forget, it is only indicated for children who require daily, round-the-clock, long-term opioids. That’s a very select indication.

But is there a possibility that some person might misuse this? There always is with these type of drugs. That’s one of the risks that you take. Most analgesics that we regulate have some liability … With the opioids, one of the huge liabilities is the addiction and abuse potential.

WP: Purdue has said it has no plans to market OxyContin to children. But the FDA’s approval for 11- to 16-year-olds would make that possible. How do you ensure that doesn’t happen?

Woodcock: What do you mean by market to children?

WP: Well, I mean that they could go speak to doctors about it [being FDA-approved for children]. I don’t mean commercials on TV.

Woodcock: Well, I think some of the post-market requirements we asked the company to look at [include] what are the uses in children? Give us all those data on a regular basis; that would be very important … Now, we have a tool to look at how this product is used in the pediatric population. Of course, it is illegal to represent drugs for off-label uses, which would be, for example, this is to treat ankle sprains or tooth extractions or something like that. That was not the indication here.

WP: Right. I think I was saying, could it potentially lead to more prescriptions overall, not for ankle sprains -- I don’t think anyone would want that -- but that it could [be prescribed] in cases that are maybe borderline whether it’s needed or not?

Hertz: We thought about that, as well. That was part of the reason we created the post-marketing requirements. What we will be watching for over time is whether the prescribing practices are changing, and if those changes are associated with undesirable outcomes … These are very large requirements that have been imposed on the company in conjunction with this action because we are sensitive to concerns about the use of opioids in this age rage.

WP: Is the FDA considering similar [pediatric] studies with other opioid drugs?

Woodcock: We are operating the program that Congress put into place. And we are trying to improve the whole armamentarium for children, so that we have evidence-based prescribing, not off-label, ad hoc prescribing for children. What we do is we look and see if any drug before [us for] evaluation, does it has a potential use in children? Or is it being used off label in children? That information informs us about what we ask a company to do. But Congress instructed us to operate this program. And as I said, it’s been considered a huge success.

WP: On OxyContin specifically, is it possible -- or probable -- that the approval gets expanded even broader, for even younger patients in the future?

Hertz: Right now, we have what we have [in terms of data], and we’ll see if anything further is called for, based on use or any other signals that might be important.

WP: One last broader question on this topic … I noticed that earlier this week a handful of governors in New England wrote to the agency, urging the FDA to put “black box” warnings on certain opioid formulations. This has happened several times, where state officials have urged the FDA to do more when it comes to opioids in general and on heroin abuse, which is linked in some ways. Is the FDA doing enough on this front? Could it do more to combat this public health issue?

Woodcock: It’s sort of another conversation. We have done extensive steps, including [risk evaluation and mitigation strategies, or REMS] in the extended-release opioids … We have re-labeled these products. We’re also doing a lot in the voluntary world, working with states on, for example, prescription drug monitoring programs and other things that we think are helping.

We’re also working with the [Obama] administration on their overall plan to combat opioid abuse. So, we are doing a lot. We often get people asking us to do one thing or another. We have to look at how effective that would be, given the entire situation and our knowledge of what intervention would be effective.

Also, of course, we’ve worked with the [pharmaceutical] firms on abuse-deterrent formulations ... We are also looking at other pain meds that don’t have the same liability.

There are two problems here. One problem has been misprescribing. And we think that emanated from the belief in the' 90s that pain was being undertreated, which was perfectly true. Everyone believed that if you treated people who really had pain, they wouldn’t get addicted or have any problems, and that actually is not the case. So, there was a broad prescribing of opioids, including immediate-release opioids ... And really, it’s just like alcohol. They’ve shown that alcohol abuse around the world correlates with how available alcohol is. 

The other problem is, there are many conditions of chronic pain that we don’t really know how well they respond to opioids. We think that many of them don’t respond that well to opioids. Some of the things we’re having the companies [involves] studies to look at the consequences of longer-term administration [of these drugs]. So there’s a very complex set of activities that we’ve done to try to help mitigate this epidemic.

Q: Anything more on the topic of OxyContin for pediatric patients? 

Woodcock: I just think the point is that seriously ill children deserve evidence-based medicine. They shouldn’t be experimented on by giving them medicines that haven’t been studied. We have established evidence-based medicine in the United States -- that’s what the FDA drug program stands for. And yet, until the '90s, we didn’t have that for children. And it really is considered a tremendous advance.

We understand there is a terrible problem with opioid abuse and addiction. Everyone understands that. But this is about evidence-based medicine for children -- seriously ill children who are suffering pain.