Shorter Approvals at FDA?
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ELIZABETH FARNSWORTH: Now we turn to two advocates involved in the debate over FDA reform. Gerald Mossinghoff is president of the Pharmaceutical Research and Manufacturers of America, a trade association. Abbey Meyers is president of the National Organization for Rare Disorders. She is also a member of the Patients’ Coalition, which includes the American Cancer Society and the American Foundation for AIDS Research, among other groups. Thank you both for being with us. Mr. Mossinghoff, what is your view of the Kassebaum bill?
GERALD MOSSINGHOFF, Pharmaceutical Research and Manufacturers of America: Well, we think that there is bipartisan support to reform the FDA to give them measurable performance standards to for the first time in history write a mission for the FDA; it doesn’t have a statutory mission. And it really needs one. It’s one of the few agencies that doesn’t have that. So we think all those things are necessary. We think there’s broad agreement that they are, and her bill is a very, very good first step to achieving those important reforms.
ELIZABETH FARNSWORTH: Abbey Meyers, what do you think?
ABBEY MEYERS, National Organization for Rare Disorders: Well, we’re very concerned that since the beginning of this century major changes and food and drug laws came about after great tragedies and people being harmed, hundreds and sometimes thousands of people being maimed and killed, and there hasn’t been a tragedy like that because we have really good laws that protect people. The FDA is the premier consumer protection agency in the United States. It protects what we eat. It protects the medicines that we take, and we really don’t want to see any kind of weakening that would subject the American public to ineffective or unsafe drugs.
ELIZABETH FARNSWORTH: Mr. Mossinghoff, what’s the problem that you think needs to be solved? The FDA says it has already sped up its review process. Do you disagree with that?
MR. MOSSINGHOFF: No, I don’t. They actually–and I personally and the association that I head in the industry that I represent, we worked very hard about four years ago to enact a user fee law which for the first time did put in accountability for the FDA. And I think they’re to be congratulated, and I agree totally with Abbey Meyers that we really do need an effective FDA. There’s no question about that. But there is a discovery approval lag. Over the last six years FDA approved about 150 drugs. Two thirds of those were first approved abroad. This past year they approved 28 drugs, two thirds again approved abroad. Five of those drugs were approved after they were approved in thirty countries and one or two of those were approved before–they had already been approved in seventy countries. There is a need for FDA to speed up it process while maintaining its diligence and its, its decisions.
ELIZABETH FARNSWORTH: What do you think about that?
MS. MEYERS: Well, Germany had to withdraw 30 drugs from the market after they hurt people. England had to withdraw twenty-some odd drugs. The, the European system is not as safe as ours, and we’ve seen a tremendous speeding up in different classes of drugs, in other words, drugs for untreatable and serious diseases and life-threatening diseases get priority at the FDA. Most of them are getting in within six months of review time. It’s the drugs for baldness, it’s the drugs for hypertension, where there’s already other, many other options on the market, the arthritis drugs, for example, it’s those drugs that are taking much longer. Does society need so many of those drugs which we call “me too” drugs? And isn’t FDA acting wisely by approving the drugs that society really needs very, very speedily?
ELIZABETH FARNSWORTH: So you’re the head of an organization of people who have very difficult to treat diseases, and yet, you feel that the drugs that you might need, people in this organization might need to get well, you can get?
MS. MEYERS: We get them very, very rapidly, before Europe, before the rest of the world; drugs such as the AIDS drugs have all been approved in the United States way before Europe. A breakthrough cancer drugs like Taxol was approved in the United States a year before Europe. A new drug for Lou Gehrig’s Disease, ALS, was approved in five months, just got on the market. It’s not approved in Europe, and they applied around the same time. So these breakthrough drugs are being handled with kid gloves at FDA. And the fast track is really working. It’s the other drugs that, that are not needed as desperately which are costing the industry a lot of money because every time their drug is delayed from getting on the market they’re not earning money.
ELIZABETH FARNSWORTH: What do you think about that?
MR. MOSSINGHOFF: Well, I’d say that this is one area where I do disagree with Abbey. Of the drugs, the 2/3 of these hundred and fifty drugs first approved abroad, 1/3 of them were identified by FDA, itself, as priority drugs. So they’re not “me too” drugs. They’re very important drugs. They may not be for rare disorders, but they’re very, very important drugs. I think two things: One is that the FDA has clearly improved its performance under the User Fee Act. No one in the industry disputes that. Two, they are the world leader in approving drugs for AIDS; there’s no dispute about that either. What that does is give us optimism that if Congress tells them again here are the performance standards we want you to meet, we believe that Dr. Kessler and his staff will very well meet them and to the good of patients here and abroad.
ELIZABETH FARNSWORTH: Okay, let’s just for a minute look at some of the specific provisions in the Kassebaum bill and discuss them. What would be wrong with requiring the FDA to review drugs within four to six months? That’s already in the statutes, they’re supposed to do that, right? Do you disapprove of that?
MS. MEYERS: Yes. I think it would lead to less drugs being approved actually because the FDA in having to make these decisions under pressure very quickly is just liable to say, no, this drug is not approvable, and send it right back to the company.
ELIZABETH FARNSWORTH: You don’t worry about that?
MR. MOSSINGHOFF: No. I believe that the time–FDA does have to have the resources, and I think that we probably have done more than any industry, any part of the regulated industry, in getting them the resources with our User Fee Act and the other provisions that we’ve worked very hard on over the years.
ELIZABETH FARNSWORTH: Just explain what the User Fee Act is.
MR. MOSSINGHOFF: That means that we–our companies will pay about $100 million this year into FDA as we file applications for them to hire reviewers, to hire the right people to review these things. There was doubt when we passed the User Fee Act. There were many, many voices saying this–they can’t do this, they can’t live off the user fees. Well, they’ve done it very successfully. With I think additional urgings by Congress, the fact that they write a new mission statement for FDA indicating approval, fast approval of safe and effective drugs is part of their mission, we believe that FDA is very capable of carrying it out and the dedicated people out there are very capable of carrying out these requirements.
ELIZABETH FARNSWORTH: What about ceding the approval to private companies if the FDA can’t meet the deadlines?
MR. MOSSINGHOFF: Well, I would jump in there. Our industry does not support that at all.
ELIZABETH FARNSWORTH: You don’t.
MR. MOSSINGHOFF: Our industry from the very start has said that the decision to put a new drug on the market is a federal decision to be made by an FDA official, an appointed official. It’s not to be delegated to industry, it’s not to be delegated to a foreign advisory. What we do is say that if a, if the United Kingdom, for example, which has an extremely good record, I don’t think even the most conservative officials at FDA don’t fault the so-called Medicines Control Agency in England as being not able to take care of the British citizens. If they approve it, what the Kassebaum bill would say is, all right, they’ve approved it, now you’ve got 90 days, make a decision one way or the other, don’t just put it back on your radiator, make a decision, is it approvable here or not and tell us why it’s not approvable. So there’s no, no thought at all in our industry that this is not a federal decision to be made by an appointed official at the Food & Drug Administration.
MS. MEYERS: I think it would be foolish to have the fox guarding the chicken coop when it comes to drug approval. We need independent, unbiased government employees who have no conflict of interest in their decisions.
ELIZABETH FARNSWORTH: What about what that they call off-label? This would be allowing manufacturers to market products which are approved for one disease that work for another one too. Currently, they can’t market them that way, they can’t go to the doctor and say, here’s the literature that shows how you can use this drug, am I right about that? Should that change?
MR. MOSSINGHOFF: And they wouldn’t be able to market it under the Kassebaum bill either.
ELIZABETH FARNSWORTH: What can they do under the Kassebaum bill?
MR. MOSSINGHOFF: They can simply disseminate a peer reviewed article, for example, in the “New England Journal” or the “Journal of the American Medical Association” or the “American College of Cardiology,” we could disseminate that to cardiologists, for example.
ELIZABETH FARNSWORTH: Give me an example of a drug that this would be useful for.
MS. MEYERS: Well, I think that probably a good example is drugs called calcium channel blockers where there are many peer review articles written in many, many journals saying that if you give this to a person after he’s had a heart attack, it’ll prevent another heart attack. Well, for years and years, this information was given to doctors unsolicited, became standard of care, and finally the NIH did a controlled trial and found out that people–
ELIZABETH FARNSWORTH: The National Institutes of Health.
MS. MEYERS: Right–people who were taking these calcium channel blockers were having more heart attacks and dying sooner than the people who were on placebo. So there is a big danger in disseminating information about the use of drugs for off-label users without the proof that they work, and that they’re safe and that they’re effective. Now, let me say that this is very important to people with rare diseases especially. Most drugs were not developed for our diseases. We used drugs that were developed for more prevalent diseases. We don’t have on the label any information about proper dosage. We don’t have any information about pediatric care. We don’t have information about the use in the elderly because they metabolize drugs differently.
ELIZABETH FARNSWORTH: This is the current situation you’re talking about.
MS. MEYERS: That’s right. The research needs to be done, and companies should not be allowed to disseminate information about off-label indications unless it has been absolutely proven.
MR. MOSSINGHOFF: I would have to jump in there and say that the example given is under existing law companies cannot do what Abbey said they did so they didn’t do that. They cannot disseminate information about off-label uses. I would give you probably the best example is speaking to an oncologist who treats lung cancer, for example, in that field, in the field of oncology, he estimates that between 80 and 90 percent of the treatment is off-label, and he definitely needs that information. We’re not talking about casual information. We’re not talking about marketing drugs. We’re talking about a company who knows more about their drug than any–anyone in the world being able to disseminate peer review literature from, for example, the American College of Oncology to oncologists, along with a warning that this is off-label, and we send the true label with that, and we leave it up to the doctor who typically doesn’t have time to read these 3,000 journals that come out every week and–
ELIZABETH FARNSWORTH: I have to interrupt you. That’s all the time we have. Thank you both so much for being with us.
MR. MOSSINGHOFF: Thank you.
MS. MEYERS: Thank you.