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  5. Panel III - Presentations by Patient Advocacy Groups, Prescription Drug User Fee Act Public Meeting November 14, 2005
  1. FDA User Fee Programs

Panel III - Presentations by Patient Advocacy Groups, Prescription Drug User Fee Act Public Meeting November 14, 2005

Panel III - Presentations by Patient Advocacy Groups, Prescription Drug User Fee Act Public Meeting November 14, 2005

Panel II - Presentations by Academic Research Groups 

MS. HENDERSON: Back on the record.

If you'd begin to return to your seats so we can get started. This panel is between you and lunch.

[Pause.]

Okay. If you'll take your seats, we'll get re-started. It may look like our FDA listening panel has emptied out. But, in fact, it's not true. Just for the information of the audience, some of our panelists have asked to move to the audience so they can see the slides for the presentations better.

MS. HENDERSON: We're going to change the order a little bit of this panel to accommodate Ellen Sigal's schedule. So we will move Dr. Sigal to the beginning, followed by Dr. Cohen, Ms. Dorman, and

Ms. Ireland.

And we will start with Ellen Sigal.

Ellen Sigal, Ph.D., is founder and chairperson of Friends of Cancer Research, a Washington, D.C.-based nonprofit organization. Friends is dedicated to accelerating the nation's progress toward prevention and treatment of cancer by mobilizing public support for cancer-research funding, and providing education on key public policy issues.

Dr. Sigal serves on the National Cancer Institute Board of Scientific Advisors, the National Institutes of Health's prestigious Director's Council of Public Representative, the National Institutes of Health Foundation Board, chairing its Public-Private Initiatives Committee, the American Society of Clinical Oncology Foundation Board, the American Association for Cancer Research Foundation Board, the Johns Hopkins Cancer Center Advisory Council, the Duke University Cancer Center Board of Overseers, and the Howard University Cancer Center Board of Visitors.

So we are very pleased to have Dr. Sigal with us.

DR. SIGAL: Good morning. And I'm sorry about leaving early, but I'm on the Board of Scientific Advisors of the National Cancer Institute, and there's a critical vote that I need to participate in. So I will try to be brief, and I will try not to be redundant, although some of my slides are redundant. But I will try.

[Slide.]

So why do we care, in the cancer community? That's pretty obvious. I mean, obviously, we have a million-three patients impacted every year on this; over 500,000 people dying every year of this disease. And, frankly, until many years ago, many of us in the advocacy and the research community didn't really focus on the FDA. It was a big black hole that was seen to be insurmountable, unintelligible, and frankly to many of us, dysfunctional. And frankly, there were a lot of bad drugs, and there not a lot of opportunities. So you know, that was the prevailing view.

And a few years ago that changed. That changed. We thought that we really had a stake in the FDA, because we talk a lot about translational research, and you can't translate anything until you have better drugs, and more effective drugs. And so we started to work with the Agency in a very collaborative way. And now, rather than a black hole, they have become friends; people we care about. They're extremely, extremely important to the new generation of drugs, and to all cancer patients.

So we have a major stake in the FDA. So we care a great deal.

I'm not going to go over PDUFA. You all know about PDUFA. You know more about PDUFA than I do. You've heard about it today.

Evaluating PDUFA is important. You know, I think that we're trying to, with the renewal of PDUFA, there's going to be a lot of metrics for evaluation. We heard some really good data today.

By and large, it's very, very successful. And I think any of the critics that are going to complain about safety or compromising integrity are not going to be very happy, because the data shows the opposite. So PDUFA has been very, very good thing for the patient community, and certainly for the cancer community. And we are enthusiastic about the continuation of PDUFA and, frankly, the enhancement of PDUFA.

[Slide.]

You know, PDUFA has allowed the FDA to expand the user fees for the management. It's added staff. And, again, it's helped understand drugs' risk profile, which I'm going to talk about in a little bit.

[Slide.]

The safety issues are a very dramatic concern to the cancer community. This is something that we are very worried about, because we think that the cancer community can be more dramatically impacted on an overzealous of safety, or interpretation or understanding of safety, and therefore we often say: safety kills. And we mean that. I mean, we're very concerned about a new pipeline of new and better and targeted drugs that will really make a difference to the patient. And if we get so consumed with safety we, in fact, will lose many, many, many more patients from this disease than not monitoring safety.

So it's extremely important for us. And we have patients from all ends of the spectrum; from people who have, you know, what can be a chronic disease, and people who have diagnosed with fatal disease. So clearly these issues are of significance. I mean, clearly, I'm going to tell you the obvious: no drug is 100 percent safe. And that we know about. And the public needs to understand that. As a matter of fact many of the nutriceuticals that people are taking in large quantities are not safe, as well. So they need to understand that.

So obviously this issue between risk and benefit is very important. Intelligent design of trials. And if the speed and the certainty of drugs is really what we care about. We certainly understand that we need to monitor drugs; that we need safe and effective drugs. And particularly as our disease becomes, hopefully, a chronic disease, this is going to be even more important.

So clearly, really, safety and efficacy are extremely important.

[Slide.]

There are things that can be done on safety. None of us would suggest that we're doing enough, or that we can't do better. But we need the resources to do that.

We are very worried about overzealous regulation that would only deal with safety and create more bureaucracy and infrastructure and really not deal with efficacy. But we can certainly collect more data. We can enhance the Med Watch. We can increase the FDA's capacity to process and analyze adverse events and get that information to the public.

But ultimately these decisions are between the patient and the doctor. And we don't want on-size-fits-all. It can't work for cancer, and it shouldn't work for cancer. We want these decisions. We want the information. And people are going to have to be informed about their choices on these drugs.

[Slide.]

Looking forward to PDUFA IV, clearly we talked about the safety of drugs. And I think that

can enhance. And if we can really deal with the

funding of FDA, and the funding of PDUFA, I think that that's going to be critical to the cancer community, and to the patients' receiving treatment or diagnostics.

Funding is important. I'm going to talk about that a little more later on. But efforts to enhance safety systems should not compromise the timely approval of many patients facing life-altering disease. And this is the consistent message that I want to discuss.

[Slide.]

Adequate Resources--that's a huge concern. FDA is one of the most underfunded agencies in the Federal government. And I don't think people understand that. And I think people need to understand that. PDUFA isn't going to entirely fix it. As a matter of fact, it's a danger. We have to be very careful that we continue to fund what we're supposed to do for FDA, and fund the science, integrated the critical path, and to really fund the agency appropriately. And that's very important.

That doesn't mean that we shouldn't continue to do PDUFA. We should enhance it. We should look for perhaps other funders other than the pharmaceutical agency, because I do think that even foundations and patient groups--there are other sources out there for funding and we should explore them. But we must continue continuing to appropriate the FDA and, frankly, enhancing that, because that's going to be important.

And we really have to look at the critical path and the funding mechanisms for the critical path. Because if we don't integrate the science and fund the critical path, we're not going to get better and safer drugs to patients. So this is a very important issue.

[Slide.]

Finally, what's important to us is that our disease, or cancer, is changing. We have a lot more information. WE have better information. WE will have better drugs. They will be more targeted. They will get to the patients faster, we hope, and they will be more effective. So continuing the science, integrating the science, working collaboratively with the NIH, with the FDA, and with patient groups is critical to this.

So we are very enthusiastic and, frankly, consumed with what happens at the FDA. Because what happens at the FDA impacts everyone who will be diagnosed with this disease, and everyone treated with a disease. And it's much more than just, you know, people who have final forms of cancer. We're talking about diagnostics, we're talking about a better class of drugs, targeted drugs.

So this is really a new day for cancer. And if we're going to achieve the 2015 goals, and if we're going to really help cancer patients, the integration of science, safety, the funding is going to be critical to us. So we care a great deal about what happens, and we're very vested in it.

You know, clearly a stronger FDA helps all of us. The predictability, the consistency of the application, greater efficiency. And frankly we need, in the patient community and the research community, to do a better job of advocating for the importance of this agency. I think a lot of people have really only talked about the science and the research. We just spend a lot of time talking about translational research, but not enough time about the entire spectrum, from early diagnosis to the importance of the interactions with the NIH and the FDA.

As a matter of fact, when Mark McClelland came to the FDA, and Andy von Eschenbach, the first thing they did is this interagency task force between the National Cancer Institute and the FDA.

And we encouraged that.

We'd like to see advocacy groups and patient groups and industry and CMS as part of that, as well.

So we want innovation. We want better science, and we care a lot about what's happening. And PDUFA IV is going to be on our radar screen. And the FDA is not a black box to us any more.

Thank you.

MS. HENDERSON: Thank you, Ellen. We'll let you set off to your vote.

Our next speaker is Dr. Perry Cohen. Dr. Cohen is an active participant and leader on the national level in advocacy activities for Parkinson's issues. He is project director for the Parkinson's Pipeline Project, which he initiated to provide the patient's perspective to clinical researchers and sponsors of new therapies.

Prior to diagnosis with Parkinson's disease in May of 1996, Dr. Cohen was a planning and evaluation consultant in the health industry for public health agencies, health insurance plans, and academic research centers. He has an M.S. and a Ph.D. in organizational development from MIT's Sloan School of Management, and a Bachelor's in Management Science and Math from Carnegie Mellon University.

Please welcome Dr. Cohen.

DR. COHEN: Thank you. And thank you for inviting me.

As you introduced me, I am a Parkinson's patient and advocate, and I've had Parkinson's disease for 10 years now, and it gets worse.

But I've been patient representative at FDA for Parkinson's since on a was on a deep brain stimulation advisory panel back in 2000. And in this role I learned some of the things that he last speaker just talked about, about the FDA. And I set up the Parkinson's Pipeline Project, which is a grass roots effort, to do everything we can do as patients to speed up and facilitate introduction of new treatments that are leading to cures.

[Slide.]

A little bit about Parkinson's Disease: briefly, in case you don't know, it's a chronic degenerative neurological condition, characterized by the loss of dopamine neurons. And dopamine is what controls movement. But it's interacting with other parts of the brain. The brain is very complicated, as you know. And there are cognitive and emotional symptoms, as well as autonomic system functioning, such as breathing and swallowing and some of the other aspects of Parkinson's.

Parkinson's is known as a lower prevalence chronic disease by CDC, with somewhere under a million patients. It's far fewer than the bigger chronic diseases such as heart, cancer and diabetes. But it's similar to, like hundreds of other neurological diseases, which I think the next speaker will speak about some more. And collectively these numbers probably add up to a significant public health problem, which is only beginning to be recognized.

And Parkinson's is on the vanguard on the last frontier of science: the brain and the central nervous system. And if the neuroscience meeting, which is downtown, which I have to go to later today, is any indication, we should be having more active discoveries and treatments in the future.

Another thing about Parkinson's is: we have a pretty active advocacy movement. And there's a number of celebraties and prominent business people, as well. And just to let you know, for the advocates, there are a lot of people in the closet still who you'd be impressed to know have Parkinson's disease, and are starting to creep into the advocacy work.

[Slide.]

As far as treatments and cure goes, it's sort of a good news and bad news situation.

First identified in 1817 by James Parkinson, the science and treatment has advanced compared to other central nervous system conditions. The good news is there are many effective treatments for Parkinson's. The bad news is the treatment may stop working after five or 10 years, and the side effects of some of these treatments are as bad as the symptoms. So we need more development.

The good news is there's more known about Parkinson's than any other brain disease, and the recent big build-up in science has put us on a path toward better treatment and cures that are promised to be right around the corner. They've been telling us "five years" for the last 15 years.

The bad news, however, is the pipeline, which we've been talking about today, 15 or 20 years or more--and I was quite impressed with that figure of neurological conditions, clinical pipeline average of 11 years. And I want some of the academics who spoke in the last panel to address that issue really quick.

But in addition to the scientific issues, there are financial markets, and business strategies, and organizational politics, and people's careers, and publication schedules and all these other things that are in between us and the cures and treatments that we need.

[Slide.]

So, what do patients want?

First: faster cures. Time is not neutral for someone with a serious or life-threatening illness. And we want to look beyond the review time. We want to look at the whole process; the whole process of drug development, and look for ways to do that.

I've learned a lot at this session. These slides were obviously prepared in advance. I've learned a lot about what's being done. And I have to admit I was not fully aware of everything that was being done.

And second, we want more emphasis on individual decision-making and choices--sort of echoing the last speaker. We need to recognize that the equation of risk-benefit tradeoff is different for each person, and there should be more flexibility for individuals to make their decisions, with advice and counsel of their own physician, or some expertise; but also with full information. And that's the key. Some of the safety problems, the coverup is worse than the problem.

And third, we want greater participation in the process. We're not rats in a cage. Our experience is valid, and it's often not captured or considered. And I've been working with several companies, and this is true.

[Slide.]

Here's my naive view of what PDUFA has done--before I got here today; which is basically correct, but now I know a lot more.

The PDUFA was established to reduce the length of time in reviews. There's extensive industry consultation, and FDA agrees to meet performance standards. And there's been little or no input from patients and consumers in the past legislation.

[Slide.]

So here's my view of the patient perspective on user fees, from the outside looking in.

As I said: little information on the time frames, on the processes. It's a black box. The FDA processes are a black box. And even when you're involved with specific treatments, the proprietary information of the companies seem to be more important than the distribution of the scientific data. And when there are meetings, we don't even know when the meetings are being held, or if they're held, much less what goes on in them.

There are a couple of simple observations I would like to make. The user fees are now greater than 50 percent of the FDA review costs, as was presented earlier. And this raises questions. Now, I don't believe this to be true, but it raises questions in the press and in the eye of the public, whether there's a lack of objectivity related to the rulings, based on this dependence on fees.

So it behooves us, as advocates, to lobby for the FDA budget, just the way we lobby for the NIH budget, and with great success. So I'm not sure if the Agriculture Committee in the Congress has enough appreciation of what the FDA does. And, unlike the rest of the Health and Human Services Department, the FDA budget is handled by the Agriculture Committee, which came as a big surprise to me.

Another little technical point is that the fees are only paid when the new drug application is submitted, yet the costs are distributed throughout the process. And that was pointed out earlier, as well. And by doing so, I think that maybe explains at least part of the reason why there are fewer applications.

[Slide.]

So we do we have for suggestions? My view is that we have an untapped resource, and there are new roles for patients. The Office of Special Health Initiatives, which included me and a number of other patient representatives and patient consultants, has been cultivating and advocacy group. And I think this is important for several reasons.

First of all, when you have patients at the panel, that could counter the appearance of a bias towards industry if the patients are actively involved, because everybody says the bottom line is the patient's safety, and the patient's treatment. And if the patient is at the table, then you get that viewpoint expressed.

[Slide.]

Next, it needs to be a transparent process. Information about the process needs to be more public; when they're having meetings, what stage of the process they're in. Certainly, companies are allowed to have proprietary information, but if we knew what was coming down,if would give us something to have some hope for, and something where, if we were involved in the process more, we could contribute to.

And I think the FDA should expand the excellent efforts of the Office of Special Health Initiatives to support community groups like the one that I work with, in education of the community about the regulatory process.

[Slide.]

So, just to summarize, this is our motto: "The missing ingredient in new therapy development is the voice of the patient."

We're partners in clinical research. We're seeing input in risk and benefit judgments. And awe have created a research participants' "Bill of Rights," which sets a framework for collaboration in relationships between industry and researchers and patients. And PDUFA should recognize these roles.

[Slide.]

Now, a couple more suggestions. And this has been covered earlier quite a bit, so I'll go over it quickly. The entire pipeline should be examined for time savings. And system changes are necessary to achieve significant reductions in the duration of the pipeline, and the maintenance of the quality. These have been brought up in other talks quite strongly and I would agree with that.

[Slide.]

Business and science as usual is not sufficient. Though we have some leadership, the Critical Path Initiative, which has been mentioned quite a lot, the NIH translational research road map, they're talking the terms--the system's terms. And my training in management and organization and systems, it seems to me that they're talking the way to go.

And "Faster Cures" is a program that starts looking at some of the assumptions in business-as-usual, and science-as-usual--like cycle times have been mentioned here; and turnaround times in getting the information back into the process from scientific research.

[Slide.]

And finally, I think my last suggestion is we should view the process in the continuous quality improvement paradigm. Rather than have one giant step to approve, which may make people be more cautious, I think we need a series of shorter steps of conditional approvals that expand the markets and the target audience for research in a more gradual way, and that way you could get some early approvals, particularly for those people who need access to potential life-saving treatments, as well as then continued maintenance of control of some of the bigger safety problems that might come up down the road.

And that's all. Thank you.

MS. HENDERSON: Thank you, Dr. Cohen. We very much want to hear the voice of the patient, and surely appreciate your input.

Our next speaker is Dianne Dorman. Ms. Dorman currently serves as vice president for public policy for the National Organization for Rare Disorders, also known as "NORD," where she develops and maintains relationships with Federal agencies, including the National Institutes of Health, the Food and Drug Administration, and the Centers for Medicare and Medicaid Services, the United States Congress, the biotechnology and pharmaceutical industries, and other health care-related agencies.

Ms. Dorman's efforts include work with other patient organizations and coalitions to accomplish initiatives that benefit people with rare diseases. Technical assistance and legislative analysis are provided to NORD's member agencies on government-related matters, as well as the training of staff and volunteers of member organizations.

Thank you for being with us--and you're on.

MS. DORMAN: I want to thank the FDA very much for giving me the opportunity to speak to you today about the impending reauthorization of PDUFA.

I'm going to be focusing my comments on a user fee issue that is very specific to the development of orphan products for patients in the U.S. affected by rare diseases. It is an issue that is of extreme importance to NORD and the people that we represent.

Now, for those people who may not know what a rare disease is, it's any disease that affects fewer than 200,000 people in the United States. And that's based on statute, the Orphan Drug Act, which was signed into law in 1983.

[Slide.]

I want to go back--if those people who may not be familiar with the statutory provisions of PDUFA as it relates specifically to orphan products. The companies do not pay one-time application fee for orphan drugs. The orphan drugs qualify for waivers of annual product and facility user fees if it is perceived that there is an unmet need, a public health need; or the fees would be a barrier to innovation; or if the company qualifies as a small business. And the waivers may be partial, or they may be complete. And that is how it's written into the PDUFA statutory language.

[Slide.]

Now the FDA is currently operating off a 1992 interim guidance that indicates that the NDA holder is the entity subject to user fees. It defines a small business to be $10 million annual gross revenue. And it also, in my opinion, reflects pre-implementation concerns of FDA that waivers would be abused--and, understandably. Because user fees would be a major revenue stream for the Agency, the interim guidance reflects the FDA concerns that waivers could be abused by larger companies.

Now I would not be standing here today if I thought for one moment that what I am about to propose to the FDA would in any way benefit large pharmaceutical and biotech companies who can well afford to pay the application, facility, and product user fees.

I'm here on behalf of small U.S. companies who are willing to develop therapies for very, very small patient populations; and for those small companies who may be even thinking about developing these products in the future.

I'm here on behalf of rare disease patients who still have no hope of any therapy to alleviate symptoms or to present a cure.

[Slide.]

Now, some orphan companies do pay user fees, because no waiver is granted if a NDA holder is a company with more than $10 million in its revenue. The U.S. licensee is evaluated based on its own revenue, the revenue of the NDA holder, and also all affiliates of the NDA holder.

Now, in 1992, I don't think anyone, including NORD, could have predicted that nations around the world would be emulating our orphan drug law, and would be encouraging the development of orphan products in Europe, Taiwan, Australia and other countries. Therefore, it was impossible for Congress or the FDA to know that foreign countries would be seeking approval of their drugs in the U.S., and would be seeking companies within the U.S. to license their markets and to market their products.

[Slide.]

Going back to 1992, it is our opinion that the Congressional intent of PDUFA is stated as follows by Senator Orrin Hatch and Senator Kennedy: "The Bill sponsors do not intend for the fees authorized under this Act to serve as a disincentive to the research and development of important prescription drug products; nor should the fees impose an undue financial burden on any company."

Now, when Dr. Kessler was asked a question from the Committee regarding whether developers working on orphan drugs for a disease that affects only 60 children, when he was asked this question he replied, "There is no way we would expect them to pay these fees."

[Slide.]

Now I'll give you a real quick case study. There is a rare genetic metabolic disorder called tyrosinemia Type I. There are less than 100 cases in the United States, and I believe only 500 documented cases worldwide. It presents itself in the first few months of life, with failure to thrive, fever, vomiting, bruising, enlarged liver, and it can lead to acute life-threatening liver failure and death within the first year of life.

There is one orphan drug, and along with this orphan drug and dietary restrictions, it offsets the deficiency, and children live, they thrive and they grow older.

Now, case in point, if you look at these numbers--and I'm assuming, of course, that DayPro has only one fee. I'm making that assumption. I'm not actually sure if it's correct or not. But if you look at the numbers, there are nearly one million patients taking DayPro. The company pays $335,000 in user fees, and the cost to the patients over one year is only 33 cents.

On the other hand, the 65 patients with tyrosinemia Type I, the company pays the same user fees, and the patients end up paying over $5,000 for the particular therapy that they have to take that sustains life.

[Slide.]

Now, what is the impact on orphan drug development? Well, number one, it reduces the incentive to develop orphan drugs for very small patient populations. It reduces funds available for post-marketing studies into more rare diseases. And it reduces funds available for other clinical research into rare diseases.

And we have a solution, which we have presented to the FDA on a number of occasions. It includes any orphan-designated product that does not qualify for complete waiver of product and facility user fees under the current user fee guidance, shall nonetheless be granted a complete waiver of such fees by the FDA if it meets all the following conditions.

Number one, it must be an FDA-designated orphan drug approved by the FDA for the designated indication. Number two, the orphan had U.S. sales in the previous year of less than $25 million for the active moiety for all indications, dosage forms, strengths for which the drug is approved, as well as for any off-label use.

Now number two, in particular, is to ensure that large companies don't take advantage of this by salami-slicing a particular drug or whatever, and taking advantage of the user fee. Because our proposal is for orphan products and rare diseases specifically, for very, very small populations.

Number three, it meets the current public health requirement of PDUFA waiver standards.

Number four, the company responsible for the user fee for the product applies for the waiver in the manner and timeframe specified by the FDA.

And, finally, the FDA shall accept and act upon the application without regard to whether the company responsible for the payment of the user fee is also the NDA holder.

[Slide.]

In summary, the problem is that the current 1992 guidance is a barrier to innovation and a disincentive to conduct clinical research on rare diseases. And NORD's solution removes those barriers to innovation, supports discovery research that is so critical to NORD and to the rare disease community.

Thank you.

MS. HENDERSON: Thank you very much.

Our next speaker is Jeannie Ireland. As director of public policy since 2002, Jeanie Ireland leads the Foundation's advocacy effortsaimed at eradicating pediatric AIDS, expanding HIV-AIDS care and treatment globally, and accelerating the discovery of new treatments for other serious and life-threatening pediatric illnesses.

Ms. Ireland brings a unique perspective to these discussions today, because from 1997 through 2001, as minority staff director for the Senate Health and Education Committee's Subcommittee on Children and Families, she advised Senator Chris Dodd on childcare and health issues and, in that role, she was involved in negotiating the Food and Drug Administration Modernization Act of 1997, and the inclusion of incentives for pediatric drug testing.

Thank you for being here, Jeannie. You're up.

MS. IRELAND: Thank you very much. And thank you for inviting me to participate today.

I very much appreciate the opportunity to share our views on the reauthorization of the Prescription Drug User Fee Act. [Technical difficulty. Discussion off mike.]

Again, we very much appreciate the opportunity to comment on the PDUFA reauthorization and how PDUFA might be improved.

I'm pleased to offer the perspective of an organization that's been focused on improving patient access to cutting-edge therapies since its inception in 1998. This issue is at the heart of the foundation's mission. In fact, the foundation's creation was sparked by Elizabeth Glaser's outrage over the lack of options for treating her two HIV-infected children in the early days of the epidemic.

Since that time our work has expanded beyond domestic HIV and AIDS, to a fight against the global pandemic. Currently we're operating programs to prevent mother-to-child transmission of HIV, and to provide care and treatment at over 750 sites in 20 countries.

Our work has also expanded beyond HIV and AIDS to encompass research and advocacy in support of accelerating treatments for a broad range of pediatric conditions through the Glaser Pediatric Research Network, a consortium of five academic medical centers and children's hospitals that conducts multi-center trials on such pediatric conditions as cancer, obesity and osteoporosis.

In keeping with that mission, we have closely followed the implementation of the user fee mechanism, and are pleased to have the opportunity to comment on ways in which we think it might be improved.

On the whole, we've been pleased with the success of PDUFA in reducing review times and speeding drugs to market. We are concerned, however, that an unfortunate consequence of the law has been to direct attention, resources away from other critical functions at the FDA; namely post-market safety activities.

In essence, the need to meet review timeframes, and the requirement that the FDA's budget not fall below a certain level has had the effect of focusing resources and attention and leadership disproportionately on drug approvals, and away from the safety monitoring of drugs once on the market. We believe that a central issue in the 2007 reauthorization must be how to give the FDA the authority and resources it needs to improve post-market safety.

It's important to state that we do not believe the answer to improving post-market safety is to return to the days of significant delays in access to new therapies for life-threatening illness. Rather, the solution lies in matching the leadership resources and attention the FDA and industry have applied over the past decade in speeding treatment to markets, with equal attention to assuring the safety of those products once on the market.

In our view, the solution must include three elements: sufficient FDA authority to conduct critical post-market safety activities; adequate resources; and performance goals as appropriate for those activities.

Under the heading of "sufficient authority," we believe it's important to give the FDA clear, unambiguous authority to require needed post market studies; also to ensure any new authorities are accompanied by flexible enforcement tools; the need for more than the nuclear option of drug withdrawal which only hurts patients; to also ensure that study findings are promptly shared with patients and providers, both through time labeling changes and, for information that doesn't make it onto the label, through a registry of clinical trials.

These new authorities must, of course, be paired with increased resources. We would suggest an increase in user fees to fund safety activities, including epidemiological studies, high-priority randomized controlled trials, and an increase in support for review of adverse events reports. Obviously, there's precedent for this in the 2002 reauthorization, which allowed some fees to go toward post-market safety activities.

A third element must be attaching performance goals to some of these activities. Simply human nature that deadlines generate attention and focus. Even if additional resources are given to post-approval activities, without deadlines, if they have to compete with pre-approval activities with deadlines, they will lose. Obviously not all activities would be appropriate for setting deadlines, but some would be, including goals for reviewed adverse event reports, and others.

I'd also like to take this opportunity to offer our recommendations for the reauthorization of two pieces of legislation we believe are essential to ensuring that the benefits of PDUFA, in terms of speeding drugs to market and greater access to new therapies accrued to children. These are the Best Pharmaceuticals for Children Act, and the Pediatric Research Equity Act. Both of these pieces of legislation expire at the same time as PDUFA, and we might expect that they would be considered as part of that reauthorization by Congress.

Like PDUFA, we believe that, in general, these two pieces of legislation are working very well. In fact, I understand that we're very close to, or we've reached, the 100th label change resulting from the Pediatric exclusivity incentives.

There are a few things we would recommend for improving these pieces of legislation. One is to improve the consistency and coordination between BPCA and PREA; the need to address both the overlap between the two and the gaps between the two. For example, currently the requirement in BPCA to make study findings public does not apply to studies conducted under PREA; nor does the requirement to report adverse events to the Pediatric Advisory Committee. Really no policy rationale why those requirements should not apply to both.

We need to improve the accuracy and speed of labeling changes. We should direct a portion of user fees to fund studies of off-patent drugs, and those on-patent studies that manufacturers have refused to perform. Our experience over the past three years has clearly shown that the existing mechanisms for support that would be helpful for funding these studies are not sufficient. And those were NIH appropriations and drug company or either private-party donations.

We'd also like to remedy what we consider to be the major flaw of the Pediatric Research Equity Act by making permanent the FDA's authority to require pediatric testing of those products clearly intended to be used in children.

So, in conclusion: this reauthorization obviously offers historic opportunity for the FDA and Congress to finally achieve the twin goals of speeding critical therapies to patients, while also improving the safety and the monitoring of those products post-approval.

I look forward to working with the FDA and Congress on this reauthorization, and hope that we can serve as a resources, and thank you again for the opportunity to speak.

MS. HENDERSON: And thank you for speaking.

And thank you for all of our panelists from this morning. We are just perilously close to being on time. We are about 10 minutes into our lunch time. And rather than penalize your lunch, we will just reconvene here at 1:10 this afternoon.

Thank you all very much.

[Off the record.]

Panel IV - Presentations by Consumer Groups

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