Annual Meeting Generic Pharmaceutical Manufacturers Association
Remarks by Dr. Margaret Hamburg
Commissioner of Food and Drugs
September 11, 2013
Good morning. It is a pleasure to be with you today. Thank you Ron [Falk] for that kind introduction, as well as for your collaborative efforts on this project, and your leadership of the Kidney Health Initiative.
I also want to recognize Dr. Patrick Archdeacon, of FDA’s Office of Medical Policy at the Center for Drug Evaluation and Research (CDER), and his co-Chair of KHI, Dr. Prabir Roy-Chaudhury, as well as Melissa West, the KHI Project Director. I know all of you have put in a lot of hard work to bring together today’s meeting, and it is an important event. This gathering represents a remarkable team effort that has involved numerous supporters and stakeholders inside and outside of government – and a great deal of creativity and collaboration.
As you know, last year when ASN (the American Society of Nephrology) expressed concerns about the slow development of products related to kidney health, as well as safety issues involving other products that could have harmful effects on kidney health, we engaged in a number of discussions on the best ways to address these challenges and focused our attention on the opportunities and potential benefits of developing a public–private partnership model. Our talks resulted in the Memorandum of Understanding that Ron and I signed last year at this time, committing us to move forward together in pursuit of opportunities for collaborative education, research, and continuing dialogue.
Today’s important forum is one outgrowth of that agreement, designed to facilitate scientific research and development, to inform regulatory science in the field of kidney health, and strengthen opportunities in clinical trial design, clinical pharmacology, and translational science. It is the beginning of what I expect will be a long, vibrant, and productive partnership.
That this effort has progressed so far, so fast is a testament not just to Ron’s vision, but to the energy and commitment of all of those who are here today and to your understanding of what needs to be done to attract greater attention to kidney disease research and development.
We know all too well the catastrophic statistics relating to kidney disease: that one in nine Americans has chronic kidney disease and more than 570,000 have end stage renal disease; that the number of people with treated kidney failure reached a new high in 2010; that kidney disease actually kills more people than some other diseases like breast or prostate cancer that get far more attention; and that even when it is not fatal, the expense of treating kidney disease, particular end-stage kidney disease, is enormous, with profound implications for our society.
These statistics are extremely troubling. But just as frustrating is the disparity between the field of nephrology and other areas of medicine as it relates to the development of innovative approaches, new therapies, and an evidence base for promoting clinical practice and scientific advancement.
There are, of course, several reasons for this imbalance, including the disproportionately large range of products that have an impact on kidney health, as well as the wide range of diseases affecting or leading to end organ damage to the kidneys, such as lupus, diabetes, hepatitis, and heart failure. The difficulty of pinning down kidney diseases through well-defined and appropriate trial designs and regulatory pathways, and a shortage of multi-disciplinary approaches are other significant factors. The bottom line, as several of my colleagues have described it, is that there exists both an awareness and, importantly, a “therapeutic gap” when it comes to kidney disease.
This initiative is designed to focus attention and resources on closing this gap and help ensure that kidney disease receives the focus it deserves. But even as we move forward in this effort, it is important to recognize and build on the real and continuing significant scientific, medical, and regulatory developments in the battle against kidney disease.
In 2011, for example, we approved the drug Nulojix to prevent acute rejection in adult patients who have had a kidney transplant. By blocking the costimulatory signal in T lymphocytes, this drug gives kidney transplant patients a new option. And in July of this year, FDA approved Astagraf, an extended-release formulation of the calcineurin-inhibitor tacrolimus, providing kidney transplant patients with the option of taking this once-daily (instead of Prograf, twice daily) to prevent acute rejection.
Even more exciting have been the strides we are making in the development of new technologies to address end-stage kidney disease. And this moves us into the device domain.
As part of our groundbreaking Innovation Pathways Initiative, which is designed to reduce the time and cost of bringing safe and effective breakthrough medical device technologies to patients, we selected three products to help patients with end stage renal disease. These revolutionary new products â€•an implantable renal assist device, a wearable artificial kidney, and a hemoaccess valve system — all of which are being developed by small businesses dedicated to innovation – offer the potential to significantly change and improve the lives of many patients.
The FDA Center for Devices and Radiological Health (CDRH) team working on this project, in conjunction with the product sponsors were able to move these innovative products forward into their next phase of development in a much more efficient and timely manner than would otherwise have occurred as a result of our collaborative “One-Team” concept.
It is all part of a broader enterprise by FDA to deepen public–private collaboration between FDA and scientific and medical innovators. The objectives of this effort, called the Critical Path Initiative (CPI), are simple, yet significant: to modernize the regulatory and product development process and to foster public–private collaboration so that new therapies can get to people who need them better, faster, and cheaper. The connection between CPI and the Kidney Health Initiative is a direct one, and I’d like to spend a few moments discussing how we got to where we are today.
CPI was launched in 2004 with the publication of a landmark report examining innovation and stagnation in the development of new medical products. The report sounded the alarm about the widening gap between scientific discoveries that have unlocked the potential to prevent and cure some of today's biggest killers and their translation into innovative medical treatments.
The Initiative catalyzed numerous collaborations with stakeholders to address specific areas where the science of product development had the greatest need for improvement and it continues to generate productive partnerships. One emphasis of the Initiative was the need to develop better evaluation tools, and, in particular, to find preclinical biomarkers that predict human kidney toxicity, which could help develop safer products and prevent kidney damage, as well as the publication of the first pilot framework for a preclinical regulatory biomarker qualification process.
Another successful example of collaboration that emerged from the Critical Path Initiative, and holds importance for kidney disease, is an ongoing partnership between FDA and the University of California at San Francisco that focuses on membrane transporters during drug development. Researchers there are gaining an increasing understanding of the vital role that membrane transporters —proteins that facilitate the movement of substances across cell and organ membranes — play in the body’s response to some therapies and have discovered ways to prevent certain interactions and toxicity that can cause kidney poisoning.
It is this successful history that we are building on today with the Kidney Health Initiative which, like CPI, recognizes that solutions to these immense scientific and medical challenges cannot readily be achieved by any one entity. Collaboration is the cornerstone for ending the stagnation in innovation and development.
KHI’s potential is evident from the broad support it has already received – from the co-chairs of the Congressional Kidney Caucus, Congressmen Jim McDermott and Tom Marino, to the 40+ pioneer members – whose ranks include health care and dialysis providers, device manufacturers, patient groups, professional groups, pharmaceutical companies, academic research organizations, and representatives of the biotech industry.
It is precisely this kind of range and diversity that is critical to building a successful coalition, and that will generate a different kind of dialogue to shape and develop from the earliest stages through to finished products and new prescription and prevention strategies. That’s why KHI is well positioned to kick start the kinds of innovative research and investment we need to eliminate the underlying disparities between kidney disease and to more effectively address the serious and under-recognized epidemic of kidney disease in this country.
I am confident that by working together across different fields and with a variety of perspectives, this public-private partnership can – and must – make a real difference. Now is the time to translate the opportunities in science and technology today – such as the dramatic advances in genomics, biotechnology and material science – into meaningful breakthroughs for kidney disease.
And thought the Kidney Health Initiative is still really quite new – after all, this is the first KHI Stakeholders meeting ever – real progress is being made. Besides the impressive membership of individuals and organizations, so many of whom are here today, important work is already underway.
By creating this kind of collaborative environment, the FDA and the greater scientific and stakeholder community committed to advancing kidney health, is engaged to support new understanding and achievements. The KHI clearinghouse, for example, a portal that allows timely and significant review of projects, offers enormous potential to optimize ideas and resources. I understand that several pilot projects are now moving forward. And there are exciting plans for think tanks, working groups, educational exchanges, and other events to promote discussion and enhanced awareness and updates related to kidney health.
In addition, KHI can bring together representatives from across FDA, as well as other relevant government agencies like CMS, NIH, and CDC, to strengthen opportunities for collaboration, which is particularly important in light of the vast expanse of research issues, regulatory concerns, and health care responsibilities associated with kidney disease. And our hope, of course, is that this new approach-- and new thinking --can enable us to find more integrated strategies, and to pursue a path, that with a realistic vision, broad engagement, and science-based strategies, can overcome barriers to innovation and optimize safety for the benefit of people with kidney disease.
In closing, I want to underscore one final important attribute of the Kidney Health Initiative – its good timing. The world of medical research and science today is in the midst of great changes. The rapid evolution of various technologies, the extraordinary forces of globalization, and the exciting emergence of new areas of science are having an enormous impact on the development of medical products, as well as on FDA’s role in evaluating and regulating them. This “brave new world” offers significant opportunities to enhance and strengthen the traditional process by building new coalitions like this one to provide support for medical innovation and advances in public health.
An eminent surgeon and researcher, Dr. Quyen Nguyen commented that, "Successful innovation is not a single breakthrough. It is not a sprint. It is not an event for the solo runner. Successful innovation is a team sport, it’s a relay race." It is creative collaborations like this one that are key to building just such a team that will identify and overcome the scientific challenges and generate the attention and innovation that chronic conditions such as kidney disease so richly deserve.