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U.S. Department of Health and Human Services

About FDA

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Overview

Previous Section: A Note from the Commissioner

Because of its role as both a regulator and as a public health agency facilitating the development of new, innovative products to meet the needs of patients and the American public, FDA has a unique perspective on the health of the biomedical industries that make up an important sector of the U.S. economy.

Today, advances in science are leading toward a fundamental change in the way medical treatments will be developed and used. Groundbreaking discoveries in complex chemistry and biosynthesis have resulted in promising new approaches for the development of drug candidates, advances in cellular and molecular biologies are creating novel strategies for new biologic therapies such as stem cell-driven products, and cutting-edge electronics, nanotechnology, and advances in materials sciences have revolutionized medical devices. As a result, incredible opportunities await both American companies and patients.

This year, FDA has approved a number of truly novel breakthroughs based on sound science to address critical health needs. These include several groundbreaking new therapies for devastating diseases, such as the first new treatment for Lupus in fifty years, two new therapies for Hepatitis C, and the first drug ever shown to be effective at treating melanoma.

However, while we have seen an increase in the quality of new drug submissions, there is a concern about a decrease in quantity. The past two decades have been marked by steadily increasing investments in basic research and development, with more than $95 billion in combined R&D by industry and the National Institutes of Health (NIH) in 2010 alone. And yet, these investments have not translated into a parallel increase in novel products submitted to the Agency for approval. For example, last year the Agency received the lowest number of applications for novel drugs in nearly two decades.

Additionally, drug companies continue to face increasing development costs, and a number of patents for blockbuster drugs are about to expire, leading to a projected loss of $114 billion in revenue over the next three years with fewer new products coming down the pipeline to cover the loss of revenue.

Medical device companies are also experiencing their share of challenges. Venture capital for small startup companies has decreased in the recession, and what investments are being made tend to come at a much later stage of development—leaving many companies and small businesses with limited resources to translate great ideas from workbench to marketplace.

There is no single cause or party responsible for this stagnation in the development of novel medical products. Rather, it reflects a number of factors affecting multiple stages of the development process, such as an uncertain and challenging economic and global marketplace, increasingly complex science and products, and the imperative to ensure the safety and efficacy of innovative medical products.

Medical Product Development Ecosystem

Translating a new idea from a discovery into a medical product is a complex process involving an entire ecosystem consisting of academia, industry, small businesses, payors, physicians, government agencies, and patient and consumer groups. Each member of the ecosystem has an important role to play in bringing a new medical product to market, and each piece of the ecosystem is currently under stress, putting America at risk of losing its competitive edge as the leader in scientific innovation.

It is possible for the U.S. to overcome the challenges ahead, but only by addressing each of the underlying problems. Just as there is no single factor responsible, there is no single solution. Innovation in medical product development transcends a single new drug or a single new device. There is a continuum of concerns that impact the environment for medical product innovation, including intellectual property and patent policies, economic policies, biomedical research and medical technology investments, regulatory reform, and reimbursement policies.

This is a holistic process that involves many different pieces and players. As such, we must all take a comprehensive, integrated approach toward a solution, involving the entire range of relevant players and approaching the ecosystem as a whole. There must be a dialogue among the various stakeholders to identify barriers to progress and better define what steps need to be taken to overcome these obstacles. By doing this, we can work together toward solutions that truly drive scientific innovation forward. As a nation, we have a unique opportunity to come together to develop an overarching strategy to spur medical product innovation.

FDA as a Driver of Innovation for Public Health

FDA has an important role to play in shaping the future of medical breakthroughs, by bringing stakeholders together to identify and overcome the challenges of product development and assessment. Within FDA, innovation can also be promoted by implementing reforms that adapt to the changing scientific and technological landscape to avoid an unnecessary delay of promising, safe and effective new treatments for patients.

Recognizing the need for action, Commissioner Margaret Hamburg, M.D., set out to develop an FDA Innovation Initiative to place a renewed emphasis on the Agency’s role as a driving force behind scientific innovation in America. This initiative is focused on identifying areas for reform within FDA that will translate to the medical product ecosystem at large.

Understanding that smart reform cannot happen without input from key players, FDA reached out to major stakeholders—the small businesses that serve as the backbone of the medical device and biotech communities, the large corporations developing the next breakthrough drugs to treat disease, the academic researchers whose work leads to new scientific discoveries, and the patients who are directly impacted by the benefits and risks associated with new medical treatments and therapies.

During the course of months of discussions with these groups to identify key areas for improvement, as well as internal analyses of current FDA processes, several common themes emerged. These include:

  • The need to do more to inform, engage, and help small businesses navigate the FDA regulatory process.
  • The need to adapt current FDA policies and procedures to address the scientific realities and opportunities presented by personalized medicine.
  • The need to take advantage of cutting edge information technology and scientific computing to enhance benefits to patients and the American public.
  • The need to address regulatory uncertainty within FDA.
  • The need to streamline FDA policies and procedures whenever possible.
  • The need to develop more efficient regulatory pathways to support devices and diagnostics, including highly innovative devices.
  • The need to build regulatory science capacity both within FDA and the broader medical development community.

In response to these themes and recommendations, the Commissioner identified the following major reforms to address the most immediate concerns in each of these areas:

  • Rebuilding FDA's small business outreach services.
  • Building the infrastructure to drive and support personalized medicine.
  • Creating a rapid drug development pathway for targeted therapies.
  • Harnessing the potential of data mining and information sharing, while maintaining strong privacy protections.
  • Increasing consistency and transparency in the medical device review process.
  • Training the next generation of innovators.
  • Streamlining and reforming FDA regulations.

As the agency responsible for the approval and regulation of medical products, FDA plays a critical role in American companies' ability to perform and compete in the global marketplace and deliver better products to patients more efficiently. We recognize the importance of maintaining America's presence as a global leader, and we are committed to modernizing our science and regulatory paths to promote innovation. The following blueprint outlines strategies to help reposition the U.S. medical product industry to adapt to the changing scientific landscape and drive innovation forward to provide Americans with cutting edge treatments and therapies that are safe and effective while simultaneously supporting economic growth and development in a critical sector of the economy.

Table of Contents: Driving Biomedical Innovation

Next Section: Rebuilding FDA's Small Business Outreach Services