Category Archives: Innovation

Early communication: A key to reduced drug development and approval times

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By: Anne Pariser, M.D.

From “test tube” to market typically takes a new drug more than 10 years.  FDA has been working hard at many points along a drug’s developmental path to reduce this time and bring safe and effective new therapies to Americans as efficiently as possible. 

Much has been said about FDA’s success in using its “expedited approval” tools, specifically Priority Review and Accelerated Approval, to support innovative new drugs. These are important tools that FDA can use once a marketing application is submitted. For instance, last year, FDA’s Center for Drug Evaluation and Research (CDER) approved 39 novel medications, almost half of which benefited from one (or both) of these expedited approval tools. According to a recent FDA report, this is a 63% increase over the average number of annual approvals since 2002. 

But less has been said about FDA’s “expedited development” tools, which help foster new drug innovation during the investigational phases of drug research and development, well before a marketing application for a new drug is even submitted to FDA. Among these tools are more frequent and earlier opportunities for communication between FDA and drug developers. FDA’s Fast Track designation for drugs with the potential to address unmet medical needs is an example. For many years, Fast Track has helped speed new drug development by encouraging more communication early in the development process. In 2012, about 40% of CDER’s novel new drug approvals were drugs that were given this Fast Track designation.     

Just this past year, the Food and Drug Administration Safety and Innovation Act (FDASIA) authorized FDA to use a new Breakthrough designation for investigational new drugs when preliminary clinical data suggest that the drug may provide a substantial improvement over existing therapies for patients with serious or life-threatening diseases. The concept behind Breakthrough is that, with increased communication, FDA will work with new drug developers to help design efficient ways to study the safety and effectiveness of their drug. This early assistance can help ensure that the results of clinical trials provide the evidence that FDA must have to determine whether or not a drug is safe and effective for approval. A growing number of drug developers are already taking advantage of Breakthrough.

But even before Breakthrough had been authorized by FDASIA, FDA was working to encourage communication opportunities for drug developers to meet with FDA to help make sure their clinical trial designs and development plans offered the best chances of efficient, safe, and timely development and approval. These opportunities are available at the start of a drug’s clinical development cycle: right before the earliest phases of human testing known as the “pre-investigational new drug (IND) phase” (fittingly called pre-IND meetings) and continue throughout drug development.

Early communication in action

Recently, FDA has taken a look at the development times of new drugs that were approved with the benefit of pre-IND meetings and compared them to the development times for drugs that were approved without such meetings. The findings underscore the value of early communication. For those new drugs for which a pre-IND meeting between the drug developer and FDA was held, average clinical development times were substantially shorter than when a meeting was not held. For instance, for all new drugs approved between 2010 and 2012, the average clinical development time was more than 3 years faster when a pre-IND meeting was held than it was for drugs approved without a pre-IND meeting.

For orphan drugs used to treat rare diseases, the development time for products with a pre-IND meeting was 6 years shorter on average or about half of what it was for those orphan drugs that did not have such a meeting. Early communication is especially important for orphan drugs because these products require special attention and thus early talks can be especially beneficial.

Many factors can influence the speed and efficiency of a drug development program. Nevertheless, FDA strongly believes in the value of effective communication during the drug development and approval process, especially for novel development programs when established regulatory pathways do not exist. FDA is committed to working with drug developers to ensure efficient and effective drug development programs whenever possible.

Thirty-nine novel new drug approvals last year is encouraging – one third of which are indicated to treat rare diseases – and many of these new drugs are now making valuable contributions to public health inAmerica. FDA will continue to do its part to help bring safe and effective new drugs to market as soon as possible. We will continue efforts to enhance communication as a critical part of the drug research, development, and regulatory process – especially since it is so clear that communications can make a big difference.

Anne Pariser, M.D., is Associate Director for Rare Diseases, Office of New Drugs, Rare Diseases Program at FDA’s Center for Drug Evaluation and Research

World AIDS Day

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By: CDR. Steve L. Morin, R.N., B.S.N.

World AIDS Day has been observed in the United States on December 1 since 1995. When I look back at early World AIDS Day observances, I remember them as a way of raising awareness of the men, women and children who had no advocates, no representation, no medicines, and practically no hope. They eventually died from the disease early in the epidemic.

In the beginning, World AIDS Day was an important platform for the HIV/AIDS community to help raise awareness among the many people who had never known or even met anyone living with HIV/AIDS. In those early years, the focus was on finding a treatment and keeping those diagnosed with the disease alive. 

Last year marked 30 years since AIDS was first reported in the Center for Disease Control and Prevention’s Mortality and Morbidity Weekly Report (MMWR), emerging as a permanent part of our lives. Today, when I think about World AIDS Day, I think of it as a day to acknowledge how far we have actually come in the fight against HIV/AIDS. We’ve come so far—not only in treatment, but also in preventing new infections, and reducing or eliminating the stigma associated with this disease. 

The Food and Drug Administration supports the fight against HIV/AIDS by promoting medical innovation, protecting the blood supply, and reviewing and regulating new and existing medical products, including devices used in prevention, such as condoms and medical gloves. Doctors, nurses, pharmacists, scientists and many others at FDA have worked hard in 2012 to make sure that there are safe and effective medical products and devices available to fight HIV/AIDS. I am happy to say that this year there were four major advances in the battle against HIV. 

  • Truvada is the first HIV drug approved for prophylactic (preventive) use. It has been shown to reduce the risk of sexual transmission of the HIV virus to uninfected adults.
  • OraQuick In-Home HIV Test is the first rapid home-use oral HIV test kit that does not require sending a sample to a laboratory for analysis. This test has the potential to identify previously undiagnosed HIV infections, especially if used by those unlikely to visit a doctor’s office or clinic.
  • Stribild is the first HIV medicine to combine four separate drugs and is the third HIV drug that can be taken once daily.
  • The number of antiretroviral drugs tentatively approved or approved for use under the President’s Emergency Program for AIDS Relief, or PEPFAR, has surpassed 150. PEPFAR is a program to treat those infected with HIV/AIDS in countries that lack the tools needed to fight the AIDS epidemic.

So today, as World AIDS Days approaches, I ask that you take a moment to remember the combined effort of patients, researchers, industry, FDA and other government agencies contributing to the successes in fighting HIV/AIDS. There are currently 36 approved therapies for treating HIV/AIDS in the United States. As new therapies are added to the list of treatments, patients’ quality of life has improved, with fewer side effects and simpler therapeutic regimens that make adhering to therapy easier. People living with HIV are now able to focus on life rather than death. Until there is a cure, we will continue to work together for an AIDS-free world.

CDR. Steve L. Morin, R.N., B.S.N., is a Health Programs Coordinator in FDA’s Office of Special Health Issues

A New Law Advances Public Health: New Web Page Tracks Progress

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By: Malcolm Bertoni and Leslie Kux

After Congress passes a law that affects how FDA carries out its public health mission, we must begin the task of implementing the law — that is, putting the law into effect and enforcing it.

For a major piece of legislation like the Food and Drug Administration Safety and Innovation Act (FDASIA), signed into law in July, this is a complex undertaking.  

Malcolm Bertoni

FDASIA is a 140-page law divided into 11 separate sections, officially known as “titles,” which address different aspects of drug and device law. FDASIA reauthorizes and makes some changes to user fee programs that provide FDA with the resources we need to maintain a predictable and efficient review process for human drugs, biological products (such as vaccines), and medical devices.  

FDASIA also creates two new user fee programs:  one for generic drugs and another for biosimilar biologics. These new programs will allow FDA to enhance its efforts to ensure that American consumers have more timely access to safe, high quality, affordable medicines. The law also gives the agency new authority to protect the safety of the drug supply chain, which is so important when these products arrive from all corners of the world; to combat drug shortages; and to improve products used to treat children. The law includes many other provisions, including those involving drug innovation and device regulation.

The requirements of the individual provisions vary; some direct FDA to write new regulations or guidance documents that will help industry meet the law’s requirements, while some call for the agency to issue reports or develop strategic plans. Some provisions set specific timetables for action, others don’t.

Leslie Kux

The successful implementation of FDASIA is one of our top priorities. To ensure its success, FDA set up a steering committee shortly after the law was passed to oversee the task of integrating the requirements of FDASIA into the agency’s ongoing workload. One of the committee’s projects has been to create a table that tracks what FDA must do to comply with the statute.

Today we are making available a website that will allow you to follow the agency’s progress in accomplishing the actions required by the new law. The website includes a table that lists information about FDASIA tasks such as the citation to the section of the law, a description of the task, the statutory due date, and the name of a primary contact person. The table will also include links to pertinent documents as they are completed and published.

Initially, the table will include only those requirements with a due date set by Congress. In 2013, other requirements that were not given a specific due date will be added, along with FDA’s target completion date.

FDASIA is an important law with significant provisions affecting industry, patients, consumers and health care providers. We will be updating the website on a regular basis as part of our commitment to transparency about our FDASIA implementation.

Malcolm J. Bertoni is FDA’s Assistant Commissioner for Planning

Leslie Kux is FDA’s Assistant Commissioner for Policy

Modernizing FDA’s Information Technology

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By: Eric D. Perakslis, Ph.D.

As I look back at my first year as chief information officer and chief scientist (informatics) at FDA, I am gratified with all of the progress that has been made. But I am also eager to transform initial wins into consistent and sustained success.

My team at the Office of Information Management (OIM) manages and advances information technology at FDA. Our mission is to provide the organizational structure and services that allow the exchange of information, communication and knowledge that enhance and sustain FDA’s ability to protect and promote the public health.

Our vision is that FDA’s technology and knowledge capability is modern, secure, accessible, cost-effective, and exceeds customer and partner expectations.

Some of our early successes include important contributions to what we call the Innovation Pathways 2.0 initiative, which provides a rapid new way to submit breakthrough medical devices to FDA for approval. This in turn would get such products faster to the patients who need them.

OIM also completed 20 projects in very short time in support of the Food and Drug Administration Safety and Innovation Act (FDASIA), signed into law by President Obama in July 2012. Many of these projects strengthen the process of reviewing and approving important new medical products, while making that process more transparent to the tax-paying public.

In addition, this year we launched a new strategic effort to improve the overall “customer experience” with FDA’s information technology, customers in this case being FDA employees stationed all over the world. Our goal is to provide the technology to ensure that FDA employees can reliably perform their daily duties efficiently and timely regardless of location, whether they’re in an office or in the field, or at home working remotely.

And our improvements to the process of analyzing the health risks presented by medical products of all kinds have helped drive the modernization of the FDA inspection process. This helps ensure that the growing number of products we regulate have a higher level of safety and quality before they reach American consumers.

While these accomplishments are significant, there is still much to be done to modernize information technology at FDA.

The new FDA Information Management Strategic Plan sets the path forward. Its priorities include strengthening real-time connectivity and access to key data and information. This is essential for daily FDA operations and for our connections to the public we serve and to our many partners outside of the agency who depend on FDA for the execution of their own public health missions.

The Strategic Plan also focuses on the availability and usability of data essential to the speed and efficiency of decision-making at FDA. The intent of this goal is to facilitate a learning and knowledge network that will enable FDA to assess the potential risks of regulated products on a scale that routinely handles global sources and large volumes of data. 

Going forward, we will do our best ensure that FDA’s technological needs today and tomorrow are fully covered and in place. There is no more inspirational public health mission than that of the FDA. Every citizen of this great nation depends on us every day and we will never forget that. In many ways this Strategic Plan is as much about what we believe, and our commitment to our customers and mission, as it is about what we deliver.

Eric D. Perakslis, Ph.D., is FDA’s Chief Information Officer and Chief Scientist for Informatics

Treating Children with Cancer

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September is National Childhood Cancer Awareness Month. Watch the below video in which two FDA experts discuss existing and new efforts to encourage the development of medicines for kids with cancer.

The conversation is between Robert “Skip” Nelson, M.D., Ph.D., deputy director and senior pediatric ethicist in FDA’s Office of Pediatric Therapeutics, and Gregory Reaman, M.D., associate director of the Office of Hematology and Oncology Products.

It begins with a discussion of FDA’s role in evaluating medications used to treat children with cancer and what measures are underway to encourage further development of these important drugs.

For More Information

Pediatrics

New Pediatric Labeling Information Database

Cancer Liaison Program

Office of Hematology and Oncology Products

Friends of Cancer Research Honors Our Dr. Richard Pazdur

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By: Margaret Hamburg, M.D.

One of the privileges of being the head of the Food and Drug Administration is being able to report on the accomplishments of the dedicated and highly skilled employees who are advancing the public health mission of our agency. Last night, I had the pleasure of participating in an event honoring one of our scientists for work that makes us particularly proud: his outstanding contribution to the fight against cancer.

Margaret Hamburg, M.D. and Richard Pazdur, M.D.Dr. Richard Pazdur, the director of our Office of Hematology and Oncology Products, joined Senator John McCain and Neera Tanden, president of the Center for American Progress, as a recipient of the Cancer Leadership Award from the Friends of Cancer Research, an important organization that helps advance medical progress.

Senator McCain and Ms. Tandem have been tireless advocates for public investment in cancer research, getting cancer drugs to market more quickly, and developing prevention strategies that are known to reduce the risk of cancer. Dr. Pazdur, who was honored for his leadership in cancer research, makes sure that the drugs used to treat cancer patients are not only available for patients who need them as fast as science will allow, but above all, that they are safe and effective.

Achieving these three goals requires highly sophisticated and frequently very difficult decisions. Thanks to Dr. Pazdur, who arrived at FDA more than a decade ago as a highly regarded physician and researcher, our agency is up to the task. He has helped make FDA a more transparent, innovative and compassionate organization grounded in solid science.

At FDA, we appreciate both his professional and personal qualities. As an FDA official and scientist, Dr. Pazdur explores hard data with industry, academia, and patient advocates in search of innovative approaches to expediting the approval of new drugs. When talking to cancer patients and their advocates, Rick Pazdur, the physician, responds with deeply-felt empathy. But even this great compassion does not weaken his resolve to perform his foremost duty, which is to ensure that the benefits of cancer drugs are not outweighed by their risks.

Dr. Pazdur’s leadership is a hallmark of FDA’s pursuit of science in the service of public health. I am proud to congratulate him on his richly deserved award on behalf of our entire agency.

Margaret Hamburg, M.D., is Commissioner of the U. S. Food and Drug Administration

FDA Voice Interviews Jesse Goodman, M.D., M.P.H., on the DARPA and NIH Project Collaboration: Human on a Chip

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FDA Voice: FDA has embarked on an exciting collaboration with the Defense Advanced Research Projects Agency (DARPA) and NIH—to develop a groundbreaking tool that could help bring new treatments to patients faster, more cheaply, and more safely. Can you talk about this new technology?

Dr. Goodman:  Yes, it’s what we’re calling Human on a Chip. This is an ambitious project to create a tool that could revolutionize toxicology testing and it’s something I’m really excited to talk about.  Scientists have relied largely on animal studies to determine if a drug is toxic before testing it in humans.  And while animal testing is useful, it’s also expensive, time consuming, and has drawbacks. For example, it doesn’t always detect toxic effects specific to humans and doesn’t usually provide information about the role that genetic differences within human populations play in toxicity. It can also generate false alarms, showing an effect in animals that doesn’t predict an actual effect in people, which leads us to abandon promising new drugs. FDA is collaborating with DARPA, NIH, and the scientific community to spur innovation in this field by exploring how tools like Human on a Chip can be integrated into our development tool box to improve testing for toxicity and potentially reduce the need for animal testing.

FDA Voice:  Can you describe Human on a Chip?

Dr. Goodman: Researchers are developing microsystems using human cells to test the effects of drugs or other substances. For example, scientists have developed a micro machine chip with human lung cells that grow on a surface to form a lung-like tissue that has both air spaces and blood circulation. FDA is supporting the coupling of this chip to a heart-like chip that beats and pumps blood. We can use this type of system to evaluate, with human cells, how specialized organs like the lung and heart react to a specific chemical.

The Human on a Chip builds on this approach. FDA’s collaboration with NIH and DARPA aims to create a 3D representation of 10 different human organ systems that mimic the processes and activities of those systems, potentially linking them to form a system with major features of human biology. For instance, in a living human, the interactions of heart, lung, kidney, and liver are crucial in the functioning of all 4 organs, and all are common targets of toxicity. A tool that creates and links organ-like systems will enable scientists to observe a substance’s effects on several interacting systems simultaneously. This can make it possible to test for beneficial effects as well as for toxicity. 

Once these systems are refined, if successful, they could not only improve testing beyond currently available tools, but could also be engineered to mimic disease states or be implanted with cells with a specific genetic background that is involved in specific diseases or drug interactions.

FDA Voice:  Why transform toxicology testing?

Dr. Goodman: Toxicity has been a major challenge in medical product development and in assessing environmental hazards. Technologies like Human on a Chip could help shrink the time frame it takes for new treatments to move to human testing and approval. These new tools can help identify toxicity earlier in product development, thus protecting patients, lowering development costs, and speeding new treatments to patients in need.

Human on a Chip could also contribute to developing medical countermeasures because the diseases and conditions we might need to treat in a public health emergency—like anthrax, smallpox, pandemic influenza, and radiation and toxin exposure—rarely occur naturally, often making animal models the only available tools for evaluating a new treatment’s effectiveness.

FDA Voice:  What makes FDA essential to this collaboration?

Dr. Goodman:  Our FDA scientists have vast experience using available tools to make tough scientific decisions about the safety and effectiveness of a multitude of products. They’ve seen what works and what doesn’t, and thus can provide insights and help solve challenges in defining how best to develop and evaluate new tools. Before accepting a new tool for use, FDA scientists must have the needed scientific data on how it performs to ensure that it is as safe and effective as possible. Once FDA accepts a scientific tool, industry can use it for its qualified purpose during product development.

FDA Voice:  In what other ways has FDA worked to drive innovation in toxicology testing?

Through the Critical Path and Advancing Regulatory Science initiatives, we are working to harness the use of new science and technology to transform regulatory science and help get needed products to people quickly and safely. FDA identified transforming toxicology as one of the eight priority areas where collaborative regulatory science research is essential and offers huge opportunities. In addition to Human on a Chip, FDA is collaborating with other Federal agencies, academia, and industry to bring new science to toxicology, such as on the cell-based Tox-21 project with EPA and NIH, and on FDA grants to evaluate cell-based approaches to evaluate risks of reproductive and developmental toxicity. 

My office has also formed a new FDA-wide council, together with scientists from across the agency, to explore, promote, and coordinate efforts concerning chemical and toxicology-related issues. FDA’s partnership throughout the development and evaluation cycle is critical to ensuring that exciting new tools and approaches like Human on a chip speed the delivery of safe and effective new treatments to people who need them.

Jesse L. Goodman, M.D., M.P.H., is FDA’s Chief Scientist and Deputy Commissioner for Science and Public Health

FDA: Reflection on 2011 and Looking Forward to 2012

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By: Margaret A. Hamburg, M.D.

The beginning of a New Year is a wonderful time to reflect on the past year and to think about what lies ahead. While 2011 had its share of challenges for us at FDA, it was also a time of significant accomplishment. We are excited about the many opportunities that 2012 will bring to the Agency.

2011 began with the most sweeping reform of our food laws in more than 70 years. FSMA promises an even safer food supply than we enjoy today and a wonderful opportunity to help promote public health.  We also continued the hard work of implementing the Family Smoking Prevention and Tobacco Control Act.

Margaret Hamburg On the medical product front, there were a multitude of outstanding activities and advances.  Importantly, we renewed our focus on the importance of innovation, in our own work and in the products we are tasked to review.  This was also a remarkable year for innovative drug approvals.  During Fiscal Year 2011, the FDA approved 35 novel drugs, targeting diseases such as late-stage lung cancer, metastatic breast cancer, and hepatitis C.

We also worked to continually strengthen the science that guides our policies, including many important collaborations with key partners and stakeholders.  Even with all of our ongoing work, when crises came in 2011, we acted swiftly to protect the American people.  We also continued to strengthen our capacity to address one of the greatest challenges in this new century – our increasingly globalized world.  “Pathway to Global Product Safety and Quality”

In 2012, FDA will meet new and complex challenges and continue our work in implementing FSMA and the Tobacco Act, promoting advancements and innovation in medical products, and broadening our strategic globalization and regulatory science initiatives. We will be working closely with Congress and stakeholders to support the reauthorization of user fees for pharmaceutical products (PDUFA), and medical devices (MDUFA), as well as to authorize new user fees for generic drugs (GDUFA) and biosimilars.

I am confident, thanks to the dedication of the employees of the FDA, that we will continue to make invaluable contributions to the lives of you, the American people.

I wish you and your family a healthy and happy 2012.

Margaret Hamburg, M.D., is Commissioner of the U. S. Food and Drug Administration.

FDA Voice

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By: Margaret Hamburg, M.D.

I’d like to introduce you to FDA Voice – FDA’s newest communications vehicle designed to give you information from behind the scenes here at FDA, and in our own words.

FDA Voice is a new forum for us.  Although I am writing the first blog post, in the future you will see posts from a wide range of FDA officials and staff, including scientists and public health professionals, employed at headquarters here in the Washington DC area, across the United States, and in posts abroad in countries as far away as China and India.

Margaret HamburgThrough FDA Voice, we hope to give you insights on some of the most pressing public health issues of the day. For example, how does globalization affect the food that we eat? How is the pace of innovation affecting the availability of new medical treatments for you and your family? What does personalized medicine mean for me? And even, what is the latest on food safety for my pet?

I believe that providing a forum like this is especially important for an agency like FDA, where the products that we regulate are directly related to public health and the issues that we work on tend to be complex in nature. FDA Voice will give FDA employees the opportunity to go beneath the surface to tell you about what they are working on and how it impacts your life.

No matter which stakeholder group you consider yourself a member of – consumer, patient, medical professional, scientist, researcher, or industry – it is our hope that you will find this medium an unparalleled opportunity to get more details directly from the source, like details of an official trip, activities in the lab, or more background information about a policy issue or regulation.

You will find FDA Voice on our homepage – and you may notice some additional changes there today as well. We have given the homepage a cleaner look and feel – by updating the design and reducing the number of links on the page. We have been working hard to improve your experience at FDA.gov, and hope that the changes we have made will help you find the information you need as quickly and easily as possible.

As Americans living in the Information Age, we are always looking for new and better ways to communicate. I am glad that you decided to take time out of your day to stop by and read FDA Voice, and I hope you will make it part of your routine in the future.

On behalf of all of the employees at FDA, I wish you and your loved ones a very happy holiday season.

Margaret Hamburg, M.D., is Commissioner of the U. S. Food and Drug Administration.