FDA’s multi-pronged approach helps meet the challenge of bringing new and innovative antibiotics to patients who need them

By: Edward M. Cox, MD, MPH

With a growing number of infections becoming increasingly resistant to our current arsenal of antibiotics, developing new antibiotics to treat serious or life-threatening infections has become a key priority.

Edward Cox interview

There are significant scientific and economic challenges inherent to the development of new antibiotics. From a scientific standpoint, many patients with bacterial infections are often very sick and need to begin antibiotic therapy immediately, without further complications that enrollment in a clinical trial might involve. Moreover, it can be difficult to conduct a clinical trial involving very sick patients.

From an economic standpoint, antibiotics may be perceived as less potentially profitable for a company because they are generally taken only for a short period of time and often only for one course of treatment, by any given patient. Compare this to the long, dependable income stream from a diabetes medicine or a blood pressure medicine that a patient takes indefinitely, often for the rest of their life. These economic realities, which are rooted in the biology of acute bacterial infections, can make it challenging for a company to justify large expenditures for the development of drugs in this area, as a recent report by Eastern Research Group (ERG) affirms.

Provisions in a law passed a little over two years ago, commonly known as the GAIN Act, or the Generating Antibiotics Incentives Now Act, is helping to stimulate the development of new antibiotics. Under GAIN, certain antibacterial or antifungal drugs intended to treat serious or life-threatening infections can be designated “Qualified Infectious Disease Products” (QIDPs). As part of its QIDP designation, a drug receives priority review and can also receive fast track designation at the sponsor’s request. At the time of approval, a product with QIDP designation may be eligible for an additional five years of marketing exclusivity, exclusive marketing rights without competing with a generic drug product. To date FDA has granted 52 QIDP designations to 35 different unique molecules. We are already beginning to approve new antibacterial drugs with this beneficial QIDP designation.

FDA is working hard to streamline requirements for clinical trials for studying new antibacterial drugs and the provisions of the GAIN act are being actively implemented, but more is needed. There are still significant economic and scientific challenges in the development of new antibacterial drugs that need to be addressed. Additional financial incentives as well as new approaches for studying antibacterial drugs such as common clinical trial protocols could provide other important means to stimulate antibacterial drug development. We also need cutting-edge science to stimulate the development of new and innovative antibacterial drugs. To help drive this effort, FDA has assembled our Antibacterial Drug Development Task Force, a group of expert scientists and clinicians from within FDA, to consider opportunities to promote antibacterial drug development.

To advance this field, our Task Force is working with many leaders including those drawn from academia, regulated industry, professional societies, patient advocacy groups and government agencies. For example, FDA has contributed to the efforts of the Biomarkers Consortium of the Foundation for the National Institutes of Health to develop new endpoints for studying antibacterial drugs. FDA also works closely with the Clinical Trials Transformation Initiative (CTTI), a key group of dedicated scientists focused on advancing clinical trials for more efficient drug development. As a result, FDA and CTTI have helped convene a variety of important scientific meetings and activities on vital topics related to efficient clinical trial designs for testing new antibiotics. Our Task Force has also helped FDA team up with colleagues at the Brookings Institution’s Engelberg Center for Health Care Reform to help galvanize the scientific community’s efforts in new antibiotic drug development. August, 2012 began the first Brookings Council for Antibacterial Drug Development (BCADD) meeting, with meetings that occur approximately twice a year.

FDA and our Task Force members have also been busy on our own.  In February of 2013 we held a public meeting focused on creating an alternative approval pathway for certain drugs, such as antibacterial drugs, that are intended to address unmet medical need. We have also asked the public for their thoughts; in March of 2013, we issued a Federal Register Notice seeking input from the public on a wide range of topics related to antibacterial drug development. FDA has generated a number of guidance documents for industry, in draft and final form, that describe FDA’s scientific thinking with regard to developing new antibacterial drugs.

As part of our Task Force’s collaborative efforts, FDA is working closely with The National Institutes of Health (NIH) to further advance the development of new antibacterial drugs. Together, we are hosting a two-day Public Workshop to identify strategies for promoting clinical trials for antibacterial drugs and encouraging partnerships to accelerate their development. The ERG report will be presented at the workshop and other specific issues will be discussed including:

  • Priorities and strategic approaches to conducting clinical trials for antibacterial drugs
  • Regulatory pathways—including streamlined development programs for antibacterial drugs for patients with limited or no treatment options
  • Clinical trial design issues such as the development of common clinical protocols; using common control groups; statistical analysis issues; sharing data across trials (and data standards); appropriate clinical trial endpoints; and lessons learned from other therapeutic areas
  • The role of public-private partnerships in advancing the scientific and clinical trials enterprises

The work of the FDA Task Force as well as the GAIN Act have provided good first steps toward strengthening the antibacterial drug pipeline, but as the findings from the ERG report indicate, the forecast for antibacterial drug development likely will include a less than robust pipeline. Thus, additional attention on both financial incentives, new approaches for studying antibacterial drugs such as common protocols, as well as streamlined development pathways, likely will be needed to improve the climate.

Edward M. Cox, MD, MPH, is Director, Office of Antimicrobial Products, in FDA’s Center for Drug Evaluation and Research

FDASIA at Year Two

By Margaret A. Hamburg, M.D.

Margaret Hamburg, M.D.Anniversaries are a time for stock-taking and today, on the second anniversary of the Food and Drug Administration Safety and Innovation Act or FDASIA, I’m pleased to report on the progress we’ve made implementing this multi-faceted law.

To date, we have completed nearly all of the deliverables we had scheduled for the first two years after FDASIA became law. And many of the new authorities under FDASIA are already having a positive impact on health. It’s difficult to cover all of our FDASIA work, but here are some highlights:

Preventing Drug Shortages: Drug shortages, which can have serious and immediate effects on patients and health care professionals, reached an all-time high in 2011, the year before FDASIA was enacted. In response to a Presidential Executive Order in December of that year, FDA issued an interim final rule to amend and broaden FDA regulations requiring certain manufacturers to give early notification of production interruptions that could cause drug shortages. FDASIA further broadened this requirement by requiring that other prescription drug manufacturers provide notification and also gave FDA additional authorities. In October 2013 FDA proposed a rule to implement these authorities and issued a strategic plan for addressing drug shortages. So far, with the help of early notifications, FDA was able to prevent 282 shortages in 2012 and 170 shortages in 2013. The number of drug shortages that did occur has also declined.

Promoting Innovation: FDASIA includes many provisions designed to encourage innovation. We have held meetings on the use of meta-analyses in drug applications; put in place a plan for implementing a benefit-risk framework for drug reviews, and issued a variety of guidance documents covering such topics as drug studies in children, abuse-deterrent drug development, antibacterial drug development and expedited review and development programs for serious diseases.

This latter guidance provided information that sponsors needed to know about our new Breakthrough Therapy designation that was part of FDASIA. This option exists for new drugs intended to treat a serious or life-threatening disease that, preliminary clinical evidence suggests, could provide a substantial improvement over available therapies. As of June 23, we had granted 52 requests for this designation, and of those, approved four new drugs and two new indications for previously approved drugs.

As part of our implementation of the FDASIA-related provisions related to medical devices, we proposed a strategy and recommendations for a risk-based health information technology (health IT) framework that would promote product innovation while maintaining appropriate patient protections and avoiding regulatory duplication; issued a proposed rule for implementing FDASIA’s streamlined new procedures for reclassifying a device; and published a final rule on a medical device unique identification or UDI with implementation in accordance with the timetable set in the law. UDIs will help the FDA identify product problems more quickly, better target recalls and improve patient safety. The riskiest medical devices will start bearing their UDI by September 24th.

Establishing and Strengthening User Fee Programs: An important element of FDASIA was reauthorizing user fees for prescription drugs and medical devices and creating new user fee programs for generic drugs and biosimilar biological drugs. User fees on some types of applications offer an important source of funding to support and maintain key activities, including FDA’s staff of experts who review the thousands of product submissions we receive every year. Since FDASIA took effect, review times for medical devices have been declining.  Our prescription drug user fee program is meeting or exceeding almost all of our performance goals agreed to with industry. We have acted on 54 percent of the generic drug applications, or amendments and supplements to generic drug applications which were pending in our inventory as of October 1, 2012. This helps ensure that consumers can have access to more low-cost drugs. And we have been able to provide advice concerning most of the 93 submissions from companies who are developing biosimilar biological drugs under a pathway that could also ultimately lower costs for consumers.

Enhancing Patient Engagement: A hallmark of FDASIA was a series of provisions intended to tap the patient perspective. Our Patient-Focused Drug Development Program allows us to more systematically obtain the patient’s perspective on a disease and its impact on the patients’ daily lives, the types of treatment benefit that matter most to patients, and the adequacy of the available therapies for the disease. In accordance with FDASIA, we have held patient meetings on eight diseases and have plans for meetings on 12 more. We have learned a great deal from patients in terms of their views of the symptoms of their condition, their feelings about how it affects their life, and their thoughts on ideal treatments and on participation in clinical trials to aid future drug development.  A FDA Voice blog post on patient reports captures these patient perspectives and much more.

Finally, Title VII of FDASIA provided FDA with numerous new authorities to protect the drug supply chain. We thought now was a good time to provide the public with a more detailed description of our work on Title VII, so we asked Howard Sklamberg, Deputy Commissioner for Global Regulatory Operations and Policy, to write a separate blog on that topic.

FDA laid out a three-year plan for implementing FDASIA and we’re on our way to achieving our stated goals. To help the public follow our progress, we set up a dedicated webpage—the FDASIA-Track. It provides useful links to each action and is updated on a regular basis.

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

FDA and Health Professionals, Safeguarding the Public’s Health

By: Anna M. Fine, Pharm.D.

At our recent third annual Health Professional Organizations Conference, some of FDA’s most senior leaders exchanged views and discussed issues of mutual interest with senior representatives from key health professional organizations.

Anna FineHeld on FDA’s White Oak campus in Silver Spring, Md., and organized by the FDA’s Office of Health & Constituent Affairs (OHCA), the event was attended by 30 professional organizations representing physicians, nurses, physician assistants, dentists, optometrists, nurse practitioners, pharmacists, and others.

An open and ongoing dialogue between these professionals and FDA is a vital part of addressing many important public health issues. In her opening remarks, FDA Commissioner Margaret Hamburg offered a few examples, such as health professionals’ contributions to the FDA’s MedWatch and Adverse Event Reporting programs and their work in interpreting and addressing medical products’ safety signals. A drug’s safety profile is continually evaluated after FDA approval, and health professionals are encouraged to report suspected adverse events to FDA which allows FDA to conduct comprehensive safety evaluations. Dr. Hamburg also emphasized the importance of health professionals’ engagement in regulatory science research, which provides essential support for the agency’s decisions and ability to bring innovative products to market.

Mitch Zeller, the Director of FDA’s Center for Tobacco Products, speaking at the third annual Health Professional Organizations Conference, on May 14, 2014

Mitch Zeller, Director of FDA’s Center for Tobacco Products, speaking at the agency’s third annual Health Professional Organizations Conference. See more photos of this event on Flickr.

Key FDA leaders who gave presentations throughout the day included Mitch Zeller, the Director of FDA’s Center for Tobacco Products; Dr. Stephen Ostroff, Acting Chief FDA Scientist; and Dr. Peter Lurie, Acting Associate Commissioner of FDA’s Office of Planning and Policy.

In addition, senior scientists from FDA’s centers for drugs, medical devices and food discussed FDA’s priorities and answered questions from the audience. The robust dialogue between the panel members and our stakeholders covered many public health issues including youth and tobacco and FDA’s proposed changes to the food label.

Feedback from the audience highlights the need for such a conference.

“It’s great to have this dialogue with FDA officials. It demonstrates that they respect our organizations and want our feedback,” said one stakeholder representative.

“I love coming to these annual meetings, not only to meet FDA personnel but to talk with colleagues in other professions. This is a one-of-a-kind forum,” said another.

As a pharmacist and team leader within OHCA, I can attest to the fact that my FDA colleagues and I benefited as well. We learned a lot about our stakeholders’ concerns and established new connections with health professional organizations—contacts that we plan to follow-up on to explore new opportunities for mutual cooperation and collaboration in the interest of the public health.

Anna M. Fine, Pharm.D., is Director of the Health Professional Liaison Program in FDA’s Office of Health and Constituent Affairs.

Ensuring Pharmaceutical Quality Through International Engagement

By: Howard Sklamberg, J.D.

As we’ve written and spoken so much about, the FDA has had to transform itself from a domestically-focused regulatory agency into a 21st century global health organization.  This transformation has come in the face of economic and technological changes that have revolutionized how we carry out our mission. We live in a world where other countries increasingly produce—at least in part—the food and medical products our consumers and patients use in their daily lives.

Howard SklambergProducts the FDA regulates now come from more than 150 countries—many with much less sophisticated regulatory systems than our own. In this international marketplace, 40 percent of our finished drugs are imported, and approximately 80 percent of the manufacturers of active pharmaceutical ingredients used in the United States are located outside our borders.

Ensuring the quality of products in a global environment is a tall order. At every stage in the production of pharmaceutical products, and all along the global supply chain, things can go wrong.  Products can be improperly formulated, manufactured, or packaged. They can be contaminated or counterfeited. And the challenges are multiplied when the supply chain stretches around the world.

FDA is on the ground, around the world, inspecting facilities, developing relationships and providing advice.

But securing the global supply chain requires more than that. It calls for a cooperative and worldwide endeavor. It means working with our regulatory counterparts abroad to build capacity. It means harmonizing our standards for the sake of safer products and greater efficiency. It means engaging with industry and with regional and international organizations.

The Food and Drug Administration Safety and Innovation Act (FDASIA), which Congress enacted in 2012, included some important provisions designed to improve the safety and integrity of imported drugs sold in the United States. Some of the provisions are focused on FDA’s inspectional activities overseas. For example, FDASIA increases FDA’s ability to partner with foreign regulatory authorities to leverage resources through increased information-sharing and recognition of foreign inspections.

We now have more than 60 agreements with foreign counterparts to share certain information in inspection reports and other non-public information that can help us make better decisions about the safety of foreign products.

This type of collaboration not only increases our ability to evaluate pharmaceutical facilities, but allows experts to learn from each other. The result: an outcome whose sum total exceeds its individual parts.

That is exactly why today we announced an initiative to expand on our existing work to ensure that the public has access to quality pharmaceuticals. Through this initiative, and in cooperation with the European Commission (EC) and the European Medicines Agency (EMA), FDA will aim to deepen our reliance on trusted regulators outside of the U.S. who provide equivalent public safety and quality protection.

This mutual reliance initiative builds on our existing relationships with the EC, the EMA, and member states of the European Union. Under this new initiative, the goal is to increase our exchange, with the EC and the EMA, of information that is critical to making decisions that protect the public health. And together we will be more efficient and effective in targeting our resources for inspecting pharmaceutical operations.

This is the latest step in our continuing efforts to improve the quality of pharmaceutical products – a step that will deploy a dedicated FDA team to work with our European counterparts on a host of issues. The team, which will focus full time on pharmaceutical quality, will include experts from our Center for Biologics Evaluation and Research, our Center for Drug Evaluation and Research, and our Office of Global Regulatory Operations and Policy.

As a public health regulatory agency with a global presence, we look forward to strengthening our mutual reliance and capitalizing on our shared interests. The initiative we embraced today signals yet another important step forward for pharmaceutical quality here in the U.S.—and around the world.

Howard Sklamberg, J.D., is FDA’s Deputy Commissioner for Global Regulatory Operations and Policy

The Commissioner’s Fellowship Program: A Win-Win for FDA and Public Health

By: Dr. Stephen M. Ostroff

As part of my FDA Voice blog series on the important work going on in FDA’s Office of the Chief Scientist (OCS), I’d like to highlight an FDA program that is giving top-tier, early career health care professionals, scientists, and engineers the chance to gain broad exposure to FDA regulatory science and scientific review opportunities. Led by OCS’s Office of Scientific Professional Development, the Commissioner’s Fellowship Program (CFP) is accepting applications from April 16 to May 26, 2014. Those who are accepted into the CFP will be joining FDA’s 7th class of Fellows.

Stephen OstroffDuring the two-year program, Fellows complete rigorous graduate-level coursework and conduct cutting-edge research on targeted scientific, policy, or regulatory issues under the mentorship of an FDA senior-scientist preceptor.

In the CFP, a Fellow is able to gain real experience in an FDA biology, physics, or engineering lab, work with a clinical review team, or work at a regional field laboratory or office. The coursework provides a common core understanding of the science behind regulatory review, encompassing activities across all FDA-regulated product areas.

Specific Fellow projects may focus on FDA review of sponsor applications for new products, monitoring product quality and safety, or other scientific or engineering topics. Fellows work closely with FDA scientists to create better research and evaluation tools and approaches, like assays for chemical or pathogen detection, or methods to assess clinical or health care data. Other science and policy areas of focus may involve foods or medical products in disciplines ranging from laboratory sciences to engineering, law, and ethics.

FDA launched the Fellowship Program in 2008 to achieve three critical goals:

1)      Attract to FDA top-tier scientists who can help tackle targeted regulatory science areas;

2)      Provide regulatory science training to expand the pool of experts;

3)      Recruit top scientific talent — scientists who may not have considered FDA in planning their career.

Since the program started, FDA has graduated 164 Commissioner’s Fellows, 75% of whom continued to work at FDA after completing the program. Our graduates have produced 175 publications based on their Fellowship work, represented FDA with 211 regulatory science presentations, authored or co-authored 917 reviews – ranging from original applications to supplements – and 26 Fellows have been the proud recipients of FDA Honor Awards.

The Fellows have brought an infusion of innovative ideas, new talents, and skills to FDA to help build the strong scientific foundation we need in our research and review activities. In turn, the CFP has enabled Fellows to develop their regulatory expertise and work confidently in the FDA environment.

Those Fellows who pursue careers outside FDA bring a deeper understanding of regulatory science and of FDA to their organizations. They enrich the regulatory science enterprise, whether by improving the quality of applications to FDA or by applying the knowledge and tools they’ve acquired through the CFP to develop practical solutions to an important public health challenge.

Stephen M. Ostroff, M.D., is FDA’s Acting Chief Scientist

For more information on eligibility criteria for the FDA Commissioner’s Fellowship Program and to apply for the upcoming class, please visit this Web link:

FDA Commissioner’s Fellowship Program Application Checklist

Visiting India: Sharing a Vision for Strengthening Food and Medical Product Safety

By: Margaret A. Hamburg, M.D.

Fresh mangos, bananas and other native fruits add a pop of color and provide the backdrop while we ride along the busy streets of Delhi. While en route to the first of several meetings I held with Indian regulators, I can’t help but marvel at the vibrant buzz of India’s capital and the progress that has been made since I traveled here years ago as a young woman. Since that time, the rapid globalization of commerce has posed significant challenges to ensuring consumer safety as the number of products and suppliers entering the U.S. has increased. India now represents the 3rd largest trade partner, 2nd largest supplier of over-the-counter and prescription drugs, and 8th largest supplier of food to the United States.

Margaret Hamburg and officials in India

(L-to-R) Arun Panda, Joint Secretary, Ministry of Health and Family Welfare; Shri Keshav Desiraju, Secretary, Ministry of Health and Family Welfare; Shri Ghulam Nabi Azad, Minister, Ministry of Health and Family Welfare; Dr. Margaret A. Hamburg, M.D., Commissioner of the U.S. Food and Drug Administration; Dr. Altaf Lal, Director of U.S. FDA’s India Office; Nancy Powell, U.S. Ambassador to India.

On Monday, I began my first official visit to the country as Commissioner of the FDA. I met with officials from the Indian government who oversee the country’s health-related matters as well as those responsible for overseeing the export of foods to the U.S. and more than 200 countries around the world. These meetings provided the opportunity for me to discuss our shared vision for strengthening the quality of the foods and medical products exported from India to the United States. Ultimately this vision is intended to enhance consumer confidence in these products both at home and abroad.

As two of the largest democracies in the world, our countries have enjoyed an enduring partnership and commitment to collaborate on initiatives designed to enhance both our economies and the lives of the people in our respective countries.

Ensuring that the products distributed in the United States meet our requirements for product safety and quality is among my top priorities as Commissioner. Unfortunately the many Indian companies that understand good manufacturing and quality processes have been overshadowed by recent lapses in quality at a handful of pharmaceutical firms.

While the FDA will take appropriate action against any company that doesn’t meet our requirements, we are also willing to work with them to address their issues. All consumers deserve access to safe and affordable drugs and should not have to sacrifice quality to get that.

Officials at India’s Ministry of Health and Family Welfare share this goal. In the spirit of continued collaboration and a commitment to quality, our agencies signed the first-ever Statement of Intent. Our organizations plan to collectively work together to improve the lines of communication between our agencies and work diligently to ensure that the products being exported from India are safe and of high quality.

While the Statement of Intent is an important milestone, I am proud to report that FDA’s Office in India has already been working closely with India’s drug regulators to reinforce the importance of producing quality products for patients. Drug and food regulators in India have participated in FDA-hosted workshops and observed FDA inspections of manufacturing facilities and clinical sites with operations in India.

During my visit I am eager to learn more about the industries that produce products for the United States and to meet with business leaders where I will reinforce our expectations that they meet our requirements for ensuring that consumers here and around the world have access to safe and high-quality products.

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

Interested in a Science Career at FDA? Our Web Portal Opens the Door

By: Jesse Goodman, M.D., M.P.H.

As a physician and a scientist, I value being part of a rich, vibrant scientific community. Since coming to FDA, I’ve been gratified to help lead and support a large group of talented, dedicated scientists who are passionate about what they do. Because science is at the core of everything we do at FDA, the majority of FDA’s staff are scientists, including engineers and medical professionals. A robust scientific workforce strongly engaged in the new sciences is critical to the success of FDA’s mission to protect and promote the public health.

Jesse GoodmanUnfortunately, I am often reminded that many people are unfamiliar with the cutting-edge research going on at FDA, and how important top-notch science and research are to our mission. Scientists, including those who may be exploring career options, are often unaware of the myriad scientific disciplines and expertise FDA must have to advance and apply the science required to assess the increasingly complex products we regulate. The outside world knows too little about the many innovative activities FDA scientists engage in, often with a wide range of collaborators, to keep our foods and medicines safe and help speed new therapies from bench to bedside.

So I’m delighted that we have now launched a comprehensive, one-stop web portal – FDA Science Careers and Scientific Professional Development - to showcase FDA’s exciting, multidisciplinary scientific work force and culture and how important science is to our public health mission. We want scientists – from students, to recent grads, mid-career, and seasoned professionals – to be aware of the diversity of FDA’s career opportunities so that we may continue to attract top scientists, including engineers and medical professionals who want to use their expertise to make a real difference in the world.

Entering our career portal, you will have access to the latest information on our fellowship programs and our internship, graduate, and faculty programs, which help us attract outstanding scientific academic talent. These opportunities include:

Once on board, FDA scientists benefit from a dynamic, state-of-the-art, scientific professional development culture, with daily opportunities to attend a variety of scientific courses, seminars, and workshops. You can find out more about FDA’s efforts to support scientific professional development through the career portal’s Training and Development section. FDA-sponsored scientific engagement ranges from cross-agency and external collaborations – including FDA’s Centers of Excellence in Regulatory Science and Innovation - to lectures by global scientific thought leaders.

For scientists and other professionals who want to make a real difference in the world, I can think of no better place to work than FDA. The opportunities to build a rewarding career and work on exciting and important issues while protecting and promoting our nation’s health are exceptional.

Jesse L. Goodman, M.D., M.P.H., is FDA’s chief scientist.

FDA Confers with International Counterparts to Advance Regulatory Science

By: Margaret A. Hamburg, M.D.

I have recently returned from the 8th International Summit of Heads of Medicines Regulatory Agencies, which was hosted on December 3-6, 2013, by the Netherlands’ Medicines Evaluation Board in Amsterdam. These annual meetings are an important forum for the exchange of information, views and regulatory strategies among the chief executives of major and like-minded medicines regulatory agencies. I was particularly pleased to be able to contribute to these discussions as a speaker on a panel on regulatory science together with Dr. Tatsuya Konda, M.D., Ph.D., the chief executive of Japan’s Pharmaceuticals and Medical Devices Agency.

Margaret Hamburg, M.D.The theme of this year’s summit was “Changes in the Regulatory Landscape,” and my foreign colleagues and I had plenty to talk about. Overcoming the challenges and reaping the benefits of regulatory science is even more critical today, when the FDA and other regulatory agencies face new and growing tasks in the global marketplace. All of us have to contend with the huge changes in the size and nature of international trade caused by emerging markets, developing economies, and increased cross-border flows of goods, information and capital.

As regulators, my international counterparts and I have many issues in common. They include the increasing complexity of new drug products and drug development; growing geographic distribution of markets; greater demands for public accountability and transparency in our work; budgetary and political challenges to regulatory oversight; and, the overriding need to keep up with the rapid changes in science and technology. Given these shared concerns, building cross-border partnerships and finding common solutions is paramount.

I reiterated to the conference our goal to encourage and strengthen cooperation and collaboration among those nations that are actively working to advance regulatory science. Regulatory science endeavors to use current and emerging knowledge to create new tools, standards and approaches for reliable assessment of the safety, effectiveness, quality and performance of medical products. At its best, this process is based on findings, evaluations, discussions and collaboration by scientists throughout the world.  And it is meetings like the recent summit in Amsterdam that help enhance this cooperation and the development of strategies that promote and strengthen the understanding, acceptance and application of regulatory science.

As the FDA embraces its international role in today’s complex regulatory environment, we fully accept the need to think and act globally more than ever before. I look forward to working with other nations’ regulators, the academic community, non-governmental organizations and industry as we join forces to advance regulatory science, the road to even better protection and advancement of the public health.

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

Personalized Medicine: The Future is Now

By Margaret A. Hamburg, M.D.

Margaret Hamburg, M.D.The difference between science and science fiction is a line that seems ever harder to distinguish, thanks in part to a host of astonishing advances in medical science that are helping to create a new age of promise and possibility for patients.

Today cancer drugs are increasingly twinned with a diagnostic device that can determine whether a patient will respond to the drug based on their tumor’s genetic characteristics; medical imaging can be used to identify the best implantable device to treat a specific patient with clogged coronary arteries; and progress in regenerative medicine and stem cell therapy using a patient’s own cells could lead to the replacement or regeneration of their missing or damaged tissues. Given these trends, the future of medicine is rapidly approaching the promising level of care and cure once imagined by Hollywood in futuristic dramas like Star Trek.

But these examples are not science fiction. They are very real achievements that demonstrate the era of “personalized medicine” where advances in the science of drug development, the study of genes and their functions, the availability of increasingly powerful computers and other technologies, combined with our greater understanding of the complexity of disease, makes it possible to tailor treatments to the needs of an individual patient. We now know that patients with similar symptoms may have different diseases with different causes. Individual patients who may appear to have the same disease may respond differently (or not at all) to treatments of that disease.

FDA has been playing a critical role in the growth of this new era for a number of years. Even before I became FDA Commissioner the agency was creating the organizational infrastructure and putting in place the regulatory processes and policies needed to meet the challenges of regulating these complex products and coordinating their review and oversight. It has been my pleasure to serve at FDA during this next exciting period and to help ensure that the agency continues to prioritize this evolution by anticipating, responding to, and encouraging scientific advancements.

I am very pleased to be able to present a new report by FDA as part of our ongoing efforts in this field. Paving the Way for Personalized Medicine: FDA’s Role in a New Era of Medical Product Development describes many of the exciting developments and looming advances in personalized medicine, lays out the historical progress in this field, and examines FDA’s regulatory role: from ensuring the availability of safe and effective diagnostic devices, to addressing the challenges of aligning a drug with a diagnostic device, to post-market surveillance.

Outside collaboration and information sharing is essential for this field to flourish. On Tuesday, the American Association for Cancer Research and AdvaMedDX held a fruitful daylong conversation on personalized medicine to treat cancer. I was one of the speakers, participating in a conversation with Dr. Francis Collins, the head of the National Institutes of Health. Our discussion focused in part on current status of drug and diagnostic co-development and the challenges and potential of whole genome sequencing, where data can be collected on a patient’s entire genetic makeup at a reasonable cost in a reasonable amount of time.

FDA is committed to fostering these cooperative efforts, as it will require the full force of government, private industry, academia and other concerned stakeholders to maximize our efforts and fully realize the promise of personalized medicine. Our new report outlines that commitment, and helps chart the way forward so that more people can live long and prosper.

Margaret A. Hamburg is the Commissioner of the Food and Drug Administration

FDA Commemorates 30th Anniversary of the Orphan Drug Act

By: Gayatri R. Rao, M.D., J.D.

Gayatri R. Rao, M.D., J.D., is Director of FDA's Office of Orphan Products Development

When President Reagan signed the Orphan Drug Act 30 years ago, he enacted a critically important piece of health care legislation. The passage of this Act on January 4, 1983, was monumental because it created—for the first time—incentives to develop desperately needed medical products for Americans suffering with rare diseases. Until that point, development of such products was very limited. For instance, in the decade leading up to the passage of the Orphan Drug Act, only 10 industry-supported products for rare diseases were brought to market.

The Office of Orphan Products Development (OOPD) was formed at FDA more than 30 years ago, prior to the passage of the Orphan Drug Act, because FDA recognized that rare diseases, when taken together, posed a significant national public health issue. Once the Orphan Drug Act was passed, OOPD became responsible for administering the incentive programs created to spur the development of medical products for rare diseases, namely the Orphan Drug Designation Program and the Orphan Products Grants Program. These products include drugs, biologics, medical devices, and medical foods for the treatment of rare diseases.

As FDA commemorates the passage of this important legislation, we look back over the last 30 years with pride. Since its passage, over 2700 products in development have been designated as orphan drugs through the Orphan Drug Designation Program and over $290 million has been awarded to clinical studies through the Orphan Products Grants Program.  These programs, along with the critical, collective efforts of the Center for Drug Evaluation and Research’s (CDER) Rare Diseases Program, and those of many individuals across FDA, have helped to bring over 400 orphan products for rare diseases to the market.

We also commemorate the more than 30 years of dedicated service from every member of the rare disease community:

  • the patient advocates, who spurred national awareness about the challenges that people with rare diseases face and who continue to support families, educate the community, and drive research into their diseases;
  • the legislators who heard the voices of rare disease advocates and worked to champion the passage of the Orphan Drug Act;
  • the research community, which continues to leverage resources and foster collaborations among academia and industry stakeholders;
  • the clinicians, who support the medical needs of families with rare diseases and work to advocate on behalf of the community;
  • and industry, including pharmaceutical and biotech companies, angel investors, and venture capitalists who, in the spirit of the Orphan Drug Act, have come together to develop products for rare diseases.

Our many successes give us a reason to celebrate 30 years of hard work to provide diagnostic or treatment options to those with rare diseases. But we are keenly aware that there is still a challenging road ahead. We at FDA remain firmly committed to working with the rare disease community to tackle those challenges and to find new diagnostic tools and treatments for the millions of patients with rare diseases.

Gayatri R. Rao, M.D., J.D., is Director for The Office of Orphan Products Development