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CDER Report to the Nation: 2005
Improving Public Health Through Human Drugs

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Director's Message



Highlights and Initiatives

1 Drug Review

2 Drug Safety and Quality

3 International Activities

4 Communications

Director's Message

I am pleased to provide our 2005 Report to the Nation from the Center for Drug Evaluation and Research.

In this report, we outline our initiatives and document our performance across our program areas during 2005. I am very proud to report that we have made substantial advances in identifying, managing and communicating safety-related issues. We have also made major advances in our efforts to improve the science of drug manufacturing.

As an organization, our Center is becoming more transparent, flexible, results-oriented and high-performing. These improvements are helping us maintain our place as the world leader in the review of the safety and efficacy of pharmaceutical products and in responding to the challenges of emerging public health threats. Through our participation in the FDA’s Critical Path Initiative, we are playing a key role in helping to enhance the efficiency of drug development. Although, we play an important role in many areas, I would like to focus on just a few key efforts to give you an idea of what we are doing to ensure the success of our mission.

Addressing public concerns about drug safety

All medicines have benefits and risks. We have continued to focus on the importance of minimizing these risks and improving the way medicines are used. For example, during 2005, we issued 16 public health advisories about important drug safety issues. To sustain a multi-disciplinary, cross-Center approach to drug safety, we elevated the Office of Surveillance and Epidemiology (formerly the Office of Drug Safety) to report directly to the Center Director, and we appointed an Associate Center Director for Safety, Policy and Communication to focus on broad drug-safety policy and communication, including overseeing MedWatch and the Drug Safety Oversight Board staff. We anticipate that this reorganization will help us successfully promote the efficient assessment of and response to emerging safety issues.

In addition, the creation last year of the Drug Safety Oversight Board continues to foster a culture of openness and enhanced oversight around safety issues within the Center. In 2005, the board, under chair Douglas Throckmorton, M.D., the Center’s Deputy Director, met five times and discussed critical safety issues including: long-acting beta agonists associated with more severe asthma episodes; Clostridium sordellii infections in patients taking mifepristone; antidepressants and suicidality; and the concerns with misuse of fentanyl transdermal patches. The board continues to work to help us improve how we identify and manage emerging drug safety issues and how we provide emerging information to health-care professionals and patients about the risks and benefits of medicines.

Improving our analytic and predictive tools to enhance drug development

One of the organizational changes announced in 2005 is a response to FDA’s efforts to improve the science of medical product development, the Critical Path Initiative. To support this priority activity and to speed the movement of innovative therapies to patients, we created an Office of Translational Sciences, which includes the Office of Clinical Pharmacology and the Office of Biostatistics. The Office of Translation Sciences will support cross-cutting science programs such as the Regulatory Science and Review Enhancement Committee, the Research Involving Human Subjects Committee and the Critical Path Initiative. This office also has the important task of coordinating our collaborations with outside groups to help identify and resolve issues that are standing in the way of efficient drug development. We anticipate that, by putting these functions together into one office, we will be better able to promote these many scientific activities.

Improving our scientific tools to ensure product quality

We are continuing our work on the transformation of product manufacturing and how it is regulated, by extending the work done in the Agency-wide Pharmaceutical cGMPs for the 21st Century initiative. Activities have included:

  • Incorporating the fundamentals of quality systems into CDER operations.
  • Restructuring the chemistry, manufacturing and controls review staff in the Office of New Drug Quality Assessment to provide for better efficiency.
  • Rolling out concepts and philosophy of the changes to industry.
  • Training chemistry, manufacturing and controls reviewers
  • Introducing a pilot program for new drugs through which industry can submit applications using a quality-by-design framework to help expand the Agency’s knowledge in this area.

All of these activities have helped strengthen the overall scientific focus for chemistry, manufacturing and controls review as well as reduce some of the regulatory burden for both the Agency and the industry.

Improving efficiency and consistency of cancer product reviews

Our efforts to improve the timely development of new drugs for the treatment of cancer have continued. To enable this, we made significant changes to our organizational structure to provide a stronger and more consistent approach to the review process for drugs and therapeutic biologics used to diagnose, treat and prevent cancer. We created a new oncology office, called the Office of Oncology Drug Products. This new office is a consolidation of three pre-existing areas within the Center: Drug Oncology Products, Biologic Oncology Products and Medical Imaging and Hematology Products. Creation of this new office will improve consistency of review and policy for oncology products and bring together a critical mass of oncologists who can help during the development of new cancer  therapies. This office will also provide technical consultation to other FDA components.

Speeding the development of medical countermeasures

We recognize the need to facilitate the development of countermeasures to protect Americans from biological, chemical, nuclear and radiological agents of terrorism. Many parts of the Center contribute to this effort, focused through the Office of Counter-Terrorism and Emergency Coordination, an office we formed in 2006. One important goal we have identified is to foster the development of medical countermeasures for vulnerable populations. For example, in 2005, following an expedited review, we approved ThyroShield, a ready-to-use, liquid medicine to block the uptake of radioactive iodine in vulnerable populations. Unlike already approved tablets, which are not ideally suited for young children, this black raspberry flavored solution is dosed using an eyedropper to enable easy treatment of young children and newborns.

We are extremely proud of the work outlined in this report and look forward to another year of important strides in approving therapies to improve the health of Americans.

Steven Galson, M.D., MPH
Center for Drug Evaluation and Research

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Who we are

The Center for Drug Evaluation and Research is America’s consumer watchdog for medicine. We are part of one of the nation’s oldest consumer protection agencies—the U.S. Food and Drug Administration. The FDA is an agency of the federal government’s Department of Health and Human Services. We are the largest of FDA’s five centers, with about 2,200 employees. Approximately half of us are physicians or other kinds of scientists.

What we do

Our best-known job is to evaluate new drugs for safety and effectiveness before they can be sold. Our evaluation, called a review, makes sure that the drugs we approve meet our tough standards for safety, effectiveness and quality. We also make sure that you and your doctor will have the information you need to use medicines wisely. Once drugs are on the market, we monitor them for problems.

Reviewing drugs before marketing. A drug company seeking to sell a drug in the United States must first test it. We monitor clinical research to ensure that people who volunteer for studies are protected and that the quality and integrity of scientific data are maintained. The company then sends us the evidence from these tests to prove the drug is safe and effective for its intended use. We assemble a team of physicians, statisticians, chemists, pharmacologists and other scientists to review the company’s data and proposed use for the drug. If the drug is effective and we are convinced its health benefits outweigh its risks, we approve it for sale. We don’t actually test the drug when we review the company’s data. By setting clear standards for the evidence we need to approve a drug, we help medical researchers bring safe and effective new drugs to American consumers more rapidly. We also review drugs that you can buy over the counter without a prescription and generic versions of over-the-counter and prescription drugs.

Watching for drug problems. Once a drug is approved for sale in the United States, our consumer protection mission continues. We monitor the use of marketed drugs for unexpected health risks. If new, unanticipated risks are detected after approval, we take steps to inform the public and change how a drug is used or even remove it from the market. We monitor changes in manufacturing to make sure they won’t adversely affect safety or efficacy. We evaluate reports about suspected problems from manufacturers, health-care professionals and consumers. We try to make sure an adequate supply of needed drugs is always available to patients who depend on them.

Monitoring drug information and advertising. Accurate and complete information is vital to the safe use of drugs. We regulate information that accompanies or is displayed with an over-the-counter drug. In the past, drug companies promoted their products almost entirely to physicians. More frequently now, they are advertising directly to consumers. We oversee advertising of prescription drugs, whether to physicians or consumers. We pay particular attention to broadcast ads that can be seen by a great many consumers. The Federal Trade Commission regulates advertising of over-the-counter drugs. Advertisements for a drug must contain a truthful summary of information about its effectiveness, side effects and circumstances when its use should be avoided.

Protecting drug quality. In addition to setting standards for safety and effectiveness testing, we also set standards for drug quality and manufacturing processes. We work closely with manufacturers to see where streamlining can cut red tape without compromising drug quality. To ensure a safe and effective drug supply, we enforce federal requirements for drug approval, manufacturing and labeling. When necessary, we take legal action to stop distribution of products in violation of these requirements. As the pharmaceutical industry has become increasingly global, we are involved in international negotiations with other nations to harmonize standards for drug quality and the data needed to approve a new drug. This harmonization will go a long way toward reducing the number of redundant tests manufacturers do and help ensure drug quality for consumers at home and abroad.

Why we do it

Our present and future mission remains constant: to ensure that drug products available to the public are safe and effective. Our yardstick for success will always be protecting and promoting the health of Americans.

Getting consumer input. Protecting consumers means listening to them. We consult with the American public when making difficult decisions about the drugs that they use. We hold public meetings about once a week to get expert, patient and consumer input into our decisions. We also announce most of our policy and technical proposals in advance. This gives members of the public, academic experts, industry, trade associations, consumer groups and professional societies the opportunity to comment before we make a final decision. In addition, we take part in FDA-sponsored public meetings with consumer and patient groups, professional societies and pharmaceutical trade associations. These help us obtain enhanced public input into our planning and priority-setting practices.

What is a drug?

We regulate drugs used to treat, prevent or diagnose illnesses. However, drugs include more than just medicines. For example, fluoride toothpaste, antiperspirants, dandruff shampoos and sunscreens are all considered "drugs." You can buy some drugs in a store without a prescription, while others require a doctor's prescription. Some are available in less-expensive generic versions.

Prescription drugs

Prescription medicines must be administered under a doctor’s supervision or require a doctor’s authorization for purchase. There are several reasons for requiring a medicine be sold by prescription:

  • The disease or condition may be serious and require a doctor’s management.
  • The medicine itself may cause side effects that a doctor needs to monitor.
  • The same symptoms may be caused by different diseases that only a doctor can diagnose.
  • The different causes may require different medicines.
  • Some medicines can be dangerous when used to treat the wrong disease.

Over-the-counter drugs

You can buy OTC drugs without a doctor’s prescription. You can successfully diagnose many common ailments and treat them yourself with readily available OTC products. These range from acne products to cold medications. As with prescription drugs, we closely regulate OTC drugs to ensure that they are safe, effective and properly labeled.

Generic drugs

A generic drug is a chemical copy of a brand-name drug. There are generic versions of both prescription and over-the-counter drugs. Generic drugs approved by the FDA have the same therapeutic effects as their brand-name counterparts, often at a much lower cost.

Scientific research

We conduct and collaborate on focused laboratory research and testing. This maintains and strengthens the scientific base of our regulatory policy-making and decision-making. We focus on:

  • Drug quality, safety and performance.
  • Improved technologies.
  • New approaches to drug development and review.
  • Regulatory standards and consistency.

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The Center for Drug Evaluation and Research promotes and protects public health by assuring that safe and effective drugs are available to Americans. The Food and Drug Administration Modernization Act of 1997 affirmed the center's public health protection role, clarified the FDA's mission and called for the FDA to:

  1. Promote the public health by promptly and efficiently reviewing clinical research and taking appropriate action on the marketing of human drugs in a timely manner.
  2. Protect the public health by ensuring that human drugs are safe and effective.
  3. Participate through appropriate processes with representatives of other countries to reduce the burden of regulation, harmonize regulatory requirements and achieve appropriate reciprocal arrangements.
  4. Carry out its mission in consultation with experts in science, medicine and public health and in cooperation with consumers, users, manufacturers, importers, packers, distributors and retailers of human drugs.

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Highlights and Initiatives

We are pleased to present our 10th performance report. Our work in 2005 offered many Americans new or improved choices for protecting and maintaining their health or new ways to use existing products more safely. We worked hard at our mission of ensuring that Americans have safe and effective drugs and also developed these initiatives to bring the latest science and technology to bear on our mission:
  • Reforming our drug safety oversight.
  • Identifying ways to improve the science of drug development.
  • Improving manufacturing practices.
  • Protecting the homeland with improved medical countermeasures to be used in the event of a terrorist attack or disaster.
  • Conducting targeted scientific research to improve our regulatory practices.
  • We accomplished our work on these initiatives while maintaining our performance on our reviews of safety and efficacy and our oversight and surveillance of the safety of products sold to Americans.
  • Reviews. We approved 80 new medicines, including 20 truly new medicines that had not been marketed in any form before in this country. We approved 141 new or expanded uses for already approved medicines. We approved 344 generic versions of existing drugs.
  • User-fee performance. We met almost all of our goals for review performance in fiscal year 2005.
  • Drug safety surveillance. We processed and evaluated more than 460,000 reports of adverse drug events, including more than 25,000 submitted directly from individual health-care providers and patients.
  • Drug promotion and advertising. We issued 60 letters on violations in drug promotion and more than 800 letters to help ensure manufacturers comply with regulations concerning drug promotion.
  • Public database of electronic labels. We required manufacturers to begin submitting drug labeling information electronically so that it can be made publicly available rapidly.
  • Public health advisories. We issued alerts on non-steroidal anti-inflammatory pain medicines and 15 other safety concerns.
  • Manufacturing compliance. We acted to halt distribution of drugs that were unapproved, poorly manufactured or improperly labeled.

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Drug Safety Initiative

Our highest priority is assuring that safe and effective medicines are available to the American public. Safe medicines, where the benefits outweigh the risks, were the focus of a number of high-profile initiatives in 2005. We worked to ensure that our systems for assessing and evaluating safety continue to be robust and that we communicate new information for health-care providers and patients in a timely and clear manner. Our efforts included:

  • Establishing the Drug Safety Oversight Board.
  • Sharing drug safety information sooner and more broadly.
  • Laying the foundations of an electronic information infrastructure that will give patients, health-care professionals and consumers quick and easy access to the most up-to-date and accurate information on medicines.
  • Obtaining public input on our drug risk communications strategies.

Comprehensive oversight of drug safety

Our professional staff spends about one-half their time addressing safety issues, including:

  • Watching for problems once we approve a drug.
  • Overseeing clinical trials.
  • Evaluating new therapies and new or expanded uses for existing therapies to balance risks against expected benefits.
  • Overseeing manufacturing, distribution and promotional activities.
  • Preventing medication errors by evaluating proposed proprietary names, labeling and packaging.
  • Developing proactive risk management strategies both before and after approval.

Drug Safety Oversight Board

The 15-member board provides drug safety oversight and recommendations to the Center Director on drug safety issues. The board held five meetings in 2005. In its initial meetings, members explored the methods we use in risk assessment of marketed drugs, including:

  • Review and analysis of spontaneous reports of adverse events.
  • Drug use data.
  • Health-care administrative data.
  • Epidemiologic and observational studies.
  • Clinical trials.
  • Active surveillance systems.

In later meetings, members discussed pre- and post-decisional risk management examples to further clarify and define their oversight and advisory responsibilities including their role in helping establish policies and managing the communication of important drug safety issues to stakeholders. Members include representatives from each of our offices with responsibility for drug safety, a representative from each of the two other FDA centers dealing with human medical products, a representative the Veterans Administration and a representative the National Institutes of Health.

More information about the board is at http://www.fda.gov/cder/drugSafety.htm.

Public summaries of board meetings are at http://www.fda.gov/cder/drug/DrugSafety/DSOBmeetings/default.htm.

Drug safety communication channels

We began sharing “emerging” drug safety information broadly through public health advisories and through specific drug safety information sheets that are tailored to the needs of health-care professionals and patients. In some cases, we are sharing safety information even before we have reached conclusions that would prompt a regulatory action. Our communications in 2005 included:

  • Public Health Advisories. We issued 16 advisories to alert health-care providers and consumers to safety concerns about drugs. Public Health Advisories.
  • Health-care professional information sheets. We published sheets on 44 drugs with detailed information about emerging important drug safety concerns, including a description of the concern along with recommendations and considerations about the concern for the prescriber.
  • Patient information sheets. We published 41 information sheets containing new information about emerging drug safety concerns for approved drugs and provided in a consumer-friendly format.

More about drug safety communication is at http://www.fda.gov/cder/drugSafety.htm.

Prescription drug information infrastructure

Accurate, up-to-date information about a prescription drug is critical for its safe and effective use. Too frequently, information about a prescription drug reads more like a legal disclaimer than useful or actionable health information. We worked hard in 2005 to bring to fruition several interconnected regulatory actions, including:

  • Electronic submission of drug labeling. We issued final guidance to assist manufacturers in submitting prescription drug label information to us in a new electronic format.
  • Launching the DailyMed public database of drug information. This multi-agency effort to improve patient safety is enabling us—through the National Library of Medicine—to provide an up-to-date electronic repository of medication labeling in a standard format. This information will be useable in computer systems that support patient safety, such as electronic prescribing and decision-support systems.
  • Working to bring out our final requirement for revised prescription drug labeling. Our final regulation, issued in January 2006, amends the content and format of prescription drug information, commonly called the package insert. The new label will provide the most important information about new and recently approved prescription drugs and new uses in a format that is better understood, more easily accessible and more memorable for physicians.

Public hearing on communication of drug safety information

Making us a reliable “trusted source” of health-care provider and consumer information about medicines emerged as the key theme at a two-day public hearing in 2005 on our risk communications. We were urged to engage health-care professional organizations, simplify our risk communications, improve provider and consumer access to our Internet site, develop consistent communication approaches and address those with limited health literacy and English skills.

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Critical Path Initiative

Our role in the Agency’s Critical Path Initiative is to stimulate and facilitate a national effort to modernize the scientific processes through which a potential human drug or therapeutic biologic is transformed from a discovery or “proof of concept” into a medical product.

In our view, the applied sciences for product development have failed to keep pace with the tremendous advances in the basic sciences. New science is not being used to guide the development process in the same way that it is accelerating the discovery process.

To focus the attention of the public, academic researchers, funding agencies and industry, an FDA report in 2004 described an urgent need to modernize the medical product development process—the Critical Path—to make product development more predictable and efficient. Because of our unique vantage point, we can work with companies, patient groups, academic researchers and other stakeholders to coordinate, develop and help disseminate solutions to scientific hurdles that are impairing the efficiency of medical product development.

Critical Path Opportunities List

During 2005, we helped the Agency describe and provide examples of how new scientific discoveries—in fields such as genomics, proteomics, imaging and bioinformatics—could be applied to improve the accuracy of the tests we use to predict the safety and efficacy of investigational medical products. The list was released in early 2006.

The list provides a concrete focus for public and private efforts and investments in new tools that could revolutionize product development. The goal is to encourage others to undertake such work in their areas of interest. It was developed based on feedback from stakeholders and the special insights of FDA’s product reviewers.

The list’s 76 scientific projects mark a starting point in identifying the essential development priorities. These are highly-targeted research projects divided into six key areas:

  • Better evaluation tools—biomarkers and disease models.
  • Streamlining clinical trials.
  • Harnessing bioinformatics.
  • Moving manufacturing into the 21st century.
  • Products to address urgent public health needs.
  • At-risk populations.

The comprehensive Web site on the Critical Path is at http://www.fda.gov/oc/initiatives/criticalpath/.

Personalized medicine

The Critical Path recognizes “pharmacogenomics” and encourages its use in drug development.

Pharmacogenomics allows health-care providers to identify differences in people’s drug risk response profiles and predict the best possible treatment options for them.

In 2005, we helped lay the regulatory groundwork for pharmacogenomics in the Critical Path by issuing:

  • A final guidance on voluntary submissions of pharmacogenomic data
  • A draft concept paper on how to co-develop a drug or biological therapy along with a device test in a scientifically robust and efficient way.

We received 15 voluntary genomic data submissions from industry in 2005.

More information is available at http://www.fda.gov/cder/genomics.

Pharmacogenomics workshop

In 2005, we held the third in a series of scientific workshops on how pharmacogenomics could enable efficient and successful drug development in such areas as:

  • The efficiency and informativeness in clinical trials.
  • Clinical trials for cancer therapy.
  •  International harmonization of pharmacogenomics guidances.
  •  Strategies to bridge pharmacogenomics information from drug development research to clinical practice.
  • Clinical qualification of genomic biomarkers for decision making.
  • Creation of diagnostic tests for clinical practice.

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21st Century Drug Quality System

Our overhaul of the regulatory and quality control systems for pharmaceutical products encourages manufacturers to modernize their methods, equipment and facilities. Our goal is to help eliminate both production inefficiencies and undue risks for consumers. Our initiative implements improved policies that are making better use of our limited resources through more targeted and effective inspections.

Collectively, our policies are known as “current good manufacturing practices” or cGMPs, and our last comprehensive revisions to them took place nearly a quarter of a century ago. Pharmaceutical cGMPs for the 21st Century is the umbrella name for this strategic initiative, and more information is available at http://www.fda.gov/cder/gmp/.

New drug quality assessment

Our chemists reorganized during 2005 to transform drug quality assessment from a checklist review to a scientifically sound, risk-based process. The mission of our new Office of New Drug Quality Assessment is to:

  • Assess the critical quality attributes and manufacturing processes of new drugs.
  • Establish quality standards to assure safety and efficacy
  •  Facilitate new drug development.

Our chemists are now focusing on critical pharmaceutical quality attributes and their relevance to safety and efficacy. These include chemistry, pharmaceutical formulation, stability, manufacturing processes, bioavailability and product performance. Our long-term goal is to:

  • Emphasize quality by design in the evaluation of critical aspects of pharmaceutical quality.
  • Have a strong focus on manufacturing science.
  • Integrate review and inspection functions.
  • Use modern statistical methodologies.

Our vision for “desired state” in manufacturing

  • Product quality and performance assured by effective and efficient manufacturing processes.
  • Product attributes based on mechanistic understanding of how formulation and process impact performance.
  • Continuous improvement and continuous real-time assurance of quality enabled.
  • Regulatory policies recognize level of product and process knowledge.

Workshop explores drug quality system

We held a three-day scientific workshop for industry to explore how we can achieve the new drug quality system. We worked with industry scientists to:

  • Identify scientific training gaps that must be filled for the successful implementation of the new system.
  • Obtain industry input on building a scientific, risk-based regulatory system that maintains high quality and facilitates continuous improvement.
  • Help determine how to best use information from the pharmaceutical development phase in the industrialization phase.
  • Identify the roles and responsibilities for industry and us in the new system.
  • Propose ways to reduce the number of post-marketing supplements.

Chemistry, manufacturing, controls pilot program

We launched a formal pilot program in July 2005 under which pharmaceutical companies can voluntarily submit new drug applications that apply quality-by-design principles and demonstrate their product knowledge and process understanding. This scientific information—more relevant than found in a traditional submission—-will enable us to:

  • Perform a risk-based assessment of product quality and process performance.
  • Consider an applicant’s proposal for regulatory flexibility in setting product specifications and post-approval changes.

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Counterterrorism, Emergency Response

We have been taking an aggressive and proactive approach to our role in helping to prepare the nation for terrorist events, emerging health threats and emergency response to natural and man-made crises, including:

  • Assuring the availability of medicines during a crisis.
  • Addressing issues on procurement, packaging, labeling, use and shelf-life extension of products in the Strategic National Stockpile.
  • Utilizing regulatory mechanisms to provide emergency access to new therapies and to approved therapies used in novel ways.
  • Assuring alternative manufacturing sites for critical medicines.
  • Protecting the nation’s drug supply from attack or deliberate contamination.
  • Leveraging with other federal agencies to answer scientific questions about treatments for emerging health threats and terrorist events.
  • Preparing ourselves to continue operations during a crisis.

Interagency collaborations

  •  Post-event surveillance planning. Along with the FDA’s other medical centers and the Centers for Disease Control and Prevention, we developed a plan to identify processes for collecting adverse event and outcome data on medical products distributed in response to an emergency.
  • Project BioShield prioritization. We participated in many interagency counterterrorism working groups that have contributed to gap analyses in medical countermeasures and authored many of the requirements documents that will be used to prioritize products for development and eventual procurement under BioShield.

Counterterrorism guidances published in 2005

  • Draft Guidance for Industry: Internal Radioactive Contamination-Development of Decorporation Agents.
  • We participated in developing FDA’s Draft Guidance: Emergency Use Authorization of Medical Products.

Medical countermeasure approvals

  • Potassium iodide oral solution (ThyroShield). This oral solution of a thyroid blocking agent for use in radiation emergencies is appropriate for children or adults who cannot swallow tablets. We advised the Department of Health and Human Services in a BioShield procurement of the product, which was approved as a generic drug.
  • Generic ciprofloxacin. We approved four additional new generic ciprofloxacin drug products, all with approved labeling for the management of inhalational anthrax (post-exposure).
  • Revised labeling for ciprofloxacin (Cipro). We approved revisions for the sections of the package insert concerning Indications and Usage, Adverse Reactions and Inhalational Anthrax—Additional Information. These changes for the tablets, intravenous solution and oral suspension were based on information obtained by the Centers for Disease Control and Prevention during the 2001 anthrax attacks. Because of these data, we released the manufacturer from its accelerated approval commitment to report data from confirmatory anthrax studies.

Facilitating medical countermeasure development

  • Plague. The Centers for Disease Control and Prevention continued to enroll patients in the second year of an FDA-funded clinical trial to assess the efficacy of the antibiotic gentamicin for endemic plague in Madagascar, where antimicrobial options for plague are extremely limited. We contributed to protocol design and the formation of a data safety monitoring board to oversee the safe conduct of the study. We are continuing our collaboration with FDA’s Center for Devices and Radiological Health to evaluate the performance of a novel, rapid, bedside plague diagnostic test kit under study conditions.
  • Pneumonic plague. We also continued our collaboration with the National Institute of Allergy and Infectious Diseases and the U.S. Army Medical Research Institute of Infectious Diseases to evaluate the safety and efficacy of five antibiotics—gentamicin, ciprofloxacin, levofloxacin, doxycycline and ceftriaxone—in a non-human primate model of pneumonic plague.
  • Inhalational Anthrax. We continued our collaboration with both institutes to establish a non-human primate natural history model for inhalational anthrax.
  • Radiological and nuclear threats. We continued to collaborate with the National Institute of Allergy and Infectious Diseases to identify promising new products for use against radiological and nuclear threats. We discussed scientific and regulatory issues with manufacturers of such products and informed them about possible funding sources, both for early development and for procurement by the federal government.

Pandemic flu preparedness

We are preparing for an influenza pandemic by:

  • Participating in the FDA Pandemic Influenza Preparedness Task Force, as well as in the Antiviral Subgroup and the Emergency Preparedness, Response and Communications Subgroup.
  • Helping to develop FDA’s Pandemic Influenza Preparedness Strategic Plan and our center’s portion of FDA’s Pandemic Influenza Continuity of Operations Plan.
  • Providing input on the HHS Pandemic Influenza Plan from the Department of Health and Human Services.

Hurricane response

We assisted in the response to hurricanes Katrina, Rita and Wilma by coordinating the Internet posting of information about:

  • The safety of medications potentially damaged by flooding or high temperature.
  •  Insulin use.
  • Safety of using combination sunscreen and insect repellent in children.
  • Reporting prescription drug sample losses.
  • Assisting investigators conducting clinical trials in hurricane-affected areas.
  • Facilitating donation of drugs.
  • Coordinating volunteer responses for relief efforts.

About 150 Public Health Service Commissioned Corps officers—one-half the total assigned to our center—were deployed to provide medical and pharmacy services in areas affected by the hurricane.

Emergency preparedness

We provided rapid responses to requests for information on medical counter­measures during a five-day interdepartmental exercise, known as TOPOFF 3 (“Top Officials”). The exercise simulated simultaneous terrorist attacks using multiple threat agents—both pneumonic plague in New Jersey as well as the release of mustard gas and a high-yield explosive in Connecticut.

Emergency use authorization

We worked with the Centers for Disease Control and Prevention to identify potential medical countermeasures in the Strategic National Stockpile as candidates for Emergency Use Authorization for an unapproved use under the Project BioShield Act of 2004.

We also began to outline the internal processes and procedures we need to handle emergency use requests.

Flu prevention for children

In December, we approved oseltamivir (Tamiflu) for prevention (prophylaxis) of influenza in children 1 to 12 years of age. This gives health-care providers an option for preventing influenza in children following close contact with an infected individual.

Our approval followed presentation of an adverse event safety update on the drug to the Pediatric Advisory Committee. The meeting provided a public forum to discuss the safety of the drug’s use in children as part of a routine drug safety update required by the Best Pharmaceuticals for Children Act.

Internet resources

We provide the most current information on medical counter­measures and vaccines, plus advice on purchasing and taking medication, at http://www.fda.gov/cder/drugprepare/default.htm.

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Scientific Research

We advance the scientific basis of regulatory practice by developing, evaluating or applying the best, most appropriate and contemporary scientific methods to regulatory testing paradigms. We provide scientific support for reviewer training, regulatory decision making and the development of regulatory policy.

We focus on creating a tighter scientific linkage between non-clinical and clinical studies, enhancing methodology for assuring product quality, building databases for improved drug development and review and providing regulatory support through laboratory testing.

Linking nonclinical and clinical studies

  • Biomarkers for organ damage. We are identifying, evaluating and establishing relevant protein biomarkers in blood in both animal models and in humans. These will help detect the very earliest damage that can be caused by certain drugs to the heart, kidney, immune system and liver.
  • Biomarkers for inflammation. To enhance safety within broad segments of patient populations and enable safe development of new drug classes, we are working on the identification and elucidation of associated serum biomarkers and mechanisms responsible for the development of vascular inflammation in specific organ systems.
  •  Evaluation of microarrays. We conduct targeted research on microarrays, a new technology that can identify thousands of genes or proteins rapidly and at the same time. We are evaluating how this technology could improve the interface between drug development and regulatory practice.
  • Medicinal plants, herbs. We established scientific research capabilities in the analyses of medicinal plant and herbal products.
  • Imaging drug targets. We continue to explore noninvasive imaging technology to extend our long-standing interest in the application of accurate dose-concentration-response principles by viewing drugs and their actions directly at the level of the drug target, rather than indirectly via plasma concentrations.
  • Better use of exposure-response data. We are developing a standardized approach for using exposure-response information to help evaluate the risks and benefits of drug therapies and recommending dose adjustments in special populations.
  • Pediatric pharmacokinetics. We are developing a pediatric population pharmacokinetics study design template to facilitate implementation of sparse sample strategies in pediatric drug development.

Biotechnology research

Our Office of Biotechnology Products consists of about 80 scientists and other staff who are responsible for evaluating therapeutic biotechnology product submissions as well as carrying out scientific research related to biologics regulatory issues.

  •  Immune responses. We review many submissions aimed at inhibiting unwanted immune responses, such as autoimmune diseases or rejection of transplanted organs, or aimed at enhancing desired immune responses, such as those against infections or cancer. To facilitate review of such immunology-related submissions, we study the mechanisms by which immune cells are activated, suppressed or channeled from one kind of active response to another.
  •  Metabolic pathways. We study the mechanisms by which various regulated products induce their intended effects, as well as unintended adverse effects. Our investigations also examine various normal and pathogenic pathways that are targeted by regulated agents.

Our research enhances the ability of our scientist/regulators to evaluate risks and benefits of biotech products, to advise industry on difficult regulatory problems, such as potency assays, and to develop hands-on expertise in the modern technologies used by sponsors of biotech products.

Informatics and computational safety analysis

  • Cancer toxicity predictive software. Our cooperative research and development agreements with several commercial software developers have resulted in the development and marketing of new computer software to predict the cancer-causing potential of chemicals based on their molecular structure. The software makes use of our extensive rodent carcinogenicity database without compromising proprietary information.
  • Safe starting dose models. We have successfully developed computer models to estimate the safe starting dose for clinical trials of drugs based on their molecular structure. The current method for estimating the starting dose is highly inexact and requires the use of multiple safety factors because it is based exclusively on an extrapolation from animal toxicity studies. We have begun studies to validate the new method.

Scientific research in pregnancy and lactation

Click here for studies to evaluate fetal safety from drug exposure or whether the dose of a drug should be adjusted during pregnancy or lactation.

Counterterrorism biotechnology research

We have used congressionally mandated special funding to initiate research in several areas relevant to counterterrorism. Our scientists are studying:

  • Microarray technologies, which could assist in identifying infectious biowarfare agents.
  • Non-specific immune boosters, which could provide transient protection against such agents.
  • Monoclonal antibodies as neutralizers of biological toxins.
  • Various strategies to defend against anthrax.
  • Development of Anthrax Toxin assays for assessment of potential therapies.

By establishing a core of scientists experienced in several areas of bioterrorism, these projects anticipate high-priority regulatory submissions likely to require rapid science-based evaluation.

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Date created: August 18, 2006

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