FDA Salutes World Sickle Cell Awareness Day

By: Jonca Bull, M.D.

Today is a World Sickle Cell Awareness Day, an annual reminder that Sickle Cell Disease (SCD) is a major area of unmet medical need that causes serious and devastating consequences to many thousands of children and adults. It is an occasion that has been commemorated each year since 2008, when the General Assembly of the United Nations adopted a resolution recognizing SCD as a global public health concern. I am happy to have this opportunity to help raise awareness about the impact of this disease on patients and their families, and to emphasize the need for additional therapies to prevent or treat SCD and its complications.

Jonca BullSCD is a genetic disorder that most commonly affects people of African descent; however, it also affects Hispanics, Asians, and people of Mediterranean and Middle Eastern descent. Millions of people are living with this disease all over the world. Here in the U.S., there are about 100,000 people with SCD and it is estimated that the disease occurs in one of every 500 Black or African American and one out of every 36,000 Hispanic-American births. Additionally, one in 12 African Americans carry sickle cell trait, the gene for the disease. People with SCD have “sickled” or abnormally shaped red blood cells that get stuck in small blood vessels blocking the flow of blood and oxygen to major organs in the body. These blockages can cause severe pain, organ damage or even stroke in some cases. SCD is a chronic and debilitating disease affecting people for their entire lives.

The Food and Drug Administration is committed to continuing the dialogue around Sickle Cell Disease to facilitate the development of safe and effective treatments to prevent the disease or reduce its complications. On February 7, 2014, our agency held a Patient-Focused Drug Development meeting to ask patients with SCD and their families, caretakers, and advocates about the various aspects of their disease and how it affects their lives on a daily basis. We heard from approximately 300 people on their treatment regimens, symptoms and complications from treatments, and what they would like to see in terms of future treatments. FDA learned a great deal from this meeting, and we hope this is the first of many successful collaborations leading to the development and approval of effective therapies for SCD.

Only limited treatment options exist for this disease, and more development is needed. In 1998, the FDA approved hydroxyurea to reduce the frequency of pain crises and the need for blood transfusions in adult patients with Sickle Cell Anemia. While the use of hydroxyurea has proven to be helpful in reducing complications in some patients, it is not universally effective and the mechanism of action is not completely understood. Other treatments, such as chronic transfusion therapy, although effective for some, can present problems for patients, which limits their use. Stem cell transplantation has been noted as a potential cure for SCD, but due to the lack of matched donors and associated risks during and after the procedure, this is also a limited option. As part of the FDA’s effort to facilitate the development of new SCD treatments, our Office of Minority Health has funded research to identify new methods to improve the safety and availability of blood for transfusion, and FDA’s drug experts are working with members of the pharmaceutical industry and outside researchers.

As we take the time today to reflect on the impact of Sickle Cell Disease, our agency encourages the search for new and better SCD therapies through medical innovation by using information gained from patients and their caregivers in the recent Patient-Focused Drug Development Meeting on Sickle Cell Disease. We will continue to join our efforts with those of patients, researchers, industry, and sister agencies such as the Centers for Disease Control and Prevention and the National Institutes of Health, to lessen the burden of Sickle Cell Disease across the globe.

Jonca Bull, M.D., is Director of FDA’s Office of Minority Health

FDA Issues Draft Guidances for Industry on Social Media and Internet Communications About Medical Products: Designed with Patients in Mind

By: Thomas Abrams

Ongoing changes in technology transform medical products – and the ways that both patients and health care providers learn about those products. In today’s world, in addition to traditional sources of medical product information, patients and health care providers regularly get information about FDA-regulated medical products through social media and other Internet sources, and those technologies continue to evolve. But regardless of the Internet source used to communicate about medical products, the public health is best served by clear, accurate, truthful and non-misleading information about them.

Tom AbramsThat’s why the agency has proposed two draft guidances for industry with recommendations to help manufacturers and their representatives accurately communicate online about prescription drugs and medical devices.

These documents strive to ensure that the information provided by drug and device companies is accurate and will help patients to make well-informed decisions in consultation with their health care providers.

Our first guidance provides recommendations for the presentation of risk and benefit information for prescription drugs or medical devices using Internet/social media sources with character space limitations, such as Twitter and the paid search results links on Google and Yahoo. These recommendations address the presentation of both benefit information and risk information in this setting. We understand that communicating on electronic Internet sites with character space limitations can be challenging. But, no matter the Internet source used, benefit claims in product promotions should be balanced with risk information. And companies should provide a way for consumers to gain direct access to a more complete discussion of risks associated with their products.

Our second guidance provides recommendations to companies that choose to correct third-party information related to their own prescription drugs and medical devices. This draft guidance provides FDA’s recommendations on the correction of misinformation from independent third parties on the Internet and through social media sites. For example, we recommend that any corrections should address all misinformation in a clearly defined portion of a forum on the Internet or social media, whether the misinformation is positive or negative.

We developed these new guidances, in part, to respond to requests for best practices from companies and other stakeholders. We gave careful thought to our draft recommendations, and we understand technology will continue to evolve. So we worked across FDA Centers and Offices to develop best practices that can be applied to existing online Internet sites — and those that have yet to be developed.

Prescription drugs and medical devices can provide tremendous benefits to patients, but they can also pose certain risks. As a regulatory agency, we are committed to ensuring that the information about these products that their manufacturers and distributors direct at patients and health care providers is accurate and balanced.

These draft guidances are the latest in a series, and the agency is very interested in receiving comments from stakeholders. Please read more about the new draft guidances on our social media guidances webpage, and share your comments and suggestions. The documents represent FDA’s current thinking on specific aspects of FDA’s evolving consideration of social media sites and other Internet-related matters. FDA continues actively to review, analyze, and develop approaches to a variety of topics related to the labeling and advertising of medical products, including the development of these and other guidances addressing the use of social media platforms and the Internet.

FDA sees social media as an important resource for industry and is committed to developing additional guidance for drug and device manufacturers that outline the agency’s current thinking. We do all of this work with the best interest of patients in mind.

Thomas Abrams is the director of FDA’s Office of Prescription Drug Promotion in the Agency’s Center for Drug Evaluation and Research (CDER)

Global Partnerships Advance the Regulatory Science That Protects Public Health

By: William Slikker, Jr., Ph.D.

In work, as in life, your success often comes down to the strength of your relationships. And as the director of FDA’s National Center for Toxicological Research (NCTR), among the most pre-eminent regulatory science centers in the world, I have found that this axiom, often so apt in daily life, is also true on a grander scale in the world of research.

William SlikkerNCTR scientists develop innovative tools and strategies to advance FDA’s mission to protect and promote public health. Our center sits on 500 acres in Jefferson, Arkansas, far from agency headquarters in the Washington, D. C., metropolitan area.

But the power of the safety assessment work done at NCTR has global reach, and it is leveraged by the global nature of partnerships we have developed across FDA and with research centers in other countries. Late this summer, Aug. 21-22, I will travel to Montreal for the Global Summit on Regulatory Science, where government, industry and academic scientists from all over the world will assess how to address emerging technologies and implement innovative ways to use them to determine the safety and effectiveness of FDA-regulated products when used in real-world applications.

If you imagine our scientific collaborations as a family tree of sorts, our international activities are one limb. In addition to the annual summit, we provide opportunities for scientists from other countries to work with experienced FDA researchers in all facets of safety assessment. NCTR also has outreach partnerships with the World Health Organization, the European Food Safety Authority and other international organizations such as the International Union of Toxicology (IUTOX).

Our internal partnerships are another limb to the science of public health. Of 200 active research projects ongoing at NCTR, over 100 are done in collaboration with scientists from other FDA centers and the Office of Regulatory Affairs (ORA). For example, we work with the Center for Drug Evaluation and Research in assessing the danger, or toxicology, of certain drugs on the most vulnerable populations— pregnant women and children.

We are partners with the ORA in the Nanotechnology Core Facility on our campus that supports the study of nanomaterials, so small that they can’t be seen with a regular light microscope, yet their effects can be profound on the increasing number of drugs, foods and cosmetics in which they are found. NCTR also works with state partners in this research.

In fact, this particular effort and other partnerships have put NCTR at the forefront of research on nanotechnology. The safety and effectiveness of nanotechnology is a focus of a Memorandum of Understanding signed by the FDA Commissioner in 2011 with the State of Arkansas that enables NCTR to collaborate with five major research institutions in the state, including the University of Arkansas for Medical Sciences.

Our state partnerships within Arkansas are invaluable as they add both laboratory and investigator expertise not normally available to FDA. In addition to work with nanomaterials, our projects with Arkansas researchers include research on the effects of anesthesia on the developing brains of young animals to emulate the possible effects in children, and the development of novel bioinformatic approaches to collect, analyze and visualize massive pharmacogenomics (the genetic response to drugs) or imaging data sets.

Our federal partners, including the National Institute of Environmental Health Sciences and the National Toxicology Program (NTP), both of which share our mission to keep you safe from chemical and environmental hazards, combine with NCTR to produce a world powerhouse for safety assessment.

This 20-year partnership between NCTR/FDA and NTP has produced numerous sets of safety data that provide the scientific foundation for FDA regulators and others around the world to establish guidance and set standards to control food contaminants and assess drugs. For example, NCTR’s work on a naturally-occurring fungal contaminant (fumonisin FB1) in the nation’s corn crop produced data for FDA’s Center of Food Safety and Applied Nutrition to provide new recommended limits for fumonisin, an action that reached across the world.

NCTR also engages in public-private partnerships to foster the development of innovative products. For example, we are working with the International Anesthesia Research Society to improve the safe use of anesthetics in children. FDA has many such partnerships to leverage the expertise and resources of industry, government, and non-profit organizations in developing tools that drive innovation.

The crux of regulatory science is this: Just as an art critic must be an expert in art, a scientist at FDA must be an expert in the science that he or she is evaluating. “It takes a village” has become almost a cliché, but in truth it does take a global village to give regulatory scientists the tools they need to ensure that the exciting new technologies will translate into products that are safe, effective and will enhance your life.

William Slikker, Jr., Ph.D., is the Director of FDA’s National Center for Toxicological Research

World Health Assembly Strengthens Regulatory Standards

By: Margaret Hamburg, M.D.

The World Health Assembly is the decision-making body of the World Health Organization (WHO), attended every year by the leading government health officials from its 194 member nations. Recently, I was pleased to participate as a member of the U.S. delegation in the 67th meeting of this important group in Geneva, Switzerland.

Margaret Hamburg at World Health Assembly

Commissioner Margaret Hamburg speaks at the World Health Assembly

One of the key topics addressed during this year’s assembly was the critical role played by regulatory systems to ensure the safety, quality and efficacy of medical products. Resolutions addressed the need to monitor and act on the rise in antimicrobial resistance; strengthen regulatory systems; and enhance access to biotherapeutic products.

As part of the resolution on antimicrobial resistance, the WHO will develop a draft global action plan to combat this problem and member states were urged to strengthen their drug management systems, support research to extend the lifespan of existing drugs, and encourage the development of new diagnostics and treatment options.

The passage of a resolution to strengthen regulatory systems is itself a milestone for global health. It endorses a comprehensive approach to strengthening medical product regulation and it represents a basic change from traditional capacity-building that has focused primarily on sharing of technical expertise. The new systems-oriented approach embraces the need for a strong legal framework, and the use of data and information technology, leadership, governance, partnership, and sustainable financing to strengthen regulatory effectiveness and efficiency.

The adoption of this resolution, co-sponsored by Australia, Brazil, Colombia, Mexico, Nigeria, South Africa, Thailand and the United States, demonstrates a true international partnership across regions, with many nations affirming their commitment to working individually and together to strengthen their regulatory systems. This is the only way governments and their regulatory authorities can continue to build a global product safety net that will benefit patients and consumers around the world.

During the assembly, I  had the privilege of co-hosting with the Mexican Secretary of Health, Dr. Mercedes Juan Lopez,  a special session entitled, “Regulatory Systems Strengthening: Mobilizing People and Resources.” The distinguished panel included Malebona Precious Matsoso, Director General, Department of Health, Government of South Africa; Dr. Mark Dybul, Executive Director, The Global Fund to Fight AIDS, Tuberculosis and Malaria; Dr. Marie-Paule Kieny, Assistant Director-General of the World Health Organization; and Dr. Vincent Ahonkhai, the Senior Regulatory Officer of the Bill and Melinda Gates Foundation. The discussions focused on several important themes, including the multiple benefits of strong regulatory systems for the public health; national security; and economic development and investment.

The panelists highlighted the unique challenges and opportunities faced by regulators because of globalization and the extraordinary increase in the global supply of medical products, and the high cost of regulatory system failures to patients and their trust in the health system. More specifically, the discussions focused on the critical role that regulators play in ensuring access to lifesaving medical products by creating an environment of good regulatory practices that support innovation and research and protect the public from threats resulting from substandard or counterfeit products.

Panelists also reported on progress that is being made as nations increasingly collaborate to share information, reduce inefficiencies, and work toward regulatory convergence. For example, participants learned about the World Health Organization’s decades-long efforts to support countries in strengthening their systems, and of its prequalification program to ensure that select priority essential medicines, diagnostics and vaccines are of quality, safety and efficacy for international procurement agencies and developing countries with limited regulatory capacity.

These kinds of collaborative strategies are essential in this global age. Only by strengthening our international partnerships and building regulatory systems that work together and support each other can we ensure the quality, safety and efficacy of the medical products that the citizens of our nations depend upon.

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

OpenFDA: Innovative Initiative Opens Door to Wealth of FDA’s Publicly Available Data

By: Taha A. Kass-Hout, M.D., M.S.

Today, I am pleased to announce the launch of openFDA, a new initiative from our Office of Informatics and Technology Innovation (OITI). OpenFDA is specifically designed to make it easier for web developers, researchers, and the public to access and use the many large, important, health data sets collected by the agency.

Taha Kass-HoutThese publicly available data sets, once successfully integrated and analyzed, can provide knowledge and insights that cannot be gained from any other single source.

Consider the 3 million plus reports of drug adverse reactions or medication errors submitted to FAERS, the FDA Adverse Event Reporting System (previously AERS), since 2004.

Researchers, scientists, software developers, and other technically-focused individuals in both the private and public sectors have always been invited to mine that publicly available data set – and others – to educate consumers, which in turn can further our regulatory or scientific missions, and ultimately, save lives.

But obtaining this information hasn’t always been easy.

In the past, these vast datasets could be difficult for industry to access and to use.  Pharmaceutical companies, for example, send hundreds of Freedom of Information Act (FOIA) requests to FDA every year because that has been one of the ways they could get this data. Other methods called for downloading large amounts of files encoded in a variety of formats or not fully documented, or using a website to point-and-click and browse through a database – all slow and labor-intensive processes.

openFDA logoOpenFDA will make our publicly available data accessible in a structured, computer-readable format. It provides a “search-based” Application Programming Interface – the set of requirements that govern how one software application can talk to another – that makes it possible to find both structured and unstructured content online.

Software developers can now build their own applications (such as a mobile phone app or an interactive website) that can quickly search, query or pull massive amounts of public information instantaneously and directly from FDA datasets in real time on an “as-needed” basis. Additionally, with this approach, applications can be built on one common platform that is free and open to use. Publicly available data provided through openFDA are in the public domain with a CC0 Public Domain Dedication.

Drug adverse events is the first dataset – with reports submitted from 2004 through 2013 available now.

Using this data, a mobile developer could create a search app for a smart phone, for example, which a consumer could then use to determine whether anyone else has experienced the same adverse event they did after taking a certain drug.

As we focus on making existing public data more easily accessible, and providing appropriate documentation and examples to developers, it’s important to note that we will not release any data that could be used to identify individuals or reveal other private information.

OpenFDA uses cutting-edge technologies deployed on FDA’s new Public Cloud Computing infrastructure enabled by OITI, and will serve as a pilot for how FDA can interact internally and with external stakeholders, spur innovation, and develop or use novel applications securely and efficiently. As we move forward with the early stages of openFDA, we will be listening closely to the public, researchers, industry and all other users for their feedback on how to make openFDA even more useful in promoting and protecting the public health.

Taha A. Kass-Hout, M.D., M.S., is FDA’s Chief Health Informatics Officer and Director of FDA’s Office of Informatics and Technology Innovation.

FDA, ONC and FCC Encourage Stakeholder Engagement on Proposed Health IT Strategy and Framework

By: Bakul Patel

Last month I blogged about a report outlining our proposed strategy and recommendations on an appropriate risk-based regulatory framework for health information technology (health IT). Issued by the Food and Drug Administration (FDA), the HHS Office of the National Coordinator for Health Information Technology (ONC) and the Federal Communications Commission (FCC), the report was followed by a three-day public workshop with consumer, patient, health care and industry stakeholders.

Bakul PatelOur goals: to promote innovation, protect patients and avoid regulatory duplication. We just posted video of the workshop discussions online so that interested parties can view (or revisit) the events; a written transcript will follow. And public comments on the strategy and recommendations are open until July 7, 2014.

At the workshop, a series of structured panel discussions guided various talks among those present, including health IT experts, representatives from industry (mobile health, medical device and electronic health records), health care providers, research organizations and consumers. Topics ranged from industry best practices to the promotion of an environment of learning and continual improvement, including the proposed risk-based framework and the categories of health IT. In fact, promoting an environment of learning—a core priority area that our report establishes under the health management function—emerged as one of the most discussed priority areas. On the last day, participants discussed how to create and sustain the learning environment, guided by the proposed safety center, led by ONC.

In general, workshop participants agreed with the core of our report, including the need for a risk-based approach that focuses on functionality rather than platform. There also was support for the concept of a multi-stakeholder, public-private Health IT Safety Center—as proposed in the report—although participants wanted additional clarity on its participants, role and governance.

Many viewed the report as “moving the ball forward,” but all recognized that the health IT community still has much work to do. (Some called the event a “starting point.”)

This collaboration with health IT stakeholders is important. The workshop was just one step in the strengthening of our nationwide health IT infrastructure, and we look forward to continued public dialogue and participation. Engagement between health IT stakeholders and the federal government is key to realizing the promise of health IT. We encourage comments on our proposed recommendations for this multifaceted area of health care.

To submit your comments on our proposed strategy, please visit regulations.gov.

Bakul Patel is senior policy advisor in FDA’s Center for Devices and Radiological Health.

More information:

Learn more about the Health IT Risk-Based Framework.

Read more about the meeting.

Watch the workshop webcast.

Read the FDASIA Health Report.

FDA’s Final Guidance on Expedited Drug Approvals: Fueling Innovation and Helping Patients

By: Janet Woodcock, M.D.

Janet WoodcockIn recent years, there have been important advances to ensure therapies for serious conditions are approved and available to patients as soon as there is sufficient data to show that the therapies’ benefits outweigh their risks. Despite the progress, there is much more work to be done. Many scientific discoveries still need to be translated into treatments, while patients are urgently waiting for new life-saving therapies.

The Food and Drug Administration (FDA) is committed to doing our part to help bridge this gap. In this context, we have been actively scrutinizing, strengthening and streamlining our regulatory processes at various steps along the path from drug discovery to delivery—including the clinical development phase, the longest and most expensive period of drug development. As part of this effort, we have developed and successfully used a number of flexible and innovative approaches to expedite the development and review of drugs—to the benefit of millions of American patients. The vast majority of the time, the United States is the world’s first country to approve novel medicines. Just last year, three-quarters of the new drugs approved by FDA were approved in the United States before any other country.

Four programs that facilitate and expedite development and review of new drugs that address unmet medical needs in the treatment of serious or life threatening conditions have been especially noteworthy. A look at recent drug approvals suggests that these programs have played an important role in bringing innovative drugs to market. Nearly half of the 27 novel drugs approved by FDA last year took advantage of at least one of these expedited drug development and review approaches. And review times were as short as 4.5 months.

After incorporating input we received from stakeholders to a draft version, we are finalizing our guidance to industry today in order to provide a more detailed explanation of these programs and help drug innovators determine whether their products are likely candidates.

These expedited programs include:

Fast track designation: Providing for more frequent meetings and communications with FDA to discuss the drug’s development plan and ensure collection of appropriate data needed to support drug approval, including such things as the design of the proposed clinical trials and use of biomarkers.

Accelerated Approval: Basing approval not on a clinical endpoint but on an agreed upon surrogate marker, that is a measure such as  blood test or urine marker, that is believed to be indicative of a disease state and treatment effect, but not demonstrative of a direct health gain to the patient.

Priority review: Acting on drug applications within 6 months instead of 10 months for standard review, and;

Breakthrough Therapy Designation: Providing all of the benefits of Fast Track designation plus intensive guidance on an efficient drug development program, beginning as early as Phase 1, and the commitment from FDA’s review staff, including senior managers, to work closely together throughout the drug development and review process.

Certainly our new Breakthrough Therapy Designation, created as part of the 2012 FDA Safety and Innovation Act (FDASIA) has been a virtual overnight success. As of May 5, 2014, we have received 186 requests for the designation, and granted 48. Six drugs have been approved, including a late-stage lung cancer drug that was approved—four months ahead of its goal date, using evidence from a trial with 163 patients.

Since its inception in 1992, more than 80 new products have been approved under the Accelerated Approval pathway. It has long been successful in driving innovation in cancer and HIV therapies, but we’re encouraging its broader application in other areas, helped by FDASIA which clarified that FDA has the authority to consider epidemiologic, pharmacologic or other evidence developed using biomarkers or other scientific methods or tools in determining whether an endpoint can support accelerated approval. We’re also exploring whether reviewer training programs and other measures might encourage greater use of this program.

It’s important to note that our own regulatory flexibility is reducing the number of approvals under the accelerated approval program. Most of the recent new drug approvals for rare diseases—approvals of drugs that would qualify for the accelerated approval program based on the serious of the disease and the lack of available therapies and that have shown effects on surrogate or intermediate endpoints—have not been approved under accelerated approval, because they were given regular or “traditional” approval. This meant that they weren’t required to do confirmatory trials required under the accelerated approval program to verify clinical benefit, because we decided that the evidence was already strong enough.

We urge drug developers and others interested in this movement to take a close look at today’s final guidance. For those drugs that qualify, participating in one of these expedited programs can reduce the time and possibly the cost of developing new therapies that can save lives. That’s a win for drug innovation and for patients.

Janet Woodcock, M.D., is the Director of FDA’s Center for Drug Evaluation and Research

FDA and Pan American Partners Work to Strengthen Regulatory Systems

By: Charles Preston, M.D., MPH

Regulatory systems are essential for good health care because they ensure safe, high quality and effective medicines. However, these systems must be strengthened in many parts of the world — a subject that is a core effort of the Pan American Network for Drug Regulatory Harmonization (PANDRH).

Charles PrestonI had the privilege of representing the FDA at a recent steering committee meeting, and I am happy to report that this network, which includes countries from all over the Western Hemisphere, is ready to adapt to new challenges like globalization, and improve its effectiveness.

In the past, the network has concentrated on upgrading regulatory standards by developing guidances and strengthening regulator capacity through training. There have been many successes, including multiple guidances issued and numerous trainings conducted.  However, there is a recognition that these efforts need new focus.

Thus, PANDRH has a strategic plan for modernizing its activities.

Rather than only developing region-specific guidances, it proposes improving standards by leveraging the work of global bodies, such as the International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use, and the Pharmaceutical Inspection Cooperation Scheme.

To strengthen capacity for oversight, PANDRH members will work to develop and implement a globally agreed upon set of regulator competencies, and use this to drive curricula and training objectives in the region. By doing this, the regulatory workforce can be professionalized, which will strengthen it for the future.

Another new emphasis will be to use evidence to help set priorities. The secretariat and PANDRH member states will now analyze data from evaluations of regulatory systems in the region to decide on future activities and benchmark success.

The importance of these changes is obvious: in today’s world of global manufacturing, trade and consumption, national regulators must be truly professional and able to implement global standards.

These are exciting developments that portend an auspicious new direction for PANDRH. FDA will continue its active engagement in the network’s efforts. They are essential to achieving a world in which everyone has access to safe, high quality and effective medical products that can protect or restore human health.

Charles Preston, M.D., MPH, is a medical officer in FDA’s Office of International Programs

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.

FDA’s “Voice of the Patient:” Listening to Those Most Affected by Their Disease and Treatments

By: Theresa M. Mullin, Ph.D.

Last year, FDA began the Patient-Focused Drug Development (PFDD) program to more systematically obtain the patient perspective on certain diseases and their treatments.  The effort is part of an FDA commitment under the fifth authorization of the Prescription Drug User Fee Act (PDUFA V).

Theresa MullinAfter conducting a public process to nominate disease areas for fiscal years 2013-2015, FDA held the first PFDD meeting on April 25, 2013. This meeting focused on chronic fatigue syndrome (CFS) and myalgic encephalomyelitis (ME), sometimes called CFS-ME, a debilitating disease for which there are currently no FDA-approved treatments.

Here, we heard directly from patients, patient advocates, and caretakers about the symptoms that matter most to them, the impact the disease has on patients’ daily lives, and the patient experience with currently available treatments. FDA staff, including members of FDA’s Division of Pulmonary, Allergy, and Rheumatology Products, listened carefully to the personal accounts of this devastating condition.

After the meeting, we released a report titled The Voice of the Patient: Chronic Fatigue Syndrome and Myalgic Encephalomyelitis, a detailed summary of the meeting. In this report we documented, in the patients’ own words, what mattered most to them in terms of impacts of the disease and treatment approaches. This summary included the patient testimony at the meeting, perspectives shared in 230 docket comments, as well as unique views provided by those who joined the meeting webcast.

These reports serve an important function in communicating, to both FDA review staff and the regulated industry, what improvements patients would most like to see in their daily life. FDA believes that the long-term impact of this program will be a better, more informed understanding of how we might find ways to develop new treatments for these diseases.

Soon after the CFS and ME meeting, in June 2013, we conducted similar meetings on HIV and lung cancer, and these summary reports are now available on our website. The reports for our recent meetings on narcolepsy and sickle cell disease will be posted soon.  Our most recent meeting was held on fibromyalgia on March 26, 2014. On May 13, we held the meeting on pulmonary arterial hypertension, and coming up next is the meeting on inborn errors of metabolism, on June 10.

By the end of FY 2015, we plan to have conducted at least 16 PFDD meetings to hear from patients suffering from a wide range of conditions. These are currently identified on our web page. For the remaining two years in PDUFA V, we will conduct another public process to identify the diseases that will be addressed during that time.

We are gratified by the enthusiastic response within the patient community to PFDD, and we look forward to continued success with these meetings — and the long-term benefit they can offer for drug development in important therapeutic areas.

Theresa M. Mullin, Ph.D., is Director of FDA’s Office of Strategic Programs in the Center for Drug Evaluation and Research