The Unique Voices of Our Patient Representatives

By: Robert M. Califf, M.D., and Heidi C. Marchand, Pharm.D.

We recently met with 21 inspirational patients and patient caregivers who have made the extraordinary commitment to become FDA patient representatives. These volunteers were in Washington to participate in our two-day Patient Representative Workshop so they can receive training that will allow them to help FDA meet its critical responsibility of guiding the development and evaluation of safe and effective medical products.

Robert Califf

Robert Califf, M.D., Commissioner of the U.S. Food and Drug Administration

The patient representative program has existed since 1999 and is integral to fulfilling FDA’s strong commitment to ensure that the needs and choices of patients – as well as their families, caregivers, and advocates – are incorporated in ever greater ways in the work we do.

Patients add context and content to the cutting-edge science and other empirical evidence that is so important in our regulatory decision-making.  Including their perspectives and voices in our work along the entire medical product continuum, from development to review and evaluation to post-market surveillance, offers opportunities to enhance our knowledge of the benefits and risks of medical products. It’s not only smart science; it just makes good sense. We know, for instance, that patients who live with a chronic disease are experts in the tangible effects of that disease and its treatments.

The training that patient representatives receive helps prepare them to serve on FDA advisory committees, meetings and workshops, where they are knowledgeable about what it is like to cope with their disease – including such topics as side effects from treatments and important lifestyle issues. They also provide valuable contributions as consultants to our review staff.

Heidi Marchand

Heidi C. Marchand, Pharm.D., Assistant Commissioner in FDA’s Office of Health and Constituent Affairs

To give you an idea of the unique set of skills and experiences patient representatives bring to their work, consider the stories and experiences we heard at the workshop.

One was an elite world class athlete, who initially thought her pain was muscular in nature before it was diagnosed as a serious blood clot. She has been on a series of different products since then and is now intimately familiar with what it is like to be on anticoagulants – reflecting on both the benefits and risks of taking these medications.

Two of our patient representatives are caregivers who have a personal experience with a rare disease, Batten’s Disease, a fatal, inherited disorder of the nervous system. Sadly, each lost a young son to the disease. But in the face of this tragedy, these two mothers have advocated tirelessly to find a cure for this disease and worked to educate other parents.

Another mother related the story of her daughter who, at age 16, survived two craniotomies to remove a lemon-sized brain tumor. The daughter went on to receive of 48 weeks of chemotherapy and 8 weeks of brain and spine radiation. The daughter is now 33 years old and doing well. And the mother told us how critical it was for her daughter to take an opioid to relieve her pain. This kind of input, from those who have experienced it first hand, is critical to our future decisions.

2016 FDA Patient Representative Group photo

FDA Patient Representatives at the 12th Annual FDA Patient Representative Workshop, hosted by FDA’s Office of Health and Constituent Affairs

The stories that these patient representatives tell are moving. But even more moving – and indeed inspirational – is their commitment to the future. That’s why they were selected – because of their individual involvement with their respective patient communities, their analytical skills, and their ability to maintain an open mind and consider options.

While we will help train them about the nuts and bolts of FDA – such as the various pathways that products take to get to market – it is their personal experience and their ability to understand and to articulate the perspectives, concerns, and experiences of patients – that makes them truly special.

As we continue to evaluate potential treatments and cures for different diseases, we must make sure that patients are more than simply statistics in this equation. They are real people, with names, faces, and, thanks to these patient representatives, important voices who represent an essential piece of the puzzle to be solved.

FDA is committed to looking for new and better ways to integrate the patient voice. Our patient representatives are an important piece of this commitment. They have an extraordinary impact. We thank them for their service and commitment, and look forward to working with them.

Robert M. Califf, M.D., is Commissioner of the U.S. Food and Drug Administration

Heidi C. Marchand, Pharm.D., is Assistant Commissioner in FDA’s Office of Health and Constituent Affairs

Addressing Global Challenges through Transatlantic Cooperation

By: Howard Sklamberg, J.D., Lou Valdez, and Donald Prater

Howard Sklamberg

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

On a recent trip to Brussels, our FDA delegation met with many of our European Union (EU) regulatory counterparts and stakeholders to discuss ways to strengthen our shared commitment to product safety and public health.  

Reflecting the broad scope of our transatlantic dialogue, we engaged on an array of issues, including supply chain safety, quality metrics, risk-based surveillance, data integrity, mutual reliance, and food safety systems.

Building on previous exchanges between FDA and the European Parliament (EP), we first met with Members of the Environment, Public Health and Food Safety Committee, known as ENVI.  ENVI Committee members visited FDA in 2013 and 2015 to share their perspective on how certain health-related topics are being addressed in the European Union. In addition, our FDA delegation exchanged views on recent trilateral cooperation with India and China on Good Clinical Practices and food safety and other approaches to cooperation on the international stage.

Lou Valdez

Mary Lou Valdez, FDA’s Associate Commissioner for International Programs

We then met with the head of the European Commission’s Directorate General for Health and Food Safety (DG SANTE), Director General Xavier Prats-Monné, and his colleagues.

We shared our observations on several topics, including:

  • How drug development has changed, including globalization of suppliers and distributors;
  • The challenges among regulatory bodies in keeping pace with risk-based allocation of inspection resources;
  • The complexity of the global supply chain and the need to collaborate on enforcement;
  • The significant progress being made on the Mutual Reliance Initiative (MRI);
  • Pharmaceutical GMP inspections; and
  • The interaction among FDA’s Europe, China, and India offices and regulatory counterparts in the EU and Governments of China and India.
Donald Prater

Donald Prater, D.V.M., Director of the Europe Office in FDA’s Office of International Programs.

We then turned to food safety. Currently, the U.S. and the European Commission are working on a Food Safety Systems Recognition arrangement, a program that FDA has developed to increase regulatory cooperation and build toward reliance on the work of regulatory counterparts.

Such cooperation is facilitated through the reciprocal assessment of one another’s food safety systems to ensure the safety of foods produced under one another’s oversight. The United States already has an arrangement in place with New Zealand and recently signed one with Canada.

We also reviewed the Food Safety Modernization Act (FSMA) and discussed ways FDA and the European Commission can assist suppliers in the EU to better understand the FSMA requirements. Acknowledging that food safety standards are quite high in the United States and the EU, we discussed ways we can leverage the systems on both sides of the Atlantic to further protect consumers and more efficiently use our oversight resources globally.

FDA and EU Delegations in Brussels

A U.S. Food and Drug Administration (FDA) delegation met with many of their European Union (EU) regulatory counterparts in Brussels to discuss ways to strengthen the shared commitment to product safety and public health. Pictured from left to right are: Karin Kadenbach, Member European Parliament (MEP); Sandy Kweder, Deputy Director, FDA’s European Office; Lou Valdez, FDA’s Associate Commissioner for International Programs; Matthias Groote, MEP; Howard Sklamberg, FDA’s Deputy Commissioner for Global Regulatory Operations and Policy; and, Susanne Melior, MEP.

Next up were meetings on medical devices and cosmetics with the Directorate General for Internal Market, Industry, Entrepreneurship, and SMEs, also known as DG GROWTH.

We were welcomed by Carlo Pettinelli, Head of the Directorate for Consumer, Environmental and Health Technologies, and we discussed the key objectives of the Medical Device Single Audit Program (MDSAP) of the International Medical Device Regulators Forum (IMDRF). Mr. Pettinelli acknowledged the importance of MDSAP, and indicated that the European Commission would continue to provide coordination and communication to support the engagement of EU Member States in the program.

We also set aside time for discussion with key industry stakeholders representing medical products – primarily drugs and devices, including, the American Chamber of Commerce Healthcare Committee to the EU and the European Federation of Pharmaceutical Industry Association (EFPIA). There we reviewed FDA’s Pharmaceutical Quality and MRI initiatives.

Our trip concluded with a media roundtable and a briefing to the Deputy Chief of Mission and staff at the U.S. Mission to the European Union. Our FDA Europe Office is based at the USEU and provides critical support to U.S. Ambassador Anthony Gardner.

Throughout all our meetings, one theme was crystal clear: Transatlantic cooperation is vitally important to address the challenges and opportunities of a globalized marketplace.  By carefully evaluating and understanding each other’s regulatory systems, there is tremendous potential to better allocate our resources based on risk, and improve the safety of food, medical products, cosmetics, and other products around the world.

Howard Sklamberg, J.D., is the Deputy Commissioner for Global Regulatory Operations and Policy

Mary Lou Valdez is the Associate Commissioner for International Programs

Donald Prater is Director of FDA’s Europe Office

The Rise in Orphan Drug Designations: Meeting the Growing Demand

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

Developing drugs for rare diseases, once considered a rare phenomenon itself, has fast become a mainstay for many companies’ drug development pipelines. This is exciting news for the 30 million Americans with rare diseases and their families.

Dr. Gayatri RaoCongress played no small role in making this a reality when it passed the Orphan Drug Act in 1983.  One of the key features of this Act was the creation of the Orphan Drug Designation Program, which provides important financial incentives to encourage companies to develop drugs and biologics for rare diseases. This legislation includes major tax credits to defray the cost of conducting clinical trials, as well as eligibility for seven years of market exclusivity. As a result of later amendments to the Act, no user fee is required for orphan drug product submissions, except when an application includes an indication for a non-rare disease or condition.

The number of requests for orphan drug designation received by FDA’s Office of Orphan Products Development (OOPD) has grown dramatically in recent years and is prompting FDA to adjust its timeframes for reviewing orphan drug designations in order to meet the demand. In 2014, we saw a 30% increase over the prior year’s record number. Yet, that record was broken the very next year when we received close to 470 requests. And the pace does not seem to be slowing. In fact, comparing the number of new requests received so far in 2016 with the corresponding date in 2015, there appears to be yet another 30% increase.

We strive to review these requests in an efficient and timely manner because we understand how critical designation can be for companies to move forward with their drug development plans. At the same time, we endeavor to safeguard the intent of the Orphan Drug Act by conducting a thorough review to ensure that the drugs we designate fully satisfy the criteria for designation and the financial incentives associated with designation.

While there is no statutory or regulatory review deadline, it has been our internal goal to review 75% of designation requests within 90 days of receipt. By streamlining our programs, modifying work priorities, and restructuring workloads, we have generally been able to meet or exceed that internal goal. However, the sustained increase in designation requests over the last three years, coupled with the increasing number of incentive programs and competing workload priorities, have forced us to reconsider our internal review target. Reviewing these applications in an efficient and timely manner continues to be a top priority, but to ensure we continue to conduct these reviews with the appropriate level of care and consideration, our current goal is to review on average 75% of designation requests within 120 days of receipt.

We will continue to evaluate workload in relation to resources, and may need to further adjust review timelines in the future.

Companies can play a critical role in ensuring that the new review timeframe does not translate into a delay in obtaining orphan drug designation by doing their part to reduce the number of review cycles needed (i.e., when OOPD needs additional information from the sponsor prior to determining the outcome of an orphan drug designation request).

On average, a request for designation today goes through two such review cycles. Sponsors can shorten this process by ensuring that designation requests are complete and fully address all requirements. We recommend sponsors review the information at www.fda.gov/orphan for helpful hints and FAQs when developing their requests.

The rise in the number of requests for orphan drug designation holds promise for the future of rare disease drug development. We remain committed to the timely and effective administration of the Orphan Drug Designation Program with the shared hope of bringing safe and effective products quickly to the patients who need them most.

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

The United Nations Sustainable Development Goals: Efficient and effective regulatory systems are the tide that raises all boats

By: Mary Lou Valdez, M.S.M., and Kristin Wedding

Lou Valdez

Mary Lou Valdez, FDA’s Associate Commissioner for International Programs

Do you think it’s possible to ensure healthy lives and promote well-being for all people of all ages by 2030? That’s just one of the United Nations 17 Sustainable Development Goals (SDGs), which the world’s leaders agreed to in September 2015. And, FDA has an important role in supporting these goals.

On June 23-24, 2016, we had the opportunity to participate in the National Academies of Science, Engineering, and Medicine’s Forum on Public-Private Partnerships for Global Health and Safety (PPP Forum). The two-day workshop focused on engaging the private sector and developing partnerships to advance health and the SDGs.

Good health and well-being are linked to many of the SDGs, including zero hunger, ending poverty, economic growth, industry, innovation and infrastructure, and reduced inequalities. But what, you might ask, are FDA’s potential contributions as a regulatory agency in achieving the SDGs?

Regulatory Systems and the SDGs: the Challenges

Kristin Wedding

Kristin Wedding is International Policy Analyst in FDA’s Office of International Programs

Strong functioning regulatory systems for food and medical products are at the nexus of public health, economic development, trade, and investment. Within our public health mission, effective regulatory systems often are a necessary precursor for economic development and growth, including private sector investment. Conversely, the absence of effective regulatory systems is an underlying threat to achieving many of the SDGs. For example:

  • unsafe food contributes to malnutrition and jeopardizes food security (Goal 2);
  • lack of access to safe and effective medical treatments exacerbates chronic diseases (Goal 3), and impedes people from getting and keeping a job (Goal 8).
  • illness hampers children’s learning and school attendance (Goal 4), and often disproportionately impacts girls (Goal 5); and,
  • insufficient access to clean water and sanitation (Goal 6) results in the death of more than 1,000 children each day from preventable diarrheal diseases.

Regulatory Systems and the SDGs: the Opportunities

At the workshop, FDA chaired an expert panel on the critical role of regulatory systems and PPPs in promoting global public health, economic development, and sustainable investments to achieve the SDGs. We were joined by Dr. Juergen Voegele of the World Bank, Dr. Rajeev Venkayya of Takeda Pharmaceuticals, and Dr. Dan Hartman of the Bill & Melinda Gates Foundation.

Despite our diversity, we sent a unified message that regulatory systems are essential drivers for the success and sustainability of global health investments to meet the SDGs. It is the responsibility of all of us – as stakeholders, donors, and partners – to make our investments matter.

UN Panel Discussion

Panel discussion at National Academies of Science, Engineering, and Medicine’s Forum on Public-Private Partnerships (PPPs) for Global Health and Safety: A Workshop on Engaging the Private Sector and Developing Partnerships to Advance Health and the Sustainable Development Goals (SDGs).
Mary Lou Valdez, M.S.M, Associate Commissioner for International Programs, FDA, and Juergen Voegele, Ph.D., Senior Director, Agriculture Global Practice, The World Bank.

FDA participates in a number of global partnerships aimed at strengthening regulatory systems, including the World Bank-led Global Food Safety Partnership (GFSP) the PPP Forum, as well as our work with multilateral institutions such as the World Health Organization.

We look forward to continuing the discussion about future pathways for collaboration and action in demonstrating the critical roles regulatory systems play in the attainment of the SDGs. The public health of U.S. citizens – and others around the globe – can benefit from our efforts now and in the future.

As Dr. Hartman so aptly noted during the panel session, in this time of globalization “efficient and effective regulatory systems are the tide that raises all boats.”

Mary Lou Valdez, M.S.M., is FDA’s Associate Commissioner for International Programs

Kristin Wedding is International Policy Analyst in FDA’s Office of International Programs

FDA Takes Action against Zika Virus

By: Robert M. Califf, M.D., and Luciana Borio, M.D.

Zika virus was first identified in 1947 in Uganda and for decades only sporadic cases and a few outbreaks were recognized in a number of locations, including parts of Africa, Asia, and the Pacific. Since 2015, the situation has changed dramatically, with 48 countries and territories reporting a first outbreak of Zika virus as of July 2016. In the United States, cases of Zika virus disease acquired by the bite of an infected mosquito have only been reported in U.S. territories; to date, cases of Zika virus infection reported in the continental United States have involved travelers and in some instances their sexual contacts. However, given the number of Zika cases among travelers visiting or returning to the United States and the increased mosquito activity in the summer months, we expect that imported cases could result in local spread of the virus in some areas of the United States.

Robert Califf

Robert Califf, M.D., is Commissioner of the U.S. Food and Drug Administration

The FDA is taking important steps to rapidly respond to the Zika virus outbreak. We are engaged with our partners across the U.S. Government, the private sector, and the international community—including the World Health Organization and ANVISA (the Brazilian Health Regulatory Agency)—to help minimize the impact of this outbreak.

Protecting Tissues and the Blood Supply

One of the FDA’s first actions was to take important steps to help protect the safety of the blood supply. The FDA issued guidance in February 2016 recommending the deferral of individuals from donating blood if they have been to areas with active Zika virus transmission, were potentially exposed to the virus, or have had a confirmed infection. The guidance also recommends that areas with active Zika virus transmission, like Puerto Rico, obtain whole blood and blood components from areas of the United States without active virus transmission unless a blood donor screening test for Zika virus is used. Because there were no blood donor screening tests available for Zika virus at the time, HHS arranged for and funded shipments of blood products from the continental U.S. to Puerto Rico to ensure an adequate supply of safe blood for residents until a blood donor screening test became available. The FDA worked closely with developers in a highly accelerated time frame to make available an investigational test for blood screening in March 2016. The availability of this investigational test, which has been in use in Puerto Rico since early April, has allowed blood establishments to safely collect blood in areas with active Zika virus transmission. A second investigational blood screening test was made available in June 2016. Together, these tests have also enabled blood donor screening to be put in place in areas of the United States where local virus transmission is anticipated, but not yet detected, helping to maintain the safety of the blood supply.

Dr. Lu Borio

Luciana Borio, M.D., is FDA’s Acting Chief Scientist

Zika virus also poses a risk for transmission by human cells, tissues, and cellular and tissue-based products (HCT/Ps) such as corneas, bone, skin, heart valves, and semen used for medical, surgical, or reproductive procedures. Because of this risk, the FDA issued guidance recommending that donors of HCT/Ps be considered ineligible if they were diagnosed with Zika virus infection, were in an area with active Zika virus transmission, or had sex with a male with either of those risk factors, within the past six months.

Supporting Diagnostic Development

The ability to accurately detect and diagnose Zika virus infection is critical for a robust response to this public health threat. The FDA is actively working with manufacturers to support their diagnostic development programs, helping to ensure that their tests are properly validated before they are used to inform patient care. This collaboration has been very successful, and since the beginning of the year, we have authorized the use of five diagnostic tests for Zika virus under FDA’s Emergency Use Authorization authority—four tests to diagnose active infection and one test to assess whether individuals who may have recently been exposed to Zika were actually infected. This test is especially important for women given the link between Zika virus infection and microcephaly and other poor pregnancy outcomes in babies of mothers who were infected with Zika virus during their pregnancy.

Strategies to Suppress Mosquito Population

FDA—as well as our colleagues at EPA— are reviewing the use of innovative strategies to help suppress the population of virus-carrying mosquitoes to help mitigate the threat of vector-borne epidemics, such as Zika virus, which is thought to spread to people primarily through the bite of an infected Aedes aegypti mosquito.

Recently, the FDA released for public comment a draft environmental assessment (EA) submitted by Oxitec, Ltd. (Oxitec). The EA assesses the potential environmental impacts of a proposed field trial of the company’s genetically engineered (GE) Ae. aegypti mosquitoes. The FDA also released for public comment a preliminary Finding of No Significant Impact (FONSI) agreeing with the conclusion in Oxitec’s draft EA that the proposed field trial of the company’s GE mosquitoes would not result in significant impacts on the environment.

The goal of the proposed field trial is to determine whether released Oxitec GE mosquitoes will mate with local wild-type Ae. aegypti and suppress their population at the release site. The FDA is reviewing the thousands of comments received during the public comment period before determining whether to finalize the EA and FONSI or prepare an environmental impact statement (EIS). Oxitec will not proceed with the field trial of the GE mosquitoes until FDA issues its final EA and FONSI or EIS. Oxitec’s GE mosquitoes are one possible approach that could be incorporated into an integrated vector control program to help mitigate the threat of vector-borne epidemics; however, it is too early to say with any certainty whether such an approach would be successful.

Facilitating Medical Product Development

There are currently no vaccines or treatments for Zika virus that have been shown to be safe and effective. Facilitating the development and availability of vaccines is one of the highest priorities for the FDA and the international community. The FDA continues to actively engage with commercial and government developers, including the NIAID and BARDA, to advance the development of investigational vaccines for Zika virus as soon as possible. We are also working with ANVISA to assist in their efforts to expedite the development of vaccines for Zika virus. As was recently reported, a commercial company announced plans to begin evaluating the first investigational Zika virus vaccine in a Phase I clinical study.

Unfortunately, during outbreak situations, fraudulent products claiming to prevent, treat or cure a disease almost always appear. FDA is monitoring for fraudulent products and false product claims related to Zika virus and will take appropriate action to protect consumers when necessary.

More than 120 FDA staff from across the Agency are  responding to the Zika virus outbreak, working together to address the complex range of issues that this evolving epidemic continues to present in order to protect and promote the public health, both domestically and abroad. This type of teamwork exemplifies the capacity of people at FDA to rally together to solve problems, often with little explicit credit other than the satisfaction of meeting the mission of promoting and protecting the public health. There are many fundamental scientific questions that need to be addressed with respect to Zika virus, and our scientists are working to help answer some of these questions in our own laboratories. We stand ready to use our expertise and authorities to the fullest extent to help facilitate the development and availability of products that may help mitigate the Zika virus outbreak.

Visit our Zika response web page for more information, including the latest Zika virus response updates from FDA.

Robert Califf, M.D., is Commissioner of the U.S. Food and Drug Administration

Luciana Borio, M.D., is FDA’s Acting Chief Scientist

FDA Advisory Committee Members and ‘Appearance Issues’

By: Michael Ortwerth, Ph.D.

FDA relies on its advisory committees as a source of independent scientific and technical expertise and advice on challenging public health issues. Most advisory committee members are appointed as “special government employees” (SGEs). Like regular government employees, these committee members are subject to Federal conflict of interest laws and regulations.

Michael OrtwerthA lack of understanding about our selection and evaluation process has, at times, resulted in confusion and misunderstandings by the public.  We’ve been working to bring greater transparency to how the financial interests of committee members are evaluated.

In 2008, we published “Guidance for the Public, FDA Advisory Committee Members, and FDA Staff on Procedures for Determining Conflict of Interest and Eligibility for Participation in FDA Advisory Committees.” That guidance describes how we apply financial conflict of interest requirements.

What has not been previously addressed in guidance is something called “appearance issues.” Sometimes FDA advisory committee members who do not have interests and relationships that are financial conflicts of interest nevertheless have interests and relationships that may create the appearance that they lack impartiality. Appearance issues are addressed in a government-wide regulation regarding standards of ethical conduct for government employees at 5 CFR 2635.502 (informally known as “Section 502”).

Some examples include:

  • When a member of the household works or is seeking to work for the sponsor with a product before the committee;
  • When a member has had past financial interests with the sponsor with a product before the committee; and,
  • When a member has a current consulting contract with a sponsor but the contract is not related to the product or issue before the committee.

We have recently published new draft guidance describing FDA’s procedures for evaluating appearance issues and how we determine whether to grant an authorization for a member with an appearance issue to participate in an FDA advisory committee.

Section 502 implements the ethical principle that a government employee should be impartial in performing their official duties, meaning that they must not give preferential treatment to any private organization or individual or use public office for private gain. To the extent that an advisory committee member’s performance of official duties might appear to benefit themselves or certain other individuals who are close to them, they must take appropriate steps to avoid an appearance of violating these ethical principles.

We also explain in the draft guidance the circumstances that FDA considers when determining whether an appearance issue may exist. We evaluate the circumstances and assess whether the interests, relationships, or circumstances would cause a reasonable person with knowledge of the relevant facts to question the advisory committee member’s impartiality in the matter before the committee. For example, if an advisory committee member serves on the board of directors of a nonprofit organization and that organization receives donations from the sponsor that is presenting before the committee, we review the details of the donation to determine whether the member should be cleared for service on the advisory committee.

FDA has flexibility and discretion in deciding whether an advisory committee member with an appearance issue should be authorized to participate in the advisory committee meeting. We evaluate whether the government’s interest in the advisory committee member’s participation outweighs the concern that a reasonable person may question the integrity of the agency’s programs and operations. If so, FDA may authorize the member to participate in the meeting.

Although FDA advisory committees provide advice and input to the Agency, FDA makes the final decisions.

The draft guidance is being issued for public comment before we issue a final guidance. Under Federal law, FDA is not permitted to disclose confidential information provided by advisory committee members related to appearance issues. But we are specifically requesting comments on whether the agency should request that advisory committee members voluntarily disclose if they have been granted an appearance authorization.

FDA is committed to ensuring that appropriate expertise and experience is brought to bear on the critical public health issues facing the agency. Often, we convene advisory committee meetings to obtain independent expert advice and perspective. At the same time, it is important that the process we use to screen advisory committee members for participation in meetings be as transparent as possible, and that we protect the credibility and integrity of advisory committee advice. We welcome your comments on how the agency can continue to meet these important goals.

Michael Ortwerth, Ph.D,. is FDA’s Director of the Advisory Committee Oversight and Management Staff

Leveraging the Power of Collaboration – FDA’s New Oncology Center of Excellence

By: Richard Pazdur, M.D.

I am honored to be selected by Commissioner Califf today as the acting director of FDA’s new Oncology Center of Excellence (OCE) in support of the Vice President’s National Cancer Moonshot Initiative.

Dr. Richard PazdurThis new center will be a place where the combined skills of regulatory scientists and reviewers with oncology clinical expertise in drugs, biologics, and devices will come together to support an integrated approach to the advancement of cancer treatment.

The OCE emulates both academia and cancer care centers, which are increasingly organized in multidisciplinary models to enhance collaboration, which is so essential when confronting a complex disease like cancer.

Such a collaborative approach – the sharing of ideas, information and best practices – closely fits my own vision for oncology at the FDA.

When I first joined FDA from the MD Anderson Cancer Center in Houston Texas in 1999, oncology products were reviewed in different divisions within the Center for Drug Evaluation and Research (CDER), in addition to those reviewed by other centers. My current Office of Hematology and Oncology Products (OHOP) was created in 2005 in an attempt to consolidate the review of oncology products within CDER. Additional reorganization into disease-specific teams followed in 2011. This reorganization greatly enhanced both our retention and recruitment of professional staff from leading academic centers. Disease-specific expertise expedited review processes and fostered multiple outreach activities to patient and professional groups. Between 2010 to the present, OHOP approved 61 new molecular entities to treat a variety of cancers – and most approvals were well before their deadlines.

The OCE will build on FDA’s integrative approach to medical product development and the collaborative work that has been a hallmark of the broader FDA oncology community for nearly a decade such as our cross-center monthly meetings to discuss key oncology issues, collaborative workshops and programs and the work we’ve done together on research and scientific publications.

This new center will also continue to facilitate the incorporation of the patient view in our regulatory decision-making, which has become a personal mission for me since my wife Mary, an oncology nurse, died of ovarian cancer last November.

And by bridging the various medical product centers, the OCE will be ideally suited to support innovation and to address the recognition that multiple treatment and diagnostic options are in the best interest of patients.

Certainly the key to OCE’s future success will be leveraging the talents of the staff at FDA. The very first thing I plan to do as acting director is to meet  with those involved in oncology medical product development and review across centers to hear their ideas for the OCE and how we can work together to enhance our efforts across the agency.

Developing the structure of the OCE is an ongoing process. Working closely with the center directors we will develop a staged approach for establishing the new center while ensuring the work across centers continues without disruption.

I look forward to guiding the agency through this initial phase, building our cross-disciplinary review staff, providing external outreach to diverse stakeholders and streamlining administrative processes to ensure rapid review of important cancer products to the American public.

Richard Pazdur, M.D., is FDA’s Acting Director, Oncology Center of Excellence

FDA: A Great Place for Science…and for Scientists on the New Frontier of Regulatory Science

By: Robert M. Califf, M.D.

Robert CaliffAs FDA Commissioner, I’m proud of our agency’s extraordinary commitment to using the best available science to support our mission to protect and promote the health of the American public. This is especially critical today, as rapid scientific and technological advances are helping to expand our understanding of human biology and underlying disease mechanisms and to identify the molecular profile of a food contaminant.

These breakthroughs offer unprecedented opportunities for us to develop new treatments and cures and to protect our food supply with a robust system that meets the challenges of globalization.

But there’s another benefit that derives from our application of cutting-edge science to the challenges we face, which has become increasingly evident to me through my conversations with some of FDA’s more than 10,000 scientists. And that’s the deep personal and professional satisfaction gained from working in FDA’s state-of-the-art laboratories on front-line issues that make a real difference in the lives of all Americans. As one FDA scientist commented, “At FDA, your work is really at the crossroads of cutting-edge technology, patient care, tough scientific questions, and regulatory science.”

Being Part of a Vibrant Collaborative Scientific Environment

Whether you’re a biologist, chemist, epidemiologist, pharmacist, statistician, veterinarian, nurse, physician, or an engineer and whether you’re a recent graduate or a seasoned scientist, FDA offers an unmatched opportunity to be a part of a vibrant, collaborative culture of regulatory science.

FDA scientists gain a bird’s eye view of the pharmaceutical and food industries, and develop a thorough familiarity and understanding of the regulatory structure that guides these industries. As one young FDA scientist recently commented, “We see a tremendous breadth of different products here, which helps us learn quickly and makes our jobs interesting and challenging.” Another newly trained FDA scientist shared, “We have the chance to work with highly trained colleagues, within and across disciplines, to build and keep our scientific training cutting-edge.”

While the work of FDA scientists helps to advance scientific understanding, it goes much further than that. That’s because our work is directly tied to regulatory decisions. As such it has a powerful and immediate effect on the health of millions of Americans. As another FDA scientist explained, “We get to see how these basic science and clinical advances get applied to producing medical treatments and devices and how these can make differences in people’s lives.”

FDA offers a number of fellowship, internship, graduate, and faculty programs through which newly-minted scientists can join FDA and continue to apply and develop their skills. Many of these individuals remain on as full-time FDA scientists. One former FDA Fellow said they appreciate how “FDA makes room for and respects voices of young, qualified scientists.”

Tackling the Most Challenging Scientific Issues

So, although I may frequently boast about FDA’s responsibility and ability to do rigorous scientific research and its importance for the American public, I’m speaking as much about our scientists as our science. And I hope that when other young talented scientists consider these testimonies from our multifaceted scientific workforce they will be encouraged to join us.

I want to see more professionals take advantage of the opportunities FDA offers to collaborate on some of the most transformative scientific issues of our times – both for their benefit and for the nation’s. We need the best scientific minds to tackle the challenges of food safety, medical product development, and to evaluate how emerging technologies are affecting FDA-regulated products so that our reviewers can make science-based decisions about a product’s benefits and risks.

That’s why we’ve successfully added thousands of qualified new employees over the last several years and worked hard to fill mission-critical positions. It’s also why we continue to seek more hiring flexibilities and other ways that enable us to be more competitive with private-sector salaries for these positions.

The career opportunities at FDA are enormous, and I look forward to welcoming the next generation of scientists of every stripe to help us fulfill our mission. It’s not only good for science and essential to FDA’s ability to protect and promote public health; it’s a unique opportunity for these talented scientists and their careers.

FDA Scientists Discuss Their Cutting-Edge Research in FDA Grand Rounds Webcasts

Robert M. Califf, M.D., is Commissioner of the U.S. Food and Drug Administration

Important steps toward streamlining access to investigational drugs for patients in need

By: Richard A. Moscicki, M.D.

FDA is only too aware that there are many patients who have a serious or life-threatening medical condition for which there is no available FDA-approved therapy. For such patients, one option may be to obtain access to an investigational drug that has not yet been approved by FDA. To do this, a physician submits an application to the FDA requesting authorization to use the investigational drug in the treatment of their patient. This is called expanded access to investigational drugs.

Dr. Richard MoscickiWhile FDA has been helping physicians navigate the system for many years, we are aware there have been physician and patient concerns about this process, which can be time consuming and difficult to understand. Consequently, FDA has recently made significant changes to streamline and simplify the process for single patient expanded access requests.

To make the expanded access process more efficient, we’ve just introduced a much simpler application form called the Form FDA 3926, which will be the form doctors now will typically fill out when they want to provide an investigational drug for a patient through expanded access. While the Form 1571 had 26 information fields and seven attachments, the new Form 3926 has fewer fields (11) and only one attachment. With this streamlined format, we estimate that physicians will be able to complete the form in just 45 minutes, as compared to the more difficult and time consuming effort required previously.

Also, as part of our commitment to streamlining the expanded access process, on May 16, 2016, the FDA and the Reagan-Udall Foundation held a meeting with interested stakeholders to explore additional options that might help patients and their physicians understand the process to request access to unapproved drugs. A common theme of the meeting was that navigating the expanded access process really does take a village. The physician, the drug company, FDA, and the institutional review board (IRB) all have important roles and must work together for the expanded access process to succeed.

The FDA and Reagan-Udall Foundation convened this forum to listen to the public express their needs about expanded access and to discuss ideas with stakeholders on ways that the complex process can be made more efficient and effective. Much work on the details remains, but in general there was agreement on the need for a central repository or clearinghouse where useful and relevant information could be stored in one place — a sort of “one-stop-shop” for physicians and patients to seek information about the expanded access process. As our thinking about this resource develops, we’ll keep the public informed.

For physicians seeking more information about expanded access to an investigational drug, we have developed an educational webinar to help them become familiar with the new application form. This live webinar will occur on July 12 at 1:00 PM EDT and will offer one hour of Continuing Education (CE) credit. The webinar will be recorded for viewing without CE credit. We also have released a guidance regarding Form FDA 3926, a guidance with Questions and Answers on expanded access, as well as a guidance directed at industry addressing questions regarding charging for investigational drugs.

Expanded access is designed for seriously ill patients who have exhausted other options.  The last thing a patient suffering from a serious or life-threatening condition needs is red tape. For many years, FDA has dedicated staff to assist physicians and patients in navigating our system. We expect these important steps will help us continue our efforts to serve patients in need and to advance public health.

Richard A. Moscicki, M.D., is FDA’s Deputy Center Director for Science Operations, Center for Drug Evaluation and Research

Be A Champion for Clinical Trial Diversity

By: Jonca Bull, M.D.

The FDA is launching a campaign to encourage minorities to participate in clinical trials for all medical conditions.

Jonca Bull, M.D., is Director of FDA’s Office of Minority HealthThe first part of the campaign will be launched on June 19, 2016, World Sickle Cell Day, observed annually to help increase public knowledge and raise awareness of Sickle Cell Disease, which primarily affects people of African and Hispanic descent. We want to encourage diverse communities to learn more about how they can become a part of the research process to bring new therapies to the market.

Clinical trials are a critical step in making new medical products available. Medical products—from vaccines to drugs for blood pressure or diabetes management — are tested in clinical trials.

Although FDA generally does not conduct clinical trials, we do the critical work in reviewing the data to assess the safety and efficacy of medical products before they can be used in medical practice. None of this is possible without clinical trials and the patients who go the extra mile by being research participants.

In order to help ensure that medical products are safe for everyone, we need a diverse pool of research participants—racial and ethnic minorities, women, even the elderly.

We know that certain diseases impact some populations differently. For example, diabetes occurs  more frequently in blacks and Hispanics, high blood pressure and heart failure occurs more frequently and severely in blacks; and, Asian American communities experience more hepatitis B.

Clinical trials participants need to more closely mirror the patients who will ultimately use the medicine. This is especially important when considering health disparities — diseases that occur more frequently or appear differently in non-white populations. But most clinical trials participants are white and male. That means we may miss vital data that could be used to be make better evidence-based, regulatory decisions. If we do not develop a more diverse pool of research participants, health disparities may persist because we will not know if a medical product is safe and effective in the actual population that will ultimately use it.

And that’s why we’re launching our campaign, which includes a series of educational aids such as videos, a blog, and an infographic. In these videos Shirley Miller, who lives with sickle cell disease, talks about her experience participating in clinical trials and encourages her peers to learn more about research studies.

In another video Dr. Luciana Borio, FDA’s Acting Chief Scientist, discusses why clinical trial diversity matters from FDA’s perspective.

This campaign is taking us one step closer to a world where health equity is a reality for all. It supports FDA’s initiative: “The Year of Clinical Trial Diversity.”

It is a part of our larger effort to improve clinical trials diversity — we also work with stakeholder groups, support research, develop multi-lingual resources, and use social media to promote a community of “Clinical Trials Champions.”

You can be a “Champion” by watching and sharing the videos and related resources.

Everyone has a stake in the game —health care providers, researchers, and patients. Share these videos and other materials. Start a conversation today.

Videos:

More information about this campaign and FDA’s OMH can be found here: www.fda.gov/minorityhealth

Follow us on Twitter @FDAOMH

Dr. Jonca Bull is FDA’s Assistant Commissioner for Minority Health, Office of Minority Health