China Joins ICH in Pursuit of Global Harmonization of Drug Development Standards

By: Theresa M. Mullin, Ph.D.

Increasingly, drug development is a global endeavor. It requires international collaboration to ensure that consistent standards are adopted and adhered to by all drug makers and regulatory authorities, regardless of country of origin or destination.

Group photo from trip to China

FDA’s Theresa Mullin (center) and the FDA delegation engaging in a discussion on ICH and generic drugs with the faculty and students at Yeehong Business School/Shenyang Pharmaceutical University in Beijing China.

I am pleased to have been in China recently, when the China Food and Drug Administration (CFDA) submitted its membership application to the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (also known as the International Council for Harmonisation or ICH). As part of a team of FDA officials whom CFDA invited to share regulatory advancements, we discussed how modernizing regulatory review systems can promote public health.

ICH, first created in 1990 by regulatory agencies and industry associations from the United States, Europe, and Japan, was established to facilitate international collaboration, and has been successful in standardizing and elevating drug development practices throughout the world. ICH’s mission helps to increase patient access to safe, effective, and high quality pharmaceuticals, and to ensure that pharmaceuticals are developed and registered efficiently.

As globalization has evolved, the ICH has kept pace and membership criteria are robust. Among those criteria new members must implement a basic set of regulatory requirements for the manufacture of pharmaceuticals, for the conduct of clinical trials, and for stability testing of pharmaceutical products. In fulfilling these and other criteria, CFDA joins the existing ICH Assembly, which includes eight regulatory authorities and six industry associations from across the globe.

Janet Mullin and CFDA Official

CFDA Minister Bi presents FDA’s Theresa Mullin a congratulatory letter for FDA Commissioner, Scott Gottlieb, personally handwritten with Chinese Calligraphy.

During our trip, we met with the head of CFDA, Minister Bi Jingquan, and some members of his senior leadership team. Our discussions revealed that CFDA faces many of the same challenges that our other ICH partners and we do. China needs to ensure that patients have access to innovative products, and that pharmaceuticals are safe and are held to a consistent quality standard. Minister Bi emphasized that CFDA has implemented reforms to align China’s regulations with global standards to address these public health concerns. CFDA has also reformed its drug review system, dedicated additional resources to carry out its important mission, and implemented ICH Guidelines.

International harmonization of regulatory standards means that pharmaceutical manufacturers and developers will be held to the same standards in different markets, which will make the development and delivery of quality pharmaceuticals to the public more timely and efficient. CFDA’s membership in ICH marks a significant milestone in expanding ICH’s impact and promoting global public health.

Officials from CFDA will visit FDA later this year to continue our conversation on regulatory modernization. FDA congratulates CFDA on their progress towards regulatory modernization and membership in ICH. Additionally, FDA welcomes their future contribution to global regulatory harmonization.

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

New System Speeds FDA Import Decisions

By: Douglas Stearn

The data is in. A new automated system for determining whether FDA-regulated products can enter the United States is allowing us to make decisions faster and more efficiently.

Douglas StearnQuick admissibility decisions are critical to commerce, especially when perishable products are involved. The new system brings better response times while still protecting consumers.

FDA has used an automated system to assist in making decisions about the admissibility of FDA-regulated products since the early 1990s. In 2015, FDA began piloting a new system, the Automated Commercial Environment (ACE). It features modernized infrastructure that can more quickly process larger amounts of data.

Part of the pilot involved the collection of additional shipment information, such as intended use codes, that could assist in automatically making informed admissibility decisions. Results were promising. So in July 2016, the use of ACE and the provision of additional data were required for everyone seeking to import FDA-regulated goods into the U.S.

Products offered for import into the U.S. must comply with the same standards as domestic products. ACE is one of many tools FDA uses to determine the admissibility of imports – tools that also include inspections of manufacturing plants abroad, physical inspection of goods arriving at our ports, and import alerts which flag manufacturers or products which have had previous violations. By better automating the admissibility process with respect to lower-risk products, FDA can focus more resources on higher-risk products.

Improvements under ACE

ACE, coupled with other enhancements to FDA systems, has brought benefits to both government and the import community. Among the benefits:

  • Due to a number of changes in FDA systems, including ACE, automated messages that an import “may proceed” into U.S. commerce without manual review by an FDA employee have increased dramatically since ACE was piloted, from 26 percent of lines to 62 percent. (A line is a single type of product in a shipment. A shipment might include one or more types of products).
  • FDA employees have less need to request additional information from the importers of record for additional documents or information. That means fewer delays in FDA admissibility decisions about shipments. Under ACE, 28,374 fewer lines needed additional documents and information than prior to ACE.
  • Although we don’t have an exact count of the average processing time before ACE went into effect, we know that times have improved. Indeed, today, automated “may proceeds” are being processed on average within 1 minute and 36 seconds. 
  • We’re also seeing improvements for products that require manual processing. Products are receiving “may proceed” within a median of one hour if no additional documentation is required. When additional information or documents are needed, decisions are processed within 72 hours, compared to 96 hours under the previous system. 

The import community, which has cooperated in submitting the data needed to optimize ACE, shares the credit for these improvements. Further cooperation, particularly by addressing common errors, will bring even greater benefits.

More Improvements through Compliance – Errors to Avoid

A study of FDA rejections between November 2016 and March 2017 found that the most common problems involved the submission of invalid or canceled food facility registration numbers and invalid FDA product codes, which identify the items in a shipment. When offering an FDA-regulated product for import, those filing the paperwork need to remember to:

  • Submit for all FDA-regulated products the correct company name and address of the importer, the manufacturer, delivered-to party, and shipper. Providing the unique number assigned to the company by Dun & Bradstreet (DUNS) or the FDA Establishment Identifier (FEI) provides additional firm-specific information, which assists in finding the companies in FDA’s database.
  • Submit “Intended use” information for FDA-regulated products.

U.S. Customs and Border Protection (CBP), which makes initial decisions before referring shipments to FDA, cites the three most common errors that lead to rejection of entries prior to their being transmitted to FDA:

  • Missing or invalid entity information, which identifies companies involved in the manufacture and importation of the product; and,
  • Missing or invalid units of measure.

The FDA ACE Error Guide details the messages FDA sends when the agency receives entries with problems that prevent further processing of entries.

FDA is standing by to help. An ACE support center is staffed from 6 a.m. to 10 p.m. EST. Contact the center by e-mail at ACE_Support@fda.hhs.gov or toll free from the U.S. at 877-345-1101. Local and international callers should dial 571-620-7320.

Upon request, FDA will assist those filing an import entry of a particular commodity for the first time. Make the request by emailing the support center.

The Division of Import Operations (DIO) also can be contacted for general import operations and policy questions, including questions surrounding the appropriate FDA product code or for more information about an entry declaration requirement. Email FDAImportsInquiry@fda.hhs.gov or call 301-796-0356.

Thank you for helping us make import operations efficient and effective as we continue to focus on our mission of protecting public health.

Douglas Stearn is FDA’s Program Director, Office of Enforcement and Import Operations, in the Office of Regulatory Affairs

FDA Announces New Steps to Empower Consumers and Advance Digital Healthcare

By: Scott Gottlieb, M.D.

When people think about personalized medicine, they often think of genetic testing and sequencing of the human genome. But the concept of personalized medicine is much broader. It includes the re-imagination of healthcare delivery. It includes empowering consumers to take more control of their own healthcare information to make better informed decisions about their medical care and healthy living.

Dr. Scott GottliebThis opportunity is enabled by a new technological paradigm of digital health tools, like apps, that enable consumers to have more active engagement and access to real-time information about their health and their activities. These tools allow consumers and providers to supersede the traditional, physical constraints of healthcare delivery and exploit the opportunities offered by mobile technology.

Historically, healthcare has been slow to implement disruptive technology tools that have transformed other areas of commerce and daily life. One factor that’s been cited, among many, is the regulation that accompanies medical products. But momentum toward a digital future in healthcare is advancing. Not all of these tools are subject to FDA regulation. For the devices we are asked to evaluate, we know that our policies must continue to empower consumers and facilitate innovation.

Digital Health Pre-Cert imageToday, I’m excited to announce, as part of our broader Medical Innovation Access Plan, a new component focused on digital health innovation—the formal launch of our Pre-Cert for Software Pilot Program. This new program embraces the principle that digital health technologies can have significant benefits to patients’ lives and to our healthcare system by facilitating prevention, treatment, and diagnosis; and by helping consumers manage chronic conditions outside of traditional healthcare settings.

At the same time we are announcing this pilot, FDA’s Center for Devices and Radiological Health (CDRH) is publishing its Digital Health Innovation Action Plan to provide details and timelines for our integrated approach to digital health technology and the implementation of the 21st Century Cures Act. We’re telling consumers and the digital health industry how we will establish clear and consistent expectations for the products FDA regulates.

The challenge FDA faced in the past is determining how to best regulate these non-traditional medical tools with the traditional approach to medical product review. We envision and seek to develop through the Pre-Cert for Software Pilot a new and pragmatic approach to digital health technology. Our method must recognize the unique characteristics of digital health products and the marketplace for these tools, so we can continue to promote innovation of high-quality, safe, and effective digital health devices.

FDA’s traditional approach to medical devices is not well suited to these products. We need to make sure our approach to innovative products with continual updates and upgrades is efficient and that it fosters, not impedes, innovation. Recognizing this, and understanding that the potential of digital health is nothing short of revolutionary, we must work toward establishing an appropriate approach that’s closely tailored to this new category of products. We need a regulatory framework that accommodates the distinctive nature of digital health technology, its clinical promise, the unique user interface, and industry’s compressed commercial cycle of new product introductions.

Our new, voluntary pilot program will enable us to develop a tailored approach toward this technology by looking first at the software developer or digital health technology developer, rather than primarily at the product (as we currently do for traditional medical products). This pilot will help us establish the most appropriate criteria for standing up a firm-based pre-certification program for these new tools.

The goal of our new approach is for FDA to, after reviewing systems for software design, validation and maintenance, determine whether the company meets the necessary quality standards and pre-certify the company. Pre-certified companies could submit less information to us than is currently required before marketing a new digital health tool. In some cases, pre-certified companies could not submit a premarket submission at all. In those cases, the pre-certified company could launch a new product and immediately begin post-market data collection. Pre-certified digital health companies could take advantage of this approach for certain lower-risk devices by demonstrating that the underlying software and internal processes are sufficiently reliable. The post-market data could help FDA assure that the new product remains safe and effective as well as supports new uses.

FDA designed the new digital health pilot program to include up to nine software firms of various sizes. Initial participants in this new pilot will range from small startups to large companies that develop both high- and low-risk software products that are devices. We want to include medical product manufacturers as well as non-traditional software developers. Given the amount of attention we’re getting, and the ongoing innovation in this space, I’m confident we’ll have strong participation in the new pilot.

Digital health product developers will be selected for the program based on the following:

  • The company must be in the process of developing or planning to develop a software product that meets the definition of a medical device;
  • The company must have an existing track record in developing, testing, and maintaining software products and demonstrating a culture of quality and organizational excellence measures that are tracked by Key Performance Indicators (KPI) or other similar measures;
  • And during participation of the pilot, companies must agree to:
    • Provide access to measures for developing, testing and maintaining software products and demonstrating a culture of quality and organizational excellence measures by KPI;
    • Collect real-world post-market data and provide it to FDA;
    • Meet with FDA for real-time consultation;
    • Be available for site visits from FDA officials; and,
    • Provide information about the firm’s quality management system.

We have intentionally left the initial criteria broad because this pilot is purposely designed to be inclusive and flexible. We want to be able to accommodate a broad range of participants and technologies. We appreciate that the experience and capabilities of a small company will be different from that of a large company and recognize that we need a pre-certification program that accommodates both.

The initiative will begin immediately. Starting on August 1, companies can submit a statement of interest that includes the qualities listed above, requesting participation in the pilot to FDAPre-CertPilot@fda.hhs.gov. Then, during the month of August, FDA’s Digital Health Team will evaluate submissions and select companies that reflect the broad range of software developers. A critical component is that we will include small and large companies, traditional and non-traditional medtech companies, and products that range in risk. This approach will create opportunities for more dynamic entrepreneurship and competition and help continue to drive product innovation.

We expect that the first four months of the pilot will better inform our regulatory team as well as product developers. FDA will hold a public workshop in January 2018 to report on and review our initial findings. The goal is to inform product developers who are not participating in the pilot, so they can understand our process and findings, to help better inform development programs underway outside of the pilot.

As we launch our Digital Health Innovation Action Plan, I am conscious of the fact that apps and app updates come to market every day. But the most powerful feature of this market may not be one revolutionary app but rather a combination of apps that provides consumers and providers with the information they need. This can help people better manage their chronic diseases, which could result in less trips to the doctor for checkups, or better awareness of illness, like prompts to a parent with a sick child on when they need to see a provider. I’m delighted to be helping lead FDA at a critical moment, when I have the opportunity to help advance innovation in digital health and to make sure patients can benefit from these remarkable technologies.

Scott Gottlieb, M.D., is Commissioner of the U.S. Food and Drug Administration

Follow Commissioner Gottlieb on Twitter @SGottliebFDA

Follow and report progress about the program on social media with the hashtag  #FDAPrecert

Patient Reps – Bringing the Voice of Patients to FDA

By: Jack Kalavritinos

At FDA we never lose sight of the fact that the work we do in evaluating and approving new medical products is done to benefit patients.

Increasingly, that means taking into account the views and expertise of patients and their caregivers, because they provide a unique voice and perspective and know best what they are living with on a day-to-day basis. Earlier this month, for instance, we announced the creation of the first advisory committee made up solely of patients and caregivers, who will provide advice on complex issues related to medical devices.

Another way we incorporate the patient viewpoint is through FDA’s Patient Representative Program. This program brings patients – and their caregivers – and the extraordinary breadth of knowledge and personal experience in more than 300 diseases and conditions they possess, directly into the regulatory medical product development and review process. They serve on 47 FDA Advisory Committees and panels to advise on drugs, devices and biologics currently being considered for approval or clearance. They also serve as a consultant for the review divisions (doctors and scientists who review data to determine whether the medical product’s benefits outweigh the potential risks), and as presenters at FDA meetings and workshops on disease-specific or regulatory and health policy issues.

Jack Kalavritinos , FDA’s Associate Commissioner for External Affairs

Members of FDA’s Patient Representative Program together with Jack Kalavritinos, Associate Commissioner for External Affairs, during a recent training session in suburban Washington, D.C.

Every year our team at FDA’s Office of Health and Constituent Affairs brings new FDA patient representatives to the Washington, D.C. area, for training and orientation. They receive briefings on everything from medical product review policies and clinical trials to the life cycles of drugs, biologics, and devices, and even a brief primer on statistical analysis.

Without a doubt, the most moving moments of the recently completed two-day workshop  were when the patient representatives told their own stories, how they are making a difference in so many lives as advocates for their own patient communities, and explaining why they had decided to make the commitment to become patient reps. One was more compelling than the next. Here are some of their stories:

  • A woman with fibromyalgia who said she joined the program “to give a voice to those who suffer with chronic, life-altering conditions.”  She believes her training in qualitative research, combined with her personal experience with her disease, will help her listen to and understand patient experiences and to “sort out the ‘noise.’”
  •  A caregiver whose husband survived for three years after being diagnosed with glioblastoma multiforme, a form of brain cancer. During that period, he received four surgeries and took 15 different chemotherapy drugs. She became a patient advocate as a result of her experience so she could be involved in the process of finding new treatments for “this daunting disease.” She also works with other caregivers to help them cope with the diagnosis. As she explained, “Being able to talk to someone who has experienced this same disease helps to reduce their level of anxiety and some of the unknowns that accompany this diagnosis.”
  • A patient with schizoaffective disorder who talked about how her disease first appeared when she was in college, when the voices she would hear interfered with her ability to learn and function. But with proper treatment, and incredible discipline and support, she was able to learn to control her disease and not let it take over her life. Today, she is a mental health therapist who works to combat stereotypes that prevent psychiatric patients from getting the help they need, when they need it. Her goal has been “to put a face to those of us who struggle with psychosis, but yet are seen as being ‘functionally well.’”
  • A father and uncle to two women with Friedreich’s ataxia, a life-shortening genetic mitochondrial nerve disorder that has no treatment. He has watched as his daughter, now 31, has become a quadriplegic. As he said with incredible honesty and pain, “she will die soon.” But he also has turned his pain into action. In addition to assisting his daughter and niece, he also has lent his energy to helping others with the disease and to generating attention and resources for finding a treatment. He became a patient representative, in part, he explained, so that he “could be at FDA on the day a treatment needs his yes or no vote.”

Space prevents including every one of their stories, but each of these remarkable individuals offered a compelling history of courage. All are committed to fighting the disease that had so directly affected them, whether as a patient or a caregiver. But, in a comment that could be applied to all of them, one woman noted, she “works hard to not let my identity be defined by my illness.” They do this, remarkably, by turning their focus outward, rather than inward, and using their strength and expertise to the benefit of others.

This understated, but courageous, spirit is echoed in one way or another by each of FDA’s patient representatives. We want to thank each of these individuals for their inspiring commitment — to the FDA, to better health, and for their role in these critical public health efforts.

Jack Kalavritinos is FDA’s Associate Commissioner for External Affairs

Dietary supplement concerns? Tell the FTC and FDA

By: Mary Engle, FTC, and Steven Tave, FDA

Ever bought a dietary supplement or other health-related product that didn’t work as promised? Maybe you had side effects, or the claims just seemed unbelievable. Know this: the government holds companies accountable for making baseless claims about products marketed as dietary supplements.

Mary Engle

Mary Engle is FTC’s Associate Director, Division of Advertising Practices

The Federal Trade Commission (FTC) and FDA have enforcement programs to protect consumers from false and misleading claims about the safety and benefits of products marketed as dietary supplements. Both agencies have authority over the marketing of these products.

You can help. Tell the FTC or FDA if:

  • You bought a dietary supplement that didn’t work as advertised – or you had an adverse reaction or illness.
  • You’re suspicious that a company is making false or overstated claims in its labeling or marketing. (Watch for claims about so-called “treatments” or “cures” for diseases like Alzheimer’s, cancer, heart disease, arthritis, and others. Dietary supplements cannot lawfully claim to diagnose, mitigate, treat, or prevent a disease.)
  • You’re concerned about the content, purity, or safety of the product.
Steve Tave

Steven Tave is FDA’s Director, Office of Dietary Supplement Programs

So who should you contact about what issue with products marketed as dietary supplements?

Labeling Claims, Content, Purity, Safety: FDA looks at whether claims on dietary supplement product labels and other packaging materials are true and accurate. They also oversee manufacturing, content, purity, and safety – including tracking any adverse reactions.

  • Concerned about a statement made on a product label or other packaging, or about the content or purity of the product? Report it to FDA.
  • If you or your doctor think you’ve had an adverse reaction to a product marketed as a dietary supplement, report it – or other safety concerns – to FDA’s Safety Reporting Portal.

Advertising Claims: The FTC looks at the truth and accuracy of any claims made in dietary supplement advertising and marketing. That means the FTC watches claims made on TV, radio, and in print ads, as well as in social media and online marketing. (More about online marketing below)

  • Think a claim seems false, unsupported, or simply unbelievable? Does it promise to treat or cure a disease? Tell the FTC. 

dietary supplements, bottles imageWebsites and Internet Marketing: The FTC and FDA work together to monitor claims made on websites or in other online marketing.

  • Dubious about claims made online about a dietary supplement? Report it to either the FTC or FDA. Then we’ll work together to figure out which agency will take the lead.

If you’re still not sure who to report to, just report it to one of us and we’ll sort it out – the important thing is that you report!

Mary Engle is FTC’s Associate Director, Division of Advertising Practices

Steven Tave is FDA’s Director, Office of Dietary Supplement Programs

FDA Sets Inaugural Meeting of First-Ever Patient Engagement Advisory Committee

By: Kathryn O’Callaghan and Jeffrey Shuren, M.D., J.D.

Imagine checking your blood sugar levels several times a day with a glucose meter to keep your diabetes under control. Or maybe you’ve had a hip joint replaced or a stent inserted in your coronary artery to treat a heart blockage. Maybe you participated in a clinical trial to help researchers or a manufacturer better understand a new device to treat a condition.

Kathryn O'Callaghan

Kathryn O’Callaghan, Assistant Director for Strategic Programs, FDA’s Center for Devices and Radiological Health

It’s possible that you had some unexpected experiences with the device or clinical trial and you’d like to share what happened with your physician, the manufacturer, other patients or even FDA.

Now, there’s a way. For the first time, an FDA advisory committee will focus on patient-related issues. On Oct. 11-12, 2017, FDA will hold the inaugural meeting of the new Patient Engagement Advisory Committee (PEAC). The topic will be the challenges of clinical trial design, conduct, and reporting identified by patients.

FDA chose this subject because patients often have concerns about participating in clinical trials or drop out once they have enrolled in a trial. Inconsistent or minimal participation in clinical trials can make it difficult to reach reliable conclusions or to determine the level of benefit for patients. It also can take longer to bring technological advances to the patients who need them.

We also are excited to announce that the nine core voting members of our patient advisory committee, including the chair and the consumer representative, all have direct experience as a patient or as a care-partner for a patient.

Jeffrey Shuren

Jeffrey Shuren, M.D., J.D., Director of FDA’s Center for Devices and Radiological Health

They are experts in the field of patient engagement, and their experience extends beyond their personal disease or condition to the broader patient perspective, which is a critical piece of FDA’s work. While patient representatives currently participate in many FDA advisory committee meetings, we’ve never had a committee that was wholly focused on patients.

The PEAC is a forum for the voice of patients. It will be asked to advise on complex issues related to medical devices and their impact on patients. The goal of PEAC is to better understand and integrate patient perspectives into our oversight, to improve communications with patients about benefits, risks, and clinical outcomes related to medical devices, and to identify new approaches, unforeseen risks or barriers, and unintended consequences from the use of medical devices.

Federal legislation enacted in 2012 instructed FDA to solicit the views of patients during the medical product development process and consider the perspectives of patients during regulatory discussion. This gave us a welcome opportunity to establish the PEAC.

Our intent is to engage with and better understand the needs, views, and concerns of patients and other users of medical devices. After all, they are, along with their caregivers, the experts in terms of what they’re living with and what they have to deal with day-to-day.

We will also need better tools to accurately capture and characterize patient views on acceptable balances of benefits and risks. And, as the nascent science of patient preferences continues to evolve, we must adapt our policies, too.

Patients are at the heart of what we do. It makes sense to establish an advisory committee built just for them.

The nine core members of the PEAC are:

Paul Conway (Chair) – President of the American Association of Kidney Patients.

Katherine Seelman, Ph.D. – (Consumer Representative) – Retired from the University of Pittsburgh as Professor Emerita in 2016.

Cynthia Chauhan, M.S.W. – Served as a Patient Advocate for over 17 years. Served as an FDA patient representative for the National Mammography Quality Assurance Advisory Committee.

Amye Leong, M.B.A. – President and CEO of Health Motivations. Extensive patient advocacy history, communications specialist, health policy advisor, author.

Monica Willis-Parker, M.D. – Director, Minority Engagement Core, Emory Alzheimer’s Disease Research Center.

Frederick Downs, Jr., M.B.A. – Decorated Vietnam veteran, who retired from the Veterans Administration as the Senior Executive Service Chief Procurement and Logistics Officer.

Bennet Dunlap, M.S. – Health communicator and advocate for people with diabetes, with experience in social media and marketing programs.

Deborah Cornwall, M.B.A. – Patient advocacy expert. She is a longtime leadership volunteer for the American Cancer Society and the Cancer Action Network.

Suzanne Schrandt, J.D. – Director of Patient Engagement at the Arthritis Foundation.

Kathryn O’Callaghan is Assistant Director for Strategic Programs, FDA’s Center for Devices and Radiological Health 

Jeffrey Shuren, M.D., J.D., is Director of FDA’s Center for Devices and Radiological Health

The Expanded Access Navigator – Helping Patients In Need of Potentially Life-Saving Drug Treatments

By: Richard A. Moscicki, M.D.

Patients with serious or immediately life-threatening diseases or conditions who have no comparable or satisfactory alternative therapy and who seek access to potentially life-saving investigational drugs will have another option to guide them through the process – thanks to the launch today of a new online tool called the Expanded Access Navigator. The development of the Navigator was a team effort led by the Reagan-Udall Foundation in collaboration with patient advocacy groups, the pharmaceutical industry, FDA, and others in the Federal government.

Dr. Richard MoscickiWhen a patient has a serious or immediately life-threatening disease or condition for which there is no FDA-approved treatment, they may be able to gain access to an investigational drug under the FDA Expanded Access process. Expanded Access permits the product’s manufacturer, with the authorization of FDA, to provide an investigational drug for a patient, even though the drug is still in development or under review for FDA approval. Each year FDA receives more than 1,000 applications requesting authorization of expanded access through individual patient, intermediate or large-size expanded access programs. FDA has worked to streamline the process, particularly with a simplified application for individual patient expanded access; however, it can still be challenging and time-consuming for patients, family members, caregivers, and health care professionals to navigate the system.

The Expanded Access Navigator helps to educate patients and physicians about the process. Most of what they need to seek expanded access is now available in this single online location, including a directory where companies can submit public links to their expanded access policies, criteria used by companies to determine whether to make a drug available through expanded access, and contact information. While not a portal for applications, the directory is the first consolidated starting point for researching available investigational therapies. To help users find information quickly, the Navigator is separated into one section for patients and caregivers, and another for physicians.

  • The patient and caregiver section provides links to resources such as how to determine if a patient can participate in a clinical trial, the difference between an intermediate-size or larger expanded access program and a single-patient expanded access program, and their physician’s role in helping them obtain an investigational drug. Patients also can reach out to FDA’s Office of Health and Constituent Affairs’ Expanded Access Team.
  • Physicians can use the tool to identify investigational treatment options for their patients, explore clinical trials on behalf of their patients, learn how to engage with FDA and pharmaceutical companies as part of the process, and read about important factors to discuss with patients when considering expanded access. After using the Navigator and deciding on an investigational drug treatment option, physicians may contact FDA’s Division of Drug Information for assistance with their expanded access application.

photo of patient and doctorThe site is a valuable resource for information about obtaining access to an investigational drug through an expanded access program, but it also offers a great deal of useful information about participation in clinical trials, which is the preferred option. Clinical trials help to ensure adequate patient protection and may provide the evidence of safety and effectiveness required to support approval of a marketing application.

The Partners who contributed to the website’s development, in addition to FDA, include the American Cancer Society, the American Society of Clinical Oncology, the Biotechnology Innovation Organization, the Pharmaceutical Research and Manufacturers of America, Foundation Medicine, Susan G. Komen, Bristol-Myers Squibb, Genentech, Janssen, Lilly, Merck, and Pfizer.

Using an investigational drug will always come with risk – and the expanded access process helps to assess whether the potential risks are appropriate for a patient. But the last thing a patient and their loved ones coping with serious or life-threating illness needs is an overly burdensome process for access to potential treatment options. The Navigator helps to reduce that burden.

We are pleased to have worked collaboratively with others to witness the launch of the Expanded Access Navigator, and are thankful for everyone’s hard work and dedication. We hope the tool will be a help to many and improve the experience for patients, their caregivers, and physicians who are seeking expanded access.

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

Keeping the U.S. Prescription Drug Supply Chain Among the Safest in the World

By: Ilisa Bernstein, Pharm.D., J.D.

The U.S. prescription drug supply is among the safest in the world, but it can be challenging to keep it that way. Criminals – both here and abroad – constantly threaten to replace safe, effective, and high-quality prescription medications with counterfeit, stolen, and otherwise substandard products.

Ilisa BernsteinRoughly 4 billion prescriptions were filled at U.S. retail pharmacies last year. That’s a lot of prescription drugs moving through the U.S. supply chain that patients are relying on. In today’s global pharmaceutical environment, in which a large percentage of FDA-approved prescription drug products are made outside of the United States, we are continuously looking for ways to keep the drug supply secure.

Substandard and falsified drugs are global problems that need global solutions and global collaboration. We cannot solve these challenges alone and we at FDA are continually looking for ways to collaborate and learn from our regulatory counterparts around the world.

I’m pleased to share an important new advancement to help protect the U.S. and global supply chain for prescription drugs and other medical products. Over the past four years, FDA led a team of international partners to create the Supply Chain Security Toolkit for Medical Products. Our collaborators included regulators from the 21-nation Asia-Pacific Economic Cooperation (APEC), non-APEC countries, industry stakeholders, representatives from non-governmental organizations, international organizations, and academia.

The goal of this collaboration was to develop strategies to better secure the medical product supply chain across APEC economies and around the world. We also aimed to enhance APEC members’ regulatory standards to secure national and global supply chains, and develop tools that regulators and industry can use for training and for implementing best practices.

The Toolkit is a comprehensive resource that covers the entire supply chain and lifecycle of medical products – from raw materials to patient use. It focuses on developing and implementing processes and procedures designed to enhance global medical product quality and supply chain security.

The Toolkit website provides detailed information and resources related to track and trace, internet sales, detection technology, and much more. The toolkit can be used by industry stakeholders and regulators from around the globe to adopt best practices and to strengthen laws and regulations to protect consumers from unsafe and substandard drug products. It also is a valuable training tool for regulators grappling with the complexities of keeping the supply chain safe.

Training will be coordinated by the United States Pharmacopeia and the University of Tennessee Health Sciences Center, which are APEC Centers of Excellence.

The tools will help regulators worldwide to PREVENT, DETECT, and RESPOND to medical products that threaten the health and safety of patients.

We all will benefit from this hard work. Together with our global partners, we will continue to combat supply chain problems as they arise and increase confidence in the legitimacy of the life-saving prescription drugs that patients rely on.

Ilisa Bernstein, Pharm.D., J.D., is Deputy Director of the Office of Compliance in FDA’s Center for Drug Evaluation and Research

Leveraging FDA Resources to Encourage Students to Pursue STEM Careers

By: Richard Pazdur, M.D.

When I was in high school, I spent summers working as a restaurant dishwasher, grocery store stock boy and gardener in northwest Indiana. The idea of spending those weeks learning about science and medicine would not have been an option for me at that time.

Dr. Rick Pazdur and Members of Summer Scholars

Richard Pazdur, M.D., Director of the FDA Oncology Center of Excellence, poses with the first class of the OCE Summer Scholars Program. Sara Horton, M.D., project lead, (far left) and Alice Kacuba, project coordinator, (second from right) joined the group, which includes a variety of backgrounds, including two childhood cancer survivors interested in biomedical careers.

Yet, it is precisely those students who may not have access to specialized learning opportunities that we need to attract to science, technology and medicine to continue progress in these fields and ensure the diversity of our scientific workforce.

In particular, oncology and hematology are falling behind other areas of medicine in the adequate representation of racial and ethnic minorities in the physician workforce. Only 2.3% of practicing oncologists self-identified as black or African American, and 5.8% self-identified as Hispanic in a 2016 survey by the American Society of Clinical Oncology (ASCO). According to census figures, 13% of the U.S. population is black or African American, and 18% is Hispanic.

That’s part of the reason why the Oncology Center of Excellence recently launched its pilot Summer Scholars Program, designed to introduce students to oncology drug development and career opportunities in government, regulatory medicine, and cancer advocacy. With the cancer incidence expected to increase 45% by 2030, according to the National Cancer Institute (NCI), we will need the talents of many more tech-savvy students from diverse backgrounds furthering their studies in our medical schools and university science labs.

Dr. Rick Pazdur and two Summer Scholars

Richard Pazdur, M.D., Director of the FDA Oncology Center of Excellence, asks Diamond McCoy, 17, of H.D. Woodson High School in Washington, D.C., and Camden Wiseman, 17, of the Thomas Jefferson High School for Science and Technology, in Alexandra, Va., about their plans for the upcoming academic year. The two were part of OCE’s first Summer Scholars Program, which seeks to encourage tech-savvy students to pursue their studies in medicine and other STEM fields.

We recently welcomed 11 Washington, D.C., area high school students to FDA’s main campus in Silver Spring for six weeks – from June 26 to August 4. The group includes students with a variety of backgrounds and experiences, including some who are part of a STEM – Science, Technology, Engineering, and Math — program at their schools and two who are childhood cancer survivors interested in biomedical careers. Our requirements for the program include that they be in good academic standing and at least 16 years old.

Sara Horton, M.D., a breast cancer clinical reviewer and one of three staff members in the FDA Office of Hematology and Oncology Products (OHOP) who collaborated to develop the Summer Scholars Program, says that partnering with the D.C.-area public schools was the first thing that came to her mind in planning this program.

She told me that we decided to focus on students who may never have had an opportunity like this, as well as childhood cancer survivors. Dr. Horton reminded me that high school is a very special stage of development when students typically start thinking about where they fit in the world, what should they do, and who should they be.

We’re excited about introducing young people with STEM aspirations to professions in science and medicine they may have never known existed.

The curriculum includes basic and translational science, drug manufacturing, clinical trials, regulatory review, patient advocacy, and marketing. Lectures in those areas will be augmented by field trips to the NIH Clinical Center, NCI, Howard University College of Medicine, and ASCO.

In addition, students will be introduced to patient advocacy lobbying with Kids v. Cancer and accompany that group on a trip to Capitol Hill. They also are invited to a workshop at the drug manufacturing company AstraZeneca. Even medical students usually don’t have this type of opportunity to learn about the work we do at FDA until they are out of medical school.

Gregory Reaman, M.D., associate director for oncology sciences in OHOP and one of the program organizers, says the program is as interactive as possible for this age group. Most of the students will not have had much, if any, exposure to the field of oncology, while the cancer survivors will have had the experience of receiving treatment. We hope they will bring their experiences to us so we can all learn to be better advocates for patients.

Dr. Reaman, a pediatric oncologist, worked at a state mental hospital one summer. He says it confirmed his interest in medicine – just not psychiatry! We hope this experience will be as transformative for these students.

The lecture curriculum covers what we are calling the “Basics of Oncology,” including cancer treatments, endpoints for clinical trials, data analysis, statistics, pharmacy, microbiology, genetics, genomics, drug promotion, and patient advocacy.

Students also have the opportunity to work on professional skills such as writing, networking, and communication, and meet regularly with their mentors from FDA staff. At the end of the program, students will give short presentations to the OCE on a topic of interest to them.

Alice Kacuba, R.N., M.S.N., chief of regulatory project management staff in OHOP’s Division of Oncology Products 1 and one of the program organizers, told me that she hopes the agency’s diverse staff will leave a lasting impression on the students. She said she excelled in science, but saw very few female role models in science in the 1970s. Since becoming a nurse, “STEM education has become my passion, as my nieces and nephews can attest,” she said.

The OCE Summer Scholars Program is a pilot this year, but could be expanded next year to high school students nationally. Cooperation with offices within FDA and external organizations has been exceptional. We hope this will be a one-of-a-kind experience for the students as well as our oncology staff here at FDA.

Richard Pazdur, M.D., is the Director of the FDA Oncology Center of Excellence

Building a Strong FDA Workforce to Bring Scientific Advances to Patients

By: Scott Gottlieb, M.D.

The key to FDA’s public health mission, and its ability to bring innovative new therapies to patients, is the technical, scientific, and clinical expertise of its people. As the products that we’re asked to review become more complex and specialized, so do the technical demands on our workforce. Our staff must remain current with the dramatic advances in science and medicine and meet the increasing demands that globalization and other trends place on our core consumer protection functions.

Dr. Scott GottliebAs a result, FDA continually faces the challenges related to building and maintaining a diverse, talented, and dedicated professional workforce. However, we’re committed to doing what’s necessary to tackle these challenges and maintain a strong FDA — one that attracts and preserves world-class talent.

Most recently, I’ve requested a comprehensive effort to evaluate our hiring practices and procedures. We know that our traditional approach to recruiting and hiring is not as efficient as it should be to attract, hire, and retain the types of experts we need now and anticipate to need over the longer term. What’s more, we’re increasingly competing with better-resourced entities in the private sector for the same limited pool of people with very specific clinical and scientific skills and training. These are challenges that our current approach to hiring did not anticipate. It’s critical that we modernize the process for recruiting personnel into these specialized positions within our Agency’s programs.

As part of a new effort, and consistent with Secretary Price’s Reimagine HHS initiative, we’ll be piloting new hiring procedures aimed at better supporting the hiring goals required to meet FDA’s evolving needs. I’m very pleased that Melanie Keller, currently head of the Office of Management in our Center for Drug Evaluation and Research, has agreed to lead this effort on a full-time basis. She’ll be running the pilot from a newly created position inside the Office of Medical Products and Tobacco.

A central part of this new effort will involve more directly aligning the administrative hiring procedures and the scientific staffing objectives of our programs. Thus, the directors of the medical product centers participating in the pilot will be closely involved in overseeing the new initiative. They’ll help ensure that the scientific objectives of our review programs are more closely reflected in the recruitment and hiring process. We want to make sure that FDA’s existing experts are more personally involved in hiring our new experts. Although we face similar challenges across many of our programs, the pilot will initially focus on PDUFA- related positions in our drug and biologics programs while we develop our new model.

To take on this new effort, we’re establishing a dedicated group of full-time staff with the responsibility to ensure that we reliably and predictably identify, recruit, and efficiently hire the scientific personnel the Agency needs. Professional staff from our centers with experience recruiting specialized scientific and medical staffing will be key members of this new pilot effort. Staff from the Office of Operations will assist with the identification of potential candidates from key scientific disciplines.

The first order of business will be to address hiring into the positions supported by our PDUFA commitments. Too many of these positions remain vacant, and the backlog is substantial. Finding the right people and bringing them on staff quickly has proved difficult. Our goals will be to speed the hiring process while improving the retention of scientific and technical experts. We’ll aim to reduce and eventually eliminate the backlog of vacant positions while demonstrating the utility of our new hiring model. I encourage scientific professionals and technical experts who wish to join an outstanding workforce serving the public health to review the available job opportunities at FDA.gov.

I’m heartened by the progress FDA’s reauthorization legislation is making through Congress, and I look forward to its final passage. In the meantime, the new efforts I’ve outlined here will provide a solid foundation for recruitment and for responsibly managing our user fee resources. The reauthorization, coupled with key provisions in the 21st Century Cures Act— which give FDA the authority to bring on top candidates at competitive salaries — will greatly assist us as we modernize our recruitment policies, systems, and procedures. All of these efforts will strengthen FDA’s core functions, enabling us to ensure that safe and effective advances can reach the patients who need them as efficiently as possible.

Scott Gottlieb, M.D., is Commissioner of the U.S. Food and Drug Administration

Follow Commissioner Gottlieb on Twitter @SGottliebFDA