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

In cancer treatment, there’s more than one way to measure patient benefit

By: Richard Pazdur, M.D.

We all want a cure for cancer. But the reality is that advances in cancer treatment rarely come in one big discovery, but rather with the continued step-by-step progress in developing new therapies. Currently, a few cancers—such as childhood leukemia and testicular cancer—can be cured. There are many ways of evaluating cancer therapies, including an improvement in overall survival, stabilizing the disease, and reducing tumor burden and tumor-related symptoms. Over the years, we have discussed with many patients facing serious and life-threatening diseases how to evaluate cancer treatments. The patients have told us there is a need for flexibility in our evaluation process.

Dr. Richard PazdurBefore a new drug is approved, FDA evaluates clinical trials in which the drug is tested in patients. We hope that the study will show a result, or endpoint, that helps us understand if the drug is safe and effective. When evaluating drugs that treat life-threatening illnesses like cancer, the risk-benefit analysis may involve weighing relatively higher risks against relatively smaller benefits.

The gold standard for determining benefit from a new cancer drug traditionally has been a randomized controlled study that demonstrates an improvement in overall survival, or OS. This is a measure of how much longer patients live who take the drug compared with patients who take another drug. An overall survival endpoint clearly demonstrates the drug’s value in extending a patient’s life.

But achieving an improvement in overall survival may not always be possible. Some cancers grow very slowly, so it might take many years for a trial to evaluate whether a potential new drug helps people live longer. Many oncology drugs target specific mutations in the tumor and there may be a limited number of patients with the mutation. Because of the small number of patients, randomized studies evaluating OS may not be possible. Also, many patients in the trial may be taking additional therapies at the time their disease progresses. This may make it difficult to accurately assess the new drug’s effect on overall survival.

When emerging data shows that a new drug demonstrates substantial benefit compared to available drugs, it may not be possible to conduct a randomized trial with certain endpoints comparing the new drug to a standard therapy with modest benefit. This is known as loss of equipoise.

Endpoints other than overall survival can shorten the duration of clinical trials so that drugs can be available sooner to patients. These alternative endpoints include progression-free survival—a measurement of how long a drug may have prevented the cancer from getting worse—and overall response rate—an evaluation of the portion of patients in the trial whose tumor size was reduced by a treatment.

Thousands of patients who previously had few therapeutic options available have already benefited from cancer therapies that were approved based on alternative endpoints, including those with renal cell carcinoma, Merkel cell carcinoma, medullary thyroid cancer, gastrointestinal stromal tumor, metastatic basal cell carcinoma, pancreatic neuroendocrine tumor, multiple myeloma, chronic myelogenous leukemia, chronic lymphocytic leukemia, and certain types of lung cancer.

We’ve held many advisory committee meetings and have heard directly from patients who believe that delaying the growth of cancer or reducing the cancer’s size are of benefit to them, since these endpoints may relate to reduced symptoms and the ability to carry on with many daily activities.

Patients have benefited, too, from the Breakthrough Therapy Designation, which was established in the FDA Safety & Innovation Act of 2012 to expedite the development and review of transformative therapies that show great promise in early clinical trials, compared to available therapy. Drugs that are designated as breakthrough therapies receive more intensive FDA consultation throughout their development period and may also qualify for other expedited development programs such as fast track and priority review. Many oncology drugs have breakthrough therapy designations, and this designation enables FDA to expedite the review of therapies that may meet patient’s needs.

There is still much more to learn about what patients need and expect from their cancer drugs. Our patient-focused drug development program has sponsored daylong meetings with patients and caregivers to discuss their views on a specific disease. A patient at our breast cancer meeting said: “As long as I can live my life and continue to work full-time, that is my goal.”

Based on these and other patient interactions, we are actively investigating ways to incorporate the patient’s experience and quality of life in benefit-risk assessments of new cancer treatments.

It’s also important to recognize that the drug approval process does not end with the drug’s approval. This is just the beginning. By looking at real-world use of the drug in the broader patient population, we may learn more about new uses for the drug, previously unknown side effects, and how different subsets of patients may respond. This information may be added to the drug labeling and can help inform our future regulatory decisions.

Our ultimate goal is to approve products that make a meaningful difference for patients and their loved ones living with the devastating effects of their disease.

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

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