Pulling Back the Curtain: Peter Marks, MD, PhD

In this edition, Peter Marks, MD, PhD, talks about career-hopping, serving the public, and landing his dream job. Dr. Marks is director of the U.S. Food and Drug Administration’s Center for Biologics Evaluation and Research.

When did you know you wanted to pursue a career in medicine?

When I started as an undergraduate at Columbia University, I thought I would become a research biochemist. But, to earn some extra cash while I was there, I got a part-time job at St. Luke’s Roosevelt Hospital (now Mount Sinai St. Luke’s). My job was to draw patients’ morning blood tests. That exposure to patients, along with the volunteer work that I did in a radioimmunoassay lab at St. Luke’s, motivated me to change my plans: Instead of going to graduate school, I went to medical school at New York University (NYU).

The summer before I started at NYU, I took advantage of a program that allowed students to work and gain experience in a lab. I was lucky enough to be accepted into in the lab with Fredrick Maxfield, PhD, who became an incredible mentor to me. His research focused on endocytosis, so that summer I worked to understand that process and became hooked on the combination of laboratory science and clinical medicine. After being accepted into NYU’s medical sciences training program, I stayed on in Dr. Maxfield’s lab, working on single-cell calcium imaging.

After I finished the program at NYU, I went on to Brigham and Women’s Hospital for my internal medicine residency and hematology/oncology fellowship. There, I connected with Thomas Stossel, MD, and David Kwiatkowski, MD, PhD, who were conducting research on basic mechanisms of cellular motility. I worked in Dr. Kwiatkowski’s lab during my fellowship and ultimately joined the attending staff at Brigham and Women’s as a clinician scientist. I was on faculty as an instructor there for several years.

At that point, why did you decide to switch career paths?

It was a mix of personal and professional reasons, but a driving factor was that, during my time at Brigham and Women’s, my spouse and I had two children and I wanted to have more family time.

So, I moved to a position in the pharmaceutical industry for a few years. It turned out to be a very productive period of my career: I was involved in the successful regulatory approval of the first oral iron chelator in the U.S. and helped to set the direction for the regulatory approval of two other products that are now on the market.

The work I did there to bring that drug to market was exciting, but the idealist in me wanted to get back to patient care and public health. So, I returned to academic medicine at Yale University. While I was there, I helped expand the adult leukemia service. Eventually, I served as the first chief clinical officer of the then-new Smilow Cancer Center.

After you returned to academic medicine, why did you decide to move to the public sector?

I enjoyed my job at Yale quite a bit, but during that time I happened to see an advertisement in the New England Journal of Medicine for the position of deputy director of the FDA’s Center for Biologics Evaluation and Research (CBER). The job description seemed to match up nicely with my training and experience, so I applied. I didn’t expect much to happen, and that’s exactly what occurred. I got busy with other work and practically forgot that I had applied.

Then, several months later, I received a letter inviting me for an interview. That process went on for months, which ended with me being offered the position. I began as the deputy director of the CBER in January 2012 and took over as director at the beginning of 2016.

If you were given a “do-over,” would you have made the same career decisions?

I wish I could say there was some rational design to my career path, but there simply was not. That said, I might have tried to organize it better – maybe only having one transition from academia to industry or to government – but I wouldn’t have changed much else. All the different positions in my career have provided me with a wealth of experience in science and medicine, as well as the opportunity to develop my skills as a manager and leader. And these experiences prepared me very well for my current position, which I view as my personal dream job.

What strikes you as the biggest differences among the fields you’ve been involved in?

One way to understand the differences between clinical medicine, the pharmaceutical industry, and government is thinking about whom you’re helping in each role. In clinical medicine, you have a devotion to the individual patient and your focus is on the care of that individual.

In industry, you are developing products that will hopefully help groups of patients, as well as bring value to the company because, obviously, pharmaceutical companies don’t just make drugs out of the goodness of their hearts. In government, you are working on behalf of the entire population and do so in a variety of ways. We are committed to serving public health, from addressing the needs of individuals in need of access to investigational medicines to working to prevent global pandemics, and everything in between.

Third, follow your heart. You need to feel a strong commitment to the advancement of public health. Compensation in the public sector is reasonable, but money usually isn’t the primary reason that people are drawn to it. Seeing forward progress in public health is really the greatest reward that you can have in such positions.

What has been the biggest accomplishment in your career?

I’ve been fortunate to work with magnificent colleagues and to contribute to many successes in the different sectors I’ve worked in, but as CBER director, I can think of two accomplishments. Also, I’d like to emphasize that these are team successes, rather than my personal successes.

One is an administrative achievement of bringing together groups of talented people to help execute our mission at CBER. Having a deep pool of talented individuals allows us to conduct the scientific and regulatory work that we need to do to ensure the safety and effectiveness of blood products, vaccines, and cellular and gene therapies.

The other is related to policy issues: During my time at the CBER, we have released a fair number of guidance documents that have helped people develop products and deal with various outbreaks, such as Zika or Ebola. But, of those, the one that stands out is establishing the Regenerative Medicine Advanced Therapy Designation for cell therapies or therapeutic tissue-engineering products, or human cell and tissue products, as directed under the 21st Century Cures Act.

We worked as a team to roll out that implementation effectively and efficiently, and, within a few weeks of the act’s passage, we had a process in place for receiving applications and reviewing them in a timely manner. That program has been quite successful: In the just-over two years of its existence, we have received nearly 100 requests for this designation.

What have been the biggest changes in the work of CBER since you started as deputy director?

The introduction of cellular and gene therapies is causing us to take a renewed look at manufacturing processes and clinical development timelines. Certain challenges and questions have come to light because we are dealing with smaller patient populations and products that are more difficult to manufacture consistently. For example, how will we adapt to products that aim to address different mutations in a gene using a specific gene-editing approach?

On the clinical side of things, we are at a place where we must look for the most relevant endpoints for many uncommon or rare diseases to understand what matters most to patients. The clinical trials of investigational agents for rare diseases are conducted with relatively small groups of individuals and within clinical areas where we perhaps don’t have extensive natural history studies.

Biologic therapies also are rapidly evolving, and we need to learn more about their optimal manufacturing and their critical quality attributes to understand how to evaluate them most efficiently. That’s a real challenge: We want to get these products out to patients in need and we don’t want to require unnecessary information for regulatory approval that overburdens investigators and industry, yet we still need enough information to ensure that the products are safe and effective.

Our responsibility is to ensure that these novel products are held to the same reasonable standards for safety and efficacy to which other medical products are held. Just because something can be treated by a cellular product or a gene therapy rather than a conventional drug doesn’t mean that the cellular product or gene therapy is the better treatment. That can be difficult to understand for people who are perhaps overenthusiastic about these new treatment modalities. At the end of the day, we want to enable the best treatment for patients – it doesn’t matter what type of treatment that is.

Does a change in administration affect your work at the FDA?

Some priorities may change with a new presidential administration, but our priority at the FDA remains the same: protecting and promoting public health. When there’s an administration change we must brief a whole new group of people, but we’ve been fortunate that people on both sides of the aisle have recognized the importance of our work. Administration changes can affect different people in different ways; my way of dealing with it – and I think that many of my colleagues have the same approach – is to stay focused on our public health mission.

With that in mind, what do you think people should be enthusiastic about? What types of therapies or approaches do you think will be considered for approval in the near future?

Research is advancing so rapidly that what I say is all speculation, but my sense is that, five years from now, we’ll probably be approving gene therapies for a wide variety of genetic diseases. We also might be evaluating novel approaches to streamline gene therapy approvals, like use of a single vector with a range of different inserts tailored to target different mutations causing a particular condition, analogous to a razor and razor blades.

Ten years from now, I suspect that we will have an array of molecularly targeted medicines and gene therapies that will address both rare and common diseases. And we also may be approving new generations of vaccines that provide broad protection against classes of viruses, such as influenza.

If these predictions come true, they will be game-changers for population health.