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About the Institute

Profile of Martin Offringa

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Dr. Martin Offringa

By: Mackenzie Hill-Strathy

Dr. Martin Offringa, MD, PhD

  • Program Head and Senior Scientist, Child Health Evaluative Sciences
  • Staff Neonatologist, Neonatology, The Hospital for Sick Children
  • Professor, Paediatrics, University of Toronto

1. Where are you from?/Where did you study?
I am from the Netherlands, and only recently moved to Toronto when I began working at SickKids in 2012. I studied medicine and clinical research methods (also known as clinical epidemiology), at the University of Rotterdam in the Netherlands and in Boston, Massachusetts in the United States. In addition, I completed my PhD and my paediatric training at the University of Rotterdam before I moved to the University of Amsterdam, where I became a professor of clinical research in paediatrics. I was actually the first research professor of paediatrics in the Netherlands; all the other paediatric professors at the time had a different sub-specialty such as paediatric cardiology or paediatric haematology.

2. What are you researching right now?
One huge problem in paediatrics is that over 50 per cent of the drugs given to sick children have never been properly researched for their effectiveness and safety. On top of that, often we aren’t sure of the dose. This is something we must do something about. There have been numerous examples of children being harmed from their medication, including the thalidomide case in the 1950s, and just a few years ago, the death of 123 children in the United States from using two types of cough syrup together.

My team and I are working on researching safe and smart ways of testing new treatments in children with our program EnRICH. We build on research conducted in the form of clinical trials and “prognostic models.” These studies compare the results of one treatment with another treatment in similar patients, with similar problems, looking for similar outcomes, and then determining which treatment had the best success in terms of improving health outcomes and reducing side effects and harm. But who is likely to benefit most from the new treatment? And for whom is the treatment probably not cost-effective? To find these answers, we need to add additional study types and find alternatives to traditionally large and expensive trials. This requires getting valid answers faster and in a form that is acceptable to decision makers, whether this be at the bedside or at the health-care system level.

3. Who is your all-time favourite scientist and why?
My favourite scientist is Iain Chalmers from the UK. He has inspired many people, including myself. He taught us that in medicine, although the intentions are good, our powerful interventions may harm patients. The intuition of doctors to choose one superior therapy over another has proved us wrong many times and the only way to prevent this from happening is to evaluate these treatments within clinical research while treating patients. In addition to developing safe and effective methods for patients, they need to be measured for their success. 

As a mentor, Iain Chalmers taught me the value of an unbiased examination and comparison of treatment options, which is our only chance to learn whether or not they are doing more good than harm. He showed me when a trial is needed, but also when to stop doing trials for a given treatment. They gave him a knighthood in England. In my opinion, he deserves a huge international prize for the difference he has made to modern medicine and to millions of patients.

4. What in your opinion is the most important scientific breakthrough and why?
To me the most important scientific breakthrough is the relatively recent development of scientific tools to test treatments in both a scientific and an ethical way. Testing treatments has always been associated with experimentation, and was usually done on animals or cell lines - not sick children. The breakthrough in my career was witnessing the development and introduction of tools to study the safety and efficacy of alternative treatments and drugs, as opposed to not studying them, which unfortunately, has led to suboptimal outcomes and harm. Change for the better has occurred recently thanks to both a mindset breakthrough and advancements in clinical measurement and statistical analytical tools. 

If we didn’t have these tools we would still be in the dark ages, using trial and error on individual patients. We now conduct comparative effectiveness research in groups of patients, with all stakeholders involved from study design to the application of the results in daily practice. In the case of paediatrics, this means involving children, their parents, the researchers, the ethicists and the hospital doctors. As I learned back in Europe, this can only be pulled off effectively if we recognize that all of these groups are in one boat together, working towards the same destination. There isn’t one boat for clinical care and one other boat for research; there is no way forward unless we all work together.

5. What are your major interests outside the lab?
I love music, particularly classical and jazz. I used to play the jazz guitar in a band, and now that I have fully transitioned to Toronto, I would like to pick up the guitar again. I just bought an amp, so the next step is to find a band – since last week I have one offer on the table! The other thing I like is cycling. As a Dutchman, I have come to realize that I can’t live without my bike, so now I’m trying to survive the roads in Toronto. Since moving to Toronto, I have joined three local action groups that advocate for separate bike lanes in the city and more bike lanes outside the city. I also love watching sports. This year, I went to my first hockey game and baseball match.

6. Why science?
There were two events that led to my decision to pursue a career in clinical research. First, as a young doctor (I was a clinician first) I was a bit frustrated that I didn’t completely understand what the reasoning was behind giving certain treatments. Choosing a particular treatment because there was one article in a famous journal on the drug, didn’t make sense to me. There was so much disagreement among experts around me. So I decided to take science courses above and beyond what I had learned in medical school to quench my curiosity.

The other event that prompted me to pursue science was that as a young doctor, I witnessed how we can inflict harm by making the wrong diagnosis and by prescribing the wrong treatment. I really wondered: how do we actually know what we know in medicine, where does all this wisdom come from? I started looking more in-depth into this, and I found that, particularly in paediatrics, the field of clinical research was quickly emerging. Instead of relying on deductive reasoning and extrapolating knowledge from adult patients, it became clear that we needed our own empirical research in child health. This includes making insights from developmental biology and developmental pharmacology and focusing on developmental and social outcomes in young children. 

This emerging research aims to provide children with safer and more effective tests and treatments, based on insights from clinical epidemiology (via. statistics) and translational research. Essentially, this enables bringing discoveries from the lab to the bedside while examining the reduction of poor health outcomes or improved livelihood due to the new treatment. Once a practicing neonatologist, I knew there could not be a clinical life for me without translational and evaluative research. Then, a few years later, I realized that research without clinical practice doesn’t work either. The two – clinical practice and research – really go hand in hand for me, which is why I try to maintain both.

7. Why SickKids?
Being a paediatric research professor in the Netherlands, there were always two places we looked up to: Great Ormond Street Hospital in the UK and SickKids in Toronto. When Dr. Janet Rossant called about an opportunity at SickKids, I was thrilled! I had to really think about whether there was something I could contribute to a place that was already so wonderful. After two visits here, they convinced me that it would be a good match. Fortunately, I am able to continue my collaborative work overseas. I really believe in collaboration and that we need to convince all stakeholders that this truly is the way forward. The opportunities to impact and really make a difference are fantastic at SickKids, which is what sealed my decision to pack up everything and move from Amsterdam to Toronto.

8. What is the most controversial question in your field right now?
The most controversial question in my field is whether children should be regarded as research objects, or whether they are subject to their own opinions about their participation in research. Researchers sometimes forget to look at what the children want to get out of the study and what health outcomes matter most to them, often ones that we may have forgotten to study. I think we will see changes for years to come surrounding this matter. Traditionally we lived in a paternalistic system, with adults deciding what is best for children, determining whether they need to be protected from inclusion into research studies. What we are now hearing from children, is that they want to be protected from exclusion; they want to be included in research! Children do have opinions on research, about how they want to be treated, what the most meaningful health outcomes are and what we should strive to improve. Multiple studies show that parents of sick babies want their babies to be included in research – especially when doctors are uncertain about the best course of treatment.

9. What are you reading right now?
I am reading How Not to Be Wrong, by Jordan Ellenberg. Jordan is the son of two friends of mine, biostatisticians Susan and Jonas Ellenberg. I like this new book because it is really funny and at the same time relevant for scientists and doctors. It demonstrates how math touches everything we do, and that with the tools of mathematics we could understand our world in a much more meaningful way. Ellenberg convinces us that mathematical thinking, which he refers to as: “the extension of common sense by other means,” is based on a science of not being wrong. He illustrates his point with some great examples, ranging from the everyday to the cosmics to the math of baseball. However the book is not just about statistics. It includes such anecdotes as the analysis of bullet-hole data from airplanes returning from World War 2 flights. The military needed to decide whether extra armour should be added to vulnerable areas, in particular where most bullets had landed. The solution turned out to be the opposite: the statistician recommended putting extra armour where there were less frequent bullet holes - planes hit in these spots didn’t return.

10. If you could give one piece of advice to someone considering a research career, what would it be?
Take time to pick your research topic. Talk to as many people as you can about the subject matter that you are interested in, and find a mentor. If you are in clinical medicine, participate in clinical practice as much as you can because that’s where the relevance begins and ends. Make sure that what you do has an impact on others and take time to decide what this will be. If you find a research field that excites you, then follow it with all your heart and make sure that you don’t lose enthusiasm and inspiration. The path is not always easy and there are always challenges, but in the end it is all worth it.

11. What does the SickKids' Peter Gilgan Centre for Research and Learning (PGCRL) mean to you?
To me the Peter Gilgan Centre for Research and Learning is a unique place, where I can easily walk up or down the stairs to meet fantastic scientists, whether they are in basic or applied research. I also love to run into people here at various stages of their research career and learn about what they are studying, and why it matters to them and to others. As a clinician-scientist, I am also very happy that we are now proceeding to further integrate the work in the hospital and in the research tower. Increased collaboration across SickKids will result in the best opportunities for progress in research, research translation and for improving child health. Therefore, not only do I travel through the new bridge connecting the main campus to the PGCRL daily myself, I have advocated for adding more bridges!

September 2014

Scientific profile