In the past years, buzzwords such as ‘precision medicine’ and ‘personalized medicine’ have
been echoing in numerous congresses and articles. It designates a fascinating field that
doesn’t want to believe that health is a matter of individual luck, but a matter of applying
medicine in an individualized and precise way, tailored to both the disease’s mechanism and
the patient’s characteristics, respectively. For instance, immunotherapies are the direct
result of our better understanding of the molecular mechanisms at fault of diseases, saving
the lives of cancer patients and making life bearable for those affected by chronic debilitating
diseases such as rheumatoid arthritis. Personalized medicine is already possible as we
already understand how our genetics can determine how our drug-metabolizing enzymes
behave and impact a drug’s effect. Hitherto, a DNA passport specifically for
pharmacogenetics has already been in use in the Netherlands: a quick check on your
passport may guide your physician in his/her treatment’s choice. Well then, this must be the
era of personalized precision medicine, right?

The truth is, that we are not yet in this era. In fact, certain observations beg the question
whether we will ever enter this glorious period.
The DNA passport, which provides pharmacogenetic information, has found disappointingly
little use into everyday clinical practice. This failure does not originate in the quality of the
scientific research that led to this tool. The Human Genome project’s goal was to generate
tools to better interpret a patient’s genetic make-up in order to scientifically guide treatment
decisions. The DNA passport in itself is practical: it is in the form of a card which easily fits in
your wallet, next to your identity and bank cards. The information and, more importantly,
interpretation can be read from the card. It has widespread use in the psychiatry field and
oncology. It is, however, not used in primary care, although the majority of drugs are
metabolized by 5 CYP-enzymes for which mutations are known to affect the metabolizing
rate. So where did we go wrong? Somewhere in the implementation process obviously.

This example shows us that there are still barriers for such tools to be implemented in
today’s clinical practice; even if it may enable personalized medicine. If we want
personalized medicine to be readily used, we need to better identify these barriers and find
solutions for absolute implementation. This starts early on, while we gather knowledge: how
do we proceed with this knowledge, who are the end-users, what are their practical needs
and how can we meet these? Of course, genotyping every citizen is too expensive. But is it
really? This depends on the advantages personalized medicine has to offer? The patient
gets better treatment from the start and doesn’t have to go through the ‘trial-and-error’
phase. So it really depends on how we define the ‘return on investment’.

In oncology, personalized and precision medicine has recently become an unprecedented
reality. Recently, chimeric antigen receptor-T cell therapies were developed and approved
by the FDA in the battle against various cancers. This treatment is the perfect example of
precision and personalized medicine: the patient’s own immune cells are used to target
tumor-specific antigens through genetic manipulation. With an astonishing survival rate, this
new therapy may dramatically change the oncology landscape. That is, if we let it. What’s

the issue here? The equally unprecedented costs: both tisagenlecleucel and axibactagene
cost 475 000 and 373 000 USD per treatment, respectively. The current payer models are
not in place to deal with such daunting numbers, and will only be complicated if these
therapies will be approved for other indications as well.

It is worrying that we already have the know-how for personalized and precision medicine,
but practical issues block our entry into this new and exciting era. It is like a never-ending
ride to Disney World, where the kids in the back ask that nagging question: are we there
yet?

This article is not meant as a negative statement, but rather an encouragement to those who
have the privilege to work in the field of medicine to join the debate. Whether you’re a
physician, a scientist or a regulatory affair specialist; ask yourself regularly the following
questions: How can we prevent a premature halt of revolution in medicine? What do we
need to make personalized and precision medicine feasible and affordable? How do we
define return on investment to enable personalized and precision medicine?

Asking these questions during scientific research is what truly enables translational medicine
and makes the utilization of scientific results more likely. Concomitant with the era of
personalized and precision medicine, we should focus on translational medicine to ensure
implementation of our knowledge as actual bench-to-bedside science.

About the author:
Lynda Grine completed her Master in Biochemistry and Biotechnology in 2010 at the Ghent
University. Right aftewards, she pursued a PhD at the Flemish Institute of Biotechnology
(VIB) with an IWT-grant. Today, she’s doctor-assistant at the Ghent University, an FWO-
project manager and senior researcher at UZ Gent. In 2018, she successfully attained the
‘Translational Medicine’ certificate at the ULB. Next to her daytime job, Lynda remains
passionate about science and wants to let the world know about scientific innovation, today’s
challenges and tomorrow’s opportunities.

Sharing a recent experience on digital health by Samia Laokri.

The complexity of health systems, including global health players and practices, calls for a greater use of integrated and multisectoral approaches to global health. In this frame, one can raise the potential role of novel technologies and practices in the provision of equitable access to quality care. Over the past years, the global health community has seen an explosion in the use of mobile technology (mHealth) and other programs promoting electronic innovations (eHealth) to improve patients’ care-seeking pathways and population’s health. A wide array of digital technologies and strategies has indeed revolutionized various aspects of our society. Health is definitely not the exception. Solutions, or part of the solutions, to several key health systems constraints are hoped to be found in the digital revolution era. For instance, greater access to information, especially in remote settings, may speed up the decisions and possibly allow real-time policy-making. Among the definitions circulating for digital health, one embraces all techniques that refer to hardware, software, communication channels, Internet, mobile for care services delivery, health financing, health education, supply chain, program M&E or data collection.

How much access do we have to information, and to preventive care, diagnosis and treatment services? How can the digital revolution realize its promises? Moreover, what do these promises look like in practice? These are vital questions in order to avoid being overwhelmed by the digital medium. To cover the issue, I have been exploring available evidence on the impact of recent digital innovations on health practices and access to care. In this study, I was interested in highlighting the role of novel technologies developed for rapid diagnostic testing, diagnosis accuracy, e-notification, medication adherence, etc. to tackle the new global health challenges, and in particular reducing the economic burden of disease.

A recent Geneva Health Forum (10-12 April 2018) held in Geneva, Switzerland was fully dedicated to Precision Global Health in the Digital Age. During the event, various stakeholders from academia, international organizations, start-up companies, private partners and NGOs exchanged their views and discussed plethora of innovative practices in digital health “with aiming to showcase innovative, accessible and sustainable practices in order to facilitate access and health equity”. The I³h team took part in it with two studies presented as poster communications: 1) Exploring the impact of novel technologies in Tuberculosis control, and 2) Fostering interdisciplinary to educate the next generation of global health actors.

The four best students of the 2017 I3h Educational Program have been rewarded a participation to the ENLIGHT-TEN summer school on Translational Research & Medicine Development in Berlin. Two of them, PhD students Ciska Verbaanderd (KUL) and Katoo Muylle (VUB), share their experience here.

Katoo Muylle
During the course, not only did we learn about the theoretical side of the drug development pipeline, we also got acquainted with real-life case studies in several stages of this pipeline.
We experienced the gap between reason and emotion, especially in the workshop about the FDA approval of eteplirsen. As scientists, the lack of evidence was clear to us, but as patients, parents, family, this lack of evidence became secondary to a glimpse of hope. Another interesting workshop was the board game “test tube to tablets” where we as a group had to decide about the dilemmas involving costs, risks and delays to get our hypothetical drug on the market. We also got interesting lectures about preclinical drug safety, the Open Targets Platform, patient involvement in drug development, and the development of biologics.

Next to this, there was sufficient time for the development of our own soft skills. Everyone had to do an elevator pitch of 3 minutes about their research project and give a poster presentation of 5 minutes. The nice thing was that it was designed as a learning activity. We got feedback on our presentation skills and on our poster design and received tips for improvement.

Lastly, the course was also broadening on a personal level. Because the participants came from all over Europe, it was interesting to discuss differences in healthcare systems, in university approaches on PhDs and PostDocs, but also in general lifestyle.

An artistic summary of the day-to-day activities of the course (click arrow to see more).

Ciska Verbaanderd 
We had the opportunity to meet and talk to experts with years of experience in different healthcare domains, like Dr. Rodger Novak,  founder of CRISPR therapeutics, who presented his career path and vision on how to translate CRISPR/Cas9 technology into innovative medicines for patients. Moreover, Jan Geissler presented his experiences as a cancer survivor and patient advocate and highlighted the importance of patient involvement in healthcare R&D.

We also got the chance to visit Bayer’s high-througput screening lab and the CoLaborator (work space for start-ups @Bayer), and to experience their 3D structural biology tool. Finally, two sessions on personal development, which were meant to explore our own strengths and interests, were included in the programme.

Overall, the speakers and the organizers were very approachable and open to discussion, which was a great added value to this course!

In the evenings, the participants had some time to see the sights.