Having graduated in Medical Clinical Science and Human Genetics, Could you please share your experience entering into clinical research?
After obtaining a Master’s degree in Human genetics (Honors) in India, my interest was to continue learning further in medical sciences and it led to pursuing a doctoral degree in Human hematopoietic stem cell functional genetics in Germany. As clinical science builds on knowledge from basic animal research, my inclination towards entering clinical research was to experience the transition of basic research findings into real world applications. The scope of the research ecosystem, that I had a chance to dwell in, had always aimed at finding pathways of effective cure or managing better of some notorious diseases. Thanks to the scientific and clinical research and community involved, that some diseases, previously thought to be incurable, are been taken care of better today.
Just as an example, Gene-therapy or even gene editing therapy did sound surreal a few years back, but today this technology is alleged to hold promise in treating a wide range of human diseases (such as CRISPER trials). In Germany, I had to carry out an additional clinical research and regulatory course, indeed a rigorous one, to prep-up for entering into the clinical research organizations. All in all, I can voice that it is conceivable to transit from bench to clinical research and it can be very smooth for an individual with scientific research background.
Can you tell us about your PHD research work?
My PhD work was a tough work but also very interesting. After being offered a PhD position at the Ludwig Maximillian University (LMU) and Helmholtz Research Center for Environment and Health, I moved to Munich, Germany. Fun fact about it is that it was my first flight ever and out of India to study. The project, that I got a chance to work on was about ‘hematopoietic PBX interacting protein’ (HPIP), one of a less investigated Homeobox gene, belonging to the class of human hematopoietic stem cell genes that are associated with regulatory networks and are among the key determinants of early hematopoietic development. The HPIP gene is functionally active in normal early hematopoietic progenitor cells as well as in blood tissue samples from patients with acute myeloid leukemia (AML) with an aberrantly high expression in molecularly defined AML subgroups. The research question that I investigated was to understand whether HPIP expression would affect normal human hematopoietic development and in vivo using a Nod Scid mouse model. Because you see, if we do not understand the functionality of the genes that exist in normal stem cells, we cannot build the basis of their functionality in aberrant stem cells as well.
You are Clinical Project Manager in a Software Company specific to Healthcare? Could you please share with us the day to day life of clinical project manager?
Yes sure! Working with a small team and a basic clinical infrastructure in a digital therapeutic start-up comes definitely with its own goodness and challenges. As the digital health care software also requires a clinical validation during their developmental lifecycle, it poses need to establish specific processes and document them. Every day, my priority at work is to maintain a good clinical practice (GCP) in all the aspects of conducting clinical research (Randomized control trials), and make sure that all the team members are guided well. To accomplish this, my day starts with different interactions with external team of clinical experts, study sites and internal clinical team members to plan and deliver the required work. Additionally, my responsibilities also extend to initiate clinical projects in the US alongside the projects that run in EU. It is a great space to work alongside a diverse team of software engineers, UX designers, digital product developers, analytics, marketing, regulatory experts and the list continues. Due to agile development, the work environment in the company is remarkably interdisciplinary, cross functional and dynamic.
In your experience, what constitutes good clinical practice and its challenges?
In my experience, protecting study participant/patient’s rights and safety in a clinical trial is paramount. A good scientific question and research gap in health care ecosystem is seen as a good basis of conceptualizing a new clinical trial. Failure to ask a right question or to stay compliant to GCP, may either put trial subjects at risk or result in the’ rejection of clinical trial data. Needless to say, the credibility of the sponsor/researcher may be questioned in such a case.
Can you tell us more about Digital therapeutics and regulations governing drug safety?
As the official definition goes, Digital therapeutics (DTx) deliver evidence-based therapeutic interventions to patients that are driven by high quality software programs to prevent, manage, or treat a medical disorder or disease. They are used independently or in concert with medications, devices, or other therapies to optimize patient care and health outcomes. Now, if we break it down to individual elements then it’s a marriage between a “High quality software programs” and “evidence-based therapeutic interventions”. The former term refers to software component responsible for the therapeutic effect, for example a digitized treatment with some kind of feedback. This component also has other elements that engage patients to help experience the therapy and complete it. The latter element of evidence comes from design of the RCTs that varies with the intended use of the DTx. DTx are also effective data-driven health management systems, the users self-report their health status and manage their health on a day to day basis (with or without assistance).
What do you see as the future of digital health? And how will it impact us?
In recent years, healthcare ecosystem has made a plenty of room for digital health products and given opportunity to demonstrate their value. The prospective to use DTx in juxtaposition with medicines to improve health outcomes has flickered the interest of big pharma, especially in the pandemic times. The Pharmaceutical industry have started to venture into the DTx space through investments and strategic partnerships leading to the integration of digital health into the existing health ecosystem.
This galvanizing advancement will create opportunities to increase patients’ awareness of their health and precisely in managing diverse chronic diseases. This creates, eventually, a chain reaction thereby generating the potential to improve patient health outcomes and reduce the demands on healthcare systems. However, this challenges in terms of how the disruptive technology is regulated, how healthcare providers respond to this paradigm shift, and how the digital technologies are reimbursed.
Thank you Dr. Pawandeep for educating us on Clinical research and the work in drug safety and digital health