Laura Iliescu, Manager of Patient Advocacy & Engagement, Center for Rare Diseases, is an adventurer and explorer. She dives into challenges the clinical development industry faces head-on, always putting patients and their experiences first. Here are some fun facts about Laura and her journey into the drug development world.
In your opinion, what is the biggest challenge our industry faces today?
Clinical research lags behind other healthcare settings when it comes to shifting towards a patient-centered paradigm. When a clinical program is envisioned, scoped, and implemented, it’s not typically approached as being a service for patients. Developing trials around patients and products instead of just products is a sophisticated, cutting-edge practice that only the most forward-thinking sponsors have mastered. It takes adopting a service design approach throughout the clinical development process and that’s a momentous shift. It places the patient’s lived experience at the center of both study development and implementation to set patients up for success as participants. That takes the combined efforts of sponsors and CROs, and my work in the Center for Rare Diseases focuses directly on this aspect to de-risk rare disease clinical programs.
Studies need to fit the lives of participants, whether that’s patients or the caregivers who support them. Studies that fail to do this face enormous risks. With thousands of rare diseases without an approved treatment to date—that’s a risk we can’t afford to take.
Why did you become involved in clinical research?
I had two very compelling reasons. The first is that working with rare disease patient advocacy communities really showed me what a clinical trial can mean for individuals living with a rare condition. It’s a long-awaited milestone for rare communities and a unique source of both healthcare and hope to patients and families. Participating in a trial can be one of the most important parts of a rare disease patient’s health journey. Working to help ensure that journey is a path to success for both patients and studies is something I’m very passionate about.
The second was PRA’s Center for Rare Diseases and the excellence of our multidisciplinary team. The Center’s vision that clinical trials must evolve to be recognized as care pathways that are as important as standard of care immediately resonated with me. The Center is a true place of innovation. It has been a pioneer in developing patient-centered approaches to designing and implementing rare disease clinical programs.
In your experience, what has been the most important factor that has changed drug development?
Within the rare disease landscape, the Orphan Drugs Act of 1983. The Act was a product of determined and strategic patient advocacy lobby by the legendary founding members of the National Organization for Rare Diseases (NORD) including Abbey Meyers, the mother of a son diagnosed with Tourettes syndrome. She realized that without changing how innovative companies were incentivized, they could not viably develop treatments for rare conditions. Without a momentous shift in the financial incentives framework for innovative companies, there could be no hope for new treatments. The Act established a framework that would encourage research and progress. It laid the foundation upon which the biotechnology industry emerged. Visionaries like the late Henry Termeer at Genzyme went on to create a completely new business model on that foundation and hundreds of treatments for rare diseases have since been developed.
What is the biggest misconception people have about clinical trials?
That sites are the only ones doing important work in a study. Patients who participate in a clinical study are performing essentially important health work. Adhering to a study protocol and completing the schedule of assessments is really a type of work that patients sign on for. That’s on top of the work they do to manage their condition and lead their day-to-day life, so it’s critical to set them up for success. When the feasibility of a study is assessed, it’s often only assessed from the site’s perspective and rarely from that of the patients it counts on as participants. That’s a very risky blind spot for a clinical program. Recruitment and retention statistics for clinical trials paint a clear picture of how much of a barrier to success this oversight can become. In rare disease, setting patients up for success in their “work” as participants can often be key to the success of a study.
How has clinical research affected your life personally?
I have both friends and family who are affected by rare and common conditions. For some there aren’t any treatments at this time. Clinical research is the only thing that will change that in the future. Working to make clinical trials more feasible for patients helps to make the long wait for a treatment as short as it possibly can be. It’s been both challenging and energizing on every level, personally and professionally.
If you had to describe yourself in three words, which would you choose?
Curious, strategic, and kind.
If you think back, at what point could you have chosen a different career path and what would it have been?
In my late 20s, I moved on from my first strategic marketing role in pharma after five years of immense professional and personal growth within an outstanding company. I moved to Europe and I asked myself whether I might want to do something else—perhaps even leave healthcare. After all, I’d spent the first five years of my career working at one company. Reflecting on it, I couldn’t think of anything more meaningful to dedicate my career to than playing a small part in the healthcare we all rely on throughout our lives. At that juncture, I decided I would stay in life sciences but I would carve a somewhat “unconventional” path for myself. As a result, my career has taken me through every phase of the product life cycle, every clinical care setting, through both the pharma and biotech sector, and into the patient advocacy landscape. It’s taken me through several countries on two continents (so far) and continues to evolve—I’m currently completing a Master’s in Human Factors Engineering. I couldn’t be happier with the choice I made.
What’s a fun fact about your childhood?
I was born in the 80s on the “other side” of the Iron Curtain, so I have a lot in common with baby boomers. All my childhood pictures are in black and white. I can remember a time before any computers whatsoever and dialing a rotary phone!
If you were to write a self-help book, what would the topic be?
Probably something like, How to Love Living Outside Your Comfort Zone! We’re living in a period of rapid change, technological and societal. I think developing the skills to thrive in times of change would be something of universal value on many levels.
What was the last trip you took and why?
It was a family trip to the north of Morocco. We drove from Casablanca to Tangiers and then to Chefchaouen, a city in the Rif Mountains known as the “Blue Pearl” for its stunning blue architecture. It’s an absolutely superb country with fantastic cuisine and warm, wonderful people. It was an unforgettable experience.
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RARE DISEASES NEWSLETTER Volume 15, December 2019
Cross-Border Enrollment of Rare Disease Patients
Clinical trials in rare diseases present unique challenges unseen in trials for more common conditions. Cross-border enrollment can be key to…