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Vivian Xie
10 Jul 2023

Women in Pharma: Advancing gender diversity & healthcare equality

In our new monthly series, we interview women from across the pharmaceutical industry and supply chain to discuss the importance of gender diversity in healthcare, the workplace, and beyond. 

This instalment shines a spotlight on Mindy Leffler, Managing Director of the Qualitative Research and Psychometrics Group at Emmes Endpoint Solutions, a patient-centric CRO dedicated to approaching rare disease clinical services grounded in an understanding of the importance of and respect for patient communities when participating in clinical research. We discuss how pharma can position themselves to have the necessary tools and collaborations when designing clinical trials and working especially closely with the industry’s key stakeholder – patients – to achieve gender parity for all. 

1. Can you please introduce yourself and share some insights into your role at Emmes? 

My name is Mindy Leffer and right now I’m the Managing Director of the Qualitative Research and Psychometrics department at Emmes Endpoint Solutions (which is what Casimir is referred to now). The impetus for starting the whole research organisation was a personal one. I have a now 20-year-old with Duchenne Muscular Dystrophy. He was a participant in clinical trials starting in 2010 (maybe a bit earlier), and it was an eye-opening experience in the way clinical trials were conducted. It was very early in the clinical trial era for Duchenne, and it gave us insight into the outcome measures that were in use and whether the resulting answers that we got from the clinical trial were definitive or not. The trial required my family and me to travel from Seattle to Vancouver, Canada every Wednesday for 2 years, and at the end of that study, we still don’t really know whether that drug works or not. Its failure could have been due to a lack of efficacy, but it could have also been due to poor study design. That is really what led to the origins of Emmes Endpoint Solutions. If families are giving up their most precious commodity in the context of paediatric rare disease, which is time, Emmes Endpoint Solutions was started to reward that contribution with a definitive answer in terms of whether that treatment is worth pursuing or not. Unfortunately, that was not my experience, and that is not the experience of a lot of families that have participated in studies for Duchenne and other rare diseases. What really drives me is this personal mission to make sure that no family ends up where mine end/ed up – giving up years of their child’s life in pursuit of answers they won’t get because of poor outcome measure selection or poor study design. 

2. What are some of the most challenging aspects you have encountered when starting a new enterprise as a woman? 

think that there’s a certain level that speaks to the experience of most women, particularly those that have families, which is essentially balancing everything. It was already a delicate balance prior to the pandemic years and then it became exponentially more challenging. I think that probably speaks to the experience of a lot of women in a lot of industries. 

Additionally, I think that parenting a child with a serious, rare disease, you’re already starting out with depleted resources and coping mechanisms to deal with different situations. Always being aware of that and making sure my response is to give myself a little grace was important on that front. That also extended to the families we were working with in studies because we understand that that is what they’re going through as well.  

3. Why are rare diseases of particular interest to Emmes? 

The measurement of efficacy is that much more complicated in rare diseases than it is in larger studies. Obviously, there’s the personal aspect, which is that my family is affected by a rare disease. But I think that the devil is in the details when it comes to rare diseases. Small disconnects between the experience of patients and, for example, the language used in a patient-reported outcome assessment would maybe wash out in a large population but for rare diseases, it would complicate a study. So, I think that is our niche – the ambiguity of some of the existing data sets and a desire to make sure there is a match between the experiences of patients and families and the resulting data. That can happen on a number of different levels: is the concept of interest being measured applicable to that disease, is the way that concept of interest is being measured relevant and meaningful to the families experiencing that disease, and is the interpretation and analysis of the results in line with the way it should be done to determine those answers? 

For example, the first outcome that was utilised in my son’s study was a 6-minute walk. That is an outcome that is used in adult cardiovascular disease to measure exercise intolerance. So, there was that huge disconnect between the role of walking in a paediatric neuromuscular patient’s life. That disconnect at the very foundational level (of the concept of interest being measured) is extremely problematic. Additionally, in other diseases, they take diagnostic tools for developmental delay and apply those to a clinical trial designed to measure treatment efficacy. We have no idea whether those diagnostic tools are sensitive enough to measure meaningful change within the context of a clinical trial. This is where Emmes Endpoint Solutions operates. 

4. As a female-led company, in what ways does Emmes champion the patient experience in ways other CROs may not? 

Because of the lived experience that myself and a lot of other people that I work with have, we’re on the frontlines as clinical trial participants at the same time that we’re working behind the scenes to improve data collection methodology for similar types of studies. We experience the problems in real-time, and that allows us a perspective on the decisions that tend to be made in pharma that may be really disconnected from that frontline experience. 

5. How can a CRO like Emmes make the most out of their work with other pharmaceutical and research organisations while championing gender parity in the workforce? 

In terms of gender parity within Emmes, in my experience in rare disease, there are some amazing dads out there that really dig in, but the heart of the operation a lot of the times becomes the mum, to a certain extent. We have a lot of people that work with us that have that experience where they are the home-based general in a family’s approach to rare disease and that lends itself to the work we do. We have mums with first-hand experience in rare disease who are study managers, study coordinators, and who help us with outcome measure design because that is their professional background. So, I think it is built into the ethos of our company not in a way that has gender parity as its primary goal but ends up achieving gender parity because of the value system we have set up. We want people that have first-hand experience and that tends to be these mums who have extensive professional expertise but who may be limited in their ability to commit to other environments because of what is going on with their family. We value those people. We want to work with them for those reasons. 

In terms, of our study participants, obviously there are a lot of fronts in which parity is an important topic: racial parity, gender parity, and socio-economic parity in terms of trial participation etc. The core of what we do in terms of data collection is data collection in the home environment. We advocate for that not only for the reduction of burden but because travel to trial sites, particularly for rare disease in the United States that requires hours of travel on an airplane, has an impact on the data integrity of a study. Travelling with somebody who is immune-compromised or non-ambulatory is very difficult. The more that we can look at patient function in the home environment that is not confounded with travel (which everyone knows has gotten more complicated) can also achieve some of those parity goals. For example, the more people don’t have to take time off work to participate in studies can achieve socio-economic parity. It just opens up study participation beyond the core group of people who can typically make it happen. 

6. Is there any advice you’d give to women looking to start their own enterprise within the pharmaceutical or healthcare sectors? 

I come from a background in software development, and this is not my initially chosen field. What’s enabled me to do that is a willingness to always be honest about what I know and what I don’t know, when I feel confident and when I need consultation. I think that allows Emmes Endpoint Solutions to bring rigour to what we do so that it can stand up under scrutiny. I am never uncomfortable to have the work that we do to be evaluated, critiqued, scrutinised, or questioned. Every time that happens, we learn something, and we can do a better job for the disease communities that we serve. So that’s the strongest piece of advice I can give – to make sure you’re very transparent about where your areas of comfort and competence are, and where you need people who know more than you do. I’ve been fortunate to be surrounded by a lot of those people and by no means would have gotten as far as we have without them. 

7. Who are some of your Heroines of Pharma? 

The number one person I would put on that list for me is on the regulatory side. I’ve had the opportunity to interact with Janet Woodcock* for a decade now, and she has so many different interests that she’s trying to balance. The way that she balances rigour with compassion, how she balances statutory adherence with the context of a particular situation and the way she is able to look at a situation and see the immediate context and the broader implications at the same time; I think she is extraordinary on that front. I’ve said to other people that I want my daughter to grow up to be her, so she would be at the top of my list.  

*Former Acting Commissioner of the U.S. Food and Drug Administration and Director of Center for Drug Evaluation and Research from 1994 to 2004 and 2007 to 2021 

Learn more about Emmes Endpoint Solutions, or check out the other instalments in our Women in Pharma series.

Vivian Xie
Editor - Custom Content

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