Your Rare Disease and Pediatric Research Partner

Why you joined Emmes?

I Felt that our company Casimir had something that was so valuable and so insightful that we had to get it out globally. We couldn’t keep it to ourselves – we needed a global footprint so we looked for a partner – one that would allow us to grow with our methodology with the focus on improving patients journeys in clinical trials.

Our experience in getting the first drug in Duchenne approved was so difficult, those learnings had to be extrapolated for the greater good. We had to share these experiences so that others could learn from them and do better for people with rare diseases - to cause a change and effect in the way that we function in this space. Becoming part of Emmes has provided that opportunity.

What role do you play within Emmes’ Rare Disease and Pediatric Center?

I utilise my personal and professional expertise, harnessing the relationships I have built and the vision I have for how we can support patient journeys. Supporting the process of how the trial can and will look for the greater good of the patients we serve. Ensuring individualised approaches wherever possible.

How does your work contribute to serving the rare disease /and or pediatric community?

We are making it more about individual and personalised care – for both the patients and the sponsors. We are bringing the real substance to the process. Humanising the clinical trial experience. Ultimately to bring more patients to treatments in a much timelier manner. Which helps patients get a treatment more quickly and sponsors too. We are also helping regulatory agencies with the contextual data they need to approve drugs.

What makes working within this space different from other research areas in your opinion?

Small patient populations and the challenges with the heterogeneity within these populations. The challenge of applying enough rigour whilst still having the flexibility to perform differently in functional tasks, but yet are still meaningful to the patient and hold up to the regulatory scrutiny. This is an extremely tough balancing act – allowing the flexibility in the data – a unique approach to this space.

What experience do you have in rare diseases/and or Pediatrics?

I founded the Jett Foundation in 2001, when my son now 27-year-old, was diagnosed with Duchenne Muscular Dystrophy. As Executive Director of the Jett Foundation, I co-founded the International Duchenne Alliance, a partnership of over 40 independent organizations that has funded nearly $15 million dollars in research to date.

As a Patient Advocate for Duchenne and other rare diseases, I have been advising other rare disease organizations, bio-pharmaceutical companies, and other stakeholders on the importance of measuring more meaningful outcomes for clinical trial participants and their caregivers.

Background about you

I have advocated for the patient perspective to be considered by the FDA when reviewing safe and potentially effective treatments for Duchenne. In April of 2016, Christine, as an advocate was granted time from the leadership team at FDA to present the Jett Foundation's Patient Centered Outcomes report at FDA's meeting of the Peripheral and Central Nervous System Drugs Advisory Committee for the review of Exondys 51, a first in FDA history.

My experience and expertise led me to co-found Casimir Trials in 2016, where learnings from my experience with the first ever approved drug for Duchenne, Exondys 51, can be utilized by drug developers, patient organizations and regulators. Casimir Trials has experience from pre-clinical through real world evidence studies in nearly 20 rare disease indications, using remote applications. My passion for rare disease studies and meaningful benefit identification fueled the recent acquisition of Casimir Trials to Emmes. Casimir, now part of Emmes, brings additional rare disease expertise, advocacy and novel outcomes in partnership with Emmes’ dedication to bring excellent management to clinical trials.

Why you joined Emmes?

I joined Emmes 30 years ago when I was working on my PhD. I was eager to apply what I had learned in classroom to the world of clinical research. Emmes, though very small at that time, already had a reputation in DC area as an excellent CRO.

What role do you play within Emmes’ Rare Disease and Pediatric Center?

What role do you play within Emmes’ Rare Disease and Pediatric Center? How does your role contribute to the Center and goals of Emmes? I oversee the Pediatrics side of the Center. Given my decades of experience working on pediatric studies, my role is to help conduct high quality research in pediatric populations and provide best value to Emmes’ clients.

How does your work contribute to serving the rare disease /and or pediatric community?

Children have long been overlooked when it comes to drug development. The goal of the program is to help our clients and partners to not only develop new drugs for children but also do studies to determine the age appropriate dose and safety profile of these drugs. We are focussed on developing patient centric studies that keep in mind not just the patient but the whole family. We support implementation science studies to enhance uptake and adherence to therapies available to children.

What makes working within this space different from other research areas in your opinion?

Pediatrics is not one uniform cohort. When designing a pediatric study we have to account for different pediatric groups: neonates, infants, children, and adolescents. Outcome assessments such as quality of life and pain measures can be difficult to assess in neonates and infants. Adherence to study visits and treatments is dependent on the parent or legal guardian.

What experience do you have in rare diseases/and or Pediatrics?

My first project in pediatrics was in liver transplantation. I helped develop the Pediatric Endstage Liver Disease (PELD) score in 2001 that is still used to allocate deceased donor livers to children in the United States. Over the past 13 years I have been leading the Best Pharmaceuticals for Children Act (BPCA) Data Coordinating Center. We have supported over 40 IND studies that have led to 14 label changes by the FDA and 2 device approvals via 510(k) pathway.

Background about you

I am a biostatistician by training. In graduate school I was fascinated by application of biostatistics to the broader field of clinical research and the potential impact it can have on public health. I joined Emmes to get practical experience in design and analysis of clinical studies. I enjoy collaborating with clinicians.