We never forget that rare diseases alter entire lives—and we’re committed to changing that.
Key Highlights
Rare changes everything
At the Center for Rare Diseases, we never forget that rare diseases alter entire lives. We’re committed to changing that.
We dedicate our time, so they don't have to
The time and effort spent by the patient and their loved ones in managing a rare disease is time better spent on living a fuller life. We dedicate our time to rare disease research so they don’t have to.
Stay up to date on PRA’s Center for Rare Diseases, our achievements, and new initiatives
Rare Disease NewslettersEvery day can be delayed, postponed, or canceled when you or a loved one has a rare disease. We understand the unique challenges rare disease patients and trials face compared to those in more common indications. Lives are uprooted and postponed in search of treatment, and not just the lives of patients, but their entire care circles.
Our experience allows us to efficiently conduct rare disease studies by anticipating and mitigating risks while maintaining focus on the patient, the family, and their quality of life. We understand that every day without treatment means another day filled with doctor’s appointments, lab work, postponed opportunities, and canceled plans.
The time and effort spent by the patient and their loved ones in managing a rare disease is time better spent on living a fuller life. We dedicate our time to rare disease research so they don’t have to.
Check out what we’re doing to fight rare disease.
Our People
Scott Schliebner, MPH
Senior Vice President, Center for Rare Diseases
In addition to more than 25 years of clinical research experience, Scott has a Master’s degree in Public Health, a background in epidemiology and biostatistics, and has held leadership positions across the biotech, non-profit, and CRO spaces. In addition to his role at PRA, Scott co-leads Global Genes’ Rare Corporate Alliance, a coalition of rare disease industry stakeholders that are committed to collaboration between industry and patient communities to improve and expedite access to effective therapies for all rare patients. He is also a member of Uplifting Athletes’ Board of Directors, and he speaks and writes regularly on innovation within clinical research.
Why rare disease?
“I find clinical research to be rather archaic, and I believe we can do better. I am inspired to look for smarter, better ways to engage patients, to ensure clinical research can be a legitimate care option, and to design clinical trials that are feasible and realistic. I want to be disruptive and help push our industry forward.”
Amy Raymond, PhD
Director of Therapeutic Expertise
Amy brings more than 20 years of experience in the bench to bedside continuum to PRA. She has a PhD in Molecular Biology, as well as an extensive understanding of research science, x-ray crystallography, and clinical operations. While much of her work is related to rare neurology indications, her experience also includes infectious diseases and extensive oncology. Amy also collaborates with ALS Association chapters across the country and is very active within her own local chapter.
Why rare disease?
“Over the course of my career, we’ve made great strides in understanding the mechanisms behind many rare neurological disorders, which is a critical step. However, the key is translating that understanding into meaningful treatments for rare disease patients. With so much unmet need, every improvement we make with new therapies has a huge impact—we can change a patient’s life.”
Juliane K. Mills, MPH, MS
Director of Therapeutic Expertise
Juliane brings a comprehensive understanding of the drug development process to the rare disease team thanks to more than 20 years in various scientific roles at CROs, pharmaceutical companies, and academic institutions. Throughout her career, Juliane has supported a wide range of indications with a primary focus on infectious diseases and immune disorders. She’s an expert at developing patient-focused scientific and operational strategies for various study types, including disease registries, natural history studies, non-interventional studies, and biomarker studies. Her goal is to make clinical research part of everyday healthcare.
Why rare disease?
“Patients with rare conditions are willing to be vulnerable. They put their trust in the scientific community to respect their sacrifices by working tirelessly towards a cure. I try to live up to my end of the bargain by making clinical trials a better experience for patients through innovative designs and new technology. It is the ultimate reward to have a direct and meaningful impact on someone’s quality of life.”
Kendall Davis, MPH
Manager of Patient Advocacy and Engagement
Kendall brings her extensive experience working with rare and chronic illness/disease in both biotech and nonprofit sectors to her role as Manager of Patient Advocacy and Engagement. Before joining PRA, Kendall served as the Director of Strategic Alliances at Global Genes. She earned her Master’s of Public Health from Michigan State University. Kendall also serves as VP – Board of Directors for Team Telomere, Inc., an organization established to provide information and support services to families affected by Dyskeratosis Congenita and Telomere Biology Disorders. She is also a Corporate Alliance Member with Global Genes and Strategic Advisor for the Rare and Undiagnosed Network.
Why rare disease?
“Every single person I have met who is impacted by rare disease is doing everything in their power to overcome the challenges that come with it. It’s a truly unique community. Working in rare disease allows me to provide those impacted with the opportunity, resources, and support to be an active participant in the development of therapies for their diseases – as well as the improvement of their quality of life. Rare disease advocates are changing the way we do clinical research and develop medicine, and it’s an honor to work in this space with them.”
Jessica Wessel
Clinical Strategy Lead
Jessica has more than eight years of experience in clinical research and holds certifications in clinical research, project management, and Ophthalmology. Before joining PRA’s Center for Rare Disease, Jessica led the Compassionate Use efforts for a large, multidisciplinary Ophthalmic practice where she was inspired by the strength of families and patients living with rare diseases. Her therapeutic expertise includes Ophthalmic indications from Anterior to Posterior Segment.
Why rare disease?
“Seeing the strength of the patients and families living with rare diseases continually inspires me to push for a better understanding of each patient’s disease and its impact on their lives. This enables more efficient and effective clinical trial development and, ultimately, can accelerate how fast we can bring meaningful therapies to patients who need it.”
Jana Benesh, PhD
Clinical Strategy Lead
Jana is a trained neuroscientist with a PhD in Neuroscience and Molecular Biology. She has more than 14 years of combined basic science and clinical research experience, with her primary focus on rare neuromuscular disorders including muscular dystrophy and rare epilepsy-related diseases such as Rett Syndrome. She’s currently a member of the ACRP Central Alabama Chapter and she frequently collaborates with patient advocacy groups on educational outreach initiatives for patients and families affected by rare neuromuscular diseases.
Why rare disease?
“Advancing treatments and awareness for rare diseases is a passion I developed from profound personal and professional experiences working with individuals battling rare disease. While these are exciting times for scientific discovery and advancement, the stark reality remains that most patients have no effective treatments and time is short. My whys are these warrior patients and families and the urgent need for disease-modifying therapies that saves lives.”
Laura Quinn (Iliescu)
Manager, Patient Advocacy and Engagement
Laura brings more than 14 years of experience in the life sciences sector to her role at PRA. With experience spanning from biotech and pharmaceutical to clinical homecare and patient advocacy, Laura’s expertise encompasses every phase of the product lifecycle and is enriched by a deep understanding of the patient journey and her passion for working with patient advocates. Partnering with Global Genes, Laura launched RareUniversity, a free online education initiative designed to support and empower the global rare disease community. She is currently completing her MSc in Human Factors Engineering with a focus on researching and reshaping complex human-system interactions in healthcare.
Why rare disease?
“Rare patient advocates are an incredible force. They’re constantly pushing the boundaries to evolve science, healthcare, and the entire drug development ecosystem. It’s absolutely inspiring to partner with the rare community to help eliminate the challenges of rare disease in clinical research.”
Patient-focused clinical development happens with PRA.
PATIENTS
47,100+SITES
11,600+Studies
250+APPROVED DRUGS
18We understand the challenges and critical success factors necessary to conduct rare disease studies, and we have the experience to back it up. Our team has built mutually beneficial relationships with patients and patient advocacy groups to ensure your study has the best chance of success. We work with you to make sure you have a feasible study strategy that will contribute to commercial success, built off real patient insights and data. Because—when we all come together—we can make clinical trials an everyday part of patient treatment and bring life-saving treatment options to patients and their families.
Read how we’re accelerating rare disease clinical development.Practical tools for sponsors, participants and champions of patient-centric trial development.
More than 90% of the 7,000 rare diseases known to science don’t have a single FDA approved treatment. There’s no time to waste in bringing meaningful treatments to lives affected by rare disease.
This toolkit was developed to support Sponsors, Patient Communities, and other stakeholders to operationalize true patient centricity in clinical development.
Leveraging advanced analytics to enhance patient recruitment.
Our medical informatics allow us to qualify and quantify rare disease patients and treating physicians through public and private data sources. We use industry-leading third-party data sources, as well as claims, pharmacy, specialty lab, and diagnostic data and electronic health records. We also examine the competitive landscape and investigator performance. This evidence-based methodology, combined with our core set of study-branded tools, helps sponsors to identify sites, and sites to identify potential patients.
With the right technology, we can reach patients where they are and, ultimately, speed the development of life-changing drugs for rare indications.
Scott Schliebner, Senior Vice President, Center for Rare Diseases
Advanced Therapies for Rare Disease Patient Communities
Currently, 95% of rare diseases have no FDA-approved treatments, and many treatments that do exist are burdensome for patients. Advanced Therapies hold some of the greatest promise for durable relief from rare disease. A greater understanding of disease mechanisms and the technologies of cell therapy, vectored gene therapy, and gene editing candidate treatments have prompted an increase in Advanced Therapy clinical trials for rare disease patient communities.
PRA understands the unique regulatory, scientific, and operational considerations that are necessary to carefully design these trials. To meet this need, we’ve established the Gene Therapy Working Group: a multidisciplinary team of dedicated professionals from the Center for Rare Diseases, Regulatory Affairs, Scientific Affairs, Study Start-Up, Patient Strategy, Real World Solutions, Mobile Health Platform, Medical Affairs, Clinical Pharmacology, and Clinical Delivery. This group provides guidance and support to all phases of Advanced Therapy clinical trials in the rare disease space, including, but not limited to:
- Assessment of appropriate technology-based solutions (ie, wearables, virtual study visits, etc.)
- Integration of evolving global regulatory landscapes
- Analysis of competitive landscapes
- Patient Journey and Patient Pathway analyses
- Training for research sites and operational teams
- Protocol refinements
Reducing the patient burden of participation.
We incorporate the patient perspective into all we do, because our work is first and foremost for the patient. We’ve built partnerships with relevant patient advocacy groups to create stronger study awareness and support, and we practice social listening and conduct patient-focused feasibility and focus groups to better understand what patients want. Our recruitment and retention strategies include the voice of patients and their caregivers during study design and throughout the entire product development lifecycle.
Watch how we’re engaging patients throughout our studies.We are rare together.
When you come to PRA with a rare disease treatment or clinical research study, you can expect to work with us. We believe clinical research happens at the person-to-person level. That’s why we want you to know who we are, so you can trust in our partnership.
Rare changes everything. Let’s change that, together.Learn more about PRA's Rare Diseases Advisory Committee
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