We never forget that rare diseases alter entire lives—and we’re committed to changing that.

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Rare changes everything

At the Center for Rare Diseases, we never forget that rare diseases alter entire lives. We’re committed to changing that.

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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.

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Stay up to date on PRA’s Center for Rare Diseases, our achievements, and new initiatives

Rare Disease Newsletters

Every 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

Rare Team Schliebner Scott

Scott Schliebner, MPH

Senior Vice President, Center for Rare Diseases
Rare Team Raymond Amy

Amy Raymond, PhD

Director of Therapeutic Expertise
Rare Team Mills Juliane

Juliane K. Mills, MPH, MS

Director of Therapeutic Expertise
Rare Team James Davis Kendall

Kendall Davis, MPH

Manager of Patient Advocacy and Engagement
Rare Team Wessel Jessica

Jessica Wessel

Clinical Strategy Lead
Rare Team Drummond Jana

Jana Benesh, PhD

Clinical Strategy Lead
Iliescu Laura

Laura Quinn (Iliescu)

Manager, Patient Advocacy and Engagement

Patient-focused clinical development happens with PRA.

PATIENTS
47,100+

SITES
11,600+

Studies
250+

APPROVED DRUGS
18

We 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.

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

Learn how technology is reshaping the rare diseases landscape.

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
Learn more about Gene Therapy in regards to rare disease.

Reducing the patient burden of participation.

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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.
Download our Guiding Principles for Interactions with Rare Disease Patient Advocacy Organizations

Learn more about PRA's Rare Diseases Advisory Committee

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