Rare Disease clinical research studies pose a different challenge in terms of project management, site selection and patient enrollment and retention. Rare disease – defined in the US as 1 in 200,000 patients – demand a full-service CRO with innovative strategies to manage a rare disease program. In particular, Rare Disease clinical research studies demand a full-service CRO with expertise in enrolling rare disease patients and maximizing site selection to create realistic study feasibility.
Putting patients first is never more important than when dealing with rare diseases. Because of our years of helping clinical development thrive in this area, we can help you navigate this specific landscape. By putting ourselves in patients’ shoes, we help you develop an approach that is laser-focused on making life easier for everyone involved.
Designing protocols and executing study design for scarce patient populations including pediatric patients who comprise 50% of these populations require medical doctors and pediatricians with specialized expertise. In addition, study design inclusive of a late phase component with access to Rare Disease patient registries is a critical tool.
- Site relationships with access to patient registries to drive patient enrollment
- Broad understanding of patient advocacy issues and organizations for support
- Physicians with Pediatric experience to understand the study design and complexity involved
- Global regulatory understanding and consultation regarding the unique environment for Rare Disease studies
BBMCT’s Medical and Scientific teams give you a single point of contact to access our global capabilities. With every project, we match you with the therapeutic area, regulatory and clinical operations experts who are the best fit for your development project.
By understanding the specific needs of families coping with rare diseases, our in-house Patient Recruitment and Strategy Group can give you solutions to help ease the burden on them. We work closely with patient advocacy groups to constantly learn and grow.
We have more than 1000 regulatory consultants on staff. Of these, more than 50 have held positions at regulatory authorities across the world. That’s why we are ready to advise you on your strategies for product development, compliance, submissions and more. Our regulatory team works closely with our clinical and operational teams for an integrated approach, informing your studies every step of the way.