Clinical Trials / Orphan Drugs

Rare diseases impact either a single system within the body or several at once, thus causing challenges in monitoring its progress. We leverage insights gleaned from the execution of over 120 rare disease clinical trials to inform the creation and execution of new research.

Our Integrated Full Service Rare Disease Solution covers the gamut of rare disease and orphan drug research, including natural history studies, pivotal trials, complex clinical trial considerations such as pediatric study populations, new biomarkers, surrogate endpoints, and the assimilation of patient perspectives and experiences. We devise novel medicines and advanced therapies for a variety of rare and ultra-rare diseases.

The success of your study depends upon selecting a team with the appropriate experience in your chosen therapeutic area. We have specialized in rare and orphan disease research for more than two decades. In the course of conducting more than 120 rare disease trials we have gained sixteen product approvals, covering a range of novel therapy classes and precision treatments such as gene therapy, GMO, stem cell therapy, and mRNA and antisense therapy.

The expertise you need

prophema’s staff has deep knowledge of rare disease research and wide experience in dealing with regulators from the US, EU and other regions. More than 90% of our PMs and CRAs have rare disease experience.

Comprehensive Knowledge Base

We maintain a knowledge base whose insights power clinical research into the most complex rare diseases and orphan drug development context. The knowledge base covers subjects such as protocol design, trial planning and development, regulatory strategies for new clinical trials, novel treatments for the rarest of diseases and patient mapping for swift study initiation. All these insights ultimately help you achieve your first-patient-in goal that much faster.