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Acquired Thrombotic Thrombocytopenic Purpura (aTTP) is a rare disease that, when left untreated leads to a 95% morbidity rate. We executed the Phase III study which lead to the marketed drug approval.
Acquired Thrombotic Thrombocytopenic Purpura (aTTP) is a rare disease that, when left untreated leads to a 95% morbidity rate. We executed the Phase III study which lead to the marketed drug approval.
For a Rare Disease study to succeed, you need well-qualified people at the helm. At Pharm-Olam, we maintain specialized staff with deep expertise in Rare Disease as well as experience working in regulations from the US, EU, and beyond. We have provided CRO services for 14 drug approvals, and roughly 88% of our PMs and 92% of CRAs have Rare Disease experience.
As new drugs are developed and treatments discovered, we can understand the underlying mechanisms of rare diseases better. To this end, our study execution repository for Rare Disease is continually evolving. We capture lessons learned from our clinical studies to leverage insights and develop tools to improve rare disease study execution and decision making. Every study is unique, every lesson valuable.
Rare disease clinical trials often require numerous supportive vendors. In rare diseases it is important to bring the research to the patients where they are. Pharm-Olam has built several relationships over the years with vendors that can support this effort, vendors like, home nursing, patient concierge services, etc. Together we go the distance for our patients and sites.
Natural history studies help sponsors understand their targeted rare disease. Pharm-Olam has extensive experience running natural history studies in rare and ultra-rare diseases to support sponsor data collection and program development. We are experienced in finding the right sites, right patients, and collecting clean data for analysis.
In this guide, we discuss several challenges that researchers must confront when conducting clinical research in rare disease and outlines best practices for a successful study.
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A rare disease article series focusing on rare disease research today and its future as clinical research has grown in leaps and bounds.
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A Pharm-Olam case study of a Phase I/II pediatric Respiratory Distress Syndrome neonate study with a gestational age between 27 and 33 weeks.
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