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International Clinical Trials

The Formula for Success

In early 2019, the FDA released a revised draft guidance for rare disease clinical trials (1). The guidance, updated from a 2015 version, provided clarification in certain areas such as natural history studies, evaluation and validation of biomarkers as surrogate endpoints, evaluation of novel drug compounds, drug manufacturing, and safety. Well-received, the 2019 guidance is intended to encourage sponsors to tackle the complex issues associated with rare disease research proactively so that research can be conducted more effectively and efficiently.

Main Challenges

Rare diseases affect a small population of people who are dispersed across the world, and it makes country and site selection, clinical trial recruitment, and long-term retention particularly complex. Furthermore, many rare diseases are difficult to diagnose, and, as a result, a misdiagnosis can occur. Although more rare diseases are being identified through recent medical advancements and development of new diagnostic techniques, it typically takes time for the diagnostic and screening standards to be established. Obtaining an accurate diagnosis is often one of the first obstacles to overcome.

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About the authors

Dr Pavle Vukojevic, Chief Medical Officer, joined Pharm-Olam in 1997 and supports clinical operations with protocol development, therapeutic training, patient safety issues, study management, and feasibility support. Pavle received his MD in 1988 from the University of Belgrade, Serbia, and also has a Masterís degree in Immunology and a specialisation in Internal Medicine. He is a former President of the Association of Clinical Research Professionalís Serbia Chapter and was an assistant professor at the Medical School University of Belgrade for internal medicine/ rheumatology. He is the author of ĎConducting Clinical Trials in Europe: An Insiderís Analysisí, written for Insight Pharma Reports.

Dr Milan Marinkov has been with Pharm- Olam since 2013 and is currently Senior Medical Director. Milan is the leading medic and head of the Rare and Orphan Disease Center of Excellence, responsible for the evaluation, medical management, and conduct of multiple global studies within the rare disease study portfolio. He received his medical degree from the University of Novi Sad, Serbia, and is currently engaged in PhD studies.

Dr Jovana Vlajkovic-Josic, project management professional, joined Pharm-Olam in 2004 and is currently Associate Medical Director with experience in both medical and project management departments. As a licensed physician with postgraduate immunology training, Jovanaís orphan clinical trial experience includes a broad range of indications and phases.
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