14th Partnerships in Clinical Trials

Building a pharmaceutical development program for the treatment of a rare disease is a monumental task. You know the challenges: poor understanding of the natural history due to few investigators studying the disease, heterogeneous patient populations with variable phenotypes and clinical courses, geographic dispersion of patients and investigators, regulatory uncertainties, and lack of prior clinical studies to establish a template for study execution. In addition, small patient populations challenge traditional methods of study operation. With >7000 rare diseases, each exhibiting diverse symptomatology, the key is expertise rather than disease-specific experience.
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Biosimilar development begins with state-of-the-art analytics to evaluate multiple batches of the originator medicinal product and select the most appropriate batch for biosimilar reference (heterogeneity between batches from the same manufacturing process is common due to biopharmaceutical complexity). Any changes to the originator marketing authorization should be reviewed, as product amendments may have altered manufacturing processes and affected product performance. Following the appropriate preclinical and comparability tests, a stepwise approach is usually employed.
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