Betsy Hughes-Formella, the Director of Business Development and Consulting at Bioskin GmbH, explains that, in dermatological models, discrete evaluation of treatment effects may be possible following simultaneous application of multiple formulations/actives to small treatment areas in parallel in one individual, without risk of cross-over effects. By making intra-individual comparisons between treatments, the inter-subject variability is reduced, allowing meaningful interpretation of results with smaller panel sizes. Further, in these models non-invasive biophysical measurement and imaging methods to monitor skin function and structure provide alternative objective endpoints to support clinical evaluation, delivering additional information on structural and functional changes in the skin.
Thomas Zwingers, Chief Operating Officer for CROS NT, explains that, in the past few years, we have witnessed an exponential increase in costs for research and development of new cancer drugs: it has been estimated that the average expenditure in R&D for new drugs has doubled in the last decade. Consequently, the question pharmaceutical companies continue to face is: “How can we make a study more efficient while maintaining the validity and integrity?” Sponsors, clinical researchers and biostatisticians are becoming more interested in designs with greater flexibility than the standards and procedures that anticipate “go/no-go” decisions.
Traditionally, clinical trial application (CTA) approval in EU member states has been subject to national legislation. As a result, the assessment of a CTA that was filed simultaneously in several member states often resulted in varying final decisions and unnecessary delays. Country-specific modifications to the application often occurred due to changes requested by the different competent authorities and ethics committees. In some cases, a clinical trial might even be approved in one member state and rejected in another. The entire procedure could be extremely time-consuming and the country-specific modifications risk jeopardising the scientific value of clinical trial results. Dr Franz Josef Buchholzer, Vice President, Regulatory Operations Worldwide at PharmaNet, concludes in this article that in Europe, the voluntary harmonisation procedure (VHP) is a new method to obtain clinical trial approval across multiple European countries in a timely manner. In comparison to the traditional standard national submissions, which must be completed in parallel and submitted to each of the different European agencies, the VHP consolidates these activities into a single submission.
Michael Federico, Vice President at ePRO, ERT Inc., states in this article that increasing concerns regarding treatment-emergent suicidality have prompted the US Food and Drug Administration (FDA) to issue draft guidance for prospective assessment of suicidal ideation and behaviour in clinical trials. The Columbia-Suicide Severity Rating Scale (C-SSRS), a free-form clinician-administered interview, is an accepted instrument for meeting this requirement. However, procedural variance in the way this and all clinical assessments are performed by human raters has been a shortcoming for many years, negatively impacting the reliability of results. Electronic patient-reported outcome (ePRO) solutions can effectively address this limitation.
Valerie Harding, Quanticate’s Communications Account Director, explains that Phase I marks a significant milestone in the development of any new medicinal product. A target has been identified, a compound has been discovered that hits the target, and it has been refined to ensure it has good properties for development. In-vivo safety pharmacology and toxicology studies have been conducted and the compound has been tested in pre-clinical models of the disease. This process will have taken many years. A multi-disciplinary team has evaluated all the data, and decided to take the plunge and invest in clinical development. And now, the drug is ready to be tested in humans.