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concerns regarding treatment-emergent suicidality have prompted the US Food and Drug Administration (FDA) to issue draft guidance for prospective assessment of suicidal ideation and behaviour in clinical trials.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.
Alison Turner, Principal Scientist at UCB (Celltech), explains in this detailed article how in the process of antibody candidate selection it is important that during manufacture and shelf-life the antibody shows aggregation stability. Hence it is necessary to be able to measure and predict the propensity of aggregation of different antibody molecules in a pre-screening assay.
Shirley Johansson of Midfield Media estimates that 6%-10% of the world’s population will suffer from a rare disease at one point in their life, and approximately one out of five people personally know an individual suffering from a rare disease. She goes on to explain that finding a cure or improved treatments for such patients is fundamental, not only to their lives, but also to society as a whole. The journey has begun, and there is great potential to improve the work amongst all stakeholders: patient organisations, governmental authorities, health technology assessment organisations, payers, and orphan drug developers. On 14th–16th September 2011, many of the key stakeholders will be represented at the Orphan Drugs Summit in Copenhagen, a hub for pharmaceutical development, to discuss challenges and alternatives to support the fight against rare diseases.
Ioannis Papasotiriou explains in this article that RGCC Ltd is a leading company in analysis of circulating tumour cells (CTCs) as well as cancer stem cells (CSCs). Through their analysis, the organisation is able to offer services in the clinical field as well as in R&D in the pharmaceutical industry. By using the most advanced and innovative technologies of molecular and cellular biology, RGCC Ltd manages to overpass several restrictions and difficulties that the analysis of CTCs and CSCs involve. Through such an approach a massive amount of information and data has been generated in order to be used for identifying new «druggable» targets as well as offering methods in clinical practice like new and precise assays, risk scale and classification of cancer patients.
A fundamental of drug development is the provision of safe and effective medicines. The safety and efficacy of a drug can only be demonstrated through large-scale trials in humans. However, as Emma Sceats, Business Development Manager at Zyoxel Ltd explains, using humans as test subjects for drugs whose safety is unknown would be unethical. Instead, legal and regulatory guidelines require a plethora of studies to be conducted prior to clinical trials, so that all reasonable steps have been taken to mitigate risk to trial participants. Drug developers are also expected to give evidence of the expected effectiveness of the drug for treating human disease. Scientists have therefore needed to develop non-human models that can help to predict how patients will respond to a drug.
Nicola Gaskell, Client Manager in Bioanalytical Sciences at Quotient Bioresearch, explains that this article provides a current overview of how high throughput diagnostic analyser platforms and research-based analysis within a regulatory environment can be used to seamlessly deliver high quality data to the pharmaceutical industry, helping to drive the drug development process for the pharmaceutical industry.
Is the insect hemolymph-fat body-Malpighian-metabolising-excretion system a relevant model for highly efficient documentation of some key ADMET parameters in the early drug discovery phase? Peter Aadal Nielsen, CEO at EntomoPharm, and Gunnar Andersson, CSO at EntomoPharm, answer this question and direct us to how in the future insect models could potentially be used as a filter between in vitro and in vivo models, filling the gap between the ‘quick’ in vitro models and the ‘slow, expensive but more reliable’ in vivo models.