SUNRISE, BATMAN and GEMSTONE, who comes up with these names?
by Lim Guan Yu
Clinical trials are research studies conducted to investigate potential treatments such as a new drug compound and its effect on humans.1
Before a new treatment is tried in humans, extensive laboratory testing and animal testing are done to identify promising compounds and toxicological (safety) tests to determine possible risks. The treatment with the most promising results is then moved into clinical trials.
Christian Dittrich, Professor of Medicine at the Vienna University School of Medicine, member of the European Society for Medical Oncology (ESMO) where he served as Chair of the Principles of Clinical Trials and Systemic Therapy Faculty group, shares the reason for the need of clinical trials, in oncology.
For instance, the prediction of anti-tumour activity and toxicity based on experiments in cell culture (in vitro) and in animals (in vivo) for the situation in humans, has inherent limitations because of differences between cell cultures and animals on the one side and the complexity of humans on the other. Toxicity seen in patients may have not been detected in animals before, hence the only scientifically sound way of generating convincing evidence are clinical trials.
Practically, clinical trials are the only tools to find out whether a new drug or therapy contributes to the most important goals of prolonging overall survival, survival without progression of the disease, or improving quality of life in cancer patients.
Clinical trials help translate results from basic scientific research and promote an evidence-based approach to healthcare leading to better health outcomes. The results of clinical trials can make a difference in the care of patients by providing information about the benefits and risks of new therapeutic, preventative, or diagnostic products or interventions.1
Dittrich says it is important to have well educated and prepared doctors to leverage this powerful tool to develop better, safer therapies for patients. In order to bridge this gap, ESMO, in partnership with the EORTC, ECCO and AACR, organises the 21st Methods in Clinical Cancer Research (MCCR) Workshop,2 designed to educate and train early-career investigators in the best practices of clinical trial design.
Academic vs Non-Academic
Dittrich explains that clinical trials can be divided into academic and non-academic ones. From the regulatory position, there is no difference, but they mainly have different goals:
- Non-academic trials are mostly sponsored by the pharmaceutical industry or at a smaller extent by developers of medical devices. These serve the development of novel drugs, biomarkers, or devices, respectively, to get approval by the regulatory bodies and thus, marketing authorisation for further market developments.
- Academic trials are sponsored by scientific societies, universities, research groups, or governmental organisations. They aim at improving a variety of facets of the global healthcare situation. Many gaps exist in evidence-based recommendations, because many of the unanswered questions are of little or no interest to companies, hence they can be addressed only by academia. Rare cancers, including childhood cancers are such examples: in consideration of the low number of patients which – at least in the past – often represented a limit to companies’ capacity and readiness, to invest in research and develop drugs in these areas.
Academic research may profit from the advantage of existing pre-competitive information in the form of quality-controlled biobanks on molecularly completely worked-up patient tumour samples and the corresponding patient data.
Regulations
In developing new drugs, Dittrich says with specific reference to anti-tumour drugs, that North America, Europe and Japan are the most active regions. The respective regulatory bodies have agreed upon a set of Guidelines, based on the International Conference on Harmonisation (ICH) of Technical Requirements for the Registration of Pharmaceuticals for Human Use.
Although the national regulatory bodies, namely the EMA (European Medicines Agency), the U.S.-FDA (Food and Drug Administration) and Japan’s PMDA (Pharmaceuticals and Medical Devices Agency) are independent from each other with regard to their decisions, they have established a mechanism for reciprocal consultation.
All procedures performed in clinical trials involving human participants have to be in accordance with the ethical standards of the 1964 Helsinki Declaration of the World Medical Association (WMA)3 and its later amendments.
Clinical Trial Registry
According to the World Health Organization (WHO), the number of clinical trials has increased across all regions.4 The number of trials listed in the WHO International Clinical Trials Registry Platform (ICTRP) reported that Americas had 1,330 trials in the year 1999, which increased to 11,013 in 2018. Europe also saw an increase from 541 trials in 1999 to 11,291 in 2018. This trend was seen across all other regions.
Clinical trial registration has greatly improved transparency in clinical trial research. WHO ICTRP is one of the many clinical trial registries out there. Another example is ClinicalTrials.gov, an online clinical trial registry that is provided by the U.S. National Library of Medicine (NLM) and consist of 306,911 research studies in the U.S. and 210 other countries.5
Because clinical trials often have very long names, for them to appear on the clinical trial registry, they are often required by the respective regulatory bodies (e.g. U.S. Food and Drug Administration) to provide a brief title to the trial.
In the Name
Who comes up with the convenient and sometimes amusing names and acronyms to identify these trials? Professor Andrew Nunn, senior scientist at the Medical Research Council’s Clinical Trials Unit at University College London and also the co-chief investigator for STREAM says different members of the trial team may do this; in the case of STREAM it was the clinical project manager. Dittrich added it is up to the sponsor or funding entity of a study to choose the name or acronym of a clinical trial.
We share some examples. Some of the studies are in progress, completed or have been terminated. [APBN]
