Clinical trials research questions

While clinical trials research incorporates many different components, the focus of this chapter is limited to study questions associated with human performance capacity variables and their measurement... 

Toxicity Amelioration. Cancer researchers traditionally have not focused their attention on the question of toxicity amehoration. This is partiy attributed to the lack of predictive animal models for human toxicities. For example, the preclinical rat model, used as a predictor of myelosuppression, has failed to predict myelosuppression in humans in clinical trials. In addition, reduction of one toxicity may result in the emergence of another, more serious problem. Research efforts to address the problem of toxicity amelioration has progressed in several directions. The three most prominent areas are analogue synthesis, chemoprotection, and dmg targeting. 

All clinical trials should have a pre-specified research question, which may be stated in the form of a primary hypothesis (or possibly a few primary hypotheses). An objective outcome measure or measures should also be clearly identified, such as the results of a biochemical test or the score on a validated scale. This allows statistical tests to be applied in order to assess the likelihood that any differences in response between treatment groups resulted from the active treatment and were not due to chance. 

The cost of completing a clinical trial to answer a particular research question will depend on a number of factors including ... 

Sometimes you may also see quality-of-life (QOL) data collected for your clinical trial. Quality-of-life data are collected to measure the overall physical and mental well-being of a patient. These data are usually collected with a multiple-question patient questionnaire and may be summed up into an aggregate patient score for analysis. Some commonly used quality-of-life questionnaires are the SF-36 and SF-12 Health Survey, but there are quite a few disease-specific QOL questionnaires available to clinical researchers. 

The most common answer to this question is that the researchers fudge the data - that the doctors who assess the patients levels of depression rate them as being more depressed than they actually are, so that they will qualify to be enrolled in the trial. It can be difficult to find enough patients for a clinical trial, and the doctors may be paid for each patient they enrol. So they can be under considerable pressure to qualify as many patients as they can. 

The basic ethical questions raised by clinical research should never be underestimated. The pharmaceutical physician will need to be aware that failure, intentionally or because of misguided enthusiasm, to protect the health and well-being of each study subject can have very serious consequences. In an age where the medical profession is constantly under scrutiny, the drug industry is heavily criticised and the communication industry extremely active, mistakes in clinical trials are pimished. Therefore, before a study is commenced, a review should be made that the scientific approach is current, the motivation is clear, the processes are imambiguous, and there should be sufficient data to judge the safety and effectiveness of the interventions proposed. 

Fundamental healthcare issues may also be involved in the situation of policy-driven therapeutic research. The future investment of society in health may require a large-scale national clinical trial to answer questions relating to the prevention of disease or premature death. This type of clinical trial will not simply relate to one manufacturer s product. As such, it is preferable that the trial is organised on a national level, not necessarily with the exclusion of the company(s) involved. An independent data-monitoring committee (IDMC) should oversee the clinical trial with as much support from the pharmaceutical company(s) as possible. The clinical data collected belongs to the state and should be treated with the same quality standards as any pharmaceutical company sponsored study. 

Clinicians, families, patients, and policy makers can be faced with various questions about the pharmacological treatment of children and adolescents with mental illness. The following are some types of questions that are often formulated into research hypotheses for clinical trials ... 

Every clinical trial is carefully designed to answer certain research questions. A trial plan called a protocol maps out what study procedures will be done, by whom, and why. Products are often tested to see how they compare to standard treatments or to no treatment. The FDA often provides extensive technical assistance to researchers conducting clinical trials, helping them design better trials that can characterize effects of a new product more efficiently, while reducing risks to those participating in the trials. 

The bottom line Know and understand the different types of trials, which questions to ask, and your rights as a trial participant. Find out what risks there may be, and determine what level of risk you are willing to accept before you agree to enroll in a clinical trial for medical research. 

---