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Lecture

Clinical Trials: Phases and Challenges

Description

This lecture explains the different phases of clinical trials, from safety testing to evaluating clinical benefits, highlighting the challenges faced in rare diseases like SMA. It also discusses the statistical evaluation of benefits and the ethical considerations in placebo-controlled trials.

In MOOC

Neuroscience Reconstructed: Genetics and Brain Development

Official source

Ontological neighbourhood

Statistics

Data collection: Experiment

Related concepts (32)

Placebo

A placebo (pləˈsiːboʊ ) is a substance or treatment which is designed to have no therapeutic value. Common placebos include inert tablets (like sugar pills), inert injections (like saline), sham surgery, and other procedures. In general, placebos can affect how patients perceive their condition and encourage the body's chemical processes for relieving pain and a few other symptoms, but have no impact on the disease itself.

Clinical trial

Clinical trials are prospective biomedical or behavioral research studies on human participants designed to answer specific questions about biomedical or behavioral interventions, including new treatments (such as novel vaccines, drugs, dietary choices, dietary supplements, and medical devices) and known interventions that warrant further study and comparison. Clinical trials generate data on dosage, safety and efficacy. They are conducted only after they have received health authority/ethics committee approval in the country where approval of the therapy is sought.

Placebo-controlled study

Placebo-controlled studies are a way of testing a medical therapy in which, in addition to a group of subjects that receives the treatment to be evaluated, a separate control group receives a sham "placebo" treatment which is specifically designed to have no real effect. Placebos are most commonly used in blinded trials, where subjects do not know whether they are receiving real or placebo treatment. Often, there is also a further "natural history" group that does not receive any treatment at all.

Treatment and control groups

In the design of experiments, hypotheses are applied to experimental units in a treatment group. In comparative experiments, members of a control group receive a standard treatment, a placebo, or no treatment at all. There may be more than one treatment group, more than one control group, or both. A placebo control group can be used to support a double-blind study, in which some subjects are given an ineffective treatment (in medical studies typically a sugar pill) to minimize differences in the experiences of subjects in the different groups; this is done in a way that ensures no participant in the experiment (subject or experimenter) knows to which group each subject belongs.

Scientific control

Informal improvements in any process or enquiry have been made by comparison between what was done previously and the new method for thousands of years. A scientific control is a modern formal experiment or observation designed to minimize the effects of variables other than the independent variable (i.e. confounding variables). This increases the reliability of the results, often through a comparison between control measurements and the other measurements. Scientific controls are a part of the scientific method.

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