Clinical Trials. Importance in future therapies. What are the Requirements to Produce New Drugs? Drug must work significantly better than a control treatment
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Importance in future therapies
All clinical trials are prospective studies in which individuals are exposed (or not) and followed for an outcome (or a few different outcomes). The outcomes must be clearly defined.
1. Internal Review Board (IRB). Forms need to be filed with the IRB. A committee determines whether the study is ethical. The committee must include some lay people as well as scientists. It should contain an "ethics expert," such as a clergy-person.
3. On site Patient Monitoring and Data collection
4. Data analysis, write results and conclusions
5. Written report: includes clinical and statistical sections
In actuality, a placebo effect is a psychosomatic effect brought about by relief of fears, anxiety or stress because of study participation.
It's not just the little white pill that brings about the effect; it's the additional attention and the belief that your condition might be being treated with a superior new treatment.
All outcomes affected by psychosomatics are prone to placebo effects.
A component of every specific treatment effect can be attributed to the placebo response.
The question that a study should be asking is whether the treatment has any effect on outcome aside from the stress-relieving effect of study participation.
If the outcome can conceivably be affected by patient or investigator expectations, then blinding is important.
Filed prior to beginning clinical trials
NDA: New drug application
Filed after pivotal trials to get drug (device) approval
Major drug companies pay a fee of $350,000 when an NDA is submitted. The money is spent to hire a qualified person to review the NDA. Drugs are now approved in less than a year.
Phase 1: Small studies conducted in healthy volunteers. These studies are usually uncontrolled and open labeled.
1. Initial tolerability and safety
5. Bioequivalence studies (these are usually double-blind crossover studies)
6. Food interaction/drug interaction studies
Phase 2. Small to moderate sized trials (usually controlled double or triple blinded) studies inpatients.
1. Safety and tolerability
2. Preliminary efficacy. These trials are done with 80% power.
3. Dose-ranging. Find the dose that produces the optimal outcome.
Two trials with sample size adequate to determine a clinically important difference with 95% power or three trials with sample size adequate to determine a clinically important difference with 90% power are required.
For things like blood pressure or cholesterol, sample sizes most often are in the vicinity of 300-600 per trial (150 to 300 per treatment group).
For a drug (does not apply to vaccines), if all trials show a significantly greater effect then placebo, the drug is considered efficacious. The magnitude of the effect does not matter.