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Drugs: From Research Lab to Pharmacy Shelf

„Before a medication is available in pharmacies it has come a long way. In all it takes approximately 12 years and a total spending of $600 million. “

Subsequent to the finding of new biological or chemical substances, new combinations of substances or usage potentialities for known substances, the most promising substances are chosen and tested in a research lab.

After a successful testing of the drug, first data is available on tolerability, safety and dosages. Based on this data one can detect how a certain form of the later drug can be applied on humans for a successful treatment of the disease. These laboratory tests are considered as "pre-clinical testing”. This phase can take up to a decade.

Only substances that are promising, and as far as is known from the pre-trials, have proven to be safe, can be applied to study participants/volunteers. The substances to be tested - as well as any comparison substances, whose effectiveness is already known - will be incorporated in a certain form of medication, such as drops or capsules. These are the investigational substances for a clinical trial.

Before the beginning of a trial a documentation of the state of the scientific knowledge about the -to be tested substance- is documented for each clinical trial in a detailed study protocol.Investigators are obliged to conduct every study, according to the prescribed guidelines. The study protocol is presented to the Research Ethics Board, as well as documents about patient education and patient consent forms.

Clinical trials take place in the following 4 phases:

Phase 1, trials with healthy volunteers:

Phase 1 trials are conducted in healthy volunteer subjects, most of the time. It is intended to gather findings about the characterisation of a drug's absorption, distribution, metabolism, and excretion. These types of studies are referred to as "pharmacological studies ". The sum of the results describe the so called Pharmacokinetics of the drug. If phase I is completed successfully and the side effects of the drug are not too grave, phase 2 will follow.

Phase 2, trials with patients:

Phase 2 trials are conducted with patients.Phase 2 trials are conducted with patients. These controlled clinical trials are conducted to gain a first evaluation of the therapeutic efficacy. Some trials aim at determining the dosage in phase 2, however this may vary from study to study, depending on the indication and other factors. Phase 2 studies are referred to as „therapeutic exploratory studies“ and the results of these studies allow a first assessment of the „Pharmacodynamics“ of the drug and identifying common short-term side effects and risks Only if phase I and II are completed successfully and the side effects of the drug are acceptable, phase 3 will follow.

Phase 3, trials with large patient groups:

Phase 3 studies are conducted with large patient groups (2000-5000 patients). During this phase drugs are often compared with the current 'gold standard' treatment and assessed in long term tests, if the drug is meant for long term use. Phase 3 studies are referred to as "therapeutic confirmatory " and have to demonstrate, or confirm therapeutic benefit Phase 3 studies are conducted with large patient groups (2000-5000 patients). During this phase drugs are often compared with the current 'gold standard' treatment and assessed in long term tests, if the drug is meant for long term use. Phase 3 studies are referred to as "therapeutic confirmatory " and have to demonstrate, or confirm therapeutic benefit aswell as collect information that will allow the experimental drug or treatment to be used safely.

Authorization after phase 1- 3:

All drugs have to be authorized for sale by the FDA (Food and Drug Administration). “Under current law, all new drugs need proof that they meet standards of quality and are effective and safe for their intended use before they can be approved for marketing. No drug is absolutely safe; there is always some risk of an adverse reaction. FDA decides—after a thorough evaluation—whether the studies submitted by the drug's sponsor (usually the manufacturer) show it to be safe and effective for its intended use. When a proposed drug's benefits outweigh known risks, FDA considers it safe enough to approve. Once a drug gets FDA approval, the drug is on the market as soon as the firm gets its production and distribution systems going.”

Phase 4, Post-Market Surveillance:

After a drug is authorized for sale, phase 4 studies are conducted. These studies are performed to gain knowledge about the therapeutic application in daily routine and to provide a broad assessment about the benefits and risks of a drug.

What are case series (clinical series)?

Besides the 4 phases of clinical trials, pharmaceutical companies also perform so called case series. In doing so the application of an approved drug or medical device is observed in clinical practice and everyday life. The objective of the case series is to review drug effects after the introduction to the market and to win further scientific knowledge.

Review of the effectiveness compared to standard therapy or placebos:

The efficacy and tolerability of a new drug is compared to existing authorized drugs or treatments (standard therapy), because an effective therapy should not be denied to patients for ethical reasons. This ensures that the disease is dealt with in any case.

If there is no alternative standard therapy, the drug is tested against a placebo, to be able to differentiate real drug reactions from independent effects (due to placebo, self-healing, etc.). Prior to conducting a placebo-controlled clinical study, it will be carefully examined whether it is ethical.

During a clinical trial, patients regularly see their investigator in the hospital or medical practice. During these appointments, they are examined by a doctor and interviewed about the efficacy and potential side effects of the drug. Sometimes the study participants are asked, to keep a kind of diary about their state of health.

After completion of the study period, the results of the treatment groups (efficacy and safety) are compared with each other, analyzed, evaluated and published.