Simplified clinical trials

Therapeutic use trials are conducted once a drug has been approved to gain additional information about the safety and efficacy, or effectiveness, of the drug The term "effectiveness" is used to describe how well the drug works in patients once the drug has been approved. One example of this type of trial is the simplified clinical trial (SCT). The intent of the word simplified... 

The introduction in clinical practice of Hib conjugate vaccines in the nineties further changed the situation. The demand for a new Hib conjugate vaccine increased, but that demand was mainly concentrated in developing countries with no rewards for large multinational companies. On the other hand, the accumulated knowledge simplified clinical trials. 

In this form, event would be replaced by some clinical finding such as myocardial infarction, stroke, seizure, or the like. This example form is extremely simplified, as there are usually a number of associated data variables captured as well. The event/endpoint page data must be clean, because it likely captures the primary efficacy data for the clinical trial. 

In some cases, the concept of a large, simple clinical trial has gained favour. This trial type would only be considered in cases where the intervention is easily administered and the outcome is easily measured. In such instances, simplified trial criteria are enforced, with the major prerequisites being recruitment of relatively large numbers of participants and randomization. 

L-asparaginase. Several pharmaceutical firms have developed greatly simplified purification schemes making it feasible to produce large amounts of highly purified enzyme for clinical trials 62-65). 

Figure 13-15. A simplified/generic representation of clinical trials involving Phases I (safety and tolerability in healthy participants and/or patients), II (to evalnate effectiveness of the drug in patients), and III (to perform expanded controlled and uncontrolled trials and gather benefit-risk data). About 70-90% of drugs entering Phase III studies successfully complete this phase of evaluation. For more details see the glossary at www.clinicaltrials.gov.

The regulatory procedure involved is a so-called Clinical Trial Authorization appH-cation (CTA) and consists, in addition to a Clinical Trial Protocol (CTP) and Investigator s Brochure (IB), of an Investigational Medicinal Product Dossier (IMPD) in CTD format. The IMPD consists of quality data, nonclinical pharmacology and toxicology data, clinical trial and previous human experience data, and an overall risk-benefit assessment. A copy of the CTA is sent to each EU Member State where the study is conducted, as well as to the Ethics Committees for approval. These also have to be notified in case of any amendments, study termination and adverse reactions. The contents of the IMPD are case specific and may be simplified or even replaced by the... 

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