Patient safety assessment clinic

Phase II studies encompass a detailed assessment of the compound s safety and efficacy in a larger patient population (a few-to-several hundreds of patients). It is important that any formulation selected for these studies must be based on sound biopharmaceutical and pharmaceutical technology principles. Phase III clinical studies, also referred to as pivotal studies, involve several thousands of patients in multiple clinical centers, which are often in multiple countries. The aim of these studies is to demonstrate long-term efficacy and safety of the drug. Since these studies are vital in the approval of the drug, the dosage form plays a very critical role. 

Approved clinical investigations follow three phases. In Phase 1, about 100 to 200 people are exposed to the drug to determine the tolerance, absorption, excretion, half-life, and other pharmacologic reactions the preferred route of administration and the safe dosage. In Phase 2, initial trials are conducted on 500 to 1000 patients to assess the treatment or prevention of the specific disease. Additional animal studies to indicate safety may be conducted concurrently. If these preliminary studies demonstrate sufficient promise. Phase 3 clinical trials are performed with several thousand patients. 

In October 2005, Osiris received approval from the US Food and Drug Administration (FDA) to conduct a non-randomized, open-label, phase II clinical trial in adult and pediatric patients with treatment-refractory severe GVHD [628422]. By November 2005 the trial had begun, enrolling 30 patients and setting a planned completion date of January 2007. At the same time, a second phase II, double-blind, randomized, placebo-controlled clinical trial, expected to enroll 75 patients to assess the safety and efficacy of OTI-010 in acute gastrointestinal GVHD, was initiated by Osiris, with an expected completion date of April 2008 [www.clinicaltrials.gov], [632720]. 

Clinical immunization safety assessment centers The National Immunization Program of the Centers for Disease Control and Prevention (CDC), Atlanta, GA, USA, is trying to set up a network of Clinical Immunization Safety Assessment Centers (CISA). Based on standardized clinical evaluation protocols the centers will assist health care providers in evaluating patients who may have had an adverse reaction after immunization. Furthermore, the centers will evaluate newly hypothesized syndromes or events identified through the routine VAERS (28). 

Misconduct or fraud is a rare occurrence in clinical research, but when misconduct or fraud is confirmed the consequences can be disastrous (Lock et al., 2001 Eichenwald and Kolata, 2004). Fraudulent practices in clinical trials can lead to trial subjects being exposed to safety risks, to submitted or published clinical data being j eopardized and, if the product has been licensed based on false data, this may result in compromised patient safety. Therefore, any suspected case of misconduct or fraud should be taken seriously and be assessed -this is when QA auditors should be involved. 

Long-term trials have now to be conducted and meet international standards, the Extent of Population Exposure to Assess Clinical Safety (it was difficult in the past to obtain long-term data). Some open phase III trials might be added to study particular patient subgroups, for example the elderly, or a specific subgroup of the disease. 

The primary study end-point was incidence of DVT and other thromboembolic events assessed clinically and by both locally and centrally assessed ultrasonography. The duplex-ultrasounds of the lower extremities were used to confirm or exclude the occurrence of DVT. These procedures were performed and interpreted by qualified specialists within the same hospi-tal/institution on a real-time basis for the timely and appropriate chnical care of the patient Furthermore, duplex ultrasound studies were videotaped for a subsequent standardized bhnded interpretation by a quahfied, independent laboratory that provided an unbiased evaluation of the incidence of DVT. ThromboemboHc events other than DVT that occurred during the study period were assessed, and the investigator estabhshed the clinical relationship of the event to treatment with rhAT. Secondary end-points were safety, adverse events and immunogenicity. 

Optimal therapeutic decisions are based on an evaluation of the patient and assessment of the evidence for efficacy and safety of treatment. Therapy must be guided by an understanding of drug pharmacokinetics and pharmacodynamics that is integrated with patient-focused information. WeU-designed and executed clinical trials provide the scientific evidence that informs most therapeutic... 

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