Patients targeting

Where compassionate use is envisaged, article 83.4 states that the Committee for Medicinal Products for Human Use (acting on behalf of the agency) may, after consulting the manufacturer or applicant, adopt opinions on various matters. The matters to be covered are the conditions for use, the conditions for distribution and the patients targeted. The opinions must be updated on a regular basis. 

Phase III Assess safety and effectiveness in a larger patient population Large number (1000-3000) patients targeted 3 yrs... 

Safety/tolerability Efficacy, find Pivotal trials and human PK in dose, safety patients target healthy volunteers in patients indication... 

One limitation of enzyme replacement therapy is the targeting of enzyme proteins to appropriate sites of substrate accumulation. Administration of a cholesterol esterase conjugated to albumin results in the degradation of pathologic cholesterol ester accumulations within the lysosomes of fibroblasts from a patient with cholesterol ester storage disease (246). 

There are also differing standards between the markets in each of these countries. For instance, the telecom market has vastly different safety requirements than the patient-contact medical market. So, it is important to determine the target market early in the product design process. These market differences will also be included in the lEC standards harmonizing efforts. 

Amyloid precursor protein (APP) is the precursor of (3-amyloid, the main component of senile plaques found in the brain of Alzheimer patients. The production of (3-amyloid from APP to the cells from abnormal proteolytic cleavage of the amyloid precursor protein. Enzymes involved in this cleavage may be suitable targets for the therapy of Alzheimer s disease. 

A higher than expected incidence of CHF is also observed in patients treated with DOX and other cytotoxics (e.g., the taxane paclitaxel) or new generation targeted agents (e.g., the humanized anti-ErbB-2/neu monoclonal antibody trastuzumab) [3]. The cardiotoxic synergism of DOX with taxanes or... 

---