Efficacy measures

Various forms of psychotherapy are regarded as effective interventions in mild to moderate depression, but studies comparing the economics of psychotherapy and pharmacotherapy are few (Rosenbaum and Hylan, 1999). One study found that the total health-care costs for patients who received psychotherapy were no different from those for patients who received an antidepressant. However, no efficacy measure was used (Edgell and Hylan, 1997). A randomized, prospective study which evaluated the treatment of depression with nortriptyline, interpersonal therapy or treatment as usual, with outcomes expressed in quality-adjusted life years, found that nortriptyline but not interpersonal therapy was a cost-effective alternative to treatment as usual (Lave et al, 1998). 

Tollefson et al. (1994] reported an American multicenter investigation of fixed-dose fluoxetine in the treatment of OCD. Three hundred fifty-five outpatients participated in two randomized, double-blind, parallel, 13-week trials, receiving fluoxetine or placebo. Fluoxetine (20 mg, 40 mg, and 60 mg] was significantly superior to placebo on the Y-BOCS total score and other efficacy measures. However, a trend was noted suggesting greater efficacy of the 60 mg/day dose. The authors reported few side effects, and most patients (79.2%] completed the study. Similarly to CMl, fluoxetine led to a significant reduction in OCD severity, regarding both obsessions and compulsions. 

The phase III Frontal Hair Loss study was conducted in parallel with the phase III Pivotal studies in order to evaluate the efficacy of finasteride 1 mg primarily in the frontal scalp area (Leyden et al., 1999), as opposed to the vertex. The frontal hair loss study used end points similar to those used in the Pivotal studies and demonstrated significant improvements in all predefined efficacy measures with finasteride as compared with placebo. Treatment with finasteride significantly... 

Both issues are widely discussed by Rieger,4 who wrote that what is actually measured is pH of the (extractable) water-soluble constituents of skin. Due to that problem, Rieger proposed to call that measured value not pH of the skin but pH on the skin or the apparent pH. That issue is also raised and described by Parra and Paye in guidelines of European Group on Efficacy Measurement of Cosmetics and Other Topical Products (EEMCO).5... 

Dr. Loden is a member of several national and international societies such as the International Society for Bioengineering and the Skin, and acts in the Committee of the European Group of Efficacy Measurements of Cosmetics and Other Topical Products (EEMCO) and the Scandinavian Society of Cosmetic Chemists (SCANCOS). She is also a member of the editorial board of the International Journal of Cosmetic Science. 

These studies evaluated the safety and efficacy of five dose strengths, three BID regimens and one QD regimen (including BID and QD for one dose level). Individual recorded 24-hour scores for baseline and for 14 day post-baseline determinations. For BID doses, 24-hour scores were calculated as an average of two 12-hour score responses between PM doses. The clinical efficacy measure was symptom score 0 (absent, symptom not present), 1 (mild, symptom was present but not annoying/troublesome), 2 (moderate, symptom was frequently troublesome but did not interfere with either normal daily activity or sleep), 3 (severe, symptom was sufficiently troublesome to interfere with normal daily activity or sleep), 4 (very severe, symptom was so severe as to warrant an immediate visit to physician). Scores were calculated for four such measures and combined with score ranging... 

It is common practice to employ outside laboratories to perform testing for safety and efficacy measures in clinical trials. Along with the results, these laboratories will also provide the units and normal ranges for the tests performed. Since the laboratories are typically utilized by many patients in a study or even across studies, it is practical for the units and ranges to be received and entered once in the system and then linked internally to the patient data to which they apply. This principle of centrally storing values that can be shared across the system is also desirable for maintaining the conversion factors used in deriving lab results into standardized units. 

Rogier V, Balls M, Basketter D, et al. (1999) The Potential Use of Non-invasive Methods in the Safety Assessment of Cosmetic Products. The Report and Recommendations of ECVAM/EEMCO (The European Group for Efficacy Measurements on Cosmetics and Other Topical Products) Workshop 36. European Commission, Institute for Health and Consumer Protection, European Centre for Validation of Alternative Methods (ECVAM). 

Both the quantity and the types of data able to be collected will be affected by the nature of the clinical study patients may be inpatients or outpatients, and this in turn will govern the nature of pharmacoeconomic data that can be recorded. It is also important whether a clinical trial is intended as a pivotal trial for registration or not if a study is pivotal, then a clinical efficacy measure will have to be the primary end point. Pharmacoeconomic parameters can still be incorporated into such a study as secondary end points, and still provide valuable information. If, on the other hand, the clinical research addresses a health system delivery issue, then the pharmacoeconomic end points may well be primary, and the study design need not be constrained by FDA-mandated requirements for the double-blind, placebo-controlled aspects of proof of efficacy. 

Biopharmaceutic studies (in vitro) Clinical study Drug release/dissolution Measurement of drug efficacy Measurement of the rate of drug dissolved under specified conditions... 

Categorical data is becoming increasingly common in population PK/PD analysis, especially ordered categorical data. Examples of such data are adverse events and efficacy measurements such as pain scales (16) or sedation scores (17). This section focuses on graphical methods for categorical type data. 

Two assays were developed that measure the potency of the FGF-4 transgene carried by Ads FGF-4. In the first case, a one-step growth-promotion assay is conducted on normal, human retinal pigment epithelial cells (ARPE-19). The assay measures metabolic activity (Alamar blue dye metabolism) following infection of ARPE-19 cells with a serial dilution of the virus. The increase in metabohc achvity was measured in relation to a mock-infected control. This increase correlates with FGF-4 produchon determined by an FGF-4 ELISA, increased de-novo DNA synthesis measured by BrdU incorporation, and an increase in cell number. This procedure is therefore an appropriate in-vitro efficacy measure, indicating that the FGF-4 transgene product is biologically active. 

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