Impact Of New Therapies Research Articles

Trends in myocardial infarction prognosis and treatment in Southwestern France between 1985 and 1990. The Monica project Toulouse

The current treatment of acute myocardial infarction (AMI) involves the use of thrombolytic therapy and, in selected cases, invasive cardiology techniques. l Published therapeutic trials involve highly selected groups. It is therefore important to determine the impact of new therapies on a population-wide basis. We report the results of a study of all AMIs recorded for the Multinational Monitoring of Trends and Determinants in Cardiovascular Disease (MONICA project) in the French region of Toulouse (Haute-Garonne, Southwestern France) from 1985 to 1990. In this study, we examined trends in AM1 frequency and prognosis from 1985 to 1990. We also compared treatment among hospitalized patients during the same period. This study allowed us to assess the practices of effective therapies in randomized trials on a population-wide basis.2 METHODS

Clinical studies to assess the economic impact of new therapies: pragmatic approaches to measuring costs

Pragmatic clinical trials using unselected patients in normal clinical situations are more appropriate for the economic assessment of new drugs. However, standard clinical studies that do not reflect current practice are useful and at present the only source of information. Anti-emetic drug studies using granisetron and ondansetron have demonstrated that the overall economic impact of these drugs is equivalent to standard therapies such as metoclopramide. Thus, an efficient anti-emetic drug with less frequent dosing, using a simplified dosage regimen and producing a reduction in anticipatory nausea and vomiting and in nursing time, may result in an overall reduction in cost. Decisions made purely on the basis of drug costs may be misleading and promote inefficient use of health resources.

Trends in high technology nutrition support

In assessing the need for a nutrition support service (NSS) in a children's hospital, central line insertions (CLI) were reviewed between 1986–91. Medical Records Department identified patients (pts) by searching its data base for procedure code 38.98 (venous catherization not elsewhere classified). Data was reviewed for number and age of pts, medical vs surgical and subspecialty services. Other objectives of review included impact of new therapies (cardiac transplant, extracorporeal membrane oxygenation). Results: 1,870 pts received CLI. Distribution by ages was similar except for 12–18 year(yr) group which increased 200%. Medicine (54%) had more CLI than surgery (46%). Medical subspecialties ranked Hematology (34%), Critical Care (16%), Cardiology (11%), General (10%), Neonatology (9%) and Gastroenterology (7%). Surgical subspecialties ranked General (75%), Neurosurgery (10%), Orthopaedic (7%), cardiovascular (6%). Analysis for trends showed: 1) no service predominated age groupings; 2) Cardiology quadrupled secondary to transplant; 3) no effect of HIV program; 4) CLI increased 60% from 1986–91, plateauing in 1988. CLI were analyzed for nutrition, chemotherapy, and antibiotic therapy and showed 58%, 27%, and 15%, respectively for 1991. Conclusions: 1) CLI averaged 336/yr since 1988, reflecting severity of diseases and diversity of specialties; 2) 58% of CLI were for nutrition, supporting the development NSS; 3) effects of new therapies and their implications for NSS can be monitored.

Parkinsonʼs Disease

Recent studies have suggested that deprenyl may exert a neuroprotective influence and thus retard progression of Parkinson's disease (PD). On the other hand, the data do not disprove that deprenyl is primarily another form of symptomatic therapy like levodopa. Longitudinal Gompertzian analysis demonstrates the absence of beneficial alteration of intrinsic or environmental influences on the epidemiology of PD mortality after the introduction of levodopa. Moreover, this method demonstrates the basis for the enhanced survival in PD that resulted from the efficacy of levodopa as symptomatic therapy. If deprenyl becomes standard therapy in PD and exerts a beneficial (neuroprotective) influence on intrinsic and environmental pathogenic mechanisms, this will become evident by a decrease in the PD mortality rate in the United States in men at age 73.4 years and in women at age 79.1 years. The ability to accurately assess the overall impact of new therapies on the general population is increasingly important in an era of upward spiraling health care costs. Longitudinal Gompertzian analysis is a simple method of detecting and distinguishing between symptomatic (competitive) and protective (intrinsic and environmental) influences on disease mortality at the population level after the introduction of new therapies.

How can epidemiology assist in guiding interventions for the acquired immunodeficiency syndrome/human immunodeficiency virus?

In a single decade, the pandemic of human immunodeficiency virus (HIV) infection has become an international health, social, and economic emergency. Early and effective intervention is urgently needed for both prevention of HIV infection and for the amelioration of clinical disease. Results of therapeutic trials have suggested expanding the population for which chemotherapy is indicated. In this paper, we first review the findings from selected recent drug trials, using zidovudine and pentamidine as examples. We then discuss six issues that we believe to be crucial for future epidemiologic research in the service of vaccine and drug development: 1. To identify which complications of HIV infection most urgently require development of new therapies, we must characterize the frequency and severity of specific medical events (outcomes) in persons taking a variety of treatments. 2. Currently, acquired immunodeficiency syndrome (AIDS) therapeutic trials gauge the effectiveness of new therapies by their impact on such clinical parameters as the time to development of AIDS or death. These approaches take too long to provide information. We urgently need to identify surrogate markers of clinical outcome that will be useful in the early assessment of treatment efficacy. 3. Progress in vaccine development is being retarded because we do not have enough data from natural history studies on host immunologic responses to suggest that a given response is protective. We therefore need to identify natural correlates of immunity, which can help set priorities in vaccine development. 4. Discovery that a therapy works in the setting of a clinical trial is only a first step in intervention. We must also assess the impact of new therapies on the health of the public, evaluating access to health care, compliance, and other barriers to treatment. 5. Clinical trials are usually associated with the effort to prevent disease in infected persons. However, other trials are needed to assess efforts to interrupt viral transmission through use of condoms, use of virucides, and treatment of sexually transmitted diseases, and by effecting specific behavioral changes. 6. Traditional methods of conducting clinical therapeutic research may not be adequate to address urgent questions in the AIDS/HIV epidemic. We must develop innovative clinical research methods, including better use of data from observational studies, to infer what we can about the effect of treatment on the clinical course.

Multiple Systems Organ Failure: Epidemiology and Prognosis

This article reviews the basic epidemiologic and prognostic data for the syndrome of MSOF. When MSOF is defined as severe physiologic abnormalities, it occurs following a wide variety of diseases. The distinguishing feature of MSOF appears not to be the underlying etiology, but the uniform and frequently fatal outcome once it develops. This suggests that MSOF may represent a final common pathway to death rather than a clinical syndrome with a single underlying etiology. Regardless of cause, evaluating the incremental impact of new therapy on MSOF will be assisted by the adoption of uniform definitions as well as by the explicit measurement of severity of disease and calculation of individual probabilities of death.

A Short Form for Clinical Assessment of Quality of Life among Hypertensive Patients

Recently, medical research studies and clinical trials have included quality-of-life assessments, which measure the biomedical, behavioral, and social dimensions of living as a major therapeutic end point. Monitoring the quality of life in routine clinical practice also has the potential to aid clinicians to evaluate the impact of new therapies on the health status of their patients. However, because quality-of-life assessment techniques are quite lengthy and often require the aid of a trained interviewer, the research format is not practical for the typical clinical setting. This study describes the formulation, construction, and testing of an abbreviated quality-of-life questionnaire suitable for the clinical assessment of hypertensive patients. The initial formulation was based on analyses of data from a large-scale clinical trial (626 hypertensive male patients). Using the data at baseline for this group, items were selected such that the variance, internal consistency, and concurrent validity of the response scales were maintained by a reduced subset of items. The sensitivity of the reduced subsets was evaluated using treatment data and found to be as sensitive to treatment differentials as the original research instrument was. A subsequent field test of 87 volunteer subjects indicated that the new shortened version had the qualities of stable internal consistency and test-retest reliability over two successive trials. The questionnaire was self-administered and required less than 10 minutes to complete. It was given as an adjunct to the history and physical exam.(ABSTRACT TRUNCATED AT 250 WORDS)

The anemia of chronic renal failure. Overview and early erythropoietin experience.

One of the most important deficiency states associated with chronic renal failure is profound anemia. While it has been possible to identify differing types of anemia in patients receiving dialysis support, and thus correct the secondary causes, most patients continue to exhibit symptoms and signs of the anemic state. This review of the anemias associated with renal failure explores the diagnostic and therapeutic possibilities that may help the physician in evaluating the anemic patient with chronic renal failure. Also described is the early clinical experience with a new genetically engineered hormone, recombinant human erythropoietin (r-HuEPo), which has been under investigation for nearly two years at The Cleveland Clinic Foundation as part of a multi-center trial. An orderly approach to the anemic patient with renal dysfunction is suggested, and conjectures about the impact of newer therapies are made.

Changes in testicular cancer in Scotland

There are two purposes to this paper. Firstly to describe the temporal pattern of germ-cell testicular cancers in Scotland, both as a single entity and as the histological sub-types (pure) seminoma and teratoma. Incidence rates rose by over 50% between 1959 and 1984, with the rates of seminoma increasing only marginally and the majority of the overall increase accounted for by the substantial increased observed among the sub-type teratoma. Secondly, to investigate the impact of new therapies on the mortality rate from germ-cell testicular cancer in Scotland in the light of improvements in survival rate reported during the last 25 years from clinical trials and clinical series. Noticeable changes have occurred in the temporal pattern of mortality which cannot be explained by changes in incidence. The ever-widening gap between the increasing incidence rate and the declining mortality rate, particularly apparent in the high-risk age group 15–44, indicates an improving prognosis for patients with this malignancy in Scotland.