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Differences in adverse drug reactions in phase III and phase IV of the drug evaluation process.

The study of medications in routine use is called "Phase IV" of the drug evaluation process. Information gathered about drugs in Phases I through III does not provide a sufficient basis for final conclusions about the clinical value of medications after marketing. Changes in preferred indications, patient characteristics (e.g., multimorbidity), treatment mode, or treatment setting in routine as compared to controlled scientific studies lead to changes in causes, incidence, prevalence, predictability, meaning, consequences, and cost-benefit ratios of adverse drug reactions (ADRs). Rare but serious ADRs are only one aspect of postmarketing surveillance; other questions are at least as important. In contrast to Phase III, which has a single study design--the controlled, randomized, double-blind study--Phase IV requires different designs for each of the many different questions. Established methodologies include spontaneous reports, stimulated spontaneous reports, comprehensive observation studies, Phase IV intervention studies, case control studies, prescription event record linkage, and data bank comparisons.

Clinical Trials, Phase III as Topic

Clinical trial design in metastatic breast cancer: a commentary.

Trial designs traditionally used in the development of new anti-cancer agents are usually categorized as phase I, phase II, phase III and phase IV. Such trials are often performed in patients with metastatic cancer. Phase I trials are conducted in small numbers of patients to determine a drug's maximally tolerated dose (MTD) and toxicity profile. In phase II studies, the anti-tumor activity of the new agent is tested in different tumor types. If a drug is found to be promising in phase II studies, it is then compared to standard therapy through a randomized trial design in phase III studies. In phase IV studies, the efficacy and safety profile of the drug are evaluated in a standard clinical setting. In the field of oncology, studies have been conducted in which agents already approved by the regulatory agency are combined with other anti-cancer drugs and evaluated for anti-tumor efficacy. These have also been termed phase II studies.

Antineoplastic Agents

Effect and efficacy--on the function of models in controlled phase III trials and the need for prospective pharmacoepidemiological studies.

While the "effect" of a drug can be observed or deduced from observational data, the concept of "therapeutic efficacy" represents mainly a theoretical construction of a high degree of abstraction which is inconceivable without reciprocal combination with other theoretical constructs. The "therapeutic efficacy" of drugs can be investigated only via clinical-pharmacological or clinical "models". Several examples are given and discussed against the background of the actual considerations for shortening phase III studies and extending pharmacoepidemiological phase IV studies for scientific, practical and economic reasons. Of special relevance is the question whether study data of phase III allow an extrapolation to the wider patient population which it is intended to treat. Thus, it is well known that the criteria for representativeness in the investigated population are rarely achieved in phase III studies. Furthermore, observations have shown that various intervening moderator variables, such as the investigated subgroup or the trial setting (e.g. Inpatient or respectively out-patient treatment), might influence therapeutic efficacy and the possibility of generalizing the results. This again raises the crucial question of clinical relevance of significant effects. Possible ways of overcoming this unsatisfactory situation are suggested.

Clinical Trials, Phase III as Topic

[Tolerance and safety of tramadol use. Results of international studies and data from drug surveillance].

This article presents a summary of drug safety data concerning the use of tramadol hydrochloride and an outline of the specific aspects of this analgesic in particular with regard to respiratory depression and dependence potential. Information from phase II to IV clinical studies, postmarketing surveillance studies (covering safety data from a total of more than 21,000 patients) and the spontaneous reporting system have been taken into consideration. The data from the spontaneous reporting system covers the period between 1977 and 1993, during which more than one billion single dose units were distributed throughout the world. The phase II to IV studies compare acute intravenous, acute intramuscular, acute oral and multiple dose oral administration Postmarketing surveillance studies provide a picture of everyday use of tramadol in general medical practice. Further analyses were performed to provide information about the gender-, age- and dose-related distribution of adverse reactions The prevalence of side effects was calculated by comparing the number of symptoms with the number of patients. The pooled data from the clinical studies and the postmarketing surveillance studies reveal that the most commonly observed side effects were nausea, dizziness, drowsiness, tiredness, sweating, vomiting and dry mouth, with an overall incidence of between 1 and 6%. In the postmarketing surveillance studies on long term and acute administration, the profile of adverse events was qualitatively almost identical to that in the phase II to IV studies. However, there were distinct quantitative differences it favour of the long term studies. In the postmarketing surveillance study on acute parenteral administration, the incidences of nausea and vomiting were only 4.2 and 0.5% respectively, which is significantly lower than the 20.7 and 11.4% in the patient-controlled analgesia studies. Nevertheless, it is important to take into consideration the different conditions in these studies. All the postmarketing surveillance studies were outpatient studies, whereas almost all of the phase II to IV studies were carried out in hospitals. The studies with intravenous and intramuscular administration were mainly postoperative, which explains the relatively high incidence of nausea and vomiting, 17.8 and 7.0%, respectively, with intramuscular administration. The different conditions in the phase II to IV studies and the postmarketing surveillance studies are also reflected in the occurrence of dizziness and postural hypotension: The incidence of dizziness in the postmarketing surveillance studies is slightly higher than that observed in the phase II to IV studies. Particularly in the studies with intravenous and intramuscular administration, the patients were confined to bed and were therefore much less sensitive to dizziness than those in the long term oral and postmarketing surveillance studies, who were all outpatients. On the other hand, postural hypotension played almost no role in the multiple dose studies, in which the oral formulation were used most frequently. It is interesting to note that diarrhoea, pruritus and gastrointestinal disorder (except nausea and vomiting) are mainly reported in the multiple dose studies in the groups receiving oral tramadol, and also in the postmarketing surveillance studies. Once again, the study conditions may well be the explanation. The adverse effects reported in both clinical and postmarketing surveillance studies are similar to those in the spontaneous reports. The most frequently documented adverse effects in clinical and postmarketing surveillance studies, i.e. nausea/vomiting, dizziness, drowsiness, tiredness, sweating and dry mouth, are noted very infrequently in spontaneous reports, since in medical practice these side effects are usually known and are described in the product information. Almost all reports referring to abuse/dependence are connected with pain therapy; they give no reason to suspect any pro

Analgesics, Opioid

Safety of the calcium antagonist lacidipine evaluated from a phase III-IV trial database.

OBJECTIVE: We reviewed the clinical safety profile of lacidipine with the help of the rather comprehensive datafile of the manufacturer. Although a number of prospective, randomly allocated trials under way at present are investigating the effects of treatment with calcium antagonists on cardiovascular morbidity and mortality, these results are not yet available. Therefore, the present approach may be useful. A fuller account of this work has been published in Blood Pressure. DESIGN: Since 1985,50 phase III-IV trials have been performed to investigate antihypertensive efficacy in patients with hypertension; 32 were controlled trials with comparison treatment and 18 were open studies of lacidipine treatment. Only data from trials completed before 1 January 1995 are presented here. SUBJECTS: In all, 16590 patients were treated with lacidipine; 13419 in open studies and 3171 in double-blind comparative trials. A total of 1810 patients were given active comparative treatment and 451 were given placebo. Altogether, 5124 person-years of data were obtained. MAIN OUTCOME MEASURES: Numbers of both fatal and non-fatal cardiovascular events were estimated. Efficacy (change in blood pressure and heart rate), adverse event rates and drop-out rates were compared for the different treatment regimens. RESULTS: In all trials, 2-6 mg lacidipine was effective in lowering blood pressure. In the controlled trials, systolic/diastolic blood pressure fell from 166/102 to 144/85 mmHg and the heart rate fell from 75.6 to 74.1 beats/min. The estimated event rate for a possible myocardial infarction in all studies was 5.46/1000 person-years; the fatal (all-cause) event rate was 5.27/1000 person-years and the estimated fatal cardiovascular event rate was 2.93/1000 person-years. There were 21 malignant events during treatment with lacidipine, for all studies yielding a crude incidence of 4.10/1000 person-years. In patients treated with lacidipine, the age-standardized (according to the world population) incidences were 1.49 (men) and 0.79/1000 person-years (women) compared with 2.74 (men) and 2.09/1000 person-years (women) in the European Community in 1990. The overall incidence in the comparative studies of one or more adverse events included 30.3% for lacidipine, 43.8% for other calcium antagonists, 18.7% for diuretics, 48.7% for beta-receptor blockers, 10.4% for angiotensin converting enzyme inhibitors and 15.7% for placebo. The adverse effects of lacidipine were, as expected, headaches, flushing, pedal oedema and palpitations. CONCLUSIONS: Lacidipine proved to be an effective and well tolerated drug in almost 19000 hypertensive patients. It displayed a reasonable adverse profile that was typical of a calcium antagonist of the dihydropyridine group. This analysis has two obvious limitations: (1) it is a retrospective analysis; and (2) the data were obtained from a large cohort of patients, but most were treated with lacidipine for a relatively short period of time. Although we found a lower fatal event rate than that reported by Collins et al., their meta-analysis included 10 times more person-years than our analysis, and therefore our event rate may be less accurate. Further prospective studies are under way at present to determine whether these drugs can produce reductions in atherosclerosis or in the incidence of cardiovascular disease.

Adolescent

Design of field trials for the evaluation of antibacterial products for therapy of bovine clinical mastitis.

In this paper, the design and statistical analysis of field trials for the evaluation of the efficacy of clinical mastitis therapeutics is covered. First, general issues underlying the design of clinical trials are reviewed. These include bias and confounding; randomization and blocking; and study objectives and choice of the corresponding hypothesis. Specific issues in the design of clinical mastitis trials are also discussed. Selection of subjects is discussed with regard to choice of experimental units, identification of reference population and study population, inclusion and exclusion criteria, and sample size calculation. Next, a section on treatment administration and evaluation of cure reviews treatment, blinding, choice of response measure, as well as compliance, withdrawal, and early termination. The statistical analysis section addresses possible statistical models, treatment of confounding, and fixed vs. random effects. In conclusion, well-conducted clinical mastitis trials represent an invaluable, albeit difficult and expensive, effort to evaluate efficacy and tolerance under usual circumstances of use.

Animals

Evaluating new vaccines for developing countries. Efficacy or effectiveness?

Despite the profusion of promising new vaccines against illnesses prevalent in developing countries, uncertainties about the balance between costs and benefits of new vaccines have retarded their use in public health practice. Conventional prelicensure trials of vaccine protection exacerbate these uncertainties by focusing on measurement of vaccine efficacy--the performance of a vaccine under idealized conditions. Vaccine effectiveness trials provide a more pragmatic perspective by addressing the performance of a vaccine under the ordinary conditions of a public health program, by capturing direct as well as indirect effects of vaccination, and by comprehensively addressing outcomes of public health concern. The use of effectiveness trials should enable more rational triaging of new vaccines for developing countries and may accelerate the introduction of new vaccines into public health practice by resolving speculative debates about practical costs and benefits.

Case-Control Studies

Nicardipine in the prevention of migraine headaches.

The subjects, 104 patients who had experienced more than two migraine episodes per month during the previous 6 months, received 60 mg of nicardipine daily for 2 months in an uncontrolled, Phase-IV study. Eighty-nine patients (mean age, 40 years; 16 with and 73 without aura; 60 women) completed the treatment regimen. The patients' blood pressure did not change during treatment. The mean number of migraine attacks was reduced significantly from 6.7 per month during the 3 months before treatment to 4.0 per month during treatment. The number of severe attacks was also reduced significantly. The patients' subjective ratings of the frequency and intensity of the attacks and their need for analgesia were reduced significantly from before to after treatment. Side effects (flushing and shortness of breath) were reported by four patients. It is concluded that nicardipine is safe and effective in the prevention of migraine attacks.

Adult

Observational versus randomized medical device testing before and after market approval--the atherectomy-versus-angioplasty controversy.

Percutaneous transluminal coronary angioplasty was developed in the late 1970s as a nonsurgical alternative for revascularization of atherosclerotic coronary arteries. It gained widespread acceptance without a controlled trial. Introduced in 1986, directional coronary atherectomy was the first of other recently developed coronary devices that sought to improve on the results of angioplasty. It was approved in 1990 by the Food and Drug Administration (FDA) on the basis of observational data. Its use expanded rapidly, reaching over 35,000 procedures in 1992, accounting for more than 10% of all interventions. After premarket approval, two major randomized trials tested the hypothesis that atherectomy would be superior to angioplasty. Their results raised a cautionary flag and stood in contrast to projections made from prior observational data. It is concluded that randomized controlled trials validate claims of relative efficacy and safety of competing medical technologies, a lesson reflected in recent changes in policy at the FDA.

Angioplasty, Balloon, Coronary

Inhalational anesthetics: desflurane and sevoflurane.

This article reviews the physico-chemical properties and performance characteristics of the two new potent inhaled anesthetics, desflurane and sevoflurane. Both drugs provide a greater degree of control of anesthetic depth and a more rapid immediate recovery from anesthesia than is currently available with other inhaled agents because of their decreased solubility. Desflurane is currently in widespread clinical use in the United States and parts of Europe. Compared with sevoflurane, it has the additional advantage of being extremely resistant to degradation and biotransformation. However, its pungent odor and tendency to irritate the respiratory tract make it unsuitable for inhalational inductions, and it has been linked to CO production in CO2 absorbents. The sympathetic nervous system activation that occurs with desflurane limits its use in patients with cardiac disease. Otherwise, its hemodynamic and physiologic effects are similar to those seen with isoflurane. Studies of the economics of using desflurane are mixed, although it may offer the advantage of shorter postoperative recovery time. Sevoflurane is currently in widespread clinical use in Japan and parts of South America. The FDA Advisory Panel has recently recommended approval of sevoflurane in the United States, and we can expect the drug to be clinically available in the United States in the second quarter of 1995. Compared with desflurane, sevoflurane has the additional advantage of being nonirritating to the airway; inhalational induction of anesthesia with sevoflurane is achieved rapidly and easily. The instability of sevoflurane with CO2 absorbents and its in vivo biotransformation produce potentially toxic byproducts. These byproducts, including Compound A and fluoride, have been extensively studied, and although the possibility for iatrogenic sequelae from sevoflurane exists, the likelihood of long-term toxicity appears quite low. Phase IV studies are indicated to determine the safety of administering sevoflurane (1) to renally impaired patients and (2) to any patient with fresh gas flows less than 2 L/min. Sevoflurane is otherwise very well tolerated and appears to offer the advantage of rapid and smooth induction and emergence from general anesthesia.

Adult

Mortality studies and the effectiveness of drugs in long-term treatment.

In scientific observations on the utilization of drugs under routine treatment conditions in patients with affective disorders, one of the main problems is the establishment of criteria for measuring treatment effectiveness. As the mortality risk of such patients is considerable, the mortality rate is an important outcome criterion for determining the long-term effectiveness of medication administered in routine treatment settings. Mortality studies are typical phase IV studies; the researcher does not interact with the treatment procedure, he only observes the results of treatment with respect to death rates. As lithium treatment is of increasing importance and interest as a major prophylactic agent in recurrent affective disorders and is by its nature a long-term treatment, such a therapy provides an excellent field for observations of effectiveness under long-term treatment conditions. Mortality studies can take into account various factors, such as type of treatment setting, treatment regimen, drop-out analysis, and comparison between times on and off treatment. Results from several mortality studies on lithium-treated patients are reported in order to demonstrate the methodology and outcome of long-term observation of drug treatment. Mortality studies conducted within the framework of phase IV studies and particularly within the concept of pharmacoeconomics can help to demonstrate the effectiveness of long-term treatment, and are an important methodological adjunct to controlled clinical trials.

Antimanic Agents

Guidelines for the implementation of drug utilization observation (DUO) studies in psychopharmacological therapy. The "Phase IV Research" Task-Force of the Association for Neuropsychopharmacology and Pharmacopsychiatry (AGNP).

The task-force on Phase-IV-Research of the Association for Neuropharmacology and Pharmacopsychiatry (AGNP) has developed guidelines for the implementation of scientifically sound drug utilisation observation studies (DUO studies). These guidelines have been adopted by the executive committee as the position of the association. DUO studies are prospective pharmacoepidemiological studies, by which prescription, illness, and patient data of individual patients are collected without interference with the routine course of treatment. They can answer questions on the interaction of treatment modalities with positive and negative treatment outcome. Scientific standards require that there is a study protocol which describes the epidemiological, statistical, procedural, and quality assurance methodology and states who is responsible for what. As such studies can violate data protection regulations or can be used for sales instead of scientific purposes, consultation of an ethics committee is recommended.

Clinical Trials, Phase IV as Topic

Financial ties as part of informed consent to postmarketing research. Attitudes of American doctors and patients.

Postmarketing research, often called phase IV trials, is intended to familiarise doctors and patients with newly approved drugs. La Puma and colleagues, in Chicago, studied doctors' and patients' attitudes to whether doctors should receive payment for taking part in such research. We asked for commentaries on their findings from four ethical experts, who put the study in a British context, present the views of patients, and examine some methodological assumptions.

Adult