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At least 19 recordsLinked to original sources

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↗

Description of controlled trials published in Methods and Findings, 1979-2002.

It is thought that the controlled trial (CT) is the most adequate research method to assess a therapeutic intervention in terms of efficacy, and it also constitutes the basis for the development of systematic reviews on health interventions. To identify and obtain the majority of published CTs is not an easy task, mainly because of limitations concerning the currently available electronic sources. The aim of the present work was to identify, describe, and assess the quality of CTs published in the journal Methods and Findings in Experimental and Clinical Pharmacology (M&F). Additionally, to assess the retrievability of both methods, a search was performed in Medline (PubMed access) through the use of an optimal search strategy for CTs. A total of 189 original studies out of a total of 2796 reviewed articles met the CT criteria according to the Jadad scale score, we could hold that only 58% of the CTs were of good quality. The present work confirms, once again, the limitations of a CT search performed exclusively through Medline (sensitivity 64% and specificity 98%). In conclusion, we suggest that the journal M&F explicitly joins the International CONSORT Statement.

Clinical Trials, Phase III as Topic↗

The science of health technology assessment--clinical effectiveness of therapeutic interventions.

Important information is not, and cannot, be available at the time a new drug enters the market. Delaying registration is not the answer because most of this information can only be obtained in a real life situation. Several types of postmarketing (phase IV studies) can be identified. The active pharmacovigilance cohort who allows large number of patients to be followed for long periods of time can answer questions about the incidence of rare events (less than one of 3000 patients). The prospective effectiveness cohort can answer questions on long term efficacy (more than two years). The simplified clinical trial, which implies randomly assigning patients and then following them with a 'naturalistic' protocol can answer questions about effectiveness (efficacy in real life). The drug use study is the only way to answer questions of key importance to drug plan managers such as 'which drug(s) is it going to replace?' and 'is it going to be used as first line or second line?'. Phase IV studies, which in some cases should be mandatory (conditional registration), are essential for the protection of the patients and the proper use of public funds to reimburse drugs.

Canada↗

[The GCP directive--consequences for clinical drug research].

The contents and implications of the EU Directive on good clinical (research) practice (GCP) regarding drug trials are described. As of May 2003, clinical researchers in Denmark must have standard operation procedures, conduct monitoring, consider quality assurance, and expect inspections. The industry may be better prepared, but the Directive makes GCP part of the law and phase IV studies become subject to GCP. Patients will be assured the same quality in trials irrespective of the industry or investigator being the sponsor and may look forward to quality improvement of drug trials.

Adverse Drug Reaction Reporting Systems↗

[Drug clinics. Clinical drug trials: the importance and role of the general practitioner].

Clinical trials are an essential step in the development of a drug. They must be conducted according to strict rules called "Good Clinical Practice" or GCP. GCP requirements aim to guarantee a perfect methodology in the planning, realization and interpretation of clinical trials. The latter can be divided in four phases: phase 1 aiming to demonstrate the safety and to investigate the pharmacokinetics/metabolism of the drug in healthy volunteers; phase 2 aiming to study the intrinsic activity (generally versus a placebo) and safety of the compound in a rather small number of (hospitalized) patients; phase 3 aiming to confirm the comparative efficacy (versus a placebo or a reference drug) and safety of the pharmacological agent in a quite large number of (ambulatory) patients; and phase 4 carried out after commercialization, to verify the clinical utility of the drug in conditions of daily practice. Because he/she occupies a crucial position in the recruitment and follow-up of outpatients, the general practitioner should play a more active role in clinical trials, provided that he/she could work in collaboration with academic centers specialized in clinical pharmacology which can help to perform studies in accordance with GCP requirements.

Academic Medical Centers↗

Update on intravenous tissue plasminogen activator for acute stroke: from clinical trials to clinical practice.

Tissue plasminogen activator (tPA) injected intravenously within 3 hours of symptom onset has emerged as a treatment option for acute ischemic stroke. Although controversial and not universally accepted, its use in carefully selected patients is supported by evidence from randomized controlled trials and by mounting community experience. In this paper we review the literature published in the past 5 years regarding the safety, clinical trial efficacy and real-world effectiveness of intravenous tPA for stroke. First we review data from the phase III clinical trials on which approval for tPA is based. Then we summarize a growing literature of postmarketing phase IV studies and discuss the limitations and challenges that lie ahead. Our aim is to provide clinicians with an overview of this evolving therapy.

Acute Disease↗

Japanese experience with dual-action antidepressants.

Milnacipran is one of the first modern antidepressant drugs to be introduced into Japan, and the first dual-action antidepressant. Placebo-controlled clinical trials with this drug have demonstrated similar efficacy and superior tolerability to imipramine and mianserin. The good safety profile of the drug has been confirmed from open-label phase IV studies. There are indications, both from the randomized clinical trials and from the phase IV programme, that milnacipran may have a comparatively rapid onset of action, showing clear signs of efficacy after one week of treatment. This observation, which needs to be confirmed in an appropriately designed study, may be the clinical correlate of the rapid desensitization of 5-hydroxytryptamine (HT)1A receptors produced by milnacipran. A series of pilot studies have demonstrated the role of milnacipran in the management of certain affective disorders not adequately treated by classical antidepressants. These include bipolar disorder, treatment-resistant depression in the elderly and post-stroke depression. These findings merit confirmation in controlled studies, and open the way to using milnacipran to provide satisfactory treatment of these condi-

Adrenergic Uptake Inhibitors↗

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↗

Drug research: from the idea to the product.

The path of a new drug from the idea to product may be divided into 2 phases, namely drug discovery and drug development. In drug discovery a dramatic change is taking place. Due to a remarkable progress in understanding and explaining the underlying cause of many diseases by identifying and sequencing the genes encoded within DNA, it has become possible with new methods, like molecular biology or gene technology, to develop simple test assays by which a large number of compounds may be tested in regard to their biological efficacy. Automation of these test systems utilizing computer-controlled robotic systems has made it possible to evaluate up to 1 million substances per robot per year on their biological effect. While the classical medicinal chemistry cannot produce such a large number of new compounds, combinatorial chemistry may offer the opportunity to screen large numbers of novel compounds rapidly and to reduce the time taken to identify drug candidates substantially. Overall, it is estimated that combinatorial chemistry techniques have resulted in a reduction in the time taken to identify drug candidates of between 18-24 months. In drug development 2 partly overlapping phases can be differentiated, namely the preclinical and clinical phase. During the first part of drug development necessary requirements for first use in man are met by performing preclinical pharmacological, toxicological, and pharmacokinetic investigations in the animal and in in vitro testing. These investigations are playing a central part for the benefit/risk evaluation of new drugs. Only if the risks connected to the clinical study are medically justifiable in relation to the likely therapeutic benefit of the compound, the clinical trial may basically take place under consideration of the legal requirements of the country in which the study is carried out. In drug research clinical pharmacology is the connecting link between preclinical and clinical research. Clinical pharmacology produces the necessary basis for the clinical trial of a new substance in the patient with the target indication. After a first clinical-pharmacological profile of the new substance has been established during phase I on the basis of which a decision for the continuation of the clinical trial and the probable effective dose range and dosing interval is made, the aim of phase II-IV is now to answer the important questions of the therapeutic efficacy and tolerability in a large number of patients with the target indication. Only with a very careful drug investigation during phase I-IV it is really possible to register the therapeutic risk and benefit of a new drug and to control resulting serious problems. An analysis of registered drugs during the last years shows that despite enhanced efforts and dramatically rising development costs the innovation potential of the classical approach is steadily decreasing. Therefore, it is absolutely necessary to develop new innovative drugs with the help of new technologies like molecular and cell biology, gene technology, or immunology as well as combinatorial chemistry and robot supported substance screening.

Animals↗

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↗

Data from regulatory studies: What do they tell? What don't they tell?

Phase III studies of antiepileptic drugs (AEDs) are specifically designed to satisfy strict regulatory criteria. As they are conducted in protocol-restricted patient populations over short treatment periods and employ fixed study designs and dosing schedules, they are not fully representative of 'real-life' clinical practice. Therefore, in order to provide an overall assessment of clinical performance, regulatory studies must be backed up by post-marketing clinical experience. Phase IV studies provide information on a drug's performance in a setting more closely representing real clinical practice, with broader patient populations and a more flexible approach to individual treatment. Prospective long-term studies allow the determination of efficacy and safety (and cost-effectiveness) over extended treatment periods; these studies and audit data provide a means of assessing idiosyncratic side effects, unusual interactions and the effects of an AED in rare patient groups. By complementing regulatory evidence with real-life clinical experience, a comprehensive assessment of the risks and benefits of an AED can be made.

Adverse Drug Reaction Reporting Systems↗

Clinical data gap between phase III clinical trials (pre-marketing) and phase IV (post-marketing) studies: evaluation of etanercept in rheumatoid arthritis.

BACKGROUND: There are fundamental differences in design between phase III clinical trials and phase IV post-marketing studies that involve patient characteristics, the clinical setting (environment) and the manner of drug use. As well, many phase IV studies are extensions of randomized clinical trials (RCTs) and suffer from selection bias. OBJECTIVE: To determine if the data obtained from RCTs of etanercept (Enbrel) in the treatment of rheumatoid arthritis would be representative of the effects attainable in community practice. METHOD: An analysis was conducted comparing data from published RCTs of etanercept use in rheumatoid arthritis patients with data collected in a community based cohort study that was not an extension of an RCT. RESULTS: Baseline clinical data, such as tender or painful joint count, patient's global assessment, the heath assessment questionnaire, physical and mental component summary of the SF-36, and rheumatoid arthritis drug profile were significantly different between the patients receiving etanercept in the phase IV community cohort study and the patients enrolled in the RCTs. Differences in the baseline data for the control patients were also noted amongst the RCT studies. The treatment outcome, American College of Rheumatology (ACR) response rate of 20%, 50% and 70% at 6 month, was the same between the cohort study and the RCTs, but at 12 months the clinical response was less for the community based patients than for the RCT patients. At 6 months there were fewer withdrawals involving community-based patients than RCT patients due to less frequent withdrawals associated with lack of efficacy. At 12 months the withdrawal rate due to either a lack of efficacy or from adverse events was similar between data sets. CONCLUSION: The data from the etanercept phase III RCTs may not reflect the characteristics of patients using etanercept in community practice, nor the clinical outcomes observed by RA patients at 12 months. These discrepancies may be derived from methodological differences in study design and patient selection. On the other hand, outcomes such as withdrawal rates at 12 months appear comparable between the two types of populations.

Antirheumatic Agents↗

[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↗

Prospective individual matching: covariate balance and power in a comparative study.

In phase II to phase IV studies, randomization has gained widespread acceptance as a methodologic tool for the allocation of patients to treatment. However, randomization is not always feasible. At times, the treatment intervention occurs universally throughout one or more units (for example, a hospital unit), while the control therapy is the only intervention provided in other units. Patients may arrive randomly at a unit, based solely on availability of the unit to accept new subjects. Thus, the treatment assignment process is out of the investigator's control and not subject to selection bias. We describe a prospective individual matching procedure through which one can achieve balanced allocation of subjects to treatment groups in this comparative study setting. In this paper, we compare balance of baseline covariates and power for this design, in which the subject is selected at random and assigned to a treatment group, and the traditional randomized block design, in which the treatment is chosen at random and assigned to a subject. We show that the prospective individual matching procedure compares favourably to the traditional randomized blocked design with respect to both baseline covariate comparability and statistical power.

Analysis of Variance↗

[Difficulties with conducting clinical trials in France].

France ranks third among European countries as regards the level of investment in clinical R&D and, overall, accounts for a contributive effort proportional to the size of its population and pharmaceutical market respectively. However, there is a trend for phase II and III studies to become proportionally fewer than in the past, while the number of phase IV studies is increasing. In a growing proportion of the mega-trials, which are instrumental for establishing evidence-based practice, French experts, investigators and, even more seriously, French patients, are insufficiently represented. Though studies in France are initiated relatively fast due to a clear regulatory framework and perform equally well as far as quantitative and qualitative factors are concerned, compared with most European countries involved in clinical research the costs incurred per completed patient are higher than those recorded in the other countries. Academic research shares most of these constraints and suffers from a lack of financial and human resources, while it faces additional delays in implementing studies because of longer administrative processes. Given the stakes in play, specific solutions should be implemented to maintain and further develop French competitiveness in clinical R&D. At the patient level, positive perception and awareness of the usefulness and safety of participating in clinical trials need to be emphasized. Education at the school level and using the lay media should be developed. Intervention of institutional and government officials is much needed. Direct patient recruitment should be developed through advertisement and the Internet, as well as within doctors' offices and through collaboration with patients' organizations. Patient information and consent forms should be made much simpler than those imposed within the framework of global studies because of FDA requirements. The French health system discourages the recruitment of patients by investigators who are not the family doctor or the usual care provider. Thus, motivation and education of general practitioners and hospital doctors may be increased by involving them during the trial design phase and in the publication process. Specific administrative solutions, within private or public institutions, need to be developed for investigators who do not personally wish to receive investigation fees. Because of lack of availability, investigators need to be assisted with study nurse services and site management organizations, particularly within hospitals and clinics, using the model of the Clinical Investigation Centres. Networks of clinical investigation centres and of individual investigators need to be created. Implementing these solutions should lead to better implications for and reputation of French clinical research.

Clinical Trials as Topic↗