Patient Groups

The time required to fully establish the safety and efficacy of a new drug in the eyes of the regulatory authority might not always be to the benefit of the proposed patient group. This is particularly so in the case of drugs intended to treat serious or life-threatening diseases where no effective therapy currently exists. Various provisions have been incorporated into the regulations to allow accelerated access to such therapies. 

Rates of smoking among patients with bipolar disorders and anxiety disorders (e.g., posttraumatic stress disorder, panic disorder) are also higher than those in the general population (Lasser et al. 2000), but there has been htde smdy of the factors associated with motivation to quit smoking or of smoking cessation interventions in these patient groups. 

Cost-efFectiveness analysis (CEA) is concerned with ensuring that resources allocated to the treatment of mental health ptohlems ate used to maximum effect. It is usually employed to help decision-makers choose between alternative interventions available to or aimed at specific patient groups ... 

A related issue is the question of which patients to include in an evaluation. The most usefirl evaluation would be one that included all patients likely to use a service in the real world. However, in order to make evaluations feasible, and because of the need to achieve the informed consent of patients, many prospective clinical trials exclude certain patient groups. For example, many schizophrenia trials exclude patients with... 

Intravenous P-blockers followed by oral p-blockers should be administered to all patients with NSTE ACS in the absence of contraindications. Benefits of p-blockers in this patient group are assumed to be similar to those seen in patients with STE ACS. p-Blockers are continued indefinitely. 

Normally, 88% to 92% of phenytoin is bound to plasma protein, leaving 8% to 12% unbound. The unbound component is able to leave the blood to produce the clinical effect in the CNS, produce dose-related side effects in the CNS and at other sites, distribute to other peripheral sites, and be metabolized. Certain patient groups are known to have decreased protein binding, resulting in an increased percentage of drug that is unbound. These patient groups include ... 

Differences in the relative proportion of f-PSA and PSA-ACT can affect the result obtained for t-PSA because of the differences in the nature of calibration and the molar response, sensitivity, and specificity of antibodies used in various immunoassays. The efficiency of these immunoassays has been evaluated by several investigators. Because the proportion of free and complexed PSA varies in benign and malignant diseases, these immunoassays measure one form or the other, giving rise to different results for different patient groups. It is very important that data from clinical studies support the proposed intended uses of these assays, since as many as 5 percent of men with a negative free PSA test (free PSA values >25 percent) will have cancer and not be recommended for biopsy. Therefore, a goal for standardization is to detect total and free PSA accurately in equimolar fractions. 

Colic is also a symptom of lead poisoning in children. EPA (1986a) has identified a LOAEL of approximately 60-100 pg/dL for children. This value apparently is based on a National Academy of Sciences (NAS 1972) compilation of unpublished data from the patient groups originally discussed in Chisolm (1962, 1965) and Chisolm and Harrison (1956) in which other signs of acute lead poisoning, such as severe constipation, anorexia, and intermittent vomiting, occurred at 60 pg/dL. 

The reduction of nausea in patients taking anti-cancer drug therapy is probably the most widely researched area for cannabis therapy. A number of these studies have shown that oral administration of isolated cannabinoids produce significant improvements, particularly for those patients who have failed to respond to standard antinausea treatments during chemotherapy (see Tortorice and O Connell, 1990 for a comprehensive review). Patients and oncologists have subjectively reported that smoked marijuana is as safe (in this patient group) and effective as isolated oral cannabinoids, but more systematic research trials are required. 

Various malignancies can also induce an anaemic state. This is often associated with decreased serum EPO levels, although iron deficiency, blood loss or tumour infiltration of the bone marrow can be complicating factors. In addition, chemotherapeutic agents administered to this patient group often adversely affect stem cell populations, thus rendering the anaemia even more severe. 

The efficacy document E7 (ICH 1993) states that the drug should be studied in all patient groups, elderly included, for which they have a significant utility. It also includes new uses, new formulations and new combinations of established medicinal products when there is specific reason to expect that conditions common in the elderly (e.g. renal or hepatic impairment, impaired cardiac function, concomitant illness or medication) are likely to be encountered. This also applies for when the geriatric patients response (safety, tolerability, efficacy) is different from the nongeriatrics. ... 

As in other patient groups, heterozygous FH patients with coronary heart disease have a significantly higher Lp(a) concentration than patients without CHD [having normal Lp(a) levels] (G16, S26). 

Patient groups or other interested parties who are not familiar with the drug development process need to realize how fantastically complex this whole story is. 

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