Intent-to-treat population

Please provide a definition of the per protocol population and the intention to treat population Per protocol population Intention to treat population... 

The term full analysis set was introduced in order to separate the practice of intention-to-treat from the principle, but practitioners still frequently use the term intention-to-treat population when referring to this set. The term modified intention-to-treat population is also in common use within particular companies and also by regulators in some settings where exclusions from strict intention-to-treat are considered. 

Another phase II study has tested rituximab monotherapy in the treatment of relapsed or refractory intermediate- and high-grade CD20(+) B-cell NHL (173). A total of 54 enrolled patients were randomized to receive intravenous rituximab at 375 mg/m2 weekly for eight doses versus 375 mg/m2 initially followed by 500 mg/m2 weekly for seven doses. In the entire intent-to-treat population of 54, the overall response rate was 31%, with CRs in 9% and PRs in 22%. No difference in response was noted between the two regimens. 

Although 213 patients actually received at least one dose of trastuzumab, results were based on the intent-to-treat population of 222. Eight complete (4%) and 26 partial responses (11%) were observed, for an objective response rate of 15%. Median time to disease progression was 3.0 months. Time to disease progression was longer for patients with HER2 overexpression at the 3+ level than for those at 2+ (3.3 vs. 1.9 months, p =. 0034). 

Critical demographic and baseline characteristics of the patient population and any other factors that might affect response should be presented comparability of treatment groups for each relevant characteristic should be documented. If there is an effectiveness subset of patients as well as an intent-to-treat population, this should be done for both groups. In a multicenter study, comparability should be assessed by center, and the centers should be compared. 

In total. 111 patients were assessable for response at final analysis 18 months after enrolment of the last patient (Table 5.4). At longer than 12 months of foUow-up, 17 of 30 responding patients and 22 of 43 patients obtaining clinical benefit (intent-to-treat population) had not progressed, precluding accurate estimates of TTP and DR in the responding group. Median OS for all enrolled patients was 24.4 months. 

Intent-to-treat population, 24-month cut-off Documented crossover = 57%... 

Which patients should be in which data set is something that should be considered before analysis data sets are created. For example, it is often decided that all analysis data sets should have a record for a subject if that subject was randomized to treatment and is considered an intent-to-treat subject. Whether this is true or not, the specifications for analysis data sets should make it clear who should be present in any analysis data set. Here is a list of common populations and their definitions ... 

In explanatory trials, we are hkely to exclude both protocol violators and treatment non-compliers. The objective behind these exclusions is to increase the efficiency. However the exclusions may give rise to bias and hence to compromise the results if too many patients are excluded. Such an analysis population is termed as completers, or per protocol, population. In pragmatic trials, we generally include all patients using the intention-to-treat (ITT) principle. 

Various analysis populations for clinical trial data can be defined and used in statistical analyses. Of relevance in this chapter are the intent-to-treat (ITT)... 

In many clinical trials, especially trials that occur later in a new drug development program, analyses are first conducted on the il l population. These analyses are considered to be the primary analyses. Intent-to-treat analysis provides a conservative strategy in the sense that it tends to bias against finding the results that the researcher hopes for (recall the discussion in Section 7.2.1 regarding hope). The ITT population therefore comprises the purest population of subjects in the... 

Module HI Presents the detailed efficacy findings including the intent-to-treat analysis population and the efficacy data listings. 

The intent-to-treat (ITT) population This comprises all participants in a clinical trial who were randomized to a treatment group, regardless of whether any data were actually collected from them. 

Therefore, an algorithm has to be developed to precisely define how each analysis population of the dataset is defined. For example, there are at least four analysis population datasets, e.g. the intent-to-treat (ITT) population, the per-protocol (PP) population, the safety population, and the microbiological population, as indicated in the diagram (Figure 22.2). During the derivation of various analysis populations, it may be necessary to issue new... 

Sanchez, M.M., Chen, X. Choosing the analysis population in non-inferiority studies Per protocol or intent-to-treat. Statistics in Medicine 25 1169-1181,2006. 

Recently, several groups have clinically evaluated compounds designed to increase or decrease synaptic levels of multiple neurotransmitters concurrently, such as norepinephrine plus dopamine or norepinephrine plus serotonin. The intent is to effectively treat a broader population with similar symptoms. Conversely, Merck and Pfizer s discontinuance of clinical trials of peptide Y antagonists for depression does not disprove the validation of that target until the results are better understood and can answer such questions as Was efficacy inadequate for a potent and selective compound Was selectivity of the compound for the target inadequate Does the molecular target not relate directly to the disease or have too many disconnects in the pathway to the disease ... 

---