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“213 This section provides general principles to consider in planning a drug development
214 programme. Efficient drug development usually requires appropriately planned interactions
215 with regulatory authorities throughout development, both in relation to planning early as well
216 as later studies including post-approval studies. This is particularly important for multiregional
217 studies to ensure the study design is aligned with regional regulatory requirements.
218 A drug development plan describes all aspects of the development of a product from the target
219 product profile through post-approval activities. The plan is usually prepared prospectively and
220 updated as the development progresses and new information becomes available. The plan
221 generally includes characterisation of formulation development, non-clinical studies required
222 to support the evaluation of the product in human clinical studies and to support product
223 approval, clinical studies designed to support the demonstration of efficacy and safety in the
224 relevant patient population, studies in special populations (e.g., paediatric populations),
225 regional considerations for product commercialisation (e.g., health technology assessments),
226 and post-approval studies.
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ICH E8(R1) Guideline
227 It is important to ensure that the experiences, perspectives, needs, and priorities of stakeholders
228 relating to the development and evaluation of the drug throughout its lifecycle are captured and
229 meaningfully incorporated into the development programme.
230 With increased globalisation of drug development programmes there is a need to consider
231 factors that impact quality of a protocol when it is conducted in more than one region (see ICH
232 E17 Multi-Region Clinical Trials). Early engagement with regulatory authorities to understand
233 local/regional requirements is encouraged and will facilitate the ability to design quality into
234 the study protocol. The results of a study are often used in regulatory submissions in multiple
235 regions, and the design should also consider the relevance of the study results for regions other
236 than the one(s) in which the study is conducted.
237 Clinical development programmes may also feature requirements for co-development of
238 validated biomarkers, diagnostic testing, or devices that facilitate the safe and effective use of
239 a drug.
240 An overview of the types of studies that may contribute to a development programme is
241 provided in the table in Annex 1.
“Exploratory
• Explore use for the targeted indication
• Estimate dose/dosing regimen for subsequent studies
• Explore dose-response/exposure- response relationship
• Provide basis for confirmatory study design (e.g., clinical endpoints, patient reported outcome measures, effect modifiers, target population, etc.)
• Randomized controlled clinical trials of relatively short duration in well- defined narrow patient populations, using surrogate or pharmacological endpoints or clinical measures
• Dose finding studies
• Biomarker exploration studies
• Studies to validate patient reported
outcomes”
“448 5.1.1 Study Population
449 The population to be studied should be chosen to support the study objectives and is defined
450 through the inclusion and exclusion criteria for the study. In practice, the study population is
451 limited to subjects available to participate and for whom consent is available (see ICH E6).
452 Recruitment efforts should ensure that the study subjects reflect the planned population for the
453 study. If objectives include obtaining information on certain subgroups, then efforts should be
454 made to ensure adequate representation of these subgroups.
455 The study population might be narrowly defined to reduce heterogeneity and maximize the
456 sensitivity of the study for detecting a certain effect. Conversely, it may be broadly defined to
457 more closely represent the population for which the drug is intended. In general, studies
458 conducted early in a development programme, when little is known about the safety of the
459 drug, tend to be more homogeneous in study population definitions, and those conducted in the
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ICH E8(R1) Guideline
460 later phases of drug development or post-approval tend to be more heterogeneous. Recruitment
461 for a precision medicine study, for example, may target the subgroup of diseased patients with
462 a particular phenotype or genotype, either exclusively or through an enrichment study design.
463 The choice of study population will depend on the study objectives, and the degree to which a
464 study succeeds in recruiting and enrolling the desired population will impact the ability of the
465 study to meet those objectives.
466 For example, a study population representative of clinical practice may be the target of a
467 pragmatic trial conducted within an existing healthcare system. In such a study, recruitment
468 procedures may differ from other types of studies, in that the inclusion and exclusion criteria
469 may be assessed based on existing medical records.
470 Because of the study objectives or because of feasibility or efficiency, there may be situations
471 in which the population unit is not an individual but a group of subjects (known as a cluster).
472 For example, some vaccine studies make use of cluster randomisation to measure their
473 protective effects on communities. The use of a cluster unit has implications for multiple design
474 elements and quality factors (e.g., intervention, analysis, consent).
475 The study should plan to have a sufficient number of subjects to make statistical conclusions
476 based on the findings either by obtaining a certain precision or by controlling the probabilities
477 of making false conclusions (see ICH E9 Statistical Principles for Clinical Trials). A larger
478 database may be needed to establish the safety of a drug (see ICH E1).”
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