Patient-centered clinical trials

Opportunities and challenges for the inclusion of patient preferences in the medical product life cycle: a systematic review

“…Potential roles of PP in industry processes 1.1 Early development • Informing ‘go/no-go’ decisions (e.g. internal prioritization portfolio decisions) [24] • Informing resource allocation decisions among multiple diseases [24] • Defining areas of unmet medical need [14, 16, 24] • Influencing which medical product will be developed [24] • Informing the design of a target product profile [14, 19, 27–29] 1.2 Clinical trial design • Quantifying how clinical outcomes, benefits and risks are perceived [14, 19, 30–34] • Indicating which clinical endpoints are of highest importance to patients [14, 31–33, 35] • Indicating which endpoints should (not) be considered [31] • Informing enrollment criteria and sample populations [19, 31, 33] • Informing clinical trial sample size [27] • Calculating acceptable levels of uncertainty (significance level and power) [36] • Analyzing clinical trials [14, 19] • Defining subgroups with different benefit-risk trade-offs [19, 24, 37] 1.3 Product labelling [14, 19, 37] 1.4 Post-marketing • Subgroup PP information for suggesting new markets for present indications [37] • Subgroup PP information for pointing to specific treatment opportunities [37] • Informing new innovations [14] • Redesigning and improving existing products [14, 19] • Informing expanded indications or populations [14] • Informing risk assessments underlying product recalls [19] • Optimizing promotional materials [19] 1.5 Pharmacovigilance activities [<...>…”

“…Potential roles of PP in BRA/MA • Highlighting patients’ needs for treatment [25, 26] • Highlighting differences in views between patients and decision-makers [19, 24, 40–42] • Highlighting situations with need for transparent communication about decision [42] • Providing quantitative measures of how patients view their choices [24] • Weighing (clinical) outcomes and attributes [14, 19, 25, 30, 34, 37, 38, 40, 43–48] • Identifying most relevant outcomes to patients [14, 19, 24, 26, 37, 48, 49] • Identifying outcomes with less perceived meaning [50] • Providing insights into patient perspectives on other aspects of treatment (e.g. dosing) [34] • Indicating patient benefit-risk trade-offs [18, 19, 24, 26, 34, 37, 38, 45, 47, 49, 51] • Indicating whether patients are likely to use therapy if approved [41] • Indicating how patients compare benefits and risks between treatment options [24] • Indicating how patients weigh benefits and risks as the disease progresses [24] • Enabling quantitative benefit-risk modelling in complex cases [19, 36, 37] • Providing information on uncertainty tolerance [24, 49] • Understanding patient heterogeneity [14, 19, 24, 37, 40, 42, 45, 52, 53] • Tailoring MA decision based on subgroups with homogeneous preferences [14, …”

“…Reasons related to the unique position of patients • Patients are the ultimate beneficiaries/end-consumers of healthcare [25, 31] • Patients are directly affected by the decision [38, 43, 53, 54, 60, 62] • Patients’ lives are affected by whether their concerns were considered [64] • Patient benefit is an objective of providing healthcare services [64] 3. Reasons related to the positive effect on quality of the decision-making process • It enables judging the consistency of decisions with patient values [64] • It enables a more patient-centered decision-making [19, 36, 40, 52, 53, 58] • It allows evidence-based consideration of patient perspectives [24, 36, 38, 40, 43, 45, 52, 58, 64, 65] • It ensures patient needs are better met [25, 53, 64] • Measurements of clinical effects usually do not sufficiently capture PP [38, 64] • It facilitates integration of patient concerns into decision-making [66] • It increases the effectiveness of patient involvement strategies [62] • It solves the issue of which patients to involve directly in decision-making [38] • It may be more representative than direct patient involvement [24, 25, 38, 40, 43, 58, 60, 62, 67, 68] • It is required for the implementation of evidence-based medicine [64]Reasons for using PP grouped into reasons related to the unique insights and position of patients and reasons related to the positive effect of including PP on decision-making (bold and underlined font). PP patient preferences, MPLC medical product life cycle…”

Patient Preferences in the Medical Product Life Cycle: What do Stakeholders Think? Semi-Structured Qualitative Interviews in Europe and the USA

“…On the HTA/reimbursement level, PP could provide information about patients’ preferred medical products and clinical outcomes [6, 24–29]. Outside the regulatory and reimbursement context, industry stakeholders are exploring how PP can inform priority setting, clinical trial design and analysis, and post-marketing risk assessments [3, 7, 15, 19, 21, 30].…”

Incorporating Patient Preferences via Bayesian Decision Analysis

“…Given the complexity of biomedicine and the consequences of both types of errors, regulators must exercise discretion in making their decisions. However, such flexibility can be made more transparent and systematic by applying Bayesian decision analysis to the regulatory approval process, as described in a series of studies (1)(2)(3)(4)(5)(6). The benefits can be best understood by contrasting it with traditional hypothesis testing in which a desired Type I error rate, say 5%, is chosen and the statistical significance of the clinical evidence is evaluated using this threshold.…”