Objectives: This study aimed to portray exemplified insights into how stakeholders across the medicinal product life cycle (such as drug developers, regulators, Health Technology Assessment (HTA) bodies, payers, and clinicians) can integrate preference study evidence into their decisions.
Methods: First, a literature review of regulatory guidelines and empirical studies eliciting stakeholder views on preference studies was performed in June 2021. Second, the results of a preference study in Multiple Myeloma (MM) were evaluated against the preference study roles identified in literature.
Results: Evaluating the MM study results against the preference study roles identified in literature revealed several potential applications for the MM study along the medicinal product life cycle. First, the MM study can be used by pharmaceutical companies to allocate resources towards developing drugs that not only improve life expectancy but as well target quality-of-life related symptoms and side-effects, such as mobility problems, pain, neurotoxicity's, and thinking problems. Second, results from the MM study may support regulators and HTA bodies in requiring drug developers to augment evidence about how MM treatments perform against these patient-relevant quality-of-life related side-effects and symptoms. Third, the MM study findings can be used by regulators and HTA bodies during benefit-risk assessment and relative effectiveness assessments respectively to increase their understanding of whether the MM treatment being evaluated address the unmet needs, symptoms, side-effects and uncertainties most important to patients. Finally, the MM study may inform healthcare providers about which (long-term) MM treatment-related uncertainties, symptoms, and side-effects should be systematically and clearly communicated to patients during individual treatment decision-making in clinical practice.
Conclusion: This study portrays multiple concrete opportunities for patient-centered and robustly conducted preference studies in informing decision-making across the medicinal product life cycle. Future efforts should seek to leverage internationally accepted guidelines, joint horizon scanning activities, early scientific advice, and parallel consultations by regulators and HTA bodies to formalize the designated role, requirement and design of patient preference studies in drug development and evaluation.

**Objectives:** This study aimed to portray exemplified insights into how stakeholders across the medicinal product life cycle (such as drug developers, regulators, Health Technology Assessment (HTA) bodies, payers, and clinicians) can integrate preference study evidence into their decisions.
**Methods:** First, a literature review of regulatory guidelines and empirical studies eliciting stakeholder views on preference studies was performed in June 2021. Second, the results of a preference study in Multiple Myeloma (MM) were evaluated against the preference study roles identified in literature.
**Results:** Evaluating the MM study results against the preference study roles identified in literature revealed several potential applications for the MM study along the medicinal product life cycle. First, the MM study can be used by pharmaceutical companies to allocate resources towards developing drugs that not only improve life expectancy but as well target quality-of-life related symptoms and side-effects, such as mobility problems, pain, neurotoxicity's, and thinking problems. Second, results from the MM study may support regulators and HTA bodies in requiring drug developers to augment evidence about how MM treatments perform against these patient-relevant quality-of-life related side-effects and symptoms. Third, the MM study findings can be used by regulators and HTA bodies during benefit-risk assessment and relative effectiveness assessments respectively to increase their understanding of whether the MM treatment being evaluated address the unmet needs, symptoms, side-effects and uncertainties most important to patients. Finally, the MM study may inform healthcare providers about which (long-term) MM treatment-related uncertainties, symptoms, and side-effects should be systematically and clearly communicated to patients during individual treatment decision-making in clinical practice.
**Conclusion:** This study portrays multiple concrete opportunities for patient-centered and robustly conducted preference studies in informing decision-making across the medicinal product life cycle. Future efforts should seek to leverage internationally accepted guidelines, joint horizon scanning activities, early scientific advice, and parallel consultations by regulators and HTA bodies to formalize the designated role, requirement and design of patient preference studies in drug development and evaluation.

Integrating Patient Preference Studies in Decision-Making Across the Medicinal Product Life Cycle: A Case Study in Oncology

DIA Europe 2022

A global, member-driven non-profit organization mobilizing life sciences and healthcare. DIA fosters innovation to improve health and well-being worldwide. Professionals from 80 countries continue to engage with DIA through our unparalleled membership network, educational offerings, and professional development opportunities.