Obtaining the preferences of patients and their caregivers is not a new concept in healthcare. Increasingly, however, industry guidance documents utilize the term ‘patient preference’ in relation to new drug development and benefit-risk assessment. For example:
- In 2022, the European Medicines Agency (EMA) published a Qualification Opinion of the Innovative Medicines Initiative (IMI) Patient Preferences in Benefit-Risk Assessments during the Medicinal Product Lifecycle (PREFER), generally endorsing their proposed research framework, which provides some guidance for researchers and industry when designing and eliciting patient preference studies.
- The ISPOR Task Force also recently published a guidance document, titled ‘Roadmap for increasing the usefulness and impact of patient-preference studies in decision making in health.’ This document also provides useful guidance for stakeholders working in research on how to devise a preference study.
- In their first Guidance in the Patient-Focused Drug Development (PFDD) series, the Food and Drug Administration (FDA) references the importance of obtaining patient preference data as part of contextualizing the patient journey through their condition. The agency refers to their 2016 Guidance for Industry on Patient Preference Information Submission — which, though a useful document, does not provide clear requirements for how to run a quantitative preference study, suggesting the Guidance is intended to be used as a catalyst for advancement in this space.
Patient preference data can be utilized at various timepoints along the drug development timeline:
- Regulators are putting more emphasis on this type of data, specifically to inform benefit-risk assessment, although there is still limited published data as to how it has been used in decision making.
- Health technology assessment (HTA) bodies also have some appetite for this information. Specifically, data can help HTA bodies in evaluating, interpreting, and deliberating the evidence in reimbursement decision making if presented alongside the economic and clinical evidence.
- Preference data clearly has a use in providing additional supporting information on patients’ condition, treatment and experience, which can aid decision making. Such data can also be utilized to call out differentiators compared to current products or the standard of care. These differentiators can inform commercial strategies.
- Preference data can also be used to help make strategic decisions internally — for example, knowing patient preferences for the mode of administration or avoidance of side effects may help with clinical trial endpoint selection or product development.
At Clarivate, our Clinical Outcome Assessment team can advise sponsors strategically and practically on obtaining the preference data they need to support their internal decisions and/or regulatory or reimbursement submissions. We utilize robust methodology, ensuring results that can be trusted and published. In one recent preference project, we partnered with a pharmaceutical client to aid their decision making for a new adjuvant therapy in a rare disease where the aim was to quantify treatment-related preferences and identify what influences patients’ treatment decisions. Data from this work were presented at conferences, including ISPOR.
Patient preferences are increasingly being utilized to inform decision making in health care. Recent publications (both from a regulatory and reimbursement perspective) only strengthen the call to action that this data should be considered at multiple time points in new product development.
For more information about how Clarivate helps companies understand patient preferences, please visit us here.