Patient Preference Studies During Early Drug Development: Aligning Stakeholders to Ensure Development Plans Meet Patient Needs

Abstract

Although patient preferences have been studied broadly for marketed products or around the time of submission to authorities and launch, patient preference studies have rarely been used during the early drug development phases. In this paper, we formulate three hypotheses supporting the use of patient preference studies in early product development: (1) integration of the patient perspective into the development process from phase 1 onwards will result in healthcare solutions with outcomes that best address patients' needs; (2) a structured process to build patient-based evidence involving partnerships between patients and other key stakeholders will improve alignment of development activities with the needs of patients; (3) quantitative patient preference research built on robust qualitative insights is necessary to strengthen development decisions in the interests of patients. To illustrate such a structured process, we describe qualitative insights research (social media analysis and online bulletin boards) and quantitative patient preference studies in dry eye disease and non-alcoholic steatohepatitis conducted during early product development by a pharmaceutical company to generate patient-based evidence. The outputs from such early patient preference studies are being used to inform patient reported outcome strategies, clinical development strategies, product design and delivery features, and form the basis for early dialog with regulators, health technology assessment (HTA) bodies and payers to ensure focus and alignment on patient-relevant endpoints. Furthermore, to discuss and theoretically substantiate our hypotheses, we review how different groups and organizations are working to embrace fully the patient perspective in product development and healthcare decision-making. The hypotheses are commensurate with the general trend toward patient-centered healthcare and the activities initiated by regulators, HTA agencies, and patient organizations. We advocate that all healthcare players should actively contribute to aligning on best practices concerning choice of methodologies and engage in multi-stakeholder dialog along the entire product development chain, to realize health technologies that best meet the needs of patients.

Gathering Structured Patient Insight to Drive the PRO Strategy in COPD: Patient-Centric Drug Development from Theory to Practice

“…Guidance for the incorporation of patient preference measurements into drug development, regulatory, and reimbursement decision-making processes is evolving [3,32] and multi-stakeholder initiatives such as IMI PREFER (Innovative Medicines Initiative's The Patient Preferences in Benefit-Risk Assessments during the Drug Life Cycle project) are actively working to fill this gap [32]. There is a need for early engagement in dialogue with regulatory agencies and HTA bodies on scientific advice to discuss patient needs, preferences, and implications thereof for clinical trial design and evidence collection [8]. This step is crucial since, eventually, HTA agencies will be one of the recipients of any product submissions that will be developed through trials using the insights generated through such a PFDD exercise and would need to make reimbursement decisions after recognizing the value that such products offer to patients.…”

Comparing the needs and preferences of patients with moderate and severe dry eye symptoms across four countries

“…In DED as in other diseases patient preference research has the potential to revolutionise healthcare practice by allowing an individualised approach to treatment in accordance with the needs expressed by the patient 7. When conducted early in the product development lifecycle, patient preference studies can inform the design of clinical trials (eg, through the inclusion of patient-reported outcomes (PROs)), ensuring that outcomes that matter to patients are captured 8. With an increasing trend towards capturing relevant PROs in clinical trials, patient-centric information becomes an important input to value frameworks for evaluating new therapeutic interventions, to ensure they deliver value to the patient 8.…”

Assessing physician preferences on future therapeutic options and diagnostic practices in non-alcoholic steatohepatitis

“…12 There have also been calls for improved alignment of stakeholders on patient needs early on in the drug development process. 13 Physicians are typically a major stakeholder that interacts directly with patients, which is why it is important for their preferences to be known and aligned with the other stakeholders. This paper takes a step further to align NASH stakeholders on their preferences and can be used in the future to guide research and develop awareness programmes for the benefit of patients.…”