EUPATI and Patients in Medicines Research and Development: Guidance for Patient Involvement in Regulatory Processes

Abstract

The importance and merits of greater patient involvement in medicines research and development (R&D) are commonly acknowledged and is thought to offer benefits for all involved parties. It improves discovery, development, and evaluation of new effective medicines, based, among others, on the collaborative identification and understanding of unmet needs, research priorities, optimization of clinical study design, as well as incorporating patient views in regulatory activities. It fosters increased transparency, trust and mutual respect between patients and other stakeholders and applies to all stages of medicines R&D, inclusive of regulation and licensing of medicines and appraisal by health technology assessment (HTA) bodies. In order to be effective and beneficial for all stakeholders, patient engagement as an integral part of medicines R&D needs clear and mutually agreed rules. Existing codes of practice for patient involvement do not comprehensively cover the full scope of patient engagement in all processes related to R&D. One specific aim of the European Patients' Academy on Therapeutic Innovation (EUPATI) was to close this gap through the development of guidance documents for pharmaceutical industry-led medicines R&D, ethics committees, regulatory authorities and health technology assessment (HTA). The guidance in this article covers patient involvement in the regulatory field and draws on the mature “Framework for interaction between the European Medicines Agency and patients and consumers and their organizations.” It expands on the EMA framework, specifically including National Competent Authorities (NCAs). It sets out objectives for patient involvement in medicines regulation and recommends concrete suggested working practices. It is primarily aimed at regulatory authorities wishing to interact with patients or their organizations in their activities but should also be considered by patients/patient organizations planning to collaborate with regulatory authorities.

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

“…Regulatory agencies such as the Center for Devices and Radiological Health (CDRH) at the US Food and Drug Administration (FDA) advocate early engagement and frequent discussions regarding design and execution of patient preference studies [31]. 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].…”

“…As active patient engagement is increasing, it has become critical to systematize the tools and approaches used to collect patients' perspectives and design a standardized process that can inform drug development based on patient-relevant end points and outcomes. Regulatory authorities do acknowledge the importance of early patient engagement and appreciate and early engagement with authorities on methods to collect patients' insights [3].…”

“…To leverage the recent advancements in digital technologies and exploit available online platforms to gain insights regarding symptoms that would be most relevant from patients' perspectives in chronic obstructive pulmonary disease (COPD), we considered a four-step approach ( Fig. 1, Table 1): (1) literature review, (2) social media listening (SML) study, (3) online bulletin board (OBB), and (4) web-based patient preference study. Steps 1-3 have already been completed, while step 4 is ongoing.…”

Moving forward from drug-centred to patient-centred research

“…Patients must remain the focus throughout the discovery process, whilst the regulatory framework for testing new interventions in a robust and meaningful way is revisited. Moreover, since the extent of patient involvement in regulatory issues varies considerably between countries and regions in Europe, the development of frameworks by the national competent authorities must also envisage an active participation and close interaction with patients, ensuring the input of their real-life experiences of diseases and their management into the process [7].…”

Improving Patient Involvement in the Lifecycle of Medicines: Insights From the EUPATI BE Survey

“…Hitherto, all stakeholders can plea with the governing authorities and regulatory bodies for an unambiguous position on how to increase patient involvement. EUPATI has guidelines that can be employed for this stance (e.g., the EUPATI Guidelines) (4,12,13). By collaborating strategically, EUPATI.be can establish a platform where patients and decision-makers can interact in a more structured and ethical way.…”