Table 2.
Barriers to Patient Engagement as Reported by Stakeholders
| Type | Description | Reported bya |
|---|---|---|
| Cultural, political | Language, cultural and political aspects of each country/region may make the adoption of PE practices difficult (e.g. disease-related stigma may be more relevant in some countries than others; changing priorities in healthcare) | PO, HTA bodies |
| Fragmentation (e.g. different diseases and geographic regions, with different needs and interests, and competing for limited funding) | PO | |
| Lack of harmonised patient input in key policy development areas due to competing priorities between POs | PO | |
| English-centricity of PE practices | Regulators, HTA bodies | |
| Regulatory and legal environment not evolving along with PE | Medicines developers | |
| PE practices may be designed with a pan-European approach (e.g. engagement at EMA, global clinical development programmes) | Multi-stakeholder CEE workshop | |
| Building and maintaining physical and virtual platforms for discussion and exchange between organisations challenging in some countries due competing strategies of organisations | Multi-stakeholder CEE workshop | |
| In some countries, lack of PE and POs’ visibility, and lack of agreed communication channels between patient organisations and other stakeholders PO can have competing priorities as to where patient input is focused | Multi-stakeholder CEE workshop | |
| Lack of knowledge, skills or experience | Lack of PE skills and limited knowledge on how to meaningfully involve patients in existing processes | POs, medicines developers, regulators, HTA bodies, ICH |
| Lack of understanding of the public about their role in medicines development and the role of regulatory authorities | Regulators | |
| Lack of PE at the early stages of a process/project | PO | |
| Practices and processes not adapted to patients’ needs | PO | |
| Lack of understanding of medical discussions and decisions | PO | |
| Lack of patient leadership | PO | |
| Lack of health and PE literacy | PO, medicines developers | |
| Lack of knowledge on how to identify the right patients for the required activity | Medicines developers, HTA bodies | |
| Methodological | Patients' needs not present | PO |
| Superficial engagement with HTA bodies and payers | PO | |
| Lack of knowledge on how to apply methodologies to capture and use patients’ insights | Medicines developers | |
| Existing evidence to prove that PE leads to better health outcomes is still immature | Medicines developers | |
| Insufficient data to demonstrate value and impact of PE for all stakeholders and that ultimately it may not be perceived as a priority to be addressed | PO, medicines developers | |
| Lack of alignment across authorities on how to define and integrate a PE framework that is applicable to the local population needs and policies | Multi-stakeholder CEE workshop | |
| Little experience integrating and weighting patients' data vs clinical data | Regulators | |
| Implementation of PE | Lack of accountability mechanism of patient engagement and of defined policies and practices | Medicines developers |
| Lack of harmonised approach to PE | Medicines developers | |
| Prioritisation of PE activities where the patients’ voice adds more value vs including patients’ voice in all activities | Regulators | |
| Practical aspects and logistics | Regulators | |
| EU guidelines and frameworks not transferable at local level | PO | |
| Lack of resources | Lack of appropriate culture and human and financial resources at organisational level | POs, medicines developers, regulators, HTA bodies |
| Lack of an organisational culture supportive of PE amongst upper management | Medicines developers | |
| Long-term efforts and relationships required for optimal patient engagement outcomes might not align with the tight timelines of medicines development, hence risking patient engagement sustainability | Medicines developers | |
| Lack of financial resources to cover the expenses incurred | PO | |
| Funding limited to short-term projects and not ensuring long-term sustainability | PO, multi-stakeholder CEE workshop | |
| Lack of funding diversification | PO, multi-stakeholder CEE workshop | |
| The lack of continuity of patient representatives (due to disease burden or low patient numbers, such as in rare or complex diseases) results in loss of knowledge and expertise and limit the availability human resources | PO | |
| Lack of funding to support development of new patient advocates and leaders for long-term activism | PO | |
| Lack of organisational capacity may prevent from incorporating good practices | PO, medicines developers, others | |
| Conflict of interest and confidentiality | Lack of public (government/health ministry) funding for PO | PO, multi-stakeholder CEE workshop |
| Funding coming from a single source (private funding) | PO, multi-stakeholder CEE workshop | |
| Risk of patients’ losing their independence due to professionalization (e.g. becoming consultants) | Regulators | |
| Confidentiality barrier makes for lack of transparency and low PE | Regulators | |
| Preconceptions | Preconceptions about the value of the contribution of some patient groups (such as children and young patients and people living with dementia) and the challenges of involving them | POs |
| In some CEE countries there may be a perception of a lack of value of any contribution of patients or patient organisations | PO | |
| Lack of understanding of the value of PE | PO | |
| Initial mistrust from engaging stakeholders | PO | |
| Negative perception of industry engaging with patients | Medicines developers |
aSources Informal interviews with regulators (European Medicines Agency, UK Medicines and Healthcare Products Regulatory Agency, International Coalition of Medicines Regulatory Authorities) and the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH). Internal consultations with PARADIGM Consortium patient organisations (PO) partners; PARADIGM industry partners (medicines developers). Multi-stakeholder CEE workshop on patient engagement (see Table 1 for further details)