ABSTRACT
The importance and merits of greater patient involvement in medicines research and development (R&D) are commonly acknowledged and are thought to offer benefits for all involved parties. It helps to improve discovery, development, and evaluation of new effective medicines, based on the collaborative identification and understanding of unmet needs, research priorities, optimization of clinical study design, outcome measures, and endpoint development. It can result in increased transparency, trust and mutual respect between patients and other stakeholders. This applies to all stages of medicines R&D, from industry-led research, to regulation and licensing of medicines, to appraisal by health technology assessment (HTA) bodies. Integration of patients into the medicines development process needs to be structured and governed by clear rules and modes of operation to be effective and yield the best results for all stakeholders. Existing codes of practice for patient involvement with various stakeholders do not comprehensively cover the full scope of R&D, with the exception of more general statements applicable to interaction. Overarching guidance on meaningful and ethical interaction is missing. One specific aim of the European Patients' Academy on Therapeutic Innovation (EUPATI) was to close this gap through the development of guidance documents for selected stakeholders. Four separate guidance documents were developed, incorporating the results from comprehensive internal and external consultation. They cover patient involvement in: pharmaceutical industry-led medicines R&D; ethics committees; regulatory authorities; HTA. Each guidance suggests where patient involvement could be adopted or strengthened. The EUPATI guidance document for patient involvement in industry-led medicines research and development covers the interaction between patients and the pharmaceutical industry within all functions throughout the medicines R&D lifecycle in relation to medicines for human use. It relates to activities pre-approval and post approval, involving individuals and groups of patients. The guideline distinguishes between the level of expertise in a disease area that is required and the different areas where patient involvement can take place; however, this is not meant to limit involvement, and these opportunities may change and increase over time. This EUPATI guidance document is aimed at the pharmaceutical industry who want to engage patients in R&D activities, however all stakeholders involving patients in pharmaceutical-led medicines R&D should understand and use this EUPATI guidance document.
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.
ABSTRACT
Involvement of patients in the research and development process (R&D) of new medicines-in all areas of indications-today is a widely accepted strategy in pharmaceutical industry to ensure relevance and suitability of the treatment under development. This may consist in, but is not limited to, patient input to achieve more patient-friendly protocol design, endpoint, and comparator selection as well as disease-adapted study conditions in a pre- or post-marketing clinical trial. Ethical aspects and especially the balance of benefit and risk in a clinical trial are frequently judged differently by clinical researchers, regulators, ethics committees, and patients due to their different focus. The final assessment of the ethical aspects of a planned clinical trial is provided by an independent ethics committee consisting of physicians and other experts in healthcare and clinical trial methodology as well as of lay persons. The participation of patients in ethics committees is a much-discussed concept, its suitability disputed in many countries, and only limited experience on best practices is available. In order to be effective and yield the best results for all stakeholders, integration of patients into the medicines development process needs to be structured and governed by clear, mutually agreed rules and modes of operation. Communication and collaboration processes need to be systematically implemented to establish transparency, trust and respect between those developing new medicines and their users, respectively between those involved in design and approval of clinical trials and participants. In particular agreement on the ethical aspects of a clinical trial and/or its overall ethical acceptability is a prerequisite before the start of a clinical trial. Existing codes of practice for patient involvement with various stakeholders do not comprehensively cover the full scope of R&D, with the exception of more general statements on interaction. Overarching guidance on meaningful and ethical interaction is missing. One specific aim of the European Patients' Academy on Therapeutic Innovation (EUPATI) was to close this gap through the development of guidance documents for ethics committees, pharmaceutical industry-led medicines R&D, regulatory authorities, and health technology assessment (HTA). This EUPATI "Guidance for patient involvement in ethical review of clinical trials" gives practical recommendations for ground rules and lists options for conditions and practices for involving patients in the work of ethics committees to enable trustful and constructive collaboration whatever the national (legal) framework for patient involvement in ethics committees might be. The guidance sets the collaboration of patients in ethics committees in the broader context of relevance and opportunities for patient input on ethics in the overall medicines R&D and specifically the overall clinical trial process from concept development to trial result reporting in lay summaries. In addition to a presentation of the full text of the Guidance, this article aims at providing additional background information on the development process of the Guidance, as well as insight into the current debate on this topic.
ABSTRACT
The main aim of health technology assessment (HTA) is to inform decision making by health care policy makers. It is a systematic process that evaluates the use of health technologies and generally involves a critical review of international evidence related to clinical effectiveness of the health technology vs. the best standard of care. It can also include an evaluation of cost effectiveness, and social and ethical impacts in the local health care system. The HTA process advises whether or not a health technology should be used, and if so, how it is best used and which patients are most likely to benefit from it. The importance of patient involvement in HTA is becoming widely recognized, for scientific and democratic reasons. The extent of patient involvement in HTA varies considerably across Europe. Commonly HTA is still focused on quantitative evidence to determine clinical and/or cost effectiveness, but the interest in understanding patients' experiences and preferences is increasing. Some HTA bodies provide support for participation in their processes, but again this varies widely across Europe. The involvement of patients in HTA is determined at the national and regional level, and is not subject to any European-wide legislation. The guidance text presented in this article was developed as part of the work of the European Patients' Academy on Therapeutic Innovation (EUPATI) and covers the interaction between HTA bodies and patients and their representatives when medicines are being assessed. Other EUPATI guidance documents relate to patient involvement in pharmaceutical industry-led research and development, ethics committees, and regulatory authorities. The guidance provides recommendations for activities to support patient involvement in HTA bodies and specific guidance for individual HTA processes. It seeks to improve patient involvement, using the outcomes of published research and consensus-building exercises. It also draws on good practice examples from individual HTA bodies. The guidance is not intended to be prescriptive and should be used according to specific circumstances, national legislation, or the unique needs of each interaction. This article represents the formal publication of the HTA guidance text with discussion about recent progress in, and continuing barriers to, patient involvement in HTA.
ABSTRACT
OBJECTIVES: To explore European-based pharmaceutical industry professionals' beliefs about patient and public involvement (PPI) in medicines research and development (R&D). SETTING: Pharmaceutical companies in the UK, Poland and Spain. PARTICIPANTS: 21 pharmaceutical industry professionals, four based in the UK, five with pan-European roles, four based in Spain and eight based in Poland. METHOD: Qualitative interview study (telephone and face-to-face, semistructured interviews). All interviews were audio taped, translated (where appropriate) and transcribed for analysis using the Framework approach. RESULTS: 21 pharmaceutical industry professionals participated. Key themes were: beliefs about (1) whether patients and the public should be involved in medicines R&D; (2) the barriers and facilitators to PPI in medicines R&D and (3) how the current relationships between the pharmaceutical industry, patient organisations and patients influence PPI in medicines R&D. CONCLUSIONS: Although interviewees appeared positive about PPI, many were uncertain about when, how and which patients to involve. Patients and the public's lack of knowledge and interest in medicines R&D, and the pharmaceutical industry's lack of knowledge, interest and receptivity to PPI were believed to be key challenges to increasing PPI. Interviewees also believed that relationships between the pharmaceutical industry, patient organisations, patients and the public needed to change to facilitate PPI in medicines R&D. Existing pharmaceutical industry codes of practice and negative media reporting of the pharmaceutical industry were also seen as negative influences on these relationships.
Subject(s)
Community Participation , Drug Discovery , Drug Industry , Interviews as Topic , Qualitative Research , Europe , Humans , Patient Participation , Poland , Spain , United KingdomABSTRACT
OBJECTIVES: To explore public knowledge of, and interest in, learning more about medicines R&D in six European countries. DESIGN: Online survey of 6931 members of the public across Europe. METHODS: The survey formed part of a public omnibus survey. A quota sampling approach was used with quotas set according to national census data on age, gender and government region. The survey explored the public's knowledge and awareness of medicines R&D, their interest in learning more and the perceived influences on this. RESULTS: The survey was completed by 6931 members of the public, over 75% of whom reported having no or less than good knowledge of medicines R&D. Males were more likely than females to report good knowledge (17% vs 15%), and knowledge appeared to decrease with age. Those who were currently or had previously been involved in medical research were almost five times more likely to report good knowledge of medicines R&D overall (43% vs 13%). Participants reported good knowledge of medicines safety and clinical trials but little knowledge of pharmacoeconomics. They were most interested in learning more about medicines safety and personalised and predictive medicine and least interested in pharmacoeconomics. Older people, women and respondents with current good knowledge of medicines R&D were most interested in learning more about medicines R&D. CONCLUSIONS: Experience of medical research appears to play a key role in increasing public awareness of and future interest in medicines R&D. Some groups may need to be specifically targeted to increase their awareness of medicines R&D, for example, women expressed great interest in learning more but reported less knowledge than men. It may be useful to explore further the views of those who are currently uninterested in learning more.
