Berlin, July 20, 2016
Almost 50 participants from regulatory authorities, patient organisations, academia, non-profit organisations and industry gathered for a EUPATI Workshop on July 20, 2016 in Berlin to advance and foster the interaction of all stakeholders on patient involvement and engagement in medicines R&D.
During this workshop, participants shared regulators’, industry and patients’ viewpoints, experiences with pilot projects and strategic approaches to patient involvement, aiming for the tri-party interaction to be understood, respected and trusted by all stakeholders, external parties and the public at large. The meeting also addressed challenges and solutions regarding patient engagement in processes and how to handle potential conflicts of interest.
Regulators shared the experience of pilot projects and processes involving patients both at European as well as at national level, while industry representatives discussed the evolution of industry processes to involve patients in all aspects of medicines R&D in a more meaningful and systematic manner, e.g. within clinical development plans, clinical trial design and trial conduct as well as compliance processes.
This report summarizes the key messages and discussions from the workshop:
Marco Greco, Chair of EPF, in his welcome applauded what has been achieved so far, and while efforts must continue, there is a need to better define the framework of interaction between various stakeholders.
Nathalie Seigneuret, IMI, in her welcome recognised the achievements of the EUPATI project and underlined the importance of continuing and developing it further. She also made reference to the considerable efforts of IMI as such to embed patient engagement more effectively in all its work.
Jan Geissler, Director of EUPATI, set the scene with the reminder that it is widely acknowledged today that patients’ contribution to the discovery, development and evaluation of medicines enriches the quality of research and development, quality of evidence and opinion and transparency, trust and mutual respect. The EUPATI project itself has contributed substantially to a more comprehensive approach. Patient involvement requires systematic involvement of all stakeholders: pharmaceutical industry, regulators and HTA bodies, patient and consumer organisations, health care professionals, non-profit organisations and academia.
He shared some preliminary data on the impact of EUPATI from a survey among EUPATI fellows, where fellows who participated in the Patient Expert Training Course have increased their advisory roles significantly, when comparing their engagement before and after the course:
- providing advice to the pharmaceutical industry increased from 8% to 52%,
- to regulatory agencies from 12% to 40% and
- to HTA bodies from 4% to 8%.
Despite this progress, challenges still remain, such as the lack of mutual learning, the lack of mutual trust, the lack of standardised metrics to measure benefits and impact, perceived and/or real barriers around conflict of interest as well as the lack of capacity in patient organisations. He highlighted that, based on the success of EUPATI in establishing the multilingual EUPATI Toolbox as well as the EUPATI Patient Expert Training course, decisions have been taken to continue with EUPATI beyond 2016 as the only dedicated training structure and trusted brand on patient involvement in medicines R&D. The long term sustainability of EUPATI is of major public interest, but securing public funding beyond the current IMI-funded project phase remains a challenge.
- The tripartite dimension of interaction of patients, regulators and industry.
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EUPATI Patients-Regulator-Industry workshop, Berlin, 20 July 2016. Presented by Jan Geissler & the EUPATI team.
Isabelle Moulon, EMA, highlighted that patients can engage in various ways in regulatory processes: they can be members of committees, act as representatives of an organisation or be individual experts and clarified how declarations of interests are evaluated for each of these roles. . EMA has established a network of European patients’ and consumers’ organisations for collaboration on various topics. The EMA Patients’ and Consumers’ Working Party (PCWP) plays a key role in enabling these interactions. To support the best possible cooperation EMA recognises that patients involved with the Agency need to have adequate knowledge of the work of the Agency and therefore provides various training opportunities, also referencing EUPATI initiatives.
- The European Medicines Agency: An example of patient engagement.
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EUPATI Patients-Regulator-Industry workshop, Berlin, 20 July 2016. Presented by Isabelle Moulon
Matthias Gottwald, Bayer, presented a practical “roadmap” for patient involvement in all steps of the R&D process. He also shared some preliminary findings from the DIA-Tufts University study which presents the following results:
- out of 20 pharmaceutical companies only three consider themselves “very patient centric” and
- four “not very patient centric”,
- most are “somewhat patient centric”.
The top planned initiatives were adaptive trial designs and adaptive licensing. The patient engagement function is placed differently in companies and mostly in clinical operations or clinical development. Corporate drivers to create a patient engagement function are company commitment and clear benefits for process improvement. Challenges for patient involvement in R&D are: concerns of patient independence, need for comprehensive guidance addressing all stakeholders, no infrastructure for “matchmaking”, no consolidated approach for patient involvement in industry and no metrics for impact assessment.
- Setting the scene: industry processes for patient involvement, organisational setup, roadblocks.
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EUPATI Patients-Regulator-Industry workshop, Berlin, 20 July 2016. Presented by Matthias Gottwald, BAYER.
Parallel workstream: Industry
Three case studies on real-life patient involvement in R&D were presented by GlaxoSmithKline, Novartis and MSD, followed by a lively discussion.
GlaxoSmithKline: directly interviewed female patients with Lupus in order to learn more about their views on the disease and on research. The information that patients shared helped the researchers to progress with designing a clinical trial, which is planned to start during 2016. The importance of always providing feedback to patients following such interactions was emphasized.
- Direct patient insight on Lupus with a focus on cutaneous aspects.
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EUPATI Patients-Regulator-Industry workshop, Berlin, 20 July 2016. Presented by Kay Warner, GlaxoSmithKline R&D, Immuno-Inflammation Therapy Area Unit.
Novartis: invited a Lymphoma Patient expert to attend an “EMA-HTA Parallel Scientific Advice Meeting” to discuss a protocol in lymphoma. For the first time the Unit engaged an expert patient for an EMA SA meeting. While it was a valuable learning experience, they suggested clarifying in advance with EMA and the patient expert invited by the company if the expert patient will in the future be eligible to collaborate with EMA and if this has any impact on a possible conflict of interest.
- Lymphoma patient representative at EMA Joint HTA Scientific Advice.
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EUPATI Patients-Regulator-Industry workshop, Berlin, 20 July 2016. Presented by Susanna Leto di Priolo, Novartis Oncology Region Europe.
MSD: sought patient input into draft phase II (proof of concept) study design in Breast Cancer to improve probability of success in terms of generating patient-relevant data, whilst also meeting current regulatory needs. The experience was considered positive and led to some protocol revisions. They did face some practical challenges around understanding sample size, compensation, bias, use of third party services, etc.
- Patient input into breast cancer study design.
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EUPATI Patients-Regulator-Industry workshop, Berlin, 20 July 2016. Presented by Paul Robinson, MSD.
Overall, the participants observe a growing trend in patient engagement, but several open questions remain that require further clarification. Some of these are:
- Where should industry go to find patient experts for projects?
- How can we measure the value of these interactions to gain further momentum?
- Can industry collaboratively develop guidance for contracting and compensating patients? At the moment every company is doing this individually, following their own rules.
- Conflict of interest: this remains a less well defined area. Should industry ask EMA about the consequences of selecting an expert patient ahead of time? If a patient gives industry feedback on a protocol, can such a patient still participate in a clinical trial? Patients are interested in providing their perspective and do not want to always have to worry about being perceived as biased or receiving (unwarranted) payments.
- Each company has their own guidance/best practices; so does it make sense then to have common standards to make it easier for patients to engage?
Parallel Workstream: Regulators
Experiences were presented by four national competent authorities followed by a broader discussion.
AIFA, Italy: involve patient organisations to debate specific products or classes of products. They focus on education of patients, signing a Memorandum of Understanding with EUPATI. The need for educating patients is truly recognized.
- Patient involvement: Italian Medicine Agency approach
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EUPATI Patients-Regulator-Industry workshop, Berlin, 20 July 2016. Presented by Silvia Cammarata, AIFA.
MHRA, UK: established an informal non-bureaucratic “Patient Group Consultative Forum” in 2014 with the purpose of creating a forum for meaningful dialogue between the agency and patients, caregivers, patient groups and members of the public. The forum has 80+ members today from different areas and they aim to eventually embed the PGCF in agency processes and projects. In addition, they have a UK Reclassification Stakeholder Platform with lay representatives where patients can also get involved to support activities like communication on alerts.
- Engagement with Patients
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EUPATI Patients-Regulator-Industry workshop, Berlin, 20 July 2016. Presented by Mike Dykes – Patient, Public and Stakeholder Engagement (PPSE) team.
Swissmedic, Switzerland: a realignment of cooperation with patient and consumer organisations took place in 2014 with two focus areas, to secure better communication via the web-page and to involve patient/consumer organisations in pre-defined non-product-related work areas; a step-by-step and 2 years’ pilot approach was adopted. The established group has 16 members at present. In August 2016 the way forward will be decided based on the results of the pilot.
- Swissmedic’s cooperation with patient and consumer organisations.
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EUPATI Patients-Regulator-Industry workshop, Berlin, 20 July 2016. Presented by Cordula Landgraf, Head of Networking, Swissmedic.
MPA, Sweden: a previous experiment with a patient/consumer group (2009-2011) fizzled out with decreasing numbers of attendees with the following reasons from participants: no interesting agendas, one-way communication, too much time investment. A new initiative, “The Patient & Consumer Advisory Council”, is under preparation which will be supported by a secretariat. Also, due to new legislation, it is expected to involve patients and consumers all across the regulatory lifecycle, and a first meeting is in preparation for early 2017.
Overall, the presentations and the discussions recognised the will and aim for patient involvement and also the need for coordination and sharing experiences and best practices between NCAs and EMA to encourage consistent approaches.
- Medical Products Agency: the Swedish experience.
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EUPATI Patients-Regulator-Industry workshop, Berlin, 20 July 2016. Presented by Lena Ring, Lia.
Summary of the parallel workstreams
The industry group shared their summarized learnings and observations with the larger audience. In general, there is a need to gain more clarity on the conflict of interest question in order to be able to engage patients in an appropriate way. There is also a need to set a standard for compensation and fees across the industry. All industry partners are interested in a common framework for patient involvement across all stakeholders- it is time now to build upon what EUPATI has already done and develop clear, more detailed processes that we all are looking for. Industry is willing to share best practices but we need a platform to do so. Demonstrating value is also of utmost importance and perhaps this could be done by extracting metrics from existing case studies. Industry is eager to move forward and to have regulators be part of this journey of taking patient engagement to the next level.
The regulatory work stream unveiled the very different approaches to patient involvement in NCAs and in EMA, as presented in case studies and discussions, and a need to continue that dialogue has been identified. What kind of patients/patient groups should be involved? And where in the work process is it useful? How to compensate participants? Sharing experience and developing best practices are needed, and the EUPATI Workshop can aid in the start of a more structured exchange of experiences on patient involvement in regulatory affairs. As a follow-up action, this exchange will be further supported. Most cooperation is informal and while there are some legal provisions already (e.g. EMA’s interaction with patients, healthcare professionals and industry, based on article 78 of Regulation EC No 726/2004), a wider consensus on processes and the legal framework may help. Commonly accepted outcome metrics on the value of patient involvement is needed.
- Reports from parallel work stream: REGULATORS.
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EUPATI Patients-Regulator-Industry workshop, Berlin, 20 July 2016. Rapporteur: BirkaLehmann – BfArM, Chair: Nicola Bedlington, EPF.
The closing plenary discussion reiterated the main purpose of the workshop which was to bring regulators’, industry and patients’ viewpoints together to ensure that the interaction is understood, respected and trusted by all stakeholders. This had clearly been successfully executed and the group concluded by discussing how it all fits together and by identifying gaps and needs.
The regulatory work stream unveiled the very different approaches to patient involvement in NCAs and in EMA, as presented in case studies and discussions, and a need to continue that dialogue has been identified. The EUPATI Workshop may have been the start of a more structured exchange of experiences on patient involvement in regulatory processes. As a follow-up action, this exchange will be further supported.
The industry work stream demonstrated that the processes of patient involvement in R&D have evolved, but the evolution of frameworks, mechanisms, metrics and processes happens individually without much alignment and sharing between the different industry players. Establishing a neutral platform to enable pre-competitive sharing and alignment was identified as a need and perhaps this could be done through credible partners such as DIA. A need to establish a working group to look into developing a guidance on compensation was also highlighted.
Widening the EUPATI target audience: training could be expanded to hospitals, regulators, industry and other healthcare professionals and doing so will also help to build collaboration and trust amongst stakeholders. Furthermore, there is a need for a matchmaking tool to connect patients to projects. EUPATI currently has a ‘match-making’ section to connect stakeholders to projects and enable a two-way dialogue between stakeholders. How can we build on this?
For IMI projects:
Call topic text has been revised by IMI to indicate where patients can engage. The IMI Joint Undertaking also proactively indicates the role of patients when it launches its calls. Currently learnings are being gathered from the two projects where patients were involved. Patients who would like to get involved in IMI projects are requested to make sure that they are registered in the database.
- Plenary discussion at the EUPATI Patients-Regulator-Industry workshop
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Plenary discussion at the EUPATI Patients-Regulator-Industry workshop, Berlin, 20 July 2016. Chair: Nicola Bedlington, European Patients‘. Forum Moderator: David Haerry, European AIDS Treatment Group.
Conclusions and next steps
The main purpose of the workshop - to bring regulators’, industry and patients’ viewpoints together to ensure that the interaction is understood, respected and trusted by all stakeholders - took an important step forward.
More clarity and common understanding is needed on the demands, expectations and restrictions on patient representatives in different roles as members of committees, acting as representatives of an organization, or being individual experts.
Questions to be explored furthers are: Should the EUPATI training be expanded to other target groups in addition to patients? And can IMI involve patients more proactively, e.g. through its new IMI Patient Engagement Strategy?
The very different approaches to patient involvement in national competent authorities and in EMA need to be aligned on the basis of more knowledge of current experiences and best practices, leading potentially to a more systematic involvement in the daily work of the national and European agencies. This will be taken forward in the European medicines regulatory network.
The processes of patient involvement in R&D in industry have evolved, but the evolution of frameworks, mechanisms, metrics and processes happens individually without much alignment and sharing. Establishing a neutral platform, as for example DIA, to enable pre-competitive sharing and alignment is needed and will be explored further.
Both regulators and industry identified a need for clarity and alignment on compensation. This should be taken forward in multi-stakeholder discussions.
Both regulators and industry identified a need for development of commonly accepted metrics on outcome of patient involvement. Such an initiative could/should be taken forward by IMI.
Furthermore, the importance of increased inter-project communication of IMI projects and Horizon 2020 projects was highlighted.
- EUPATI Case Report on meaningful patient involvement in R&D and regulatory affairs.
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EUPATI Patients-Regulator-Industry workshop, Berlin, 20 July 2016.