Authors: Teresa Finlay, Giorgio Barbareschi, Maria Dutarte, Bojan Cigan, Lidewij Vat
Acknowledgements to Giulio Maria Corbelli (EATG), Luis Mendao (EATG), Daniel De Schryver (Janssen), Lyudmyla Maista (Medicine Patent Pool) for their input.
In 2007, the European AIDS Treatment Group (EATG) launched a series of multidisciplinary meetings to address the needs of people living with HIV and the Hepatitis C virus (HCV) for whom inclusion in clinical trials and treatment options were limited. The objective of the meetings was to obtain access to clinical trials and new drugs for people with HIV/HCV co-infection. The annual meetings were held in Sitges in Catalonia, Spain, over a period of 10 years. They were attended by representatives of different stakeholder groups with an interest in medicines development. The attendees included patient representatives from different organisations, healthcare professionals, pharmaceutical companies, academics, local health authorities, regulators and representatives of international organisations.
Unmet patients’ needs
Hepatitis C (HCV) is highly prevalent, progresses more rapidly, and causes significant morbidity and mortality among HIV-positive people. When the Sitges Series started, people co-infected with HIV and HCV were excluded from participation in clinical HCV studies, and there were no regulatory requirements or recommendations for studying novel treatments in co-infected people. These meetings with all stakeholders focused on how people with HIV/HCV co-infection could be included in medicines’ development and subsequently have access to the medicines. Collaboration between the patient community and all relevant stakeholders was seen as crucial to meet the needs of people with HIV/HCV co-infection.
Evaluation of the Sitges initiative
The Sitges Series was one of the cases of patient engagement used to test a draft version of the PARADIGM project’s Patient Engagement Monitoring and Evaluation Framework. Developed from published evidence (Vat et al 2019) the Framework has been created by project partners using 24 cases of patient engagement initiatives in medicines R&D (Vat et al 2020 – forthcoming). The Framework aims to improve the understanding of the steps on the pathway to impact. These include input, activities/process, learnings and changes, and impacts, and how these are influenced by context. (See Figure 1. For the EATG’s tailored framework). Researchers at the University of Oxford and the VU Athena Institute in Amsterdam collaborated with EATG members and staff to co-create a tailored version of the Framework by applying an early draft to an evaluation of the Sitges Series, including the development of metrics for a formal evaluation, using qualitative and quantitative methods. A preliminary qualitative assessment of the impact of the initiative was conducted during a focus group meeting with a representative of each stakeholder group who had attended one or more of the Sitges Series meetings.
Figure 1: PARADIGM Monitoring and Evaluation Framework for Patient Engagement tailored to the Sitges Series for EATG (click here for full view)
Focus group participants reported that the Sitges Series led to increased awareness and knowledge and altered attitudes of all who attended. Co-infected people’s needs were made clear as were the processes of medicines development. Barriers to participation in clinical trials and the work of companies developing medicines for HCV infection were presented. Consequently, clinical trial designs changed to include people with co-infection, medicines R&D has speeded up as a result of patients’ input, a strategy for medicines access was developed and the resulting treatment meets patients’ needs more directly. Notably, significant regulatory change has been achieved in this area including the European Agency for the Evaluation of Medicinal Products (EMEA) guidelines for drug development for co-infection with HIV/HCV.
However, in addition to achieving the Sitges Series’ principal objectives, wider impacts resulted from the collaboration. One patient representative described the direct benefit for patients in their country as a direct result of their representation at the meetings:
“Because I was there, I could have an impact in xxxxxxxx [home country]… being able to have an impact on medicines development [was achieved] by all the stakeholders together.”
Impact for one industry representative was described as:
“Patient-centricity for us [Pharma company] is really the main outcome… [the Sitges Series] adds understanding and how you can solve [problems] in a collaborative way by putting all [stakeholders] around a table.”
There was a perception that the difference between the long-term Sitges Series and the European Community Advisory Board meetings (ECABs) on HCV, was that the Sitges Series was able to tackle broader issues around patients’ needs and access to medicines than the detailed clinical trial designs that the ECAB meetings discuss. Broader issues include what is ethically acceptable to patients and potential participants in medicines R&D; this was possible within the Sitges Series framework because representatives from all stakeholder groups, including regulators, were represented.
Metrics for evaluation of each component of the Framework were selected by the focus group (see Figure 2). This was a theoretical exercise because the initiative had been completed and the opportunity for evaluation had passed. However, it is hoped that similar long-term initiatives that the EATG is organising will make use of the tailored framework and metrics for their planning, monitoring and evaluation.
Figure 2: Menu of metrics for evaluation (click here for full view)
Overall value assessment
Focus group participants were asked to rate the value of the initiative on a scale from 1 (negative impact) – 5 (high impact). Everyone reflecting on the initiative rated the Series’ value very highly (5). That value was felt to be linked to the repeated meetings with all stakeholders around the table; this facilitated collaboration, reciprocal relationships, trust and understanding of others’ perspectives. The success of this collaboration was summed up by an industry representative’s observation
“Society is in dire need of understanding that health is made by putting people around a table and co-creating solutions … the model could be shared to advantage.”
Patient engagement has the power to benefit all the stakeholders included, both directly in achieving the objectives of the initiative, and more widely by positively impacting individuals and organisations. As a result of developing relationships between stakeholders through meeting around the same table over a long period of time, a shared and profound experience of learning engenders a willingness to tackle the challenges, builds trust and co-creates solutions. The PARADIGM M&E Framework has been developed to enable stakeholders in patient engagement initiatives to plan, monitor and evaluate their activities. The aim of this is to realise the returns on patient engagement in order that it becomes meaningful and sustainable. Applying the Framework to the Sitges Series both framed its impacts and value successfully but also supports the evidence (Vat et al 2019) that multi-stakeholder collaboration is often fruitful for effective R&D.