2021 Patient Focused Medicines Development Annual Report

New additions to Patient Engagement Management Suite

The Patient Engagement Management Suite (PEM Suite) - your global hub for practical tools to plan, assess and execute any patient engagement initiative was significantly enriched in 2021.

New practical How-To Guides, have been finalized and added to the suite in 2021, driving the organizational adoption of patient engagement.

Pilots are now ongoing, with both EMD Serono and Ipsen utilizing the Guide for Patient Engagement in Early Discovery & Preclinical phases.

Following the completion of nearly 1400 surveys by patients and healthcare professionals across 5 continents, an infographic was developed to further disseminate and communicate this work.

Mobilizing a co-created, agile Monitoring & Evaluation (M&E) metrics builder for better patient engagement intelligence

Appropriate Monitoring and Evaluation (M&E) tools and approaches are essential for systemic impact of patient engagement. In 2021 a PEOF session was organized to build on the M&E framework started as part of the PARADIGM project. It enabled insight gathering to provide direction and shape to an operational and actionable M&E tool.

A new multi-stakeholder workstream was initiated, with 19 people, representing the patient community, industry and academia. They worked towards a simplified framework for M&E, encompassing PARADIGM identified criteria, as well as the Patient Engagement Quality Guidance dimensions. The goal: turning them into an easy-to-use and actionable digital tool, fully integrated into Synapse.

The group co-developed the first mock-ups for the tool, and layed out the plans for the beta testing coming in 2022.

Communication was also enhanced via a presentation made at the Patient Centricity & Collaboration conference on November 9. Underpinning this was the increasing recognition that stakeholders require additional support and resources to implement an organizational M&E strategy beyond a metrics builder - therefore, this will guide our work in this area during 2022.

PFMD was also approached by several organizations to collaborate on further developing a benchmarking tool for industry - this represents an exciting opportunity and leads to further consideration of organizational benchmarking and opportunities for PFMD to advance the development of a sector-wide rating/ranking system to further support organizational needs with regard to monitoring.

Securing Patient Engagement in Patient Experience Data to drive momentum & better decision-making for all stakeholders

Significant efforts have continued to fuse PE and patient experience data (PED), through our Patient Engagement & Patient Experience Data (PED) project.

370+ people got involved in the project development, through two dedicated sessions at the Patient Engagement Open Forum (PEOF) in 2021 and various working groups. A Steering Committee for the Collective Value Publication and Taxonomy workstreams was established. This multi-stakeholder, global group includes over 40 members representing industry, patient community, healthcare professionals, academia, regulatory bodies and HTA agencies.

Last year also saw the publication of our Highlighting Recent Trends in the Fast-Evolving Patient Engagement & Patient Experience Data Landscape report, which taught us that as patient engagement has become more prevalent in medicines development, the development and use of patient experience data is another critical way to further engage patients.

During 2021 IMI-PREFER and PFMD initiated a collaboration to support the IMI-PREFER sustainability goals and we remain in ongoing conversations to discuss patient preference studies, which are a valuable type of Patient Experience Data.

This key area for PFMD was a busy one during 2021. PFMD actively contributed to HTA & regulatory dialogue in Asia, working with the Center of Regulatory Excellence (CoRE) and Coalition to Accelerate Patient Engagement in Asia-Pacific (CAPE).

The PFMD PE & PED project also delivered a presentation at the 2021 CAPE Regional Multi Stakeholder Roundtable: Embedding Patient Engagement for Sustainable Health Systems. 50 + participants representing regulatory and HTA Asian agencies were present.

In the background, dialog with regional partners was ongoing as we sought to develop and support capacity & capability-building programs, including the sharing and translation of PFMD PE resources

Forging the first large-scale co-creation conference in Patient Engagement Open Forum

Building our capacity and capability was of course a priority once again last year. We poured our resources into our networks and tools that bring those with an interest in PE together.

For example, in 2021, the Patient Engagement Open Forum celebrated its first full and successful year beyond Paradigm. The event continued to prove its sustainability as a co-project organized by PFMD, EUPATI and European Patients’ Forum (EPF). Together, we have created a forum to shape and co-create real solutions FOR patients and WITH patients while providing a holistic perspective of patient engagement, its landscape and actors.

The goal of this project is to establish a trusted process to remunerate the patient community for interacting with the pharmaceutical industry through patient engagement activities. Over 20 pharmaceutical companies and 40 patient organizations have been involved in the co-creation process until now, together with key bodies such as the IFPMA Patient Centricity Working Group, the Efpia Ethics and Compliance Committee, and the Efpia Patient Think Tank.

A dynamic and interactive PEOF session on the topic, with over 90 engaged participants, took place in October 2021. Check out the session recording here.

A set of Harmonized Global Principles for remunerating the patient community for interactions with the pharma industry were co-created. A public consultation was launched. It involved 200+ individuals, including 95 survey responses and more than 15 organizational feedback reports.

Ongoing efforts to disseminate the outputs of the co-creation activities included the publication of two articles on the PFMD blog. As we look towards 2022, the next steps are to bring forward international standards on patient community remuneration through the co-creation of a Global Frameworks and a Methodology for determining remuneration at market value.

Clinical Trial Information Network: connecting information providers with information users with new distribution capacity

The Clinical Trial Information Network Project addresses core issues within clinical trial information sharing and aims to empower all potential beneficiaries with a view to better performance and ultimately better health outcomes.

By creating a new distribution capacity to optimize the exchange of standardized clinical trial information, the project’s goal is to improve patient matching to clinical trials and enable all players to further innovate.

The focus during 2021 was to create a solid foundation for the project by bringing together the right stakeholders and mix of expertise with whom to analyze the current ecosystem in order to draft a sustainable concept and plan. This was achieved by landscape mapping of the current clinical trial information ecosystem and in-depth interviews with both patient organizations and pharma collaborators on existing challenges and bottlenecks.

This allowed us to identify existing gaps and opportunities for collaboration, helping shape the Project Roadmap and incremental project phases, as well as the Proof of Concept Pilot. As a result, new multi-stakeholder collaborations were established with Codex, Trialscope, Breast Cancer Trials and Efpia.

Over 850 participants were engaged to co-create and design the patient needs and priorities of the digital project through three live Patient Engagement Open Forum sessions:

As a result of our efforts three distinct work streams were formed:

• Structuring the discussion: digital health landscape mapping from a PE lens, which will focus on preparing a CTA landscape paper on the need for PE in Digital and creating a set of PE in Digital Health tools for new initiatives.
• Equipping stakeholders with PE data governance resources, which will focus around health data management and the empowerment of the patient.
• In 2022 we will interview a multitude of digital health actors to develop a stakeholders expectations matrix, which will enable us to better understand the environment and the particular needs of each involved champion.