Patient Engagement That Enables Regulatory Decisions
Co-Creating Next Steps in Patient-Focused Drug Development
Magda Chlebus
Nicholas Brooke
The Synergist
Juan García Burgos
European Medicines Agency
Sharon Gorman
Annette Bakker
Children’s Tumor Foundation Europe
Almath Spooner

egulators’ expectations of patient involvement in drug development are growing, while new tools are helping to remove practical barriers to working with patients. A multistakeholder group is co-creating a repository of resources that can accelerate progress ahead of the DIA Europe 2023 DIAmond Session on 24 March 2023. The road ahead is clear; only the speed of progress is in doubt.

Patient experience data (PED) and patient-focused drug development (PFDD) are set to become mainstream features of our healthcare systems. Medicines regulators expect that new products have a meaningful impact on patients, including quality of life, and that this can be demonstrated by including robust evidence designed, generated, and analyzed with patients’ perspectives. This is a step change in the transition from past rhetoric on intended patient-centricity toward a new model of partnership in drug development in which patients and patient advocates are equal and active partners.

The pace of progress has been impressive and needs to accelerate further. A growing number of successful examples have illustrated the value of patient engagement as well as new tools to help overcome practical and technical challenges that might slow the march of progress. These advances are welcome, but there is still a long way to go before the journey is complete.

Collaboratively developed by our multistakeholder group, the Patient Engagement Action Plan published in July 2022 set out Seven Steps for Boosting Patient Engagement, including multiple examples of patient engagement initiatives by regulators, patient organizations, multistakeholder nonprofits, and the Innovative Medicines Initiative (IMI), among others.

Ironically, we are pleased to report that this Action Plan is already going out of date, thanks to the multistakeholder efforts we describe here below.

For example, in September 2022, the European Medicines Agency hosted a Multi-Stakeholder Workshop on patient experience data in medicines development and regulatory decision making. This meeting addressed several of the steps outlined in our 2022 article, including the need for progress on common definitions, the need for guidance, and transparency on how patient data is used in decision making. EMA has committed to developing a reflection paper to provide advice on the best approach to generating and collecting patient experience data.

In December 2022, the Innovative Health Initiative (IHI), a large EU public-private partnership, published a funding call for projects focused on “patient-generated evidence to improve outcomes, support decision making, and accelerate innovation.” This is significant given the funds available to the IHI, its diverse consortium of public and private stakeholders, and its role as the successor to the IMI, which supported several projects in this area.

In the US, the Milken Institute published its report on The Current Landscape of the Science of Patient Input in November 2022. The institute’s Future of Health Summit in December further addressed these issues from multiple perspectives, including the potential of partnerships between nonprofit and for-profit organizations in patient engagement research.

Co-creating Solutions to a Shared Challenge

This article reports from an interactive session hosted jointly in November by DIA and the Patient Engagement Open Forum, which explored how to move the Seven Steps outlined in our July article from theory to practice. This exploration was conducted through GroupMap, a co-creation tool that allowed almost 100 participants to contribute to an open, cloud-based collaborative document. This diverse group shared existing resources that can support these Seven Steps, identified gaps that must be filled, and debated whether additional steps will be required to complete the journey. The GroupMap remains open for further input.

  1. Meaningful involvement of patients in research priorities, protocol design, and regulatory decision making
  2. Participants highlighted the need to build capacity among patient organizations and patient advocates as well as their counterparts in all stakeholder groups to enable patients’ full participation in engaging with a wide range of decision makers. Practical support is essential. The “How To” guides developed by the nonprofit collaborative Patient Focused Medicines Development (PFMD) can help to equip patients to be effective, equal partners by providing information about preclinical development, clinical outcome assessment (COA), and protocol design.

  3. Find and share examples of good practice and existing tools
  4. Showing practical examples of how to conduct patient engagement, and how to do it well, is vital to sparking wider uptake. A number of publications showcase patient engagement in action:

    • The Council for International Organizations of Medical Sciences (CIOMS) has published a report on a working group examining Patient involvement in the development, regulation, and safe use of medicines. It provides a global overview of current knowledge and existing initiatives, with multiple examples and recommendations for the future.
    • A briefing note for researchers by the National Institute for Health and Care Research (NIHR) in the UK provides practical examples on how members of the public can be involved in research.
    • IMI Paradigm has published a suite of tools and recommendations which bring together a wide range of guidance documents, recommendations, and practical documents including patient engagement agreements and a code of conduct.
    • Testimonials and tips for involving consumers and community groups in health research are available from the Western Australian Health Translation Network.
    • The EUPATI Toolbox provides patient engagement case studies along with guidance on patient involvement with regulators, HTA bodies, ethics committees, and the pharmaceutical industry.
    • PFMD has published Patient Engagement Quality Guidance which maps existing guidance on patient engagement and identifies gaps to be addressed.
    • PFMD has also published a Book of Good Practices covering patient engagement at various stages of the medicine development process.
  5. Promote patient engagement training
  6. The wide variety of new approaches to patient engagement is the result of a welcome expansion in activity in this field. Increasing the quantity and quality of patient engagement activities is vital to its credibility and usability by decision makers. Training plays a vital role in extending the practice of patient engagement and raising its standards. It is essential that training is viewed as a two-way street: Patients and patient advocates need new skills and knowledge to play a full part in medicines development, while health professionals, regulators, HTA bodies, industry, and others should be educated in good patient engagement practice. Crucially, all parties must be trained to view other stakeholders as partners rather than subjects, servants, or subordinates.

  7. Measure patient engagement
  8. Monitoring and evaluating patient engagement using established and accepted metrics will drive quality and learning. The goal of measurement is not to find fault with the performance of patient engagement but to drive quality improvement. This will ultimately deliver a more rigorous form of patient engagement that produces data upon which decision makers can rely. Several tools help practitioners evaluate their work:

  1. Agree on a common language and terminology for patient evidence
  2. If diverse groups of stakeholders are to communicate with a single purpose, they must speak the same language. This requires shared definitions and precise terminology; to ensure a universal lexicon, education is crucial. It is important that this common language remains accessible and inclusive. The lingua franca of patient engagement must be patient-centered so that it is within reach of patients, parents, and other advocates whose skills do not include high levels of clinical or medical knowledge. A minimum level of key definitions, without asking patients to embrace new jargon before taking part in dialogue, is required. The goal of harmonized terminology is to lower, not raise, barriers to engagement:

Co-Creating Next Steps in Patient-Focused Drug Development

  • The value of patient input is clear to all key stakeholders.
  • Regulators expect sponsors to incorporate the patient voice early during drug development and to turn it into patient experience data that supports regulatory decision making.
  • Existing guidelines, training, and metrics will facilitate progress, with more tools and support to come.
  • The EU Innovative Health Initiative can be a catalyst for change.
  • DIA’s July 2022 Global Forum proposed a Patient Engagement Action Plan: Seven Steps to Move the Needle.
  • A subsequent joint DIA and Patient Engagement Open Forum session identified gaps and brainstormed ways to accelerate action in these seven key areas.
  • An upcoming DIA Europe DIAmond Session is an opportunity to explore the role of patient-generated data in regulatory decisions.
  1. Collaborate on guidance to help sponsors generate the right evidence
  2. Solid, explicit definitions feed into clear guidance. Clear guidance provides clarity to industry and investigators on what data to collect and how to collect it. There has been positive movement on this topic on both sides of the Atlantic, but further guidance is anticipated. In the meantime, industry and others can engage with regulators on a case-by-case basis to receive scientific advice and input on their drug development plans.

    There is a risk that industry could slow its progress while waiting for signals from regulators, and that regulators may wait for action from industry. The best option is for all stakeholders to jump in together and share any perceived risk associated with making the first move.

    • FDA is developing a series of four guidance documents on incorporating the patient voice in medical product development and regulatory decision making.
    • EMA has committed to publishing a reflection paper on patient data.
    • Within the IMI2 grant EU-PEARL as well as in the Children’s Tumor Foundation-supported REiNS collaborative, patients actively participate in clinical trial design and clinical trial endpoint development.
    • The PFMD Global Patient Experience Data Navigator is clarifying, integrating, and streamlining the design, generation, analysis, and decision making about patient experience data through (and with) patient engagement.
  3. Communicate on whether and how patient input and experience data have been or will be used in decision-making processes
  4. Regulators and industry benefit from greater transparency on how they use patient insights in decision making. Patients also need to see their contribution make an impact if they are to continue to engage in medicines development. Multistakeholder organizations can also play a role in sharing practical experiences of how decision makers responded to patient data, including examples of what was not accepted. This will further drive quality improvement and help to make PE more mainstream.

    • Regulators are exploring ways of increasing transparency on how patient data is used.
    • The Understandings, Learnings, Guidances: What do we know so far on the Use of Patient Experience Data (PED) for Healthcare Decision-Making? session at the Patient Engagement Open Forum 2021 included a proposed checklist based on regulator feedback.
    • DIA Europe 2023 will present Patient Generated Data – Does it Influence Decisions? on 24 March 2023.

An Eighth Step to Patient Engagement?

The co-creation exercise also explored whether there are gaps or resources not captured in the Seven Steps previously outlined. In response, participants suggested improving education on compliance and legal aspects of PE; better coordination within and between organizations to ensure synchronized approaches to working with patients; embedding patient involvement to make it an enduring feature of drug development; and devising mechanisms to value and acknowledge patients’ input.


Patient engagement in medicines development is shifting from intention to practicalities and operational implementation. For many stakeholders, including regulatory decision makers, factoring in the patient perspective is on the path to becoming a natural reflex, as a range of tools exist, experience is building, and skills are multiplying.

There is still work to do, but there is also a clear willingness among stakeholders to travel together at the same pace. This collective effort is laying a foundation, not only for improved involvement of patients in how the next generation of medicines are developed, but in other areas of healthcare. There is an opportunity to move beyond focusing only on medicine development and to embark on sustainable and resilient collaborations in all aspects of healthcare, with patients as key players in innovation and in their care. But we must first get the patient engagement model right in drug development, then build from there to optimize outcomes for all.

You can contribute to this work by adding your input to the open GroupMap document upon which this article was based. Please share your resources and ideas to play your part in the next chapter of the global patient engagement story.