he task of promoting the generation and use of meaningful patient-generated data in drug development is a shared one. An interactive DIA Europe 2023 session concluded that collaborations between regulators, industry, academia, and patient advocates will drive patient-focused medicines development.

The role of patients in drug development and the associated decision-making continues to expand. Patient perspectives are important in setting research priorities and in planning clinical trials to ensure medical innovation addresses unmet needs. This is driving efficiencies by focusing all stakeholders on patients from the outset. There is further potential to harness the power of the patient voice in the regulatory process as well as in health technology assessments (HTAs) and in payers’ decisions about reimbursement.

The European Medicines Agency (EMA) has indicated that it wants robust patient experience data (PED) to be part of the marketing authorization process, and researchers and patients stand ready to embrace this opportunity. While several challenges remain to be addressed—notably in the areas of guidance, terminology, incentives, predictability, and transparency—the momentum is moving PED toward the center of decision-making. Industry, regulators, HTA bodies, research foundations, and patient advocates all play their part in catalyzing progress.

Inspired by a session at DIA Europe 2022, our diverse group of stakeholders has published a seven-point Patient Engagement Action Plan. In September 2022, the EMA held its workshop on Patient Experience Data in EU Medicines Development & Regulatory Decision-Making. Among the key outcomes of the EMA event was a commitment to produce a reflection paper that would advise on the generation and collection of PED.

Building on this foundation, our multistakeholder collective published a report in February 2023 capturing co-created solutions that were explored at a joint webinar organized by DIA and the Patient Engagement Open Forum. This prepared the ground for a DIA Europe 2023 DIAmond session which brought together those who generate patient data (patients, and public and private sponsors) and those who assess these data (regulators, HTA bodies, and healthcare authorities). Together, this diverse group identified practical ways to derisk and incentivize the use of patient-generated data, focusing on predictability, guidance, and taking responsibility for building a patient-focused drug development ecosystem in Europe.

Five Take-Home Messages

Panelists and participants agreed by consensus on five priority areas. Many of these priorities can and should be addressed through multistakeholder collaborations and/or early engagement:

1. The earliest possible patient involvement in design, starting from clinical trial strategy and evidence-generation plans.
2. Terminology is foundational: EMA’s Reflection paper on generation and collection of PED is an important opportunity to consider terminology, taking account of the global environment for clinical evidence generation.
3. Shared confidence in methodologies and predictability in requirements: development of a roadmap for proportionate methodological guidance to increase predictability of interpretation and support international harmonization.
4. Greater visibility of how PED has informed assessment conclusions will drive shared learnings, quality, and impact in serving the needs of all stakeholders.
5. Use of PED in HTA: Decision-makers should embrace patient evidence in their recommendations and be transparent about their needs and expectations of PED.

There are platforms that can host this type of collaboration, and EMA already signaled at the DIAmond session [at the meeting] its intent to lead some of them.

How can Regulators Contribute?

Fostering PED generation and use is of increasing importance to regulators. However, despite the regulators’ clear signals, the systematic collection of PED is not yet common practice for some medicines developers.

Through scientific advice mechanisms and the qualification of new methodologies, European regulators can engage with investigators to support the development of high-quality patient evidence. The patient voice should be heard in developing scientific advice, evidence-generation plans, and regulatory assessment to ensure that they generate meaningful PED. Positive lessons can be learned from the pilot phase of the EMA’s CHMP (Committee for Medicinal Products for Human Use) early contact with patient and consumer organizations.

Providing guidance and clarity on the need for PED will send a strong signal to drug developers. Transparency also needs to be increased on how PED is assessed and how it supports benefit-risk decision-making. Transparency is vital to enhancing the quantity and quality of PED. It is important that the work of reviewers at regulatory bodies reflects the positive view of PED expressed in high-level regulatory policy.

In addition, incentives can further encourage the generation of meaningful PED that are valued by regulators and other decision-makers. Along with action by European regulators and others, ongoing work at the ICH can further help to harmonize the generation and uptake of PED across the globe.

How can HTA Authorities Contribute?

The inclusion of PED by HTA bodies and reimbursement authorities varies across Europe. In Norway, for example, patient evidence is used to illustrate the benefit of intervention, and quality of life is considered in HTA assessments. The variety of approaches poses challenges for marketing authorization holders, who are often unclear about what patient evidence would be of value to HTA decision-makers.

The role of patient evidence in HTA decision-making may be influenced by the new HTA Regulation (EU 2021/2282) which will apply from January 2025. Work is under way to coordinate standards between HTA bodies, presenting opportunities to harmonize approaches to PED.

Engagement between stakeholders, and guidance to investigators, can help to streamline the development of robust PED. To ensure that the right data are collected from the earliest stages of clinical development, it is also important that the priorities of regulatory decision-makers, HTA, and reimbursement authorities are aligned.

How can Industry Contribute?

It is vital that industry promotes the systematic incorporation of the patient voice in drug development. Companies have a role to play in raising awareness within their organizations of the value of patient engagement in general, and the importance of generating patient evidence in particular. They can also help to normalize PED in the scientific community, and to build capacity for data gathering and handling in patient organizations.

Listening to the patient voice allows medical product developers to ensure that therapies address what is important to patients. PED can particularly help developers recognize concepts of importance. Even in well-known conditions, thoughtful interpretation of PED can yield fresh insights and demonstrate differences in perspective between patients and clinical experts.

The treatment experience itself may also be explored through the collection of PED, with preferences and priorities identified by patients informing medical product development. Preferences for drug or product attributes may be instructive in optimizing adherence to treatment regimens and improving patient outcomes. PED can also enable sponsors to identify opportunities to support healthcare providers and patients in shared decision-making and working together to manage disease.

Finally, a patient-centric development program embraces opportunities for sponsors to communicate PED-based insights through publications. Such communications may serve as the basis for payer consideration as well.

How can Academia Contribute?

Fostering research on novel methodologies, frameworks, and measures for the generation of PED in academic institutions can catalyze efforts to raise and harmonize standards. European multistakeholder collaboration, with strong academic contributions, can play a key role in this endeavor, as evidenced by Innovative Medicines Initiative projects such as PREFER (Patient Preferences in benefit risk assessments during the drug life cycle) and PARADIGM (Patients Active in Research and Dialogues for an Improved Generation of Medicines).

In addition, academic research can also be a key driver in the generation of PED which is specific to indication areas but not targeted to specific product characteristics, for example, by running patient preference studies on patients’ unmet needs and priorities in a given therapeutic area or generating PED in platform trials.

How can Patient Advocates Contribute?

Patient organizations have unparalleled knowledge of patient communities. Their role as advocates and allies positions them as a trusted source of high-quality information and evidence on the lived experience of patients. These organizations already generate large volumes of PED through surveys and in gathering patient preference data. They are well-placed to do more. For example, patient advocacy groups deliver disease mapping on quality of life, burden of disease, and treatment adherence; patient preference surveys on needs, expectations and values; and insights on inequalities of outcomes and access.

This applies to large patient communities, such as diabetes and cancer, as well as to organizations advocating for rare disease communities. For example, the Duchenne Data Platform amplifies the patient voice using high-quality data. The initiative has been presented as an example at the EMA Patients’ and Consumers’ Working Party (PCWP) and Healthcare Professionals’ Working Party (HCPWP), as well as at the EURO-NMD project developed by the European Reference Network (ERN) on rare neuromuscular diseases. In addition, the pan-European cancer and hematology patient communities are currently collecting evidence on the generation, use, and publication of PRO data in clinical trials across the European Union in the WECAN EuroACT project.

The patient community can raise awareness of the value of PED in the regulatory process among patients and advocates. Investing in skills development and capacity building will unlock opportunities to collaborate with investigators and decision-makers. Guidance from decision-makers is vital to informing this work.

How can we all Work Together?

The task of advancing the use of PED in regulatory and reimbursement processes is a shared one. Co-creation can ensure that all stakeholders are on board during this crucial period. At the DIA Europe 2023 DIAmond session, participants had an opportunity to discuss and vote on priority actions. Key areas for impactful collaboration include the development of aligned terminology, guidance, international convergence, and managing uncertainties.

Next Steps

A multistakeholder initiative in Europe to advance patient experience data should serve as a catalyst to addressing these challenges together. To be inclusive of all stakeholders, to achieve synergies with existing initiatives, and to avoid duplication of efforts, the host, governance model, and interfaces of such an initiative must now be discussed and agreed. We are pleased to initiate this conversation and look forward to the widest possible engagement from all stakeholders.