EuropaBio Patient engagement with the European Medicines Agency

September 2020

Aim: This position paper aims to emphasize the importance of obtaining patient input in the development of medicines and of reflecting this input in the regulatory and HTA decision making process. EuropaBio recognises the huge benefits that patient engagement in drug development can bring to patients' care and quality of life, as well as to the entire healthcare system. Among other aspects, patient involvement helps to improve protocol research questions, understand appropriate comparators, and identify patient relevant endpoints. The efforts made by Regulators and HTA bodies in Europe to consult with patients when discussing medicinal products are much appreciated and fully supported. EuropaBio would like to advocate that patient engagement and the patient's voice is also more consistently and transparently reflected in regulatory/HTA assessment and decision-making processes which should be supported by appropriate guidance. EuropaBio is sharing this position paper with the European Medicines Agency (EMA) to advocate a European guidance framework on patient engagement that allows sufficient flexibility but enables alignment on cornerstone requirements with key stakeholders and draft FDA guidance, as well as considering the outcome of initiatives, such as the IMI PREFER project.

EuropaBio policy position
There is an urgent need to develop specific EMA guidance around patient engagement in drug development and how this is evaluated during regulatory assessment of medicinal products. A European perspective should be designed to consider global initiatives and align whenever possible with other Agency guidance.

1. Clear guidance on PFDD which cover all stages of development and permits flexibility in how stakeholders engage with patients and collect their perspectives.
2. Guidance should provide recommendations for capturing and incorporating patient input at clearly defined stages of the regulatory review timeline and how this input will be considered during the approval and reimbursement process, as well as proposed timelines for the additional dialogue.
3. Guidance should address both patient experience and patient preferences (the latter should consider/be revised based on IMI PREFER output).
4. Europe needs to develop an approach to enable three-way discussion between patients/patient groups, regulators and industry on drug development that meets the needs of patients.
5. European stakeholders should explore the opportunities offered by externally led PFDD meetings and consider whether this can be adapted to the European setting.
6. Focused and more technical guidance is needed on COAs/PROs. The guidance should provide recommendations for development and modification/use of existing COAs, the latter being particularly important in rare diseases. Guidance should also address how to translate COAs into endpoints in clinical trials to inform regulatory decision making. In addition, more alignment between EMA and HTAs is required with respect to tools accepted for regulatory approval and reimbursement.
7. Patient input and data should be formally acknowledged and its use by regulators and value in decision making made transparent.

Proposed EuropaBio actions on patient engagement in the near future:

a) Engage with the patient community, EU pharmaceutical trade associations, EMA and other EU stakeholders (such as HTA and payers) to co-develop an EU roadmap for patient engagement, which will include:
i) EU guidelines aligned in terms of key principles with FDA guidelines as well as considering potential updates to existing guidance documents. Europe should develop a clear vision of what its needs are in this space and work with the US at minimum to design aligned guidance based on the same methodology and systematic inclusion of keys principles in terms of patient engagement activities. ii. Developing an approach to enable discussion across patient community, regulators, payers and industry to ensure development programmes meet the needs of patients and that the evidence needed by HTA and payers is incorporated early in drug development.
ii) Determining what the patient community needs are in terms of providing their input and experience, understanding how this informs drug development and decision making, and what is important to communicate back to the patient community.
b) EuropaBio continues to support ongoing patients' involvement in EMA activities, Committees, working parties and the EuropaBio Patient BioForum.
c) EuropaBio will strengthen the dialogue with EFPIA to ensure activities are aligned and mutually supportive.
d) EuropaBio will find ways to be updated on the outcomes of IMI PREFER project on Patient Preferences in Benefit-Risk Assessments during the Drug Life Cycle and of its potential impact in order to tune with other trade associations on the final position on patient engagement.
e) During future EMA/European Commission stakeholder meetings focused on patients, EuropaBio will support a global convergence approach concerning patient engagement in EU regulatory procedures and impact on regulatory decision-making.
f) Proactively engage with regulators and HTA bodies to ensure the proposed new ICH guidelines on clinical outcome assessments (COAs) and patient preference methods are fit-for-purpose and allows sufficient flexibility whilst harmonising requirements for the qualification of new instruments across regions.

Download the file below to read the full Position Paper.