Patient Engagement for Medicines Development in a nutshell

Framework building working groups

PFMD set up three Framework Building Working Groups (WGs) that each focused on different phases of medicines development. WG1 focused on research and pre-discovery phases, WG2 focused on clinical trial phases and regulatory approval, and WG3 focused on post-approval phases.

Together the action items from these three WGs formed a combined plan for next steps, which were taken on by task forces formed by WG volunteers and other professionals.

Past meetings

The first two framework building Working Group meetings were held in Brussels on the 16 and 17th of  November 2016 at The Synergist offices in  Brussels. The meeting served to mobilise a dynamic community of stakeholders with diverse expertise, extensive networks and experience, and who are motivated to contribute to the PE journey.

A common theme of the meeting was agreement on the need  to maintain momentum in driving patient engagement forward and secure participation from stakeholders that are not yet around the table. Working Group 3 was held in February 2017 with the same objective.

Objectives of working group meetings

Although the growing understanding for the importance to have patient engagement from the very early to the latest phases of medicines development, there are no industry wide standards or agreement on how it should be done. These working group meetings aimed at identifying key gaps, opportunities, key stakeholders to be involved and the next steps to take in order to co-create the Patient Engagement Meta-Framework – a guidance and practical toolkit that would help all stakeholders, disregard of how advanced they are in PE, to do more meaningful PE and get measurable results out of it.

13 participants:

  • 3 patients / patient organisation representatives
  • 3 independent experts
  • 7 industry representatives

16 participants:

  • 4 patients / patient organisation representatives
  • 1 independent expert
  • 9 industry representatives (2 absent)
  • 2 regulator / HTA representatives

19 participants:

  • 4 patients / patient organisation representatives
  • 2 independent experts
  • 1 university hospital representative
  • 3 regulatory / HTA representatives
  • 9 industry representatives

Outcomes of the WG meetings

The discussions had many overlapping themes which were turned into prioritised action items, that were later take on by specific task forces. Main topics of discussion revolved around the following:

  • There is a strong need for a  guidance, or a “book of good practices” to clarify HOW, WHEN and WHY to involve and engage meaningfully with patients – In addition, there is also a need to create a set of guidelines to describe and identify good PE practices and a clear and generally accepted criteria or standards for representative and high quality patient input
  • There is a need to create and enforce a structured/ systematic feedback loop in all phases of medicines development – In addition, there is a need to develop a broadly endorsed framework for securing support for patient adherence (a holistic framework beyond a product/ company)
  • There is a need to clarify roles/responsibilities of patients and other stakeholders in PE, and to align stakeholders’ objectives
  • There is a need to create a platform/ hub where researchers can connect with patients or patient organisations
  • There is a need to include regulators, payers, HCPs HTA , academia more strongly in all phases of medicines development cycle
  • There is a need to build the capabilities, knowledge and training for all stakeholders based on existing material and experiences

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