The European Federation of Pharmaceutical Industries and Associations (EFPIA) has issued this week new guidance on working with patient groups. The document has been prepared by the EFPIA Patient Think Tank, which is a forum comprised of representatives of patient organisations and the research-based pharmaceutical industry.

The new guide provides an additional point of reference and supplements the 2011 EFPIA Patient Organisation Code of Practice. It underlines the rationale for interactions between the pharmaceutical industry and patient organisations, providing clear principles that justify engagement between these two groups and a practical list of potential hurdles and proposed solutions. It is not applicable to interactions with individual patients but the rationale underpinning the guide may be applied to such interactions too.

Principles for Engagement

The guide recognises the benefits of effective collaboration between patient organisations and industry in ensuring that the patient perspective is taken into account when decisions which affect patients are made. However establishing an appropriate engagement between the industry and patients organisations without breaching the legal restrictions governing these interactions represents a challenge. The following 5 principles have been distilled by the Think Tank as necessary to ensure compliance:

  • Clarity of Purpose – a legitimate need for the interaction and the desired outcomes must be identified in advance.
  • Transparency – of aims and in particular of financial relationships between the two groups; any compensation paid to patient organisations and their representatives, should be proportional and commensurate with experience, expertise and time invested.
  • Independence – of decision-making, policies and communications helps to ensure credibility; funding from a wide range of sources is therefore always preferable. (NB: the EFPIA Patient Organisation Code prohibits its members from requiring to be sole funders of patient organisations or patient organisation’s activities.)
  • Mutual Respect – reflecting a collaborative approach, valuing the contribution of both parties.
  • Non-interference in the healthcare professional-patient relationship.

In Practice: examples of collaboration

Providing information to individual patients: The guide reminds stakeholders that, while European law prohibits the advertising of prescription only medicines to members of the public, companies may provide non-promotional information on human health and diseases and collaborate with Patient Organisations on educational initiatives. Companies are also required to provide information about specific products to patients through patient information leaflets supplied with every pack of product and the Clinical Trials Regulation provides that clinical trial data, including summary results and clinical study reports should be made public.

Research and Development and Clinical Trials: Patient Organisations may provide companies with information regarding the challenges of living with a disease, which can be used to guide R&D processes, aims and objectives. Patient Organisations may also work with regulators, ethics committees and investigators, influence trial design, e.g. through contributing to the design and content of patient information and consent and assist with trial recruitment and retention.

Marketing authorisations: The guide notes the increasing participation of patients in regulatory processes, including acting as experts in their disease area, to input into pharmacovigilance, to provide real world evidence that may further inform the regulation of medicines and to contribute to communications on medicines.

Health Technology Assessment: The guide also recognises that although patient involvement in Health Technology Assessment (HTA) processes is still developing in Europe, there is a clear role for patients to play in defining the value of medicines. The Think Tank advocates for patients to be able to serve as members of HTA boards, committees and workgroups. It emphasizes that they should also contribute to the design and preparation of patient-friendly HTA report summaries and the dissemination of HTA findings to policy makers.

Appropriate use of medicines, healthcare systems and use of Real World Data; Patient led data may be used to develop patient focused/patient friendly guidance on the appropriate use of medicines to prevent abuse, reduce medication errors and wastage, and improve patient education, as well as shaping healthcare delivery in the context of the outcomes that make the most difference to patients. The guide also acknowledges the use of health informatics to support the delivery of improved health outcomes, relevant to patients and the collection and use of Real World Data/Big Data in decisions that affect patients’ access to medicines, while emphasising the importance of adequate data protection to ensure patient privacy.

The final section of the guide provides a list of common challenges or “hurdles” to the interaction between industry and patient organisations together with proposed solutions. The list, which is not intended to be exhaustive, identifies the following key challenges:

  • Interactions may be perceived as commercially motivated.
  • Assumptions of inappropriate influence of the industry on patient organisations decision-making.
  • Difficulty of patients groups identifying the right contact points within companies.
  • Lack of resources, mainly for smaller patient groups, to engage in HTA processes.
  • Managing multiple codes of practice and terms of engagement for the different sectors.
  • Lack of inclusion of patient groups in the discussions conducted by regulatory bodies.

The solutions proposed by the guide include actions for the various stake holders:

  1. For companies: clear identification of patient benefit and legitimate needs of the interaction, ensuring good governance in reporting and adherence to laws and codes, publicly displaying code of conducts, support for diversity of funding sources and ensuring transparency of financial relationships and establishing company online resources to sign-post patient groups to the appropriate company contact points.
  2. For patient organisations: publicly displaying codes of conduct and articulate decision-making processes independent of industry, ensuring transparency of financial relationships and diversity of funding sources, building their own capacity through initiatives like the European Patients’ Academy (EUPATI) and their understanding of the applicable legal and regulatory requirements.
  3. For competent authorities: creating and resourcing patient engagement processes (taking EMA’s processes as a model) in particular for HTAs.
  4. For industry associations: working on a greater alignment of codes of practices across the different sectors and ensuring knowledge and understanding of this guide.

As with other guidance issued by the EFPIA, this guide is mandatory for EFPIA members, which includes 33 national associations and 42 leading pharmaceutical companies, and a good point of reference for non-members. Further practical examples of industry, Patient Organisations and healthcare system collaboration may be found in the EFPIA Health Collaboration Guide.