Table 2

Overview of frameworks and resources assessed for PE quality guidance co-creation

FrameworkFormat, objectives and approachesStakeholdersKey learnings and outputs
Clinical Trials Transformation Initiative33 Set of recommendations to improve participation of patient organisations in the work of clinical trial sponsors (the industry and academia), which includes meaningful engagement of patients throughout all stages of the Medicine Development ContinuumMultistakeholder
  • Early involvement

  • Comprehensiveness

  • Expectations of mutual transparency and confidentiality

  • Diversity and representativeness

  • Creation of standard metrics to assess partnership effectiveness

Patient Focused Drug Development (PFDD) Conceptual Framework or University of Maryland Centre of Excellence in Regulatory Science and Innovation (M-CERSI)34 Forum for patient groups, Food and Drug Administration (FDA), biopharmaceutical industry, payer and other organisations to voice views, challenges, activities and aspirations for PFDD, as well as future direction and opportunity for collaboration. The Framework Supports FDA PFDD concept (Prescription Drug User Fee Act VI and 21st Century Cures Act)Multistakeholder
  • PFDD definition

  • Defining engagement level by the gradual criteria:

  • Patient role

  • Continuity

  • Meaningfulness

  • Representativeness

  • Temporality

  • Importance of harmonisation among patient groups and national regulators/health technology assessment bodies

  • Persistent challenge of measuring success

National Health Council/Genetic Alliance Framework35 Recommendations of the multistakeholder working group to reach a consensus on what it entails to meaningfully engage patients and identify key gaps and barriers in patient engagement across drug research, development and approvalMultistakeholder with action plans for:
patients/community, industry, academia
  • Three categories of barriers: culture, communication and regulation

  • Focus on meaningfulness and its criteria/questions to assess engagement

  • Criteria of best practice examples

  • Complex solutions to overcome the barriers

Patient-Centred Outcomes Research Institute (PCORI) engagement rubric36 37 PCORI engagement principles are developed to illustrate and provide guidance around how input from patients and other stakeholders can be incorporated throughout the entire research process with the particular focus on study planning, conduct and disseminationMultistakeholder
  • Reciprocal relationships

  • Co-learning

  • Transparency, honesty and trust

  • Partnerships throughout study planning, conduct and dissemination

  • Supported by real-world examples

Evidence adoption framework38 This case-supported framework examines the factors that may affect the pace of evidence adoption and application into routine clinical practice. Aday, Andersen and Rogers’ framework/model of innovation diffusion and healthcare utilisation were used as baselinesMultistakeholder categorised by sectors:
public, private and academic
Key factors to be taken into consideration:
  • Validity, reliability and maturity of the science

  • Communication of the science

  • Applicability

  • Economic drivers

  • Integration into guidelines

FasterCures Value Framework39 This value-based framework was developed to determine gaps in assessing value of treatment options and based on the four earlier established frameworks/ approaches within oncology (American Society of Clinical Oncology, Institute for Clinical and Economic Review, MSK Drug Abacus and National Comprehensive Cancer Network)MultistakeholderFive domains of patient value and technical criteria:
  • Patient preferences

  • Patient-centred outcomes

  • Patient and family costs

  • Quality and applicability of evidence

  • Usability and transparency

National Institute for Health Research (UK NIHR) INVOLVE21–23 The set of recommendations agreed by INVOLVE—the national advisory group to bring together expertise, insight and experience in the field of public involvement in research, with the aim of advancing it as an essential part of the process by which research is identified, prioritised, designed, conducted and disseminated. The six national standards for public involvement in research21 and the guidance on co-producing research project22 were based on the INVOLVE’s values and principles framework23 Multistakeholder with the focus on the joint working between researches, health care professionals and public sectorThe six national standards for public involvement are:
  • Inclusive opportunities

  • Working together (which incorporates co-production)

  • Support and learning

  • Communication

  • Impact

  • Governance

Key elements of co-producing a research project are:
  • Sharing of power

  • Reciprocity

  • Respecting and valuing the knowledge of all those working together on the research

  • Including all perspectives and skills

  • Building and maintaining relationships