The purpose of medicines is to improve patients' lives. Stakeholders involved in the development and lifecycle management of medicines agree that more effective patient involvement is needed to ensure that patient needs and priorities are identified and met. Despite the increasing number and scope of patient involvement initiatives, there is no accepted master framework for systematic patient involvement in industry-led medicines research and development, regulatory review, or market access decisions. Patient engagement is very productive in some indications, but inconsistent and fragmentary on a broader level. This often results in inefficient drug development, increasing evidence requirements, lack of patient-centered outcomes that address unmet medical needs and facilitate adherence, and consequently, lack of required therapeutic options and high costs to society and involved parties. Improved patient involvement can drive the development of innovative medicines that deliver more relevant and impactful patient outcomes and make medicine development faster, more efficient, and more productive. It can lead to better prioritization of early research; improved resource allocation; improved trial protocol designs that better reflect patient needs; and, by addressing potential barriers to patient participation, enhanced recruitment and retention. It may also improve trial conduct and lead to more focused, economically viable clinical trials. At launch and beyond, systematic patient involvement can also improve the ongoing benefit-risk assessment, ensure that public funds prioritize medicines of value to patients, and further the development of the medicine. Progress toward a universal framework for patient involvement requires a joint, precompetitive, and international approach by all stakeholders, working in true partnership to consolidate outputs from existing initiatives, identify gaps, and develop a comprehensive framework. It is essential that all stakeholders participate to drive adoption and implementation of the framework and to ensure that patients and their needs are embedded at the heart of medicines development and lifecycle management.

IntroductionMeaningful patient engagement (PE) can enhance medicines’ development. However, the current PE landscape is fragmentary and lacking comprehensive guidance.MethodsWe systematically searched for PE initiatives (SYNaPsE database/publications). Multistakeholder groups integrated these with their own PE expertise to co-create draft PE Quality Guidance which was evaluated by public consultation. Projects exemplifying good PE practice were identified and assessed against PE Quality Criteria to create a Book of Good Practices (BOGP).ResultsSeventy-six participants from 51 organisations participated in nine multistakeholder meetings (2016–2018). A shortlist of 20relevant PE initiatives (from 170 screened) were identified. The co-created INVOLVE guidelines provided the main framework for PE Quality Guidance and was enriched with the analysis of the PE initiatives and the PE expertise of stakeholders. Seven key PE Quality Criteria were identified. Public consultation yielded 67 responses from diverse backgrounds. The PE Quality Guidance was agreed to be useful for achieving quality PE in practice, understandable, easy to use, and comprehensive. Overall, eight initiatives from the shortlist and from meeting participants were selected for inclusion in the BOGP based on demonstration of PE Quality Criteria and willingness of initiative owners to collaborate.DiscussionThe PE Quality Guidance and BOGP are practical resources which will be continually updated in response to user feedback. They are not prescriptive, but rather based on core principles, which can be applied according to the unique needs of each interaction and initiative. Implementation of the guidance will facilitate improved and systematic PE across the medicines’ development lifecycle.

Patient Focused Medicines Development (PFMD) is a not-for-profit independent multinational coalition of patients, patient stakeholders, and the pharmaceutical industry with interests across diverse disease areas and conditions. PFMD aims to facilitate an integrated approach to medicines development with all stakeholders involved early in the development process. A key strength of the coalition that differentiates it from other groups that involve patients or patient groups is that PFMD has patient organizations as founding members, ensuring that the patient perspective is the starting point when identifying priorities and developing solutions to meet patients’ needs. In addition, PFMD has from inception been formed as an equal collaboration among patient groups, patients, and pharmaceutical industry and has adopted a unique trans-Atlantic setup and scope that reflects its global intent. This parity extends to its governance model, which ensures at least equal or greater share of voice for patient group members. PFMD is actively inviting additional members and aims to expand the collaboration to include stakeholders from other sectors. The establishment of PFMD is particularly timely as patient engagement (PE) has become a priority for many health stakeholders and has led to a surge of mostly disconnected activities to deliver this. Given the current plethora of PE initiatives, an essential first step has been to determine, based on a comprehensive mapping, those strategic areas of most need requiring a focused initial effort from the perspective of all stakeholders. PFMD has identified four priority areas that will need to be addressed to facilitate implementation of PE. These are (1) culture and process change, (2) development of a global meta-framework for PE, (3) information exchange, and (4) training. This article discusses these priority themes and ongoing or planned PFMD activities within each.

PURPOSE: Participation of racial and ethnic minority groups (REMGs) in cancer trials is disproportionately low despite a high prevalence of certain cancers in REMG populations. We aimed to identify notable practices used by leading US cancer centers that facilitate REMG participation in cancer trials. METHODS: The National Minority Quality Forum and Sustainable Healthy Communities Diverse Cancer Communities Working Group developed criteria by which to identify eligible US cancer centers—REMGs comprise 10% or more of the catchment area; a 10% to 50% yearly accrual rate of REMGs in cancer trials; and the presence of formal community outreach and diversity enrollment programs. Cancer center leaders were interviewed to ascertain notable practices that facilitate REMG accrual in clinical trials. RESULTS: Eight cancer centers that met the Communities Working Group criteria were invited to participate in in-depth interviews. Notable strategies for increased REMG accrual to cancer trials were reported across five broad themes: commitment and center leadership, investigator training and mentoring, community engagement, patient engagement, and operational practices. Specific notable practices included increased engagement of health care professionals, the presence of formal processes for obtaining REMG patient/caregiver input on research projects, and engagement of community groups to drive REMG participation. Centers also reported an increase in the allocation of resources to improving health disparities and increased dedication of research staff to REMG engagement. CONCLUSION: We have identified notable practices that facilitate increased participation of REMGs in cancer trials. Wide implementation of such strategies across cancer centers is essential to ensure that all populations benefit from advances in an era of increasingly personalized treatment of cancer.

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