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Background: Working with patients through meaningful patient engagement (PE) and incorporating patient experience data (PXD) is increasingly important in medicines and medical device development. However, PE in the planning, organization, generation, and interpretation of PXD within regulatory and health technology assessment (HTA) decision-making processes remains challenging. We conducted a global review of the PE and PXD landscape to identify evolving resources by geography to support and highlight the potential of integration of PE and PXD in regulatory assessment and HTA. Methods: A review of literature/public information was conducted (August 2021–January 2023), led by a multistakeholder group comprising those with lived or professional experience of PE and PXD, to identify relevant regulatory and HTA initiatives and resources reviewed and categorized by geography and focus area. Results: Overall, 53 relevant initiatives/resources were identified (global, 14; North America, 11; Europe, 11; Asia, nine; UK, six; Latin America, one; Africa, one). Most focused either on PE (49%) or PXD (28%); few (11%) mentioned both PE and PXD (as largely separate activities) or demonstrated an integration of PE and PXD (11%). Conclusions: Our analysis demonstrates increasing interest in PE, PXD, and guidance on their use individually in decision-making. However, more work is needed to offer guidance on maximizing the value of patient input into decisions by combining both PE and PXD into regulatory and HTA processes; the necessity of integrating PE in the design and interpretation of PXD programs should be highlighted. A co-created framework to achieve this integration is part of a future project.

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.

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.

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.

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.

To raise awareness and promote dialogue leading to action, this article provides proceedings on ethical and legal considerations associated with medicine use during pregnancy discussed during the 2014 DIA Medicines and Pregnancy Forum. A key focus of discussion at the forum was “When is it ethically appropriate to include or unethical not to include pregnant patients in clinical studies, and how can ethical barriers be addressed?” Also debated was the question “What are the most appropriate methods to collect and share data on medication use in pregnancy, and what is the best process for sharing such information?” Goals of the forum were to gain participant alignment on answers to these ethical questions, offer rationale for the answers, and provide insight into which stakeholders might be needed to facilitate discussion and action. Participants felt that under the right circumstances, drug research in pregnant women is justified and necessary. Multiple ideas and opinions on the handling of pregnant patients in clinical research, treating pregnant women in clinical practice, and communicating data to physicians and patients are presented.

To raise awareness and promote dialogue leading to action, this article provides proceedings on ethical and legal considerations associated with medicine use during pregnancy discussed during the 2014 DIA Medicines and Pregnancy Forum. A key focus of discussion at the forum was “When is it ethically appropriate to include or unethical not to include pregnant patients in clinical studies, and how can ethical barriers be addressed?” Also debated was the question “What are the most appropriate methods to collect and share data on medication use in pregnancy, and what is the best process for sharing such information?” Goals of the forum were to gain participant alignment on answers to these ethical questions, offer rationale for the answers, and provide insight into which stakeholders might be needed to facilitate discussion and action. Participants felt that under the right circumstances, drug research in pregnant women is justified and necessary. Multiple ideas and opinions on the handling of pregnant patients in clinical research, treating pregnant women in clinical practice, and communicating data to physicians and patients are presented.

Engagement is increasingly recognized as a decisive factor for health-related outcomes in people living with a medical issue. It is their experience that drives this engagement. Therefore, providers who seek to develop better solutions, including medicines, must gain a deeper understanding of the patient experience. Beyond pathology, such understanding requires direct engagement with patients, something that has been historically avoided in the pharmaceutical industry. Whereas clear and comprehensive engagement frameworks are in place for direct engagement with health care professionals, such guidance does not yet exist for engagement with patients. A patient engagement framework has been developed at UCB to fill this gap, and it is herewith shared publicly as a contribution to setting and raising standards in patient engagement, with the ultimate aim of fostering the development of better solutions for people living with medical issues.

Engagement is increasingly recognized as a decisive factor for health-related outcomes in people living with a medical issue. It is their experience that drives this engagement. Therefore, providers who seek to develop better solutions, including medicines, must gain a deeper understanding of the patient experience. Beyond pathology, such understanding requires direct engagement with patients, something that has been historically avoided in the pharmaceutical industry. Whereas clear and comprehensive engagement frameworks are in place for direct engagement with health care professionals, such guidance does not yet exist for engagement with patients. A patient engagement framework has been developed at UCB to fill this gap, and it is herewith shared publicly as a contribution to setting and raising standards in patient engagement, with the ultimate aim of fostering the development of better solutions for people living with medical issues.

The objective of this paper is to communicate a proposed framework for addressing research limitations and communication barriers that contribute to a lack of data for making clinical treatment decisions about medication use in pregnancy. To address this global public health concern, a cross-stakeholder coalition composed of several workstreams is proposed. The intent is to foster collaborative discussion regarding potential solutions to address gaps in communication, engagement, and data generation and collection. Topic areas that require focus include development of awareness initiatives, cultural transformation efforts, collaboration initiatives, research standards, data compilation projects, and new data capture methods. Objectives to aid these efforts are outlined, and collaboration among researchers, regulators, health care providers, and patients is emphasized.

The objective of this paper is to explore the strengths, weaknesses, gaps, and needs in research on medication use in pregnancy, where opportunities have been bypassed to develop standards and collaborations for collecting data to better understand how medications can impact clinical outcomes in pregnant women and developing fetuses. The availability of existing data and the methods of its capture are reviewed, including registries, claims and health record databases, and meta-analyses. The paper focuses on why these efforts have not fundamentally provided benefit-risk information and clinical treatment algorithms for medication use in pregnant women. Methodological issues, such as lack of standardization and central data collection, are discussed. Common barriers are examined, including a lack of awareness and education, cultural hurdles, collaboration deficiency, and an insufficient development of new data collection methods.

The objective of this paper is to communicate a proposed framework for addressing research limitations and communication barriers that contribute to a lack of data for making clinical treatment decisions about medication use in pregnancy. To address this global public health concern, a cross-stakeholder coalition composed of several workstreams is proposed. The intent is to foster collaborative discussion regarding potential solutions to address gaps in communication, engagement, and data generation and collection. Topic areas that require focus include development of awareness initiatives, cultural transformation efforts, collaboration initiatives, research standards, data compilation projects, and new data capture methods. Objectives to aid these efforts are outlined, and collaboration among researchers, regulators, health care providers, and patients is emphasized.

The objective of this paper is to explore the strengths, weaknesses, gaps, and needs in research on medication use in pregnancy, where opportunities have been bypassed to develop standards and collaborations for collecting data to better understand how medications can impact clinical outcomes in pregnant women and developing fetuses. The availability of existing data and the methods of its capture are reviewed, including registries, claims and health record databases, and meta-analyses. The paper focuses on why these efforts have not fundamentally provided benefit-risk information and clinical treatment algorithms for medication use in pregnant women. Methodological issues, such as lack of standardization and central data collection, are discussed. Common barriers are examined, including a lack of awareness and education, cultural hurdles, collaboration deficiency, and an insufficient development of new data collection methods.

OBJECTIVE: To determine which patients with ankylosing spondylitis (AS) have radiographic spinal damage and to investigate the relation between radiographic spinal changes and limitations in physical function. METHODS: A cross-sectional nationwide study in Belgium of patients with AS under the care of a rheumatologist. The treating physician completed a questionnaire including clinical disease manifestations and laboratory findings (HLA-B27 and C-reactive protein), and classified spinal radiographs into 3 categories: (1) no AS-related spinal abnormalities; (2) syndesmophytes; and (3) spinal ankylosis. Patients completed the Bath AS Disease Activity Index (BASDAI) and the Bath AS Functional Index (BASFI). Ordinal regressions were performed to quantify the relationship between clinical manifestations and spinal radiographic changes. Generalized linear models were computed to quantify relationships among clinical manifestations, radiographic spinal changes, and functioning (BASFI). RESULTS: A total of 619 patients fulfilled modified New York criteria for definite AS and had evaluable radiographic data; 68% were male and disease duration was 17.5 (SD 12.2) years. Male sex, younger age at symptom onset, and hip involvement were associated with radiographic changes; but HLA-B27, peripheral arthritis, and extraarticular disease status (uveitis, psoriasis, and inflammatory bowel disease) were not. Older age, BASDAI, hip involvement, and spinal change contributed to BASFI; but sex, disease duration, peripheral arthritis, and extraarticular manifestations did not. CONCLUSION: Radiographic spinal changes in patients with AS are seen more often in men and those with hip involvement. BASFI status indicates the influence of radiographic changes and hip involvement, but does not reflect the presence of peripheral arthritis and does not differ between men and women.