EUPATI Library
Effective patient–researcher partnerships are central to EUPATI’s vision. As patient involvement in health innovation advances, more research is emerging in this field. EUPATI collects and curates peer-reviewed and other publications on the importance, value, and methods of patient involvement, with support from its Sustaining Partners, National Platforms, and Fellows.
The EUPATI Library compiles recent scientific publications reflecting the different phases of the EUPATI Roadmap for Patient Involvement, including the full lifecycle of medicines R&D, but also extending to medical devices, digital health, real-world evidence and other aspects of health innovation. All materials are available for use with full references. New submissions to the library can be sent to [email protected].
Publications list
1.
Patient engagement in drug development: dialogically problematizing participation Journal Article
In: Science as Culture, vol. 34, no. 1 , pp. 31–51, 2024, (Source: Taylor & Francis Online (tandfonline.com)).
Abstract | Links | Tags: Pharmaceutical Development
@article{nokey,
title = {Patient engagement in drug development: dialogically problematizing participation},
url = {https://www.tandfonline.com/doi/full/10.1080/09505431.2024.2410719},
doi = {10.1080/09505431.2024.2410719},
year = {2024},
date = {2024-10-28},
journal = {Science as Culture},
volume = {34},
number = {1 },
pages = {31–51},
abstract = {For many years, the field of drug development remained insulated from patient engagement initiatives that became commonplace in healthcare, it is currently experiencing a participatory turn. This has been informed by a re-evaluation of the patients' insights and of the roles they can play in drug development, with both democratising and technocratic arguments invoked in support of their more substantial engagement. This article problematises the democratising potential of drug development through a knowledge co-production process between the authors of this paper - two scholars and two practitioners in this field. It scrutinises what patients can participate in drug development by zooming in on the Patient Expert Training provided by EUPATI, arguably the most influential and reputable patient education provider in Europe currently. Through their dialogue, the authors highlight the considerable epistemic requirements placed upon patients and warn about the risk of the emergence of participatory practices that rely exclusively on a narrow patient elite. They also reflect upon the benefits and challenges of close collaborations between researchers and practitioners and call for the systemic transformations needed for such knowledge co-production initiatives to become commonplace in academia.},
note = {Source: Taylor & Francis Online (tandfonline.com)},
keywords = {Pharmaceutical Development},
pubstate = {published},
tppubtype = {article}
}
For many years, the field of drug development remained insulated from patient engagement initiatives that became commonplace in healthcare, it is currently experiencing a participatory turn. This has been informed by a re-evaluation of the patients' insights and of the roles they can play in drug development, with both democratising and technocratic arguments invoked in support of their more substantial engagement. This article problematises the democratising potential of drug development through a knowledge co-production process between the authors of this paper - two scholars and two practitioners in this field. It scrutinises what patients can participate in drug development by zooming in on the Patient Expert Training provided by EUPATI, arguably the most influential and reputable patient education provider in Europe currently. Through their dialogue, the authors highlight the considerable epistemic requirements placed upon patients and warn about the risk of the emergence of participatory practices that rely exclusively on a narrow patient elite. They also reflect upon the benefits and challenges of close collaborations between researchers and practitioners and call for the systemic transformations needed for such knowledge co-production initiatives to become commonplace in academia.
2.
C. Egher, Zvonareva
Knowledge-based representation: Patient engagement in drug development Journal Article
In: 2023, (Source: Published in Health Expectations).
Abstract | Links | Tags: Pharmaceutical Development
@article{nokey,
title = {Knowledge-based representation: Patient engagement in drug development},
author = {Egher, C., Zvonareva, O.},
url = {https://onlinelibrary.wiley.com/doi/10.1111/hex.13912},
year = {2023},
date = {2023-12-22},
abstract = {Introduction: Recently, different actors have intensified their efforts to make drug development more participatory. They have produced many frameworks, tools and dedicated fora, where patients are portrayed as relevant stakeholders to be involved throughout the entire drug development trajectory. To better understand what such participatory efforts entail, in this article, we investigate how patient representation is configured in drug development and what patients can engage as representatives in this field.
Methods: This is a qualitative study based on the thematic analysis of 40 semistructured interviews with different stakeholders in the field and three patient engagement How-To guides (HTGs) complemented by observations of two sessions of the Patient Engagement Open Forum (PEOF) and a patient expert training of the European Patients' Academy on Therapeutic Innovation (EUPATI).
Findings: The emerging practices of patient engagement in drug development configure representation as hinging upon three types of knowledge—drug development knowledge, autobiographical knowledge and community knowledge—and a specific set of skills. We discern a new kind of representation based on these findings, termed ‘knowledge-based representation’, which appears to more accurately describe how patients are expected to represent others in drug development.
Conclusion: Even though knowledge-based representation may be understood as an attempt to downplay the political aspects of representation in favour of its epistemic elements, the political processes involved in patient representation in drug development cannot be ignored. The extent to which reliance on knowledge-based representation will contribute to democratic decision-making is likely to depend on the resources needed to develop the types of knowledge relevant to representation work and on how these types of knowledge are determined.
Patient or Public Contribution: Patient representatives and practitioners in the field of patient engagement (including 13 interviewees, representatives of EUPATI and HTG developers) gave feedback on the interpretation of the findings during a multistakeholder workshop we organised. We also sent an interviewee an extended draft and discussed it during an online meeting. Claudia Egher presented these findings at a PEOF session in June 2023, which further contributed to their validation.},
note = {Source: Published in Health Expectations},
keywords = {Pharmaceutical Development},
pubstate = {published},
tppubtype = {article}
}
Introduction: Recently, different actors have intensified their efforts to make drug development more participatory. They have produced many frameworks, tools and dedicated fora, where patients are portrayed as relevant stakeholders to be involved throughout the entire drug development trajectory. To better understand what such participatory efforts entail, in this article, we investigate how patient representation is configured in drug development and what patients can engage as representatives in this field.
Methods: This is a qualitative study based on the thematic analysis of 40 semistructured interviews with different stakeholders in the field and three patient engagement How-To guides (HTGs) complemented by observations of two sessions of the Patient Engagement Open Forum (PEOF) and a patient expert training of the European Patients' Academy on Therapeutic Innovation (EUPATI).
Findings: The emerging practices of patient engagement in drug development configure representation as hinging upon three types of knowledge—drug development knowledge, autobiographical knowledge and community knowledge—and a specific set of skills. We discern a new kind of representation based on these findings, termed ‘knowledge-based representation’, which appears to more accurately describe how patients are expected to represent others in drug development.
Conclusion: Even though knowledge-based representation may be understood as an attempt to downplay the political aspects of representation in favour of its epistemic elements, the political processes involved in patient representation in drug development cannot be ignored. The extent to which reliance on knowledge-based representation will contribute to democratic decision-making is likely to depend on the resources needed to develop the types of knowledge relevant to representation work and on how these types of knowledge are determined.
Patient or Public Contribution: Patient representatives and practitioners in the field of patient engagement (including 13 interviewees, representatives of EUPATI and HTG developers) gave feedback on the interpretation of the findings during a multistakeholder workshop we organised. We also sent an interviewee an extended draft and discussed it during an online meeting. Claudia Egher presented these findings at a PEOF session in June 2023, which further contributed to their validation.
Methods: This is a qualitative study based on the thematic analysis of 40 semistructured interviews with different stakeholders in the field and three patient engagement How-To guides (HTGs) complemented by observations of two sessions of the Patient Engagement Open Forum (PEOF) and a patient expert training of the European Patients' Academy on Therapeutic Innovation (EUPATI).
Findings: The emerging practices of patient engagement in drug development configure representation as hinging upon three types of knowledge—drug development knowledge, autobiographical knowledge and community knowledge—and a specific set of skills. We discern a new kind of representation based on these findings, termed ‘knowledge-based representation’, which appears to more accurately describe how patients are expected to represent others in drug development.
Conclusion: Even though knowledge-based representation may be understood as an attempt to downplay the political aspects of representation in favour of its epistemic elements, the political processes involved in patient representation in drug development cannot be ignored. The extent to which reliance on knowledge-based representation will contribute to democratic decision-making is likely to depend on the resources needed to develop the types of knowledge relevant to representation work and on how these types of knowledge are determined.
Patient or Public Contribution: Patient representatives and practitioners in the field of patient engagement (including 13 interviewees, representatives of EUPATI and HTG developers) gave feedback on the interpretation of the findings during a multistakeholder workshop we organised. We also sent an interviewee an extended draft and discussed it during an online meeting. Claudia Egher presented these findings at a PEOF session in June 2023, which further contributed to their validation.
3.
Zvonareva, Olga
Patient engagement in drug development: configuring a new resource for generating innovation Journal Article
In: 2023, (Source: Published in Critical Public Health).
Links | Tags: Pharmaceutical Development
@article{nokey,
title = {Patient engagement in drug development: configuring a new resource for generating innovation},
author = {Olga Zvonareva},
url = {https://www.tandfonline.com/doi/citedby/10.1080/09581596.2023.2188140?scroll=top&needAccess=true},
year = {2023},
date = {2023-03-17},
note = {Source: Published in Critical Public Health},
keywords = {Pharmaceutical Development},
pubstate = {published},
tppubtype = {article}
}
4.
Olga Zvonareva, Constanța Craveț & Dawn P. Richards
Practices of patient engagement in drug development: a systematic scoping review Journal Article
In: 2022, (Source: Published in BioMed Central, 2022).
Abstract | Links | Tags: Pharmaceutical Development
@article{nokey,
title = {Practices of patient engagement in drug development: a systematic scoping review},
author = {Olga Zvonareva, Constanța Craveț & Dawn P. Richards},
url = {https://researchinvolvement.biomedcentral.com/articles/10.1186/s40900-022-00364-8},
year = {2022},
date = {2022-06-29},
abstract = {Background
During the past decade, patient engagement (PE) has attracted significant attention in the field of drug development. Readiness to accept the central importance of patients’ knowledge and contributions has become evident. This study aimed to synthesize evidence on the current state of PE in drug development: what is actually being done and how.
Methods
A systematic scoping review was conducted based on a PRISMA-informed protocol. Search was performed in PubMed, EMBASE and Web of Science, covering the period between 2011 and 2021. For analysis of extracted data, we developed a framework for analyzing PE in Drug Development. The Framework distinguishes a number of different PE types that take place at different stages of drug development and are characterized by the different degrees of power patients have in the process. It allowed us to assess depth and intensity of PE initiatives included in this review.
Results
Most included PE initiatives took place at the stage of designing studies (40 in total). At this stage drug development goals are already set, but the mode of reaching them has not yet been fully determined. PE initiatives on the finetuning details stage followed (16 in total). The finetuning details stage covers the last parts of the drug development trajectory, when only relatively minor issues are still open for patients’ contributions. The least numerous were PE initiatives on the stage of setting up R&D program (13 in total). This stage refers to the early steps in drug development where PE has the potential to make the most impact on shaping the subsequent process. In terms of intensity of engagement, most PE initiatives included in this review align with consultation and involvement types, 26 and 30 initiatives, respectively. Partnership was less frequent in the published accounts of PE (13 initiatives).
Conclusions
This review delineated a contemporary landscape of PE in drug development. Although attention to PE in drug development is relatively recent, a wide range of PE practices has already been initiated. The results indicate the necessity of distinguishing between different types of PE in order to understand consequences of choices regarding depth and intensity of PE.},
note = {Source: Published in BioMed Central, 2022},
keywords = {Pharmaceutical Development},
pubstate = {published},
tppubtype = {article}
}
Background
During the past decade, patient engagement (PE) has attracted significant attention in the field of drug development. Readiness to accept the central importance of patients’ knowledge and contributions has become evident. This study aimed to synthesize evidence on the current state of PE in drug development: what is actually being done and how.
Methods
A systematic scoping review was conducted based on a PRISMA-informed protocol. Search was performed in PubMed, EMBASE and Web of Science, covering the period between 2011 and 2021. For analysis of extracted data, we developed a framework for analyzing PE in Drug Development. The Framework distinguishes a number of different PE types that take place at different stages of drug development and are characterized by the different degrees of power patients have in the process. It allowed us to assess depth and intensity of PE initiatives included in this review.
Results
Most included PE initiatives took place at the stage of designing studies (40 in total). At this stage drug development goals are already set, but the mode of reaching them has not yet been fully determined. PE initiatives on the finetuning details stage followed (16 in total). The finetuning details stage covers the last parts of the drug development trajectory, when only relatively minor issues are still open for patients’ contributions. The least numerous were PE initiatives on the stage of setting up R&D program (13 in total). This stage refers to the early steps in drug development where PE has the potential to make the most impact on shaping the subsequent process. In terms of intensity of engagement, most PE initiatives included in this review align with consultation and involvement types, 26 and 30 initiatives, respectively. Partnership was less frequent in the published accounts of PE (13 initiatives).
Conclusions
This review delineated a contemporary landscape of PE in drug development. Although attention to PE in drug development is relatively recent, a wide range of PE practices has already been initiated. The results indicate the necessity of distinguishing between different types of PE in order to understand consequences of choices regarding depth and intensity of PE.
During the past decade, patient engagement (PE) has attracted significant attention in the field of drug development. Readiness to accept the central importance of patients’ knowledge and contributions has become evident. This study aimed to synthesize evidence on the current state of PE in drug development: what is actually being done and how.
Methods
A systematic scoping review was conducted based on a PRISMA-informed protocol. Search was performed in PubMed, EMBASE and Web of Science, covering the period between 2011 and 2021. For analysis of extracted data, we developed a framework for analyzing PE in Drug Development. The Framework distinguishes a number of different PE types that take place at different stages of drug development and are characterized by the different degrees of power patients have in the process. It allowed us to assess depth and intensity of PE initiatives included in this review.
Results
Most included PE initiatives took place at the stage of designing studies (40 in total). At this stage drug development goals are already set, but the mode of reaching them has not yet been fully determined. PE initiatives on the finetuning details stage followed (16 in total). The finetuning details stage covers the last parts of the drug development trajectory, when only relatively minor issues are still open for patients’ contributions. The least numerous were PE initiatives on the stage of setting up R&D program (13 in total). This stage refers to the early steps in drug development where PE has the potential to make the most impact on shaping the subsequent process. In terms of intensity of engagement, most PE initiatives included in this review align with consultation and involvement types, 26 and 30 initiatives, respectively. Partnership was less frequent in the published accounts of PE (13 initiatives).
Conclusions
This review delineated a contemporary landscape of PE in drug development. Although attention to PE in drug development is relatively recent, a wide range of PE practices has already been initiated. The results indicate the necessity of distinguishing between different types of PE in order to understand consequences of choices regarding depth and intensity of PE.
5.
Starling B Parsons S, Mullan-Jensen C
In: 2015, (Source: Published in BMJ Open).
Abstract | Links | Tags: Pharmaceutical Development
@article{nokey,
title = {What do pharmaceutical industry professionals in Europe believe about involving patients and the public in research and development of medicines? A qualitative interview study},
author = {Parsons S, Starling B, Mullan-Jensen C, et al.},
url = {https://bmjopen.bmj.com/content/6/1/e008928},
year = {2015},
date = {2015-10-16},
abstract = {Objectives: To explore European-based pharmaceutical industry professionals’ beliefs about patient and public involvement (PPI) in medicines research and development (R&D).
Setting: Pharmaceutical companies in the UK, Poland and Spain.
Participants: 21 pharmaceutical industry professionals, four based in the UK, five with pan-European roles, four based in Spain and eight based in Poland.
Method: Qualitative interview study (telephone and face-to-face, semistructured interviews). All interviews were audio taped, translated (where appropriate) and transcribed for analysis using the Framework approach.
Results: 21 pharmaceutical industry professionals participated. Key themes were: beliefs about (1) whether patients and the public should be involved in medicines R&D; (2) the barriers and facilitators to PPI in medicines R&D and (3) how the current relationships between the pharmaceutical industry, patient organisations and patients influence PPI in medicines R&D.
Conclusions: Although interviewees appeared positive about PPI, many were uncertain about when, how and which patients to involve. Patients and the public's lack of knowledge and interest in medicines R&D, and the pharmaceutical industry's lack of knowledge, interest and receptivity to PPI were believed to be key challenges to increasing PPI. Interviewees also believed that relationships between the pharmaceutical industry, patient organisations, patients and the public needed to change to facilitate PPI in medicines R&D. Existing pharmaceutical industry codes of practice and negative media reporting of the pharmaceutical industry were also seen as negative influences on these relationships.},
note = {Source: Published in BMJ Open},
keywords = {Pharmaceutical Development},
pubstate = {published},
tppubtype = {article}
}
Objectives: To explore European-based pharmaceutical industry professionals’ beliefs about patient and public involvement (PPI) in medicines research and development (R&D).
Setting: Pharmaceutical companies in the UK, Poland and Spain.
Participants: 21 pharmaceutical industry professionals, four based in the UK, five with pan-European roles, four based in Spain and eight based in Poland.
Method: Qualitative interview study (telephone and face-to-face, semistructured interviews). All interviews were audio taped, translated (where appropriate) and transcribed for analysis using the Framework approach.
Results: 21 pharmaceutical industry professionals participated. Key themes were: beliefs about (1) whether patients and the public should be involved in medicines R&D; (2) the barriers and facilitators to PPI in medicines R&D and (3) how the current relationships between the pharmaceutical industry, patient organisations and patients influence PPI in medicines R&D.
Conclusions: Although interviewees appeared positive about PPI, many were uncertain about when, how and which patients to involve. Patients and the public's lack of knowledge and interest in medicines R&D, and the pharmaceutical industry's lack of knowledge, interest and receptivity to PPI were believed to be key challenges to increasing PPI. Interviewees also believed that relationships between the pharmaceutical industry, patient organisations, patients and the public needed to change to facilitate PPI in medicines R&D. Existing pharmaceutical industry codes of practice and negative media reporting of the pharmaceutical industry were also seen as negative influences on these relationships.
Setting: Pharmaceutical companies in the UK, Poland and Spain.
Participants: 21 pharmaceutical industry professionals, four based in the UK, five with pan-European roles, four based in Spain and eight based in Poland.
Method: Qualitative interview study (telephone and face-to-face, semistructured interviews). All interviews were audio taped, translated (where appropriate) and transcribed for analysis using the Framework approach.
Results: 21 pharmaceutical industry professionals participated. Key themes were: beliefs about (1) whether patients and the public should be involved in medicines R&D; (2) the barriers and facilitators to PPI in medicines R&D and (3) how the current relationships between the pharmaceutical industry, patient organisations and patients influence PPI in medicines R&D.
Conclusions: Although interviewees appeared positive about PPI, many were uncertain about when, how and which patients to involve. Patients and the public's lack of knowledge and interest in medicines R&D, and the pharmaceutical industry's lack of knowledge, interest and receptivity to PPI were believed to be key challenges to increasing PPI. Interviewees also believed that relationships between the pharmaceutical industry, patient organisations, patients and the public needed to change to facilitate PPI in medicines R&D. Existing pharmaceutical industry codes of practice and negative media reporting of the pharmaceutical industry were also seen as negative influences on these relationships.
6.
Eleanor M. Perfetto, Elisabeth M. Oehrlein
Assessing Meaningful Patient Engagement in Drug Development: A Definition, Framework, and Rubric Technical Report
2015, (Source: CERSI: University of Maryland).
Abstract | Links | Tags: Pharmaceutical Development
@techreport{nokey,
title = {Assessing Meaningful Patient Engagement in Drug Development: A Definition, Framework, and Rubric},
author = {Eleanor M. Perfetto, Elisabeth M. Oehrlein},
url = {https://www.pharmacy.umaryland.edu/media/SOP/wwwpharmacyumarylandedu/centers/cersievents/pfdd/mcersi-pfdd-framework-rubric.pdf},
year = {2015},
date = {2015-04-01},
abstract = {A movement to include the patient voice in health care research and decision making is underway. In light of broad stakeholder interest in patient-focused drug development (PFDD), a range of stakeholders are considering approaches to increase the scope of PFDD and enhancing patient engagement. On March 9, 2015, the University of Maryland Center of Excellence in Regulatory Science and Innovation (M-CERSI), with the support of many partner organizations, held the “M-CERSI Conference on PatientFocused Drug Development.” The objective was to allow stakeholders from patient groups, the US Food and Drug Administration (FDA), the biopharmaceutical industry, payer, and other organizations to voice their views on, activities in, and aspirations for PFDD. During the day-long program, participants discussed the challenges to successful PFDD including regulatory challenges, the patient and patient advocate role, the emerging payer role, along with future directions and opportunities for collaboration. This document summarizes the outputs of the conference including a suggested definition, rubric, and framework for PFDD.},
note = {Source: CERSI: University of Maryland},
keywords = {Pharmaceutical Development},
pubstate = {published},
tppubtype = {techreport}
}
A movement to include the patient voice in health care research and decision making is underway. In light of broad stakeholder interest in patient-focused drug development (PFDD), a range of stakeholders are considering approaches to increase the scope of PFDD and enhancing patient engagement. On March 9, 2015, the University of Maryland Center of Excellence in Regulatory Science and Innovation (M-CERSI), with the support of many partner organizations, held the “M-CERSI Conference on PatientFocused Drug Development.” The objective was to allow stakeholders from patient groups, the US Food and Drug Administration (FDA), the biopharmaceutical industry, payer, and other organizations to voice their views on, activities in, and aspirations for PFDD. During the day-long program, participants discussed the challenges to successful PFDD including regulatory challenges, the patient and patient advocate role, the emerging payer role, along with future directions and opportunities for collaboration. This document summarizes the outputs of the conference including a suggested definition, rubric, and framework for PFDD.
7.
Eleanor M. Perfetto, Burke
Patient-Focused Drug Development: A New Direction for Collaboration Journal Article
In: 2015, (Source: Published in Medical Care: Official Journal of the Medical Care Section, American Public Health Association).
Abstract | Links | Tags: Pharmaceutical Development
@article{nokey,
title = {Patient-Focused Drug Development: A New Direction for Collaboration},
author = {Perfetto, Eleanor M., Burke, Laurie, Oehrlein, Elisabeth M., Epstein, Robert S.},
url = {https://journals.lww.com/lww-medicalcare/abstract/2015/01000/patient_focused_drug_development__a_new_direction.3.aspx},
year = {2015},
date = {2015-01-01},
abstract = {Context: Patient-Focused Drug Development (PFDD) is a new initiative from the Food and Drug Administration (FDA) intended to bring patient perspectives into an earlier stage of product development. The goal is that patients will be able to provide context for benefit-risk assessments and input to review divisions, and also aid in the development of new assessment tools, study endpoints, and risk communications. This paper provides a summary on what is known to date about FDA’s PFDD initiative and describes implications for patients, researchers, payers, and the biopharmaceutical industry. It also provides a roadmap for stakeholders to consider in defining their role in and in shaping PFDD’s direction, and for expanding PFDD principles to conditions beyond the current 20 under FDA consideration.
Methods: A search was conducted of the peer-reviewed and gray literature using PubMed and Google. This included laws, FDA guidance documents, the peer-reviewed literature, and FDA presentations for content relevant to the search term “patient-focused drug development.”
Findings: Currently, FDA activities within PFDD are limited to gaining patient insights through 20 disease-specific meetings. However, many stakeholders see the initiative much more generally as representing a broad shift toward patient centeredness in biopharmaceutical product development.
Conclusions: Depending upon the trajectory taken and whether or not all PFDD aims are eventually addressed, the initiative has the potential to change product development in fundamental ways. Further research should explore how patient input on disease manifestation and treatment options is best ascertained from patients and documented before initiating and during drug development.},
note = {Source: Published in Medical Care: Official Journal of the Medical Care Section, American Public Health Association},
keywords = {Pharmaceutical Development},
pubstate = {published},
tppubtype = {article}
}
Context: Patient-Focused Drug Development (PFDD) is a new initiative from the Food and Drug Administration (FDA) intended to bring patient perspectives into an earlier stage of product development. The goal is that patients will be able to provide context for benefit-risk assessments and input to review divisions, and also aid in the development of new assessment tools, study endpoints, and risk communications. This paper provides a summary on what is known to date about FDA’s PFDD initiative and describes implications for patients, researchers, payers, and the biopharmaceutical industry. It also provides a roadmap for stakeholders to consider in defining their role in and in shaping PFDD’s direction, and for expanding PFDD principles to conditions beyond the current 20 under FDA consideration.
Methods: A search was conducted of the peer-reviewed and gray literature using PubMed and Google. This included laws, FDA guidance documents, the peer-reviewed literature, and FDA presentations for content relevant to the search term “patient-focused drug development.”
Findings: Currently, FDA activities within PFDD are limited to gaining patient insights through 20 disease-specific meetings. However, many stakeholders see the initiative much more generally as representing a broad shift toward patient centeredness in biopharmaceutical product development.
Conclusions: Depending upon the trajectory taken and whether or not all PFDD aims are eventually addressed, the initiative has the potential to change product development in fundamental ways. Further research should explore how patient input on disease manifestation and treatment options is best ascertained from patients and documented before initiating and during drug development.
Methods: A search was conducted of the peer-reviewed and gray literature using PubMed and Google. This included laws, FDA guidance documents, the peer-reviewed literature, and FDA presentations for content relevant to the search term “patient-focused drug development.”
Findings: Currently, FDA activities within PFDD are limited to gaining patient insights through 20 disease-specific meetings. However, many stakeholders see the initiative much more generally as representing a broad shift toward patient centeredness in biopharmaceutical product development.
Conclusions: Depending upon the trajectory taken and whether or not all PFDD aims are eventually addressed, the initiative has the potential to change product development in fundamental ways. Further research should explore how patient input on disease manifestation and treatment options is best ascertained from patients and documented before initiating and during drug development.