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Patient Perspectives - Patient Reported Outcomes Drive Drug Development, but Improving Patient Involvement Remains a Challenge
GBI Research, May 2012, Pages: 46
GBI Research, the leading business intelligence provider, has released its latest research, “Patient Perspectives - Patient Reported Outcomes Drive Drug Development, but Improving Patient Involvement Remains a Challenge”. The report examines the role of patients in changing the research agenda and helping pharma companies to bring effective medicines that meet real unmet medical needs into the market place. It explores the use of patient reported outcomes in drug development, with a particular focus on their use in the regulatory setting. The report investigates whether the measures used in clinical development are relevant to patients and describes current initiatives to improve patient involvement. The final chapter explores the way in which patient involvement is changing as a result of online interactions.
The report is built using information from primary and secondary research including interview with experts in the field.
GBI Research analysis shows that regulators and reimbursement authorities are driving efforts to involve patients in drug development through initiating projects to find out what matters most to patients and including this in their own benefit-risk or value assessments. Pharmaceutical companies will need to take this changing environment into consideration more carefully when designing strategies across the board, as well as in defining the particulars of individual development programs and clinical trials. Company strategies should depend on identifying key areas of unmet need as patients in these areas may be willing to take more risks and reimbursement authorities will be more willing to finance new, and potentially expensive, treatments for drugs in these areas.
Scope
- History of patient involvement in drug development, regulation and reimbursement.
- Definitions and regulatory status of Patient-Reported Outcome measures and electronic Patient-Reported Outcome measures.
- Detailed analysis of Patient-Reported Outcome instrument development and collaborations underway to accelerate their development in key therapeutic areas.
- Analysis of patient relevance in the assessment of a new drug’s value by health technology assessors and reimbursement authorities.
- Growth of online communities of patients with particular diseases and their relevance in drug development.
Reasons to buy
- Identify the best ways to work with patients to improve drug development.
- Identify key projects that are developing methods for involving patients more closely in decision making and agenda setting activities.
- Develop strategies for Patient-Reported Outcome instrument development.
- Understand the current thinking amongst regulators and reimbursement authorities with regards to Patient-Reported Outcome measures.
- Explore ways in which to interact with patients online or to use information generated by online communities to support new research strategies.
1.1 List of Tables
1.2 List of Figures
2 Introduction
3 Patient Perspectives – Regulatory Focus
3.1 The US FDA
3.2 European Medicines Agency
3.3 Patient Perspectives – Reimbursement Focus
3.4 Patients and the Pharmaceutical Industry
3.4.1 The EU Patient Partnership Project
3.4.2 The Drug Information Association (DIA) Patient Fellowship Program
3.4.3 The James Lind Alliance
3.4.4 Engaging the Pharmaceutical Industry
3.5 Overcoming Barriers to Patient Involvement
4 Patient Reported Outcomes
4.1 Introduction
4.2 Regulatory Guidance
4.2.1 FDA Guidance
4.2.2 EMA Reflection Paper
4.2.3 Influence of EMA and FDA Guidance Documents
4.3 Using PROs in drug development
4.3.1 PRO Instrument Development and the FDA
4.3.2 Contract Research Organisations with PRO Expertise
4.3.3 PRO Instruments: Sharing and Cooperation
4.3.4 Electronic PRO instruments
4.4 Conclusions
5 Ensuring Patient Relevance in Value Assessments
5.1 Introduction
5.2 Patient Preference Data in Quantifying Benefit-Risk Decisions
5.3 Patient-preference Data in Reimbursement Decision-making
5.3.1 Quality Adjusted Life Years and HRQL Data
5.3.2 Patient involvement with NICE
5.3.3 Patient Preference Methods
5.3.4 Conjoint analysis
5.3.5 Qualitative Studies of Patient Preference
5.4 Patient Perspectives and Comparative Effectiveness Research in the US
5.4.1 The US Patient Centered Outcomes Research Institute (PCORI)
5.5 Conclusions
6 Patients Online
6.1 Introduction
6.2 Tracking, Monitoring and Sharing Data
6.2.1 Quantified Self
6.2.2 wikiLife
6.3 E-communities
6.3.1 PatientsLikeMe.com
6.3.2 CureTogether.com
6.4 Patients, Social Media and Personalized Medicine
6.5 Conclusions
6.6 Future Outlook
7 Appendix
7.1 Key Definitions
7.2 Abbreviations (in alphabetical order)
7.3 References (in alphabetical order)
7.4 Methodology
7.4.1 Primary Research
7.4.2 Secondary Research
7.5 Contacts Us
7.6 Disclaimer
1.1 List of Tables
Table 1: Patient Perspectives, Role of Patient-reported Outcomes and Health-related Quality of Life Data in Product Labelling in the US and EU, 2006-2010
Table 2: Patient Perspectives, Examples of Contract Research Organizations with Expertise in Patient-Reported Outcomes
Table 3: Patient Perspectives, Potential Benefits of ePRO Solutions
1.2 List of Figures
Figure 1: Patient Perspectives, Patients’ and Consumers’ Organizations Working with the EMA
Figure 2: Patient Perspectives, Potential Barriers Towards Patient Involvement: Results of the Patient Partner Project Survey
Figure 3: Patient Perspectives, Schizophrenia Research Priorities Identified through the James Lind Alliance
Figure 4: Patient Perspectives, Examples of Disease Areas and Relevant Primary Endpoints
Figure 5: Patient Perspectives, Process for Including Patient Reported Outcome Measures in Drug Development
Figure 6: Patient Perspectives, Pharmaceutical Companies Working Together for Patient-Reported Outcome Instrument Development in the Critical Path Institute’s PRO Consortium
Figure 7: Patient Perspectives, The Process Used By The Critical Path Institute’s PRO Consortium for PRO Instrument Development
Figure 8: Patient Perspectives, History of Regulatory Actions on Quantitative Benefit-Risk Analysis
Figure 9: Patient Perspectives, Schematic of the BRAT Framework,2011
Figure 10: Patient Perspectives, Steps in the BRAT Framework, 2011
Figure 11: Patient Perspectives, Quality Adjusted Life Years in Practice, 2009
Figure 12: Patient Perspectives, Difference in Focus for HRQL and Patient Pereference Methods for Determining Value, 2007
Figure 13: Patient Perspectives, PatientsLikeMe.com
Patient Perspectives Influencing Drug Development.
Support is growing for increased collaboration between pharmaceutical companies and patients in research processes worldwide, according to a new report by healthcare intelligence company GBI research.
The new report* states that patient perspectives are increasingly being sought by drug regulators and reimbursement authorities, who hope to better meet the needs of their customer base.
Patient Reported Outcomes (PROs) are increasingly being seen as valuable assets to drug regulators, who recognize that patients should be brought in early in the regulatory process to help define an acceptable benefit-risk balance for particular disease areas. Both the FDA and European Medicines Agency are working to establish processes to support this, and reimbursement authorities are looking to patients to help them define truly valuable new medicines. Pharmaceutical companies hope to measure PROs, but this requires a great deal of upfront planning, particularly in cases where a new appropriate measurement instrument must be developed and validated as part of the drug development program.
Successful patient involvement in setting research agendas can be seen in the James Lind Alliance, which was established in 2004 as a non-profit-making initiative. The alliance brings together patients, carers and clinicians who share an interest in a particular health problem, in order to identify ‘unanswered questions’ about the effects of treatments.
The EU Patient Partnership Project (PatientPartner) also promotes the role of patient organizations in the clinical trials context, creating a virtual network called the European Network of Patients Partnering in Clinical Research (ENCPR) to empower European patient organizations to interact with the other stakeholders in clinical trials. Their guide for sponsors and researchers demonstrates how to build effective partnerships, and highlights the fact that patients and their representatives can offer unique perspectives based on their own or collective experiences from dealing with the effects of their condition on a daily basis.
PatientPartner also identified barriers within patient organizations and pharmaceutical companies that need to be overcome for successful partnerships to be built. Patient organizations can suffer from a lack of expertise and awareness of clinical research, and a lack of organizational structure. In contrast, pharmaceutical companies can suffer from concerns over potential negative publicity, possible delays of clinical trial processes, and loss of control when proprietary information is shared.
Nevertheless, most large pharmaceutical companies have integrated PRO strategy within their drug development planning activities, with guidance from the FDA and EMA providing support. GBI analysis suggests that companies should aim to consider what will be of most relevance to patients in the development of their overall strategy, as well as within each drug development, so that clinical trials can be designed with these points in mind. In this way, companies will be able to better meet the needs of patients, regulators and reimbursement authorities.
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