兰德-National-Patient_164页_7mb

PCORnet Phase I Final Evaluation Summary

Background

The National Patient-Centered Clinical Research Network (PCORnet) was established by the Patient-Centered Outcomes Research Institute (PCORI) to address limitations in the current research paradigm by accelerating, reducing the cost, and improving the patient-centeredness of clinical research. PCORI invested over $100 million to create PCORnet, a distributed network combining clinical data from electronic health records (EHRs) and patient-generated data from participating networks across the United States. The goal is to enable large-scale comparative effectiveness research (CER) and improve health outcomes by involving patients, clinicians, and health systems in the research process.

Evaluation Objectives

The external evaluation of PCORnet's Phase I (March 2014 – August 2015) aimed to:

• Identify key accomplishments and challenges in implementing PCORnet
• Gather stakeholder perspectives on implementation progress
• Assess the extent to which PCORnet was on track to achieve research readiness

Governance Infrastructure

PCORnet's governance structure was developed by the Steering Committee, Executive Committee, and an advisory Patient Council. The structure included 11 task forces to address policy development and implementation strategies across various topics such as data privacy and biobanking. Despite initial challenges with transparency and perceived decision-making authority, the governance model demonstrated adaptability and course correction during Phase I.

• A revised governance model was approved on the last day of Phase I.
• The new model aims to enhance decision-making efficiency and promote self-governance.
• Concerns remain about the need for strong executive leadership to ensure alignment with PCORnet-level goals.

Data Infrastructure

PCORnet's data infrastructure involved standardizing data using the common data model (CDM) and establishing secure DataMarts for cross-network querying.

• CDRNs: 89% of CDRN DataMarts were set up by the end of Phase I, with data standardized for 72.6 million patients (over 3 billion encounters).
• PPRNs: 68% of PPRN DataMarts were established, but they standardized fewer domains due to challenges in obtaining EHR data and lower requirements.
• Common Data Model: Limited data elements pose a challenge for research scope and may require expansion in Phase II.
• Data Quality: Only two DataMarts were tested for quality by the end of Phase I. Data quality assessments will remain a priority in Phase II due to the need for one-on-one engagement and process refinement.
• Data Completeness: CDRNs aimed to achieve complete data on their million-member populations, but challenges with data use agreements, costs, and payer collaboration limited progress. A framework for improved collaboration with payers was developed during Phase I.
• Data Privacy: Networks developed privacy protections, but some delayed further policy development due to lack of guidance. Continued work on PCORnet-level privacy standards is essential.

Patient-Generated Data

All networks were required to collect core patient-reported outcomes (PROs) and could supplement with additional data.

• PPRNs reported active engagement in collecting substantial patient-generated data, using multiple instruments and modalities.
• CDRNs had limited quantitative data on PRO collection, but many had begun integrating PROs into their DataMarts.
• Future updates to the common data model may include entire PRO measurement scales to improve utility.

Biobanking Infrastructure

Biobanking was not a primary focus during Phase I, but many networks began planning for its development.

• CDRNs and PPRNs that already had biobanking capabilities continued specimen collection.
• Developing an inventory of biospecimen data and standard operating procedures (SOPs) should be a priority in Phase II to expand research capabilities.

Research Infrastructure

PCORnet focused on streamlining research processes and enhancing patient engagement.

• Multi-Site IRB Review: All CDRNs made progress in implementing multi-site IRB models. Some had delays in obtaining buy-in, but many were already pioneers in this area.
• Patient-Centered Consent: CDRNs and PPRNs explored innovative consent methods, including electronic tools and multimedia-based apps.
• Patient Enrollment: CDRNs enrolled patients into obesity and rare-disease cohorts, while PPRNs met specific enrollment targets. This helped prepare the network for future research activities.

Collaboration and Stakeholder Engagement

Collaboration and stakeholder engagement were central to PCORnet's development.

• Collaborations: CDRNs and PPRNs reported numerous collaborations, with 89% of CDRNs and 68% of PPRNs establishing DataMarts.
• Stakeholder Engagement: PCORnet aimed to involve patients, clinicians, health systems, and federal and industry stakeholders. The evaluation highlighted the importance of continued engagement and communication to ensure the network's sustainability and effectiveness.

Conclusion

PCORnet's Phase I laid a strong foundation for a national patient-centered research network, but several challenges remain. The governance structure, data infrastructure, and research processes are now more robust, and the network is better prepared for Phase II. However, ongoing efforts are needed to address issues related to data completeness, privacy standards, and stakeholder engagement. The success of PCORnet will depend on its ability to continue evolving and addressing these areas in the next phase of development.