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. Author manuscript; available in PMC: 2022 Apr 27.
Published in final edited form as: J Am Coll Cardiol. 2021 Apr 27;77(16):2040–2052. doi: 10.1016/j.jacc.2021.02.056

Table 1:

Summary of Workshop Outcomes

KEY PRINCIPLE COMPONENTS GOALS
1. Develop novel analytical approaches to large datasets
  • Integration of clinical & multi-omics data

  • Encourage development & validation of bioinformatic methods

  • Support multidisciplinary research teams

  • Support discovery science

  • Maximize high-value outputs of complex data initiatives in pulmonary vascular disease

  • Move quickly from molecular discovery to clinical trials in pulmonary vascular disease

  • Provide innovative analytical frameworks applicable across biomedical research

2. Incentivize dataset integration & facilitate data accessibility
  • Incorporation of existing data resources (e.g., PHBI, PH Biobank, PHAR, others)

  • International partnerships to increase power and cross-validation

  • Harmonization of present & future data creation endeavors

  • Secure, web-based exposure of data resources

  • Assurance of implementation of FAIR principles

  • Enable more advanced data analytical approaches (e.g., machine learning) in pulmonary vascular disease

  • Extend impact of pulmonary vascular disease research across all of biomedical research

3. Organize preclinical studies of new targets and new experimental disease models
  • Consortia of preclinical investigators

  • Shared core facilities

  • Common analysis methods for physiologic & molecular data types

  • Streamline consensus prioritization & validation of findings

  • Prioritize development of novel experimental models of pulmonary vascular disease

  • Cost savings via economies of scale

  • Improve data standardization & harmonization

4. Support acquisition of longitudinal data
  • Molecular measures over time

  • Clinical measures over time

  • Identification of integrated pulmonary vascular disease trajectories

  • Enhanced predictive pulmonary vascular disease modeling

  • Informed development of precision trials & endpoints

5. Lay the foundation for a master protocol for pulmonary vascular disease trials
  • Define roles for biomarkers/molecular signatures

  • Incorporation of multiple data types, including patient-centered data

  • Consideration of adaptive designs & drug withdrawal trials

  • Definition of consensus measures & common data elements

  • Harmonization of robust, clinically meaningful consensus endpoints across studies

  • Enhance efficiency of trial execution & progression by incorporating molecular with clinical data types

  • Facilitate impactful trials oriented toward the rarer types of pulmonary vascular disease