Science of Patient Input Resources

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In early 2016, FasterCures completed an environmental scan of collaborative initiatives that have generated resources for advancing the science of patient input. We documented our findings in an article titled, On the Path to a Science of Patient Input,” published on April 27, 2016 in Science Translational Medicine. The table below displays resources that may be helpful to individuals launching patient-centered efforts as well as to seasoned practitioners who wish to identify where more work is needed. We will update this table when new initiatives are announced and as new resources become available. Please bring additions or corrections to our attention by sending an email to

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Methods & Toolkits
Sources of Patient Data
Regulatory & Legislative Activities
Training Programs
Measurement & Metrics
ALS Association
      Draft regulatory guidance on development of drug therapies for ALS to submit to FDA    
American Institutes for Research
  A Roadmap for Patient + Family Engagement in Healthcare       Project to identify measurement principles that are meaningful to patients and their families
Amyloidosis Foundation
      Draft regulatory guidance on development of drug therapies for amyloidosis to submit to FDA    
Association of Medical Charities
  Paths to Patient and Public Involvement for Medical Research Charities        
Biotechnology Innovation Organization (BIO)
Lifecycle Approach to FDA’s Structured Benefit-Risk Assessment

Key Considerations for Developing and Integrating Patient Perspectives in Drug Development (with PPMD)

Guiding Principles for Interaction with Patient Advocacy Organizations
BIO Annual Convention Alliance Pavilion   BIO Patient & Health Advocacy Summit  
Bristol-Myers Squibb
  The Universal Patient Language        
Cancer Support Community
  Working with Regulators: A Focus on the FDA

Working with Regulators: A Focus on CMS
Clinical Trials Transformation Initiative (CTTI)

Patient Engagement Across the Research & Development Continuum

Effective and Efficient Clinical Trial Recruitment Planning

Patient Group Organizational Expertise & Assets Evaluation Tool       Project to measure the return on investment of patient engagement in clinical trials
Community Catalyst
  Meaningful Consumer Engagement: A Toolkit for Plans, Provider Groups and Communities        
      21st Century Cures Act (H.R. 6) Medical Innovation legislation and the Patient-Focused Impact Assessment of 2015 (S. 1597)    
Visual Model of Patient Engagement in Benefit-Risk Assessment through the Medical Product Life Cycle Global Forum, 2016 June Issue featuring special section on patient engagement     Patient Advocate Fellowship Program Patient Engagement in Benefit-Risk Assessment throughout the Life Cycle of Medical Products Case studies to quantify the adoption and impact of patient centric initiatives (with Tufts)
European League Against Rheumatism (EULAR)
  Patient Involvement in Research - A Way to Success        
European Medicines Association & FDA
      Terms of Reference for the EMA-FDA Cluster on Patient Engagement    
European Patients’ Academy on Therapeutic Innovation

Case Reports on meaningful patient involvement in industry-led medicines R&D

Patient involvement in Industry R&D
    The A to Z of howmedicines are developed Expert Training Course for Patients and Patient Representatives on the Medicines Research & Development Process  
Everylife Foundation
Patients As Critical Partners in Rare Disease Drug Development          
From Anecdote to Actionable: The Case for Patient Perspective Data Patient-centered framework for assessing the value of care TRAIN Central Station Building Smarter Patient Registries   Benefit Risk Boot Camp  
Food and Drug Administration (FDA)
        Patient Representative Program  
FDA Center for Drug Evaluation and Research

“Voice of the Patient” reports

Diabetes Outcome Measures: Beyond HbA1C
Patient Focused Drug Development initiative

Pilot Clinical Outcome Assessment Compendium

Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events

Drug Trial Snapshots

Provisions in Sixth authorization of the Prescription Drug User Fee Act (PDUFA-VI)

Draft guidance on conducting natural history studies
FDA Center for Devices and Radiological Health

Patient Preference Initiative

Draft Guidance for Submitting Patient Preference Information

2016-17 Strategic Priority: Partner with Patients

Patient Engagement Advisory Committee

Draft Guidance: Dissemination of Patient-Specific Information from Devices by Device Manufacturers
Food and Drug Law Institute
        Introduction to Drug Law & Regulation  
Friends of Cancer Research
        Training program to facilitate patient involvement in clinical trial design and risk-benefit decisions  
Genetic Alliance
Advancing Meaningful Patient Engagement in Research, Development, and Review of Drugs (with NHC)   PEER Platform Draft guidance on Integrating Patient Perspective in the Drug Development Process (with NHC)    
Global Genes
  RARE Toolkits        
  Resources for payment and recognition for public involvement in health research        
Innovative Medicines Initiative
   A Short Guide to Successful Patient Involvement in EU-Funded Research        
International Society for Pharmacoeconomics and Outcomes Research (ISPOR)
  Patient Centered Special Interest Group: Patient Engagement in Research     Patient Representatives Roundtables  
Medical Device Innovation Consortium (MDIC)
Framework for Incorporating Information on Patient Preferences Regarding Benefit and Risk into Regulatory Assessments of Medical Technology Catalog of Methods for Assessing Patient Preferences for Benefits and Harms of Medical Technologies        
Multi-Regional Clinical Trials (MRCT) of Harvard and Brigham & Women’s Hospital
  Report and Guidance on Return of Individual Results        
National Breast Cancer Coalition
        Project LEAD  
National Health Council (NHC)
Advancing Meaningful Patient Engagement in Research, Development, and Review of Drugs (with Genetic Alliance) Patient Information Tool & Implementation Guide   Draft guidance on Integrating Patient Perspective in the Drug Development Process (with Genetic Alliance)    
National Institutes of Health (NIH)
    Million-person Precision Medicine Initiative cohort      
NIH-National Center for Advancing Translational Sciences (NCATS)
Clinical and Translational Sciences Awards Consortium Principles of Community Engagement Office of Rare Diseases Research Toolkit for patient groups wishing to engage in translational research Patient advocacy portal for existing tools and resources Global Rare Disease Registries      
National Organization of Rare Diseases (NORD)
    Natural Histories Patient Registry Platform      
National Pharmaceutical Council
Guiding Practices for Patient-Centered Value Assessments          
National Quality Forum
  International Patient Decision Aid Standards        
Patient Centered Outcomes Research Institute (PCORI)
Patient Engagement Rubric Compensation Framework for Engaged Research Partners Methodology Standards PCORnet: The National Patient-Centered Clinical Research Network   PCORI Ambassadors Program Patient Engagement Rubric & Evaluation Framework
    PatientCrossroads Patient Registry Platform      
Patient-Focused Medicines Development
Global Framework for Patient Engagement Mapping patient engagement: online collection tool        
  Complete publications list  PatientsLikeMe health data-sharing platform      
Parent Project Muscular Dystrophy (PPMD)
  Assessing and Integrating Patient Views into Drug Development: Patient Preference Study Considerations (with BIO)   Duchenne Muscular Dystrophy and Related Dystrophinopathies: Developing Drugs for Treatment Guidance for Industry    
Parkinson’s Disease Foundation
        Parkinson's Advocates in Research  
Reagan Udall Foundation
        Big Data 4 Patients  
  Patient Awareness & Access initiative       Method to define and measure patient burden in clinical trial participation
Tufts Center for the Study of Drug Development
          Case studies to quantify the Adoption and impact of patient centric initiatives (with DIA)
    Unitio Community Application for Research and Engagement (CARE) platform      
University of Maryland's Center of Excellence in Regulatory Science and Innovation (M-CERSI)
Assessing Meaningful Patient Engagement in Drug Development: A Definition, Framework, and Rubric         Assessing Meaningful Patient Engagement in Drug Development: A Definition, Framework, and Rubric


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Last updated: August 2016