Empower Your Patient Community Voice

Making a Difference

Pharmaceutical companies and academic researchers are realizing that they need to start with the patient in mind in order to create a successful therapy. Although aligning the understanding of disease biology and the science of therapeutic approaches with patient needs seems self-evident, this realization is relatively newly accepted in the research community. However, this means that opportunities for patient group involvement now exist in each stage of therapy development and continues to grow. 

  • Benefits of involvement:  When you are engaged in the entire therapy development process, you help make certain that potential therapies will address the needs of your disease community and will be more readily adopted. This can be achieved by:
    • Ensuring research on the cause and pathology of your disease is funded and that researchers have the necessary DNA/tissue samples and information needed.
    • Advising  Sponsors on their therapy development program including:
      • Identifying which symptoms patients deem are in the most need of a treatment.
      • Conveying what patients would look for in an ideal treatment based on their experiences with existing ones, for example, fewer or no side effects, less frequent administration, or different modes of administration.
      • Informing the design of clinical trials and the informed consent process
    • Helping identify or develop tools that assess the benefit of potential therapies, such as clinical outcome assessments (COAs).
    • Raising awareness and channel engagement within the patient community which, in turn, could increase registry and clinical trial participation. 
    • Supporting FDA staff in conducting patient preference studies for therapies under review by informing them what is important to your patient community.
  • Resources to learn more: Partners in therapy development and the rare disease community have developed multiple resources that can help you understand more about both the importance of the disease community voice and ways in which you can begin to harness its power.  Although mainly focused on rare and genetic disease communities, the tips and strategies in the resources may be helpful to others as well.  
    • Global Genes , a nonprofit with the mission of connecting, empowering, and inspiring the rare disease community, provides several resources focused on the power of the patient voice and data.
    • FDA Patient-Focused Drug Development Guidance Series for Enhancing the Incorporation of the Patient’s Voice in Medical Product Development and Regulatory Decision Making (2017 to 2021) is being developed by the FDA to address, in a stepwise manner, how stakeholders can collect and submit patient experience data, as well as other relevant data from patients and caregivers for therapy development and regulatory decision making.
    • FasterCures , a nonprofit, non-partisan think tank devoted to accelerating and improving the medical research system, has several resources focused on the patient voice.
      • Nonprofits: Innovators in Preclinical Drug Development (2019) is a webinar presenting innovative approaches by several patient groups to support the translation of a medical therapy from lab bench to clinical trials. 
      • FasterCures Webinars (2011 to present) lists the focus of other past webinars, several of which may also be of interest. 
      • From Anecdotal to Actionable: The Case for Patient Perspective Data (2015) is a fact sheet that highlights ideas for collecting and using patient input. It includes suggestions for types of data to collect and how to go about capturing it. This document also describes how the data can be used to inform research priorities and influence drug development.
      • FasterCures Patients Count Program is a set of initiatives aimed at improving health by promoting the integration of patient perspectives in the development of medical therapies and services. These initiatives include tips for navigating the legal landscape, a patient foundations directory to facilitate visibility among stakeholders, and a resource database to encourage patient engagement
      • On the Path to a Science of Patient Input (2016) is an article that assesses the progress and unmet needs associated with patient engagement in research and development. The paper includes information about current resources and initiatives in the area of patient engagement.
      • Innovator Spotlight (2011–2016) is an online platform containing Q&A’s with individuals from patient advocacy organizations regarding specific steps in the drug development process. These Q&As include best practices and resources for other patient advocacy groups. 
    • Discovery Documentary: First in Human (2017) is a 3-part video documentary capturing the real-life experiences of doctors, researchers, staff, patients and their caregivers, at the NIH Clinical Center. The documentary provides an in-depth look at the reality of experimental medicine in clinical trials.
    • Assessing Meaningful Engagement in Drug Development: A Definition, Framework, and Rubric is a summary of a 2015 Conference on Patient-Focused Drug Development. The document defines patient-focused drug development and includes a conceptual framework diagram. It also includes a rubric for evaluating the quality of patient engagement in drug development. The conference was organized by the University of Maryland Center of Excellence in Regulatory Science and Innovation.
    • Principles of Community Engagement: Second Edition (2011) is an online book that provides tools for those who are leading efforts to improve population health through community engagement. It includes a scientific literature review, key principles, and practical guidance for engaging partners with successful examples, management frameworks, social networking, and program evaluation. The book was developed as part of the work of the Clinical and Translational Science Awards (CTSA) Consortium’s Community Engagement Key Function Committee. CTSA is now a program within the NIH National Center for Advancing Translational Science.


Finding a Group
GARD Organization Database Genetic and Rare Diseases Information Center (GARD) (link)
Starting a Patient Group
GARD Guide: Support for Patients and Families Genetic and Rare Diseases Information Center (GARD) (link)
Steps to Nonprofit Formation National Organization for Rare Disorders (NORD) (link)
Nonprofit Resources National Organization for Rare Disorders (NORD) (link)
What is a 501(c)(3)? Foundation Group (link)