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.  


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)