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.
    • From Molecules to Medicine: How Patients Can Share Their Voices Throughout the Drug Development Process (2018) provides information and case studies regarding areas where patients can be engaged to advance disease treatments. It includes information about forming communities, sharing data, and participating in clinical trials.
    • Data DIY – Your Involvement in Driving Understanding, Discovery, and Treatments for Rare Disease (2019–2020) is a 4-part webinar series focused on educating patient group leaders to become empowered data owners and stewards.
  • 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

If you are not currently involved in a patient group and wish to be involved in the therapy development process, you first want to find any patient groups already involved in advancing research for your disease, whether they are disease-specific or larger umbrella organizations. Competition for patients is of particular concern for rare diseases, because the patient population may be very small. Having one united patient group fosters collaborations and prevents fracturing within your rare disease community, allows for the pooling of resources, reduces duplication of efforts, and avoids sources of competition.

• Genetic and Rare Disease (GARD) Organization Database can help you find other patient groups for specific diseases. The database can be filtered by associated disease or country where the group is located.
• If you locate a group, their mission statement can help you determine whether the group is supporting or driving therapy development for your disease. 
  • If yes, contact the group either by email or phone to find ways you can become involved.
  • If not, they may be interested in expanding their mission to include therapy development, especially with someone willing to lead the way.
  • If the group is not interested in supporting therapy research, you may consider forming a group that solely focuses on supporting therapy development while still working with the established group to avoid duplicating efforts or splitting the patient population. 
• If the group is an umbrella organization, you may be able to form a subgroup and work within their established system. Alternatively, you may need to form a separate group, but again continue to work with the larger umbrella group whenever possible.
• If a patient group does not exist for your disease, you may wish to consider establishing one.

Starting a Patient Group

Although there are ways for patients to engage in different aspects of the therapy development process without belonging to a patient group, that is not the focus of NCATS Toolkit. In general the voice of a patient group offers clinicians, researchers, and industry a more diverse perspective and has a greater impact. If there is a patient group that already exists, working within the existing group can keep your disease community united, avoid duplication of efforts, and reduce competition for resources. If you are certain there is not an existing group that includes your disease, you may consider starting one.

• Social media groups: Today, many nonprofit patient groups for rare and genetic diseases begin as social media groups. Social media groups can quickly connect people/families who are affected by the same rare disease. 
  • If there is already a social media group, reach out to the administrators and moderators (on Facebook these can usually be found on the About page for the group) to see whether there is interest in forming a nonprofit with a mission to advance the development of medical therapies. 
  • If there is not a social media group, you may wish to start one. Using the disease name or associated gene in the title of the group can help others find it. 
  • Facebook has information about starting a group located at:
    • Group Basics
    • What are the privacy settings for groups?
• Forming a nonprofit: Once you have connected with other patients/families, you may want to see if anyone is interested in forming a nonprofit group. The tasks involved in forming a new group are best accomplished by having several people willing and able to dedicate the necessary time and effort. 
  • It may be a good idea to consider what roles you and the other members would like the group to fill. 
    • For example, a group’s mission may include:
      • Supporting research and medical therapy development.
      • Allowing families to connect and share resources.
      • Building a registry.
      • Establishing centers of excellence.
    • For more information about the many roles filled by support and advocacy groups, please see the GARD guide, Support for Patients and Families
  • The following resources can help you learn more about forming a nonprofit patient group:
    • Global Genes is a leading rare disease patient advocacy group whose mission is to “eliminate the challenges of rare disease.” Two of their toolkits are devoted to starting non-profit advocacy organizations:
      • So You Think You Want To Start A Nonprofit
      • Starting A Nonprofit: The First Steps
    • National Organization for Rare Disorders (NORD) is another leading  patient advocacy group dedicated to individuals with rare diseases and the organizations that serve them. They offer several resources for those considering starting a patient group.
      • Steps to Nonprofit Formation is a  review of the steps necessary to achieve 501(c)(3) nonprofit status. The guide also addresses frequently asked questions, such as the cost and time associated with establishing a disease foundation.
      • Nonprofit Resources is a list of resources to help provide information about managing, establishing, or finding nonprofit foundations. The resources include newsletters, nationally-based centers, and lists of charities.
    • Genetic Alliance is an international coalition comprised of advocacy, research, and health care organizations representing individuals with rare and genetic conditions. Most of the groups associated with Genetic Alliance are disease specific and many have driving therapy research for their specific disease as one of their main missions. 
      • Community forum (sign up) is a  forum where advocacy groups can mentor and learn from one another.
      • WikiAdvocacy: Information Regarding Organization Development provides a guide for developing a support and advocacy organization.
    • The Foundation Group also has helpful information about starting a nonprofit, including an explanation about gaining 501(c)3 status including the benefits and limitations imposed by this status. The Foundation Group is a company however, not a nonprofit. 
      • What is a 501(c)3?

Resources