Translational research is a team sport. When you and other patients collaborate with researchers, healthcare professionals, and industry, you help drive research forward. Virtual or in-person meetings give you and other participants the opportunity to learn from one another and develop personal relationships. Meetings also help encourage strategic planning and collaboration on new projects.
Patient registries and natural history databases are proving to be invaluable tools for stimulating research. The type of data collected will vary depending on the purpose of the registry. Contact registries typically include contact information and demographics and are useful for organizing the community and keeping people informed of opportunities to participate in research. Registries for research are often called natural history databases and contain more detailed clinical information over time, such as age at diagnosis, symptoms, medical images, and test results. Data may be entered by patients, their caregivers, or healthcare professionals. These databases are helping engage patients early, often, and effectively. Documenting the experiences of patients and their caregivers provides valuable information for researchers, industry partners, and regulators.
Patient groups often choose to raise money to support research or seek other funding sources from the government or nonprofits. Some groups form their own grant program, where researchers submit a proposal and a scientific review committee recommends which projects to fund. Another option is to team up with other organizations where the science overlaps to support mutually beneficial projects.
Your patient group can help accelerate research by working with patients, scientists, and industry partners to develop the translational research tools (for example, assays, biomarkers, cell and animal models, or biorepositories). These tools are needed to better understand a disease process, identify therapeutic targets, and test investigational therapies in preparation for clinical research in humans.
The U.S. Food & Drug Administration (FDA) encourages patient organizations to organize meetings to generate public input on a disease area, using the process established through the FDA's Patient-Focused Drug Development (PFDD) Initiative. Your patient group can submit a Letter of Intent to the FDA to request that FDA staff attend the meeting. The FDA hosted 24 meetings from 2012-2017 and now suggests that patient groups host their own meeting following a similar format to capture the patient perspective on specific diseases and their treatments.
The therapy development space is a dynamic, innovative field. New therapeutic approaches are constantly under investigation. The following tools will help you learn about new research areas and technologies.