What Can NCATS Toolkit Do Better?
Interventional clinical trials study the efficacy and safety of a potential therapy in humans. Studies are first performed in small groups of patients and then in larger patient populations. The goal of these clinical trials is to obtain data to support marketing approval. Marketing approval allows a medical product to be manufactured and sold outside of research studies.
In the United States, the U.S. Food and Drug Administration (FDA) regulates clinical trials and the sale of medical products. Your group can play an essential role in educating your community about clinical trials to ensure potential participants can make an informed decision. You can also contribute to the FDA monitoring and marketing review processes by providing input on the patients’ perspective.
Support Clinical Trials
Serve On Review And Monitoring Boards
Participate In FDA Advisory Meetings
Prepare For Clinical Trials
In addition to supporting preclinical trial research, your group can work with key stakeholders to lay the foundation for successful clinical trials.
What Can NCATS Toolkit Do Better?
Preclinical studies can help scientists learn how a potential therapy interacts within living systems before being tested in humans. Testing is carried out using cell and animal models of the disease, samples of human or animal tissues, and possibly computer simulations. When a therapy has the potential to be both safe and effective in humans, a Sponsor, often from the pharmaceutical industry, will begin to move forward towards clinical trials.
In addition to supporting preclinical studies, multiple opportunities exist for your group to help prepare for clinical trials. Data from your patient registry and natural history study can help Sponsors design clinical trials. Established clinical centers for your disease may serve as sites for the clinical trial. Your patient group can also perform patient preference studies and participate in initial meetings between the Sponsor and the FDA.
Establish Clinical Sites
Help Industry With Clinical Trial Design
Participate In Initial Industry-FDA Meetings
Perform Patient Preference Studies
Give Input On The Informed Consent Process
Discovery
Your group can support the early phases of therapy development in multiple ways, including collecting and sharing data from your patient community and helping to fund a and prioritize research activity.
What Can NCATS Toolkit Do Better?
Discovery is the scientific exploration to find therapies that may be able to treat or cure your disease. Potential therapies can include drugs, medical devices, and biologics. Researchers often discover potential therapies through new scientific insights or by testing compounds for beneficial effects. After early testing of many potential options, researchers will select promising ones for further study.
It is ideal for your group to engage in research from the beginning. When researchers and industry partners understand your patient community’s needs and priorities and can track the natural history of your disease, they can target their research projects accordingly. You can often accelerate research by encouraging collaboration among scientists, government, and industry. You can also support research through efforts to increase funding for the development of translational research tools and discovery of potential therapies.
Understand Translational Research Tools
Determine Patients' Needs
Facilitate Scientific Research
Fund Discovery Research
Getting Started
If your patient group is new to supporting research, Getting Started can help you understand therapy development, the importance of patient input, basic disease research, and ways to connect with partners.
What Can NCATS Toolkit Do Better?
Whether you are leading a new patient group formed to drive the development of a treatment or cure for your disease or an established patient group that is considering adding research initiatives to your group’s mission, the information and resources in the Getting Started section will provide an overview of the medical therapy development process, the increasing role of patient groups in the process, basic research questions that need to be understood prior to therapy development, and methods to make the connections needed to drive the process forward.
The different stages of the therapy development process are described in more detail in subsequent sections, so please don’t be overwhelmed by the information in the Getting Started overviews. Instead, congratulate yourself on taking this first step to join other patient groups who have become an integral part of the therapy development process.
If you are leading a group that has already been involved in therapy development for your disease, you may wish to skip to one of the later sections to find information and resources that can help you develop next steps, overcome challenges, or fill in pieces of the process that may further your efforts.
Understand The R&D Process
Empower Your Patient Community Voice
Demystify Your Disease R&D Readiness
Build Relationships With Key Partners
This enables the test sub-module
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