Natural history study databases or registries that contain extensive clinical information can provide valuable insights on how the disease evolves over time. They can be used to help scientists fully understand a disease and determine which symptoms will impact patients the most. They may also help find biomarkers and clinical outcome measures that will show how well a patient responds to a treatment in clinical trials. These databases can be used for identifying patients who may be eligible for each research project and informing clinical study designs and procedures. Natural history databases are also important for attracting investment by industry partners and educating funding agencies and regulators.
Developing clinical centers that have expertise in diagnosing and caring for patients with your disease are for conducting clinical trials and optimizing care. When clinicians and staff have experience with your disease and establish relationships with patients, they are better prepared for conducting clinical trials. It is also helpful when clinical centers have been uniformly trained in evaluation procedures so as to minimize variability among the sites in the trial network.
Your patient group can draw from your patient registries, natural history databases, animal and cell models, biorepositories, benefit-risk assessments, patient surveys, and clinical site networks to help industry partners design clinical trials in terms of subject and site selection, clinical outcome measures, and study procedures that will be well tolerated by the patients and most likely to capture meaningful data. When you and your patient group are involved in clinical trial design, you may improve recruitment, retention, endpoint selection, and study completion.
Talk to your industry sponsor about participating in their meetings with the FDA. In most circumstances, before an industry sponsor can administer an investigational product to humans, they must submit an Investigational New Drug (IND) Application to request authorization from FDA. The sponsor may request pre-IND meetings with FDA to request feedback on their development program. FDA regulators are increasingly interested in having patients and patient advocates participate in these early discussions. However, it is up to the industry sponsor to invite patient groups to participate because the FDA cannot invite patients or patient advocates directly.
Capturing data on how patients perceive potential risks and benefits of an investigational therapy can inform product development and regulatory review decisions. These patient-preference studies can help identify what is most important to patients, particularly when assessing benefits and risks is challenging.
Informed consent allows clinical trial participants to understand what will happen during a clinical study, possible risks, and whether or not they will receive their personal results. Your patient group can provide input during development of informed consent documents and help clinical trial participants better prepare for the consenting process.
Your patient group can help improve the study process. For example, enhanced funding for patient recruitment services may help to speed enrollment. Financial assistance for support staff, materials, or other infrastructure needs can help make the study process run smoothly and efficiently.