Community Webinar: Clinical Trials Basics with Jennifer Panagoulias
In this webinar, Jennifer Panagoulias provides an introduction to clinical trials and discusses key considerations for parents considering trial participation. She explains the different types of clinical studies, the phases of clinical trials, and the importance of controls in ensuring unbiased results. Jennifer also discusses the informed consent process and the role of advocacy organizations in clinical trials. She emphasizes the importance of asking questions and engaging with regulators and companies to ensure the best outcomes for patients. Jennifer concludes by addressing specific questions from the audience regarding genotype-specific trials, the approval process in different regions, and the role of pediatricians in trial enrollment.
Welcome to the community webinar on clinical trials basics! In this webinar, Jennifer Panagoulias, head of regulatory and policy at FAST in the US, will provide an introduction to clinical trials and discuss important considerations for parents. The webinar is a joint event between FAST UK and Angelman UK, and we have 45 minutes of Jennifer’s time for the presentation followed by 15 minutes for questions. Let’s get started!
Irina, a FAST UK trustee, introduces Jennifer Panagoulias and thanks her for joining the webinar. Jennifer is an expert in regulatory affairs and has extensive experience in rare disease clinical trials. She is also the aunt of a young lady with Angelman syndrome, which has brought her to this community. Jennifer will be sharing her knowledge and experience in clinical trials from a parent’s perspective.
Clinical Trial Basics
Jennifer begins by explaining the different types of clinical studies. There are observational studies, where researchers observe patients without intervening, and interventional studies, where patients are assigned to different groups and receive an intervention, such as a drug or device. The focus of this webinar is on interventional studies, specifically clinical trials testing the safety and efficacy of interventions for Angelman syndrome.
Clinical trials have different phases, typically ranging from Phase 1 to Phase 3. Phase 1 focuses on safety, Phase 2 on safety and efficacy, and Phase 3 on confirming safety and efficacy in a larger population. In rare diseases like Angelman syndrome, sometimes phases are combined due to the small patient population. There is also a Phase 4, which occurs after approval and involves post-approval studies to gather additional information about the drug.
Jennifer explains the importance of controls in clinical trials. Controls are used to minimize bias and help establish the safety and efficacy of the drug being tested. Placebo controls involve giving one group no treatment, while active controls compare the new drug to an already approved drug. Sham controls are used in invasive procedures, such as gene therapy, to maintain objectivity. Historical controls compare the treatment group to a reference group from previous studies.
Informed Consent and Enrolling in Trials
Jennifer emphasizes the importance of informed consent in clinical trials. The consent form should cover various aspects of the trial, including the purpose, duration, procedures, risks, benefits, alternatives, confidentiality, compensation, and contact information. Participants should have the opportunity to ask questions and fully understand the trial before enrolling.
She also explains the process of enrolling in a trial. Eligibility criteria are defined in the study protocol and must be followed to ensure patient safety. Companies can amend the criteria with scientific rationale, but it requires approval from regulatory agencies. Jennifer encourages participants to stay informed about ongoing trials through resources like clinicaltrials.gov, Be Part of Research, and advocacy organizations.
During the Q&A session, Jennifer addresses questions about the inclusion of different genotypes in trials, the approval process in different countries, and the role of patient advocacy groups. She explains that companies can expand eligibility criteria based on scientific rationale and encourages advocacy groups to engage with regulators to ensure the needs of the community are considered.
The webinar concludes with a reminder to participants to continue advocating for expanded eligibility criteria and to stay informed about ongoing trials. Jennifer thanks everyone for their participation and encourages them to reach out to companies and advocacy organizations for more information.
- Title: Community Webinar: Clinical Trials Basics with Jennifer Panagoulias
- Author(s): Jennifer Panagoulias
- Author(s)’ affiliation: FAST
- Publication date: 2023-06-20
- Collection: FAST UK Webinars