The Food and Drug Administration (FDA) recently released a new draft guidance entitled, “Enhancing the Diversity of Clinical Trial Populations – Eligibility Criteria, Enrollment Practices, and Trial Designs: Guidance for Industry.” As with other clinical trials, inclusion and exclusion criteria in rare disease trials limit who can participate for safety, analysis, or other reasons. Sponsors and researchers struggle with the balance between increasing the chance of detecting real effects of the investigational product an limiting the generalizability of the trial findings. In this new guidance, the FDA recommends that sponsors consider removing inclusion criteria in later phase trials, using enrichment strategies, and increasing supports offered to trial participants. You can read the draft guidance here and enter your comments before August 30th.
TRiNDS offers expert clinical trial services to sponsors conducting clinical trials throughout the development process. We customize services to each project to meet its unique needs. Contact us for more about how TRiNDS can help your project succeed.
The Food and Drug Administration (FDA) has recently released
a new draft guidance entitled, “Adjusting for Covariates in Randomized Clinical
Trials for Drugs and Biologics with Continuous Outcomes – Guidance for
Industry.” This document would build on the ICH guidance for industry, “E9
Statistical Principles for Clinical Trials” by specifying the appropriate uses
of analysis of covariance (ANCOVA) in randomized clinical trials. The draft
guidance is open for public comment until June 24th.
Clinical trials testing investigational drugs and biologics
in rare disease populations are more difficult than in more common diseases.
Rare disease trials must be successful despite smaller sample sizes of patients
who may have had varying disease severity, routine clinical care, and impact on
quality of life prior to enrollment in the trial. Industry sponsors struggle to
balance increasing diversity of trial participants while maximizing a trial’s
statistical power. This new draft guidance includes recommendations for using
ANCOVA to increase power without increasing statistical error.
TRiNDS offers expert data management and biostatistical support to rare disease clinical trials and research studies. Are you struggling with the analysis plans for your study? Contact us to request a quote today.
TRiNDS is dedicated to clinical trials and research for
people with neuromuscular and other rare diseases. Rare disease research is
evolving quickly – the science is complex, the populations are small, and
everyone wants to finish trials quickly. It takes a team of experts to reach
rare disease research goals.
TRiNDS is guided by an expert board of advocacy group
leaders, researchers, and clinicians who help us do our best work for our
clients. The experts on our board bring perspectives from industry, academia,
major clinical centers, and non – profit groups. We rely on them to help steer
our work in the right direction. This
week, our teams are travelling to the main TRiNDS offices for our annual
company – wide meeting. We’ll be taking time to reconnect, to reflect on our
accomplishments, and to plan for the year ahead.
Are you starting a rare disease research project in 2019? Email us to learn how our unique business model can support your projects’ success.
TRiNDS has two job postings open in two cities at TRiNDS Careers. In Washington, DC we are looking for a contract Clinical Data Manager to join our data management team. The second opening is for an in-house Clinical Research Associate to work in our Pittsburgh, PA office with our clinical operations team.
TRiNDS is a great place to work and grow. Our expert teams specialize in neuromuscular research and clinical trials conducted around the world. We collaborate with innovative clients, expert researchers, and key opinion leaders to generate research success that matters to families affected by rare diseases.
Are you interested in joining the team? Do you know the perfect person for these positions? Apply today at the link above. We look forward to hearing from you!
Clinical trials can be difficult to manage. Trial sites around the world have different local institutional and ethical rules. Central study teams work with multiple different scientific and company stakeholders. Regulations governing clinical trials and drug development require comprehensive and accurate record keeping. And everything needs to be finished on time and on budget.
Rare disease clinical trials present unique management challenges. In rare disease clinical trials, eligible participants are much harder to find and sample sizes are smaller, making each detail of the trial even more critical to its success.
TRiNDS Clinical Operations offers customized trial management to researchers around the world. We assign a lead project manager to your project from the first feasibility assessment to final closeout. Our experts follow established guidelines and procedures to help trials stay on track throughout the course of the project. We’ll work with you to create the plans, strategies, and metrics that your trial will need. We help your team work together effectively for the rapid start up, on target enrollment, regulatory compliance, and accurate data that your research program requires.
TRiNDS offers expert clinical operations support to research studies and clinical trials working with neuromuscular and other rare diseases. Contact TRiNDS today to request a quote.
Every year, on the last day of February, the world celebrates Rare Disease Day. One day a year, we honor the patients and families living with a rare disease around the world. Rare diseases affect 1 in 20 people at some point in their lifetime.
Even though there are many different rare diseases, everyone faces the same kinds of struggles. A lack of awareness and scientific knowledge delays diagnosis. Lack of funding for research slows development of effective treatments. Families struggle to access high quality health care where they live.
All of us at TRiNDS are so grateful to work with scientists, families, and community groups to help find new approaches to rare diseases around the world. Join us in celebrating Rare Disease Day by visiting www.rarediseaseday.org to learn more.
TRiNDS offers customized services for rare disease clinical trials and research studies. We work with companies large and small to find research success. Contact us today to request a quote for your project.
Rare disease clinical trials are not the same as other trials. The paperwork and research rules might be the same, but everything else is different. The tests are different, the providers are different, but most of all, the families are different. People who are coping with a rare disease have different experiences, different fears, and different hopes than people with other kinds of diagnoses.
These differences are at the core of why we do what we do at TRiNDS. We know that conducting clinical trials are both essential to helping people with rare diseases and ask families to add stress to already stressful lives. Helping researchers navigate these issues is a core part of what we do.
TRiNDS Patient Science Liaison services offer customized advocacy, patient outreach, and research education services to researchers working with rare diseases. We can help connect you to the patient groups working in the community and help connect families to your trials. We’ll help you make the materials and strategies that you’ll need to recruit and retain eligible patients to your studies. Do you have an advocacy team? We can help extend your team by attending more events, creating special materials, or answering central inquiries.
TRiNDS offers expert research patient advocacy supports to research studies and clinical trials working with neuromuscular and other rare diseases. Contact TRiNDS today to request a quote.
The first step of any research study is the informed consent process. Every patient and their family is asked to decide whether or not to enroll in a study through a discussion with site staff and study doctors.
Even though informed consent is important, finding the right balance of enough information at the right time is difficult. Too little information, and the decision isn’t truly informed. Too much information, and the participant becomes overwhelmed. It’s even harder when your participant is a child, or speaks English as a second language, or has limited health literacy.
Researchers at the Michigan Institute for Clinical and Health Research have been working on different ways to make informed consent better. Join the Trial Innovation Network for a webinar on March 20th. You can learn more by visiting the events page.
TRiNDS offers expert clinical operations support to research studies and clinical trials around the world. Contact TRiNDS today to request a quote.
We specialize in neuromuscular and rare disease clinical trials. The science is fascinating, but there are a lot more complexities than in other types of clinical trials. Many neuromuscular and other rare diseases have genetic causes, which makes everything even more complicated.
It’s important to design your clinical trial protocols to minimize variability in your study population up front. For many TRiNDS studies, that means confirming participants’ genotypes during study screening. Whether you’re testing an investigational product that acts at the DNA or RNA level, or if you need to exclude certain diagnostic subtypes, having expert help is critical.
We offer central diagnostic confirmation services to research projects and all phases of clinical trials. Our licensed genetic counselors combine their clinical training and research expertise to provide consistent review and analysis of test results. After informed consent, sites or central laboratories submit de – identified reports to our team for review. Our genetic counselors interpret the results and inform the site and the medical monitoring team of the participant’s eligibility for the study.
Contact us to request a quote for central diagnostic confirmation services.