Our rare disease team shows up — for patients, sponsors, and families. It's important for us to hear directly from these invaluable voices so we can be the best allies.
Thanks for sharing Laura Fernandes. Great presentation.
The Bhatt article is very detailed and well-organized.
Anyways, as I prepared a presentation last week based on the Bhatt article, I realized “the reasons for and ways of complementing RCTs with RWE”.
Example
Reasons:
1. It may not be possible to conduct an RCT for a rare disease with a small number of population.
2. It may not be appropriate to conduct an RCT for medical devices unless it claims to possess significant benefits, etc.
In both situations, RWE can help.
Ways:
RWE has to achieve randomization (via matching strategies), be controlled (inclusion and exclusion criteria) and use trial-like econometric methods such as Survival analysis and difference in difference.
I can’t wait to share my presentation here as well.
#RWE#RCT
Ex-FDA biostatistician specializing in oncology/hematology clinical trial designs and RWD research.
Did you know the first recorded clinical trial was in 500BC, that James Lind conducted the first controlled trial in 1747 and the first double blind trial was conducted only in 1943?
I did a mini presentation on the past, present and future of clinical trials on national clinical trial day on 5/20, browse through the slides and let me know if you find anything new or interesting. Would love to know if you participated in a clinical trial yourself - thank you in advance for your participation. #clinicaltrials
Happy Clinical Trials Day everyone!
Organized and promoted by ACRP since 2014, Clinical Trials Day arrives each May 20 as a joyful opportunity to pause in reflection, recognition, and admiration of all that has been accomplished thanks to clinical trials and the people behind them. Learn more here: https://lnkd.in/gNVnmqGX#CTD2024#CTD24#ILoveClinicalResearch
Looking forward to reviewing the science of IgG4-RD next Friday with Cory Perugino. Though IgG4-RD was recognized as a unique autoimmune disease only this century, we've learned a lot about what makes this disease tick already. Cory will review that in a way that facilitates patients' understanding about just how far we've come - and, by extension, how our therapeutics should be advancing quickly because of trials ongoing now.
Register through this link: https://rb.gy/aks71a#IgG4RD#IgG4relateddisease#raredisease
Have you registered for our upcoming Fireside Chat on Friday, February 23, from 12 - 1:30 p.m. EST., with Dr. Cory Perugino and John Stone MD, MPH?
Dr. Perugino will provide a patient-centric overview of the science of IgG4-RD, and Dr. Stone will share information on the current two phase 3 IgG4-RD clinical trials: the MITIGATE Trial and the INDIGO Trial.
In addition to those presentations, Drs. Perugino and Stone will be answering some pre-submitted patient questions during a Q and A.
Register for the event and submit your questions for consideration during the Q&A here: https://rb.gy/aks71a#IgG4RD#IgG4relateddisease#raredisease
We've got some exciting opportunities for you through Practice Point CME:
✔️ Talking COPD: Challenged and recommendations for improving access to evidence-based COPD Management
✔️ Talking PAH: Evidence from ATS, ERS and CHEST 2023 for Improved Clinical Care
✔️ What's New Doc: What does it take to start an expert PH referral center and become a clinical investigator?
Access activities at https://bit.ly/472R5n9
The Clinical Trials Platform team have been attending the Australian Clinical Trials Alliance (ACTA) International Clinical Trials Symposium over the last three days. They have been learning how novel clinical trial design approaches can be utilised to improve efficiency in clinical trials, and how public involvement can generate greater impact. It has been great to hear from some fabulous international guest speakers about the different approaches to accelerate clinical trial growth.
#ACTA2023#clinicaltrials#publicinvolvement
Happy Clinical Trials Day!
Today we celebrate all of the hard work that goes into clinical trials on the anniversary of the first clinical trial in 1747.
While we celebrate all of those that put in hard work to make a clinical trial happen - we have to remember the patients. Without them there would be no trial!
Ask me how we can help enhance patient retention in your clinical trial!
mdgroup#clinicaltrialsday2024#patientretention#patientadvocate
The purpose of RMI Clinical Trials Unit.
Starting with COVID-19 and efforts for its vaccine development, which led to essential clinical trials.
With our patient-focused approach, every step is guided by the recognition of the participant's crucial role.
RMI CTU was designed to enhance medical understanding, conduct high-quality research, and bring real benefits to the community.
For details, visit our website:
ctu.rmi.edu.pk#RMI#RMICTU#ClinicalTrials#clinicaltrailsunit#healthadvancements#healthcareresearch#medicalresearch
Felix Perez, CEO Research Director
1moThat's wonderful to hear! Direct insights from patients, sponsors, and families truly make a difference in rare disease advocacy and support.