Sciensano

MAY 18, 2022

During this event, Dr. Tim Dallman (University of Utrecht) lectured on specific case studies, advantages and challenges of routine genomic surveillance of gastrointestinal pathogens. BE READY project coordinator, Transversal activities in Applied Genomics ( TAG )). Dr. Marie-Alice Fraiture ( TAG ). Dr. Nancy Roosens (.

Genome 52

Genome Genomics Research 52

Rethinking Clinical Trials

JUNE 14, 2023

The goal with this aim is to determine if the same conclusion can be drawn from a database study and a trial. The second aim is to test the transparent and reproducible process with the FDA to evaluate Real World Evidence (RWE) studies. The study team used three predefined binary agreement metrics that they applied to each trial.

Clinical Trials 130

Clinical Trials Clinical Trials Trials Research 130

Intouch Solutions

NOVEMBER 17, 2021

blue links) of the organic results appeared to be pulling from content outside of the HTML title tag at a much higher frequency. Title Tag – This is an HTML element that provides a title for the page. The title tag is not otherwise visible to users on the page. Specifically, title links (i.e., The SERP Title is the blue link.

Marketing 52

Marketing Engineer Branding 52

Rethinking Clinical Trials

DECEMBER 6, 2023

But this assumption may not be true in all studies or for all types of treatment. Researchers need to think very carefully about what is the possible exposure time curve and what is it the study is trying to estimate in the stepped-wedge trial? A key question is what is the estimand and what is most scientifically meaningful?

Trials 162

Trials Clinical Trials Clinical Trials Research 162

Rethinking Clinical Trials

OCTOBER 19, 2022

Due to the low referral rates of fibromyalgia patents to PT (~30%), FM-TIPS had an emergent ethic issue related to patient recruitment and whether the study team could include the American College of Rheumatology criteria to screening to increase eligibility. There is a possible for risk-signaling data to “slip between the cracks.”.

Clinical Trials 130

Clinical Trials Clinical Trials Trials Medicine 130

Rethinking Clinical Trials

OCTOBER 11, 2023

Moyer, PhD Statistician, NIH Office of Disease Prevention Slides Keywords Implementation; Study design; Hybrid; Clustered; DECIPHeR Key Points People often contest that hybrid designs are not as rigorous as they should be. In implementation studies, how can you blind outcome assessor? Learn more Visit the DECIPHeR website.

Clinical Trials 162

Clinical Trials Clinical Trials Trials Research 162

Rethinking Clinical Trials

NOVEMBER 6, 2023

Finally, in a single agent structure, there’s only one agent present per arm in the fully nested case or in the trial as a whole for the partially nested or crossed structures. Since power in small studies is less adequate, a power analysis with realistic and data-based estimates is key. Are most of these trials being done incorrectly?

Trials 130

Trials Clinical Trials Clinical Trials Contamination 130

Pharmaceutical Technology

APRIL 13, 2023

Two gene therapies up for approval this year for sickle cell disease could be cost effective in some cases at a $2 million price point, based on a draft evidence report published by the Institute for Clinical and Economic Review (ICER).

Gene Therapy 147

Gene Therapy Gene FDA Approval Licensing 147

Camargo

AUGUST 22, 2021

To help sponsors understand how the FDA uses the eCTD technical validation rules to verify conformance, the FDA developed the technical rejection criteria (TRC) for study data. Effective September 15, 2021, the FDA will begin enforcing the TRC for study data. Where Are the Technical Rejection Criteria for Study Data Applied?

Containment 190

Containment Bioavailability Cosmetics Packaging 190

Rethinking Clinical Trials

OCTOBER 27, 2022

Case study: The U.K. coroner in the case of Molly Russell, a teenager who committed suicide, determined that she was suffering from a depressive condition, and, based on the sites she was visiting, the algorithms were feeding her very negative images without her request. Learn more. Discussion Themes. What is the path forward?

Clinical Research 130

Clinical Research Research Clinical Trials Clinical Trials 130

pharmaphorum

JANUARY 5, 2022

Teliso-V is currently in a 233-patient phase 2 study (LUMINOSITY) involving subjects with c-Met-positive, non-squamous NSCLC as a second- or third-line treatment after chemotherapy, immunotherapy with checkpoint inhibitors or drugs targeted at specific mutations. Preliminary results from that study showed a 53.8%

Antibody 109

Antibody Drugs Protein Gene 109

The Pharma Data

FEBRUARY 22, 2022

As part of its on-going work to track variants, WHO’s Technical Advisory Group on SARS-CoV-2 Virus Evolution ( TAG-VE ) met yesterday to discuss the latest evidence on the Omicron variant of concern, including its sublineages BA.1 Studies have shown that BA.2 1.1), there is still a reported decline in overall cases globally.

Protein 52

Protein Vaccination Vaccine Genetics 52

The Pharma Data

NOVEMBER 29, 2021

1.1.529 a variant of concern, named Omicron, on the advice of WHO’s Technical Advisory Group on Virus Evolution (TAG-VE). Researchers in South Africa and around the world are conducting studies to better understand many aspects of Omicron and will continue to share the findings of these studies as they become available.

Vaccination 52

Vaccination Vaccine Genome Genomics 52

Rethinking Clinical Trials

DECEMBER 14, 2022

Ethics, Stepped Wedge Cluster Randomized Trial, Study Design. Trialists should work with a biostatistician to come up with the most scientifically robust design given the practical constraints of the study. In most cases, a parallel arm or cluster cross would be a better choice. Director, Stanford Center for Biomedical Ethics.

Trials 130

Trials Clinical Trials Clinical Trials Medicine 130

XTalks

AUGUST 19, 2022

Register for this free webinar to learn why the agile clinical trial model is a great fit for many rare disease categories, how to navigate the regulatory landscape for agile clinical trials in rare diseases, how to design studies and more. Zynteglo’s hefty price tag of $2.8 The results were maintained as of last follow-up.

Gene Therapy 95

Gene Therapy FDA Approval Gene Clinical Trials 95

Rethinking Clinical Trials

OCTOBER 26, 2023

with the rate of reported acute Hepatitis C cases increasing 400% during 2010-2020. PCORnet is engaging with sites to support the ITAP clinical study for de novo clearance of a qualitative POC HCV test-to-treat platform. Rates are the highest among 20-39-year-olds. A manuscript is under development.

Clinical Research 147

Clinical Research Clinical Trials Clinical Trials Research 147

Rethinking Clinical Trials

NOVEMBER 9, 2022

CardioNerds collaborated with ACA to invite every fellowship to share case-based learning and the training opportunities of their program. In the future, we would like to study this in greater detail and find out what happened at CardioNerd sites compared to other sites. Forty-four programs participated. Learn more.

Clinical Trials 130

Clinical Trials Clinical Trials Trials Development 130

Rethinking Clinical Trials

JULY 19, 2023

Results of the study showed a positive correlation between prescription of the therapies and the patients actually using the medications. Key study limitations were the need for remote delivery due to the COVID-19 pandemic and the lack of total representation of the broader U.S. They randomized the clinics, then enrolled the patients.

Trials 130

Trials Cardiology Medicine Nurses 130

pharmaphorum

FEBRUARY 22, 2021

Bluebird Bio’s decision to hit pause on the launch of Zynteglo for beta thalassaemia after two cases of cancer were seen in a clinical trial could see fears over the safety of viral vectors used to deliver gene therapies resurface. . million in Europe.

Gene Therapy 52

Gene Therapy Gene Gene Sequencing Gene Expression 52

Rethinking Clinical Trials

SEPTEMBER 21, 2023

Default options have been shown to increase healthy food orders in restaurants, but no studies had not been previously conducted to represent the effects of online shopping carts pre-filled with tailored fruit and vegetable items. The results showed that about 20% of the cart total was spent on fruits and vegetables across all cases.

Clinical Trials 130

Clinical Trials Clinical Trials Sales Trials 130

XTalks

SEPTEMBER 27, 2022

The record-breaking price tag of Skysona is not surprising for one-time gene therapies like it. Case in point, bluebird had to pull both Zynteglo and Skysona and cease commercial activities in Europe after it failed to reach reimbursement agreements with local governments. Together, this amounts to about 40 cases a year in the US.

FDA Approval 52

FDA Approval Gene Therapy Drugs Gene 52

pharmaphorum

NOVEMBER 24, 2022

Yesterday, the drugmaker added to the evidence behind Spravato – an intranasal formulation of esketamine – with the release of top-line data from a head-to-head study with a long-acting formulation of the antipsychotic quetiapine, which is also used to treat TRD. and 17.6%, respectively, according to J&J’s pharma unit Janssen.

Trials 52

Trials Sales Drugs Marketing 52

XTalks

DECEMBER 13, 2023

Related: Roctavian Becomes First Gene Therapy for Severe Hemophilia A to Get FDA Nod Casgevy and Lyfgenia Pricing and Warnings The price tags of both gene therapies are steep, given the complexity of developing gene therapies and as therapies meant to be one-time treatments. Vertex-CRISPR’s Casgevy has a US list price of $2.2

Gene Therapy 98

Gene Therapy FDA Approval Gene In-Vivo 98

pharmaphorum

APRIL 27, 2022

It was the first time ever that a HER2-directed therapy has shown a benefit in patients with HER2-low breast cancer, a group which accounts for around 55% of all cases of the disease versus 25% for HER2-positive tumours. The post Enhertu gets breakthrough tag in HER2-low breast cancer appeared first on.

Sales 57

Sales Drugs Antibody Licensing 57

pharmaphorum

AUGUST 18, 2022

million price tag attached to the therapy when it was first made available in Europe, and is also well above the proposed $2.1 Offsetting the cost of the therapy is the lifetime cost of transfusion-dependant thalassaemia, which in a recent study was estimated to be $5.4 bluebird has opted to launch it at a cost of $2.8

Gene Therapy 52

Gene Therapy FDA Approval Gene Clinical Trials 52

pharmaphorum

OCTOBER 4, 2022

The author of the study noted that researchers from anywhere in the world can now request access to the growing dataset that is being generated by the Biobank effort. Informed consent is crucial to recruiting the patient to the study. Up in the clouds. Facilitating patient understanding and consent.

Biobanking 103

Biobanking Clinical Trials Clinical Trials Trials 103

Pharmaceutical Technology

APRIL 5, 2023

But access to these treatments continues to remain limited due to high price tags and variable availability across regions. But while companies continue studying allogeneic CAR-T therapies, including for their coveted use in solid tumours, such advancements remain challenging.

Gene Therapy 147

Gene Therapy Gene Engineer Production 147

XTalks

MAY 2, 2024

In a case study on inspections, Sonny Brar of Fiera Foods highlighted how technology can transform food safety inspections from mere compliance to excellence, offering practical insights into the application of these technologies. .

Compliance 110

Compliance Regulation Production Containment 110

XTalks

JUNE 8, 2021

With a price tag of over $2.5 The researchers conducted a long-term follow-up study of the START trial that included patients who completed phase I of the study. The study also found that early treatment was better. Moreover, the drug is a one-time dose; “the fewer the cases the higher its price will be,” Dr.

Gene Therapy 110

Gene Therapy Gene Protein Trials 110

XTalks

NOVEMBER 24, 2022

Many women carriers of the disease have no symptoms while 10 to 25 percent have mild symptoms and in rare cases, women may have moderate or severe symptoms. The FDA approval was based on results of two studies with adult men, including the HOPE-B trial, which had 54 participants, the greatest number of patients in a gene therapy trial.

Gene Therapy 52

Gene Therapy Gene FDA Approval Genetics 52

pharmaphorum

JANUARY 5, 2021

It’s been granted on the strength of data from the phase 2 CITYSCAPE study – reported at the ASCO congress last year – that showed the pairing shrank tumours in 31% of patients, twice the response seen with Tecentriq (atezolizumab) alone. The post Roche nabs breakthrough tag for TIGIT cancer immunotherapy appeared first on.

Immune Response 52

Immune Response FDA Approval Drugs Sales 52

XTalks

JUNE 9, 2021

While the FDA approval does not indicate that the drug is for a specific group of patients, aducanumab was studied in a specific subset of patients with early disease and who were positive for a component found in amyloid brain plaques. Aducanumab Controversy and Price Tag. Evidence for aducanumab’s efficacy has been highly contested.

Drugs 98

Drugs Clinical Trials Clinical Trials Trials 98

pharmaphorum

MAY 10, 2022

The FDA awarded a fast-track designation to lecanemab in December and has also given the drug breakthrough tag as a treatment for early-stage Alzheimer’s, and Biogen and Eisai will be hoping the drug can sidestep the controversy that engulfed Aduhelm from the moment it was approved.

FDA Approval 40

FDA Approval Trials Clinical Trials Clinical Trials 40

XTalks

DECEMBER 21, 2022

Bluebird is also now back on track in evaluating its candidate gene therapy for sickle cell disease (SCD) after the FDA lifted a clinical hold on studies of the drug this month. Thus, in some cases, the expense of a gene therapy may be lower than the lifetime cost of treating patients with conventional treatments.

Life Science 52

Life Science Gene Therapy Gene Clinical Trials 52

Drug Discovery World

MAY 22, 2023

But are these price tags a barrier to access? In the case of Hemgenix, the assurance behind the price point is that the drug would reduce long-term healthcare costs as patients would have fewer healthcare incidents and need fewer clotting treatments. million per dose.

Gene Therapy 52

Gene Therapy Gene Drugs Contamination 52

XTalks

FEBRUARY 27, 2024

Addressing the high price tag, Iovance’s commercial chief Jim Ziegler said, “Payers have expressed their appreciation for the value proposition for Amtagvi.” The immune cells in both cases are then infused back into the patient. With growing interest in TIL therapies, investors have invested $2.7

FDA Approval 52

FDA Approval Manufacturing In-Vivo Trials 52

Delveinsight

FEBRUARY 23, 2021

The company plans to use the capital to expand its pipeline of protein-based vaccines and biologic cancer therapies utilizing its innovative and proprietary Trimer-Tag technology platform. The company now plans to study more about the biomarkers in ALS and hopes to come up with broadly effective therapy.

In-Vitro 52

In-Vitro Vaccination Vaccine Licensing 52