Agency IQ

JUNE 28, 2024

BY COREY JASEPH, MS, RAC New guidance from the European Commission outlines alternatives for full pre-market clinical trials for orphan devices, defined by the Commission for the first time. Notably, this is also the first guidance to describe the possible use of a conditional Notified Body certificate.

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Crucial Data Solutions

DECEMBER 19, 2023

The growth of decentralized clinical trial (DCT) strategies may be happening slower than many expected, but it is definitely happening. The post The Advantages of Decentralized Clinical Trials in Diabetes Research appeared first on Crucial Data Solutions.

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FDA Law Blog: Drug Discovery

MARCH 7, 2024

Clissold — The trio of CDER, CBER, and CDRH released a new draft guidance titled “ Use of Data Monitoring Committees in Clinical Trials ” that revises the 2006 guidance “Establishment and Operation of Clinical Trial Data Monitoring Committees” and, when final, will replace the 2006 guidance.

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Cytel

APRIL 22, 2024

Kyle Wathen, Vice President, Scientific Strategy & Innovation One of the pivotal metrics considered when designing a clinical trial is the study’s probability of success, which can be measured in several ways.

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Agency IQ

JUNE 28, 2024

New guidance provides definition for orphan device, offers alternative trial designs New guidance from the European Commission outlines alternatives for full pre-market clinical trials for orphan devices, defined by the Commission for the first time.

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PPD

MARCH 11, 2025

While there is no universally accepted definition for rare disease, it is estimated that more than 10,000 distinct rare conditions exist, and people living with these rare diseases represent as much as 10% of the global population. Moreover, in some rare disease trials, it is unethical to design a control group of patients with a placebo.

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Conversations in Drug Development Trends

MAY 1, 2024

This process can be daunting, but understanding how to manage feedback effectively is crucial for developing and ultimately gaining approval for new therapies, especially in oncology clinical trials. Additionally, consider the locations of your trial; if it is multiregional, you can start planning for your overall product approval strategy.

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Drug Target Review

OCTOBER 30, 2024

Now take a step further: envision testing drugs in these organoids to identify the ones that can treat disease safely and effectively without needing to run expensive clinical trials first. Further still, think about implanting these mini organs into the patient to restore lost function.

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Advarra

JUNE 18, 2024

Defining Feasibility Feasibility is “a process of evaluating the possibility of conducting a particular clinical program/trial in a particular geographical region with the overall objective of optimum project completion in terms of timelines, targets, and cost.” Take a clinical trial management system (CTMS) example.

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The Premier Consulting Blog

OCTOBER 21, 2024

However, accelerated approval comes with a condition: sponsors must conduct confirmatory trials to verify the drug’s anticipated clinical benefits using robust outcome measures. Concerns have arisen over delays—sometimes spanning over 7–8 years—that may expose patients to risks before confirmatory trials are completed.

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Vial

JUNE 12, 2024

PROs in clinical trials are important as they capture the patient’s perspective and ensure that the impact of an intervention is comprehensively evaluated. What are PROs in clinical trials? In a study on cancer clinical trials, Pe et al. From a regulatory standpoint, agencies like the U.S.

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FDA Law Blog: Biosimilars

FEBRUARY 9, 2024

Overall, the final guidance provides many editorial clarifications as well as some noteworthy updates, including discussions on DHTs that meet the definition of a device, selection of a DHT and rationale for use in a clinical investigation, retention and protection of data collected by DHTs, and DHT updates.

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Advarra

JUNE 9, 2022

Optimizing clinical trial access for potential patients is a critical goal for researchers and sponsors. Decentralized clinical trials (DCTs) can meet that need, but they come with some challenges. Designing a DCT comes with certain challenges of course, particularly when proposing to utilize outside groups or locations for the trial.

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The Pharma Data

AUGUST 5, 2020

Following an interim analysis of the phase 2 ELARA trial, researchers found that Kymriah met the primary endpoint of complete response rate (CRR) – a standard measure of response to therapy in FL patients. Source link.

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PPD

AUGUST 9, 2023

The increased adoption of decentralized clinical trial (DCT) designs has revolutionized how the industry approaches clinical studies. In short, for clinical trial design to truly benefit from the digital revolution, data flow, management and analysis are the next frontiers.

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Conversations in Drug Development Trends

NOVEMBER 5, 2024

Their virtual platform is fantastic, as well as their collaboration with the European Reference Network are all things CROs should leverage to support their trials. Both approaches need experts in the drug process, and CROs can definitely make an impact. Looking ahead to 2025, what are your hopes for rare disease research?

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The Pharma Data

SEPTEMBER 23, 2020

The halting of AstraZeneca’s COVID-19 vaccine trial around the world was cause for much initial concern over the eventual safety and efficacy of the therapy. Trials have resumed in the UK, Brazil and South Africa, but US trials remain on hold. Matt Fellows. Source link.

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Advarra

OCTOBER 4, 2022

Clinical trial site selection can make or break a trial’s success before it even begins. When you consider around 11% of sites 2 fail to even accrue one participant on a given study, cost savings become a major consideration when evaluating which sites to partner with for a trial.

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Antidote

FEBRUARY 28, 2023

In the United States, a rare disease is defined as a condition that impacts less than 200,000 people — by that definition, about 1 in 10 individuals (roughly 30 million people) in America have a rare disease

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Drug Target Review

OCTOBER 5, 2023

This study sought to shed light on the safety and effectiveness of aHSCT in routine healthcare settings, moving beyond the confines of clinical trials. It’s important to note that this study is observational in nature and lacks a comparative group, which limits the ability to draw definitive conclusions.

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Alta Sciences

SEPTEMBER 13, 2023

Four Pain Models Altasciences Uses to Assess Treatments During Clinical Trials pmjackson Wed, 09/13/2023 - 17:01 September is pain awareness month, and the importance of pain management and continued research into effective analgesics is integral to helping patients suffering from various pain conditions. Tags Clinical Trials Weight 1

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New Drug Approvals

JULY 8, 2025

Clinical Trials: Pritelivir is currently in phase II clinical trials, with ongoing research into its effectiveness and safety. Clinical Trials: Pritelivir mesylate is currently under extensive study to evaluate its efficacy and safety profile, with promising results in early clinical trials. Johnston, C. 2016.18189.

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LifeSciVC

JANUARY 16, 2024

Whether trial design, execution, or otherwise, drug development even where there is precedent is a challenging road and should not be taken for granted. Additional trials (e.g., The incretins as a class also look interesting across a number of follow-on indications, as evidenced by clinical trials in these areas (below).

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Conversations in Drug Development Trends

FEBRUARY 28, 2023

Though cancer ran in my family, we definitely weren’t expecting this news, and the survival rates left little to be hopeful for,” says Rob. Over the next few decades, that drug made its way through clinical trials, securing approval in 2007—just 36 months before Rob was diagnosed in 2010. To say it was devastating is an understatement.

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Drug Target Review

MARCH 3, 2025

“We believe this will definitely make its way into preclinical research and practice.” This approach has the potential to revolutionise clinical trial design and lead to more effective, personalised treatments. “Then, the organoid model would be tested with, say, five, six, or seven drugs available on the market.

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Conversations in Drug Development Trends

FEBRUARY 21, 2024

To proactively manage subject expectations, we provide participants with the following: Dry restrooms No definitive stopping date Dietary requirements 24/7 collection of urine & fecal matter Blood sample collection 2. This process involves a careful assessment of medical history and daily bowel habits.

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Cytel

APRIL 25, 2023

This program includes a definition of maximum fair price, based on key elements including comparative effectiveness data and information about a drug’s impact on specific Medicare populations.

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LifeSciVC

JULY 8, 2025

This essay takes up those questions, not to provide definitive answers, but to explore the contours of a new leadership landscape. Not because they’re definitive, but because they feel aligned with the realities biotech leaders now face. It’s no longer just about accessing lower-cost manufacturing or early-phase trial speed.

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DrugBaron

JANUARY 31, 2024

Consider target selection, which is probably the most critical decision for any drug R&D project (after all, if the target is wrong, then everything that follows will by definition be wasted). Can AI help make better decisions here?

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Alta Sciences

JUNE 27, 2023

Cancer Treatments, Clinical Trials, and the Motivation to Innovate nbartlett Tue, 06/27/2023 - 20:58 Getting to the Heart of Science with Michelle Newby At the heart of science are stories—stories of courage, challenges, successes, lessons, and people. A: I went with the clinical trial. A: I wouldn’t be here without clinical trials.

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LifeSciVC

AUGUST 21, 2024

Clinical Trials Are your clinical trials on track? What has the impact of summer been on recruiting efforts and the ability to manage clinical trial participants? Have you identified and reached out to the PIs and potential clinical trial sites? Or maybe you are nearing the clinic. Do you have ample drug supply?

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Agency IQ

APRIL 12, 2024

Landscape analysis: A look at the regulation of decentralized trials and what’s coming soon In the past few years, regulators and researchers have shown increased interest in leveraging decentralized trial methods – especially following the pandemic.

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FDA Law Blog: Biosimilars

AUGUST 4, 2023

DASCA amended the CSA by revising and adding specified substances to the definition of “anabolic steroid,” providing for the temporary and permanent scheduling of anabolic steroids and adding labeling requirements for products containing anabolic steroids. Implementation of the Designer Anabolic Steroid Control Act of 2014 , 88 Fed.

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The Pharma Data

NOVEMBER 9, 2020

Trial Also Met the Primary Endpoint in Patients With Low Levels of Eosinophils. In the subgroup of patients with baseline eosinophil counts less than 300 cells per microliter, the trial met the primary endpoint with tezepelumab demonstrating a statistically significant and clinically meaningful reduction in AAER.

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Drug Target Review

JANUARY 12, 2024

Deep phenotyping and AI could assist in the production of patterns which describe a set of patients – in effect, creating a new disease definition. Much of the time that is not the same as what is measured in a clinical trial for an outcome. Surrogate endpoints can mimic endpoints used in clinical trials.

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The Pharma Data

OCTOBER 27, 2021

Kymriah was preliminarily granted orphan medicinal product designation by the European Commission (EC) forFL.However, Kymriah would have the occasion to present an important treatment option for those cases with r/ r FL in need of potentially definitive issues, If approved in this implicit third suggestion.

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FDA Law Blog: Drug Discovery

DECEMBER 6, 2024

Here, the draft guidance states that the appropriate timing for an INTERACT meeting should be when a sponsor has identified a specific product and has conducted some preliminary proof-of-concept (POC) studies but has not yet designed and conducted definitive toxicology studies.

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