Clinical Development, Production and Trials

Regulator and Funder? FDA’s Orphan Products Grants Program awards significant funding to help move promising treatments through clinical development

FDA Law Blog: Drug Discovery

NOVEMBER 10, 2024

Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. Each year, FDA selects a limited number of clinical trials to fund to help sponsors pursue development of medical products for rare diseases and advance their field.

Clinical Development

Clinical Development Production Clinical Trials Treatment

Coordinating the Cell Journey: Essentials in Early Clinical Development

PPD

MARCH 13, 2024

In every early phase clinical trial, the transportation, chain of custody, environmental monitoring and subsequent analysis of biological samples are the cornerstones of a successful, robust and quality outcome. This significance and focus are ever more apparent when the starting cellular material is imperative to drug product success.

Clinical Development

Clinical Development Clinical Trials Therapies Trials

Deconstructing the Diligence Process: An Approach to Vetting New Product Theses

LifeSciVC

APRIL 24, 2024

Additionally, for illustrative reasons this is geared towards a single target / product focus vs. broader platform diligence, though many of these mental models will apply for selecting targets and indications for a platform. In order to start building a case for or against a target, I like to start with genetics – first human and then mouse.

Production

Production Small Molecule Regulations Trials

Hybrid FSP/FSO Solutions Enable Flexibility and Agility to Keep Biotech Trials On Time and On Budget

PPD

OCTOBER 8, 2024

The highly dynamic biotech industry has a core need to remain as flexible and agile as possible across the spectrum of clinical development activities. One primary way these companies create a nimble and adaptive environment is by outsourcing some portion of clinical development functions.

Trials

Trials Clinical Trials Clinical Development Clinical Research

The Growing Role of CROs in Clinical Trials

PPD

JULY 6, 2023

It’s estimated that nearly three out of every four clinical trials are conducted by contract research organizations (CROs), highlighting just how much sponsors value — and rely on — the work that CROs perform. Surveys show that CROs improve trial efficiency and increase productivity.

Clinical Trials

Clinical Trials Trials Drug Development Testimonials

How FSP Models Help Biotech Companies Augment Their Clinical Development Needs

PPD

JANUARY 2, 2025

Its ability to maneuver persistent drug development challenges, like patient recruitment, trial complexity and rising costs, will ultimately determine its success. This blog explores the value of functional service provider (FSP) models and how they help biotech companies augment their clinical development needs.

Quantitative Strategies for Rare Disease Clinical Trials

Cytel

MARCH 11, 2024

In 2023, rare diseases accounted for 30% of product pipeline under development, about half of which comprising non-oncology rare diseases. Clinical development in rare diseases has specific challenges. Effective clinical development strategy for rare diseases requires agility to adapt to accumulating learning.

Clinical Trials

Clinical Trials Trials Disease Clinical Development

How to Run DMC? It’s Tricky – FDA’s New Draft Guidance Provides Updated Recommendations on How to Best Use Data Monitoring Committees in Clinical Trials

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.

Clinical Trials

Clinical Trials Trials FDA Regulations

How to Create and Optimize a Clinical Development Plan

Cytel

AUGUST 28, 2023

A clinical development plan — a comprehensive strategy for developing an investigational product through regulatory submission — is a critical component of drug development and helps ensure that new therapies are safe, effective, and of high quality.

Clinical Development

Clinical Development Drug Development Therapies Production

Navigating CAPA in Clinical Development: Ensuring Compliance and Quality Assurance

Advarra

OCTOBER 4, 2023

When it comes to clinical development, precision, compliance, and quality assurance are paramount. For clinical development organizations, an effective CAPA serves as an essential compass, directing a path towards continuous improvement while maintaining steadfast regulatory compliance.

Clinical Development

Clinical Development Compliance Clinical Trials Trials

AlgoTherapeutix Receives Regulatory Approval to Initiate Clinical Development of ATX01

The Pharma Data

DECEMBER 14, 2020

The trial, which will explore the pharmacokinetics and safety of ATX01 in healthy volunteers, is due to start in January 2021. AlgoTherapeutix recently raised a 12M€ Series A that will fund the Phase 1 and 2 clinical development of ATX01. More information at www.algotx.com. View source version on businesswire.com: [link].

Clinical Development

Clinical Development Clinical Supply Pharmacokinetics Clinical Trials

The Evolution of Adaptive Protocols: Early Clinical Development

PPD

NOVEMBER 9, 2023

Our dedicated, global and highly experienced cross-functional early development team is ready to support you through this journey. The partner you select for your early phase studies is critical in enabling you to achieve your overall development timeline efficiently and within budget.

Clinical Development

Clinical Development Drug Development Clinical Research Treatment

Novavax Announces COVID-19 Vaccine Clinical Development Progress

The Pharma Data

NOVEMBER 30, 2020

Two of the three planned late-stage efficacy trials for NVX-CoV2373 sponsored by Novavax are fully enrolled, and more than 20,000 participants have been dosed to-date. In alignment with Novavax’ commitment to transparency, Phase 3 clinical trial protocols are posted to the company’s website at Novavax.com/resources upon finalization.

Clinical Development

Clinical Development Vaccine Clinical Trials Immune Response

Scott Gaines on the Power of Simulation-Guided Design to Handle Increasingly Complex Clinical Trials

Cytel

MAY 5, 2023

As clinical trials become more complex, simulation-guided design approaches are crucial.

Clinical Trials

Clinical Trials Trials Clinical Development Production

Clinical Trials Nexus 2023 Reflections: The Clinical Trial Industry and Vendors

Conversations in Drug Development Trends

DECEMBER 5, 2023

Author: Lona Sheeran, SVP, Clinical Operations, Early Phase At this year’s Clinical Trials Nexus, I had the privilege of representing Worldwide Clinical Trials as the sole CRO on a panel discussion: “Reversing the Conversation: What the Clinical Trial Industry Really Wants from its Service Providers.”

Clinical Trials

Clinical Trials Trials Clinical Development Drug Development

Commercial and Open-Source Software Synergy for Clinical Trial Design

Cytel

APRIL 5, 2024

Written by Sydney Ringold, Customer Success Manager, and Kevin Trimm, Chief Product Officer In an ever-changing clinical development environment, sponsors face many challenges when designing clinical trials. Both options can be utilized to effectively design a trial, but which approach is best?

Clinical Trials

Clinical Trials Trials Clinical Development Regulations

Bayer extends clinical development program for finerenone with Phase III study in patients with non-diabetic chronic kidney disease

The Pharma Data

SEPTEMBER 29, 2021

Heerspink, Professor of Clinical Trials and Personalized Medicine and a clinical pharmacologist/trialist at the Department of Clinical Pharmacy and Pharmacology at the University center Groningen, Netherlands, and Co-Chair of the study’s Executive Committee. In the U.S., 1 in 3 adults is at risk for the disease.

Clinical Development

Clinical Development Disease Clinical Trials Trials

Accelerated Approval and Confirmatory Trials: Timing is Everything

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.

Trials

Trials FDA Clinical Trials Disease

The science of ageing and restoring healthspan

Drug Target Review

JANUARY 8, 2024

We are at the forefront of drug development in an area of research called cellular rejuvenation, which is an approach that has the potential to address many diseases of ageing by restoring aged and injured cells to a more youthful and resilient state. About the author Jerry McLaughlin CEO, Life Biosciences, Inc.

Science

Science Disease FDA Approval Clinical Trials

Analytical Testing Considerations for Gene Therapy Products

The Premier Consulting Blog

MARCH 7, 2024

The analytical package, consisting of release, stability, and characterization tests, includes data generated throughout the product development and manufacturing process. For gene therapy vectors, this testing encompasses production cell lines, master and working cell banks (MCB, WCB), and virus banks.

Production

Production Therapies DNA Virus

Sandoz confirms late-stage clinical development plans for proposed biosimilar aflibercept, a key ophthalmology medicine

The Pharma Data

MAY 8, 2021

Sandoz, a Novartis division, today announced progress in the late-stage clinical development program for its proposed biosimilar aflibercept. Sandoz will begin enrolling the first patient in MYLIGHT, a clinical Phase III confirmatory efficacy and safety study, shortly 1. You should not place undue reliance on these statements.

FSP Engagements Continue to Gain Popularity, Drive Success

PPD

MARCH 26, 2024

A large majority of drug developers utilize FSP or hybrid FSP/FSO models, half are “heavy users” Heavy users: Those that use FSP or hybrid FSP/FSO models for most of their clinical development outsourcing. Heavy users leverage these models for nearly three-quarters of their outsourcing.

Clinical Research

Clinical Research Pharmaceutical Companies Clinical Development Drug Development

How to Save Time and Limit Costs toward First-in-Human Clinical Trials

Cytel

JANUARY 22, 2024

Regulatory guidelines outline all crucial studies and documentation that should be in place before a drug product can be tested in humans for the first time. Here, I share ways to save time and limit costs on your way to clinical trials.

Clinical Trials

Clinical Trials Trials Production Drugs

The Expansion of Cell Therapies in Asia-Pacific

PPD

OCTOBER 17, 2024

There has also been an increase in government and regulatory support for CGT trials, in conjunction with an increase in investments for these products to get to market. There is a race in the field to overcome these challenges to enable the success of CGT products in Asia-Pacific.

Therapies

Therapies Clinical Trials Clinical Research Trials

How Many Clinical Trials Are Run by CROs?

Vial

FEBRUARY 29, 2024

Contract research organizations (CROs) are an integral partner of the drug development process, as they play a pivotal role supporting clinical trial conduct for pharmaceutical, biotechnology, and medical device sponsor companies. That is, how many clinical trials are actually managed by these organizations?

Clinical Trials

Clinical Trials Trials Clinical Research Drug Development

Defining Probability of Success in Clinical Trial Design with Commercial Software and R Coding

Cytel

APRIL 22, 2024

Adler, Director, Global Product Engagement, and J. 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. Written by Boaz N.

Clinical Trials

Clinical Trials Trials Production Clinical Development

Macrophage cell therapy: a new hope for chronic liver disease patients

Drug Target Review

AUGUST 28, 2024

The preclinical findings, together with the early evidence of tolerability and efficacy coming from the MATCH Phase I study, laid the foundations for our characterisation and engineering platform, used to develop Resolution Therapeutics’ lead product, which will be tested in patients starting later this year.

Therapies

Therapies Disease Engineering Medical Schools

The Competitive Edge of Biosimilars

DrugBank

SEPTEMBER 26, 2024

Due to the less extensive clinical trial requirements and the competitive nature of the biosimilar market, these biologics can be produced and marketed at significantly lower prices than their reference counterparts. The primary advantage is cost-effectiveness.

EMA finalizes clinical anticancer therapeutic guidance update

Agency IQ

FEBRUARY 2, 2024

In a newly finalized revision, the EMA’s guideline covering all aspects of clinical development of anticancer therapeutics has expanded its already broad scope. The revisions flesh out considerations for use of biomarkers at all stages of product development and add new content on rare cancers and platform trial designs.

Clinical Development

Clinical Development Trials Production

6 Principles of Our Rare Disease Methodology That Drive Success for Our Sponsors

Conversations in Drug Development Trends

MAY 14, 2024

As the strategists for Worldwide Clinical Trials’ rare disease sponsors, we design customized solutions that make a difference in achieving successful outcomes. This philosophy is encapsulated in our belief: “If you’ve seen one trial for a rare indication, you’ve seen one trial.”

Disease

Disease Clinical Trials Trials Pharmaceutical Companies

4 Unique Challenges of Oncology Trials

Advarra

JUNE 20, 2023

Additionally, in 2021, a literature review estimated the average capitalized research and development (R&D) costs per new cancer medicine at between $944 million and $4.54 Typical clinical development timelines for anticancer drugs average an estimated 6.7

Trials

Trials Clinical Trials FDA Approval Therapies

Supercharging Quantitative Decision-Making with Simulation-Guided Trial Design

Cytel

FEBRUARY 9, 2023

Those familiar with simulation-guided design (SGD) know that it can be used for a wealth of clinical trial options: endpoint selection, number and timing of interim analyses, hitting recruitment targets, managing risk, and building in trial efficiencies.

Trials

Trials Clinical Trials Production Clinical Development

Sentiment & Themes Emerging From JPM 2024

LifeSciVC

JANUARY 16, 2024

Rise of the best-in-class biologics plays: Are you looking to in-license or invest in “de-risked” biology that is already in the clinic? The past year has seen an explosion in “fast follower” approaches to improve on existing target product profiles for select mechanisms, and was exceptionally apparent for antibodies and other biologics.

Small Molecule

Small Molecule Trials Therapies Disease

China National Medical Products Administration Approves Tislelizumab in Combination with Chemotherapy in First-Line Advanced Squamous Non-Small Cell Lung Cancer

The Pharma Data

JANUARY 13, 2021

This is our sixth global approval for an internally-developed product, and our first approval for tislelizumab in a lung cancer indication, an area where we believe tislelizumab can have a large impact for patients.”. Chief Medical Officer, Immuno-Oncology at BeiGene. Chief Medical Officer, Immuno-Oncology at BeiGene.

Production

Production Trials Clinical Trials Treatment

Knopp Biosciences Announces Positive Top-Line Results from the Phase 2 EXHALE Trial of Oral Dexpramipexole in Moderate-to-Severe Eosinophilic Asthma

The Pharma Data

JANUARY 13, 2021

PITTSBURGH–( BUSINESS WIRE )– Knopp Biosciences LLC today announced positive top-line results in a Phase 2 dose-ranging trial of the novel oral drug dexpramipexole in patients with moderate-to-severe eosinophilic asthma. The trial was conducted at 28 U.S. 14, 2021 11:00 UTC. study centers. ABOUT THE EXHALE STUDY.

Trials

Trials Small Molecule Clinical Trials Treatment

How Worldwide and Every Cure Are Using AI To Unlock New Rare Disease Treatments

Conversations in Drug Development Trends

NOVEMBER 21, 2023

As a provider of end-to-end clinical development solutions, we plan to leverage our expertise to refine drug-disease matches identified by Every Cure’s use of AI and data analysis. At Worldwide, we deliver full-service clinical development to support drug approval and commercialization.

Disease

Disease Treatment Clinical Trials Clinical Development

Industry Voices: Yannis Jemiai on Simulation-Guided Design and the Changing Landscape of Clinical Trial Strategy

Cytel

MARCH 13, 2023

And despite initial hesitancy in the clinical trials space, simulation-guided design has revolutionized, and will continue to transform, the landscape of clinical trial development. Many industries have long since adopted the practice of modeling and simulating experimental scenarios.

Clinical Trials

Clinical Trials Trials Drug Development Production

BioNTech announces first patient dosed in Phase 2 clinical trial of mRNA-based BNT111 in patients with advanced melanoma

The Pharma Data

JUNE 26, 2021

BioNTech SE (NASDAQ: BNTX, “BioNTech” or “the Company”), announced that the first patient has been treated in its BNT111 Phase 2 cancer vaccine trial (2020-002195-12; NCT04526899). The trial is enrolling a total of 120 patients and will evaluate the effects of the combination as well as single agents alone.

Clinical Trials

Clinical Trials Trials Immune Response Vaccine

Why Proactive AME Studies are Critical to Accelerating Your Approval Journey

Conversations in Drug Development Trends

FEBRUARY 21, 2024

During clinical development, new chemical entities (NCEs) require an absorption, metabolism, and excretion (AME) study. The post Why Proactive AME Studies are Critical to Accelerating Your Approval Journey appeared first on Worldwide Clinical Trials.

Clinical Trials

Clinical Trials Pharmacy Clinical Pharmacology FDA

Johnson & Johnson Posts Interim Results from Phase 1/2a Clinical Trial of its Janssen COVID-19 Vaccine Candidate

The Pharma Data

SEPTEMBER 25, 2020

Johnson & Johnson Posts Interim Results from Phase 1/2a Clinical Trial of its Janssen COVID-19 Vaccine Candidate. Johnson & Johnson Posts Interim Results from Phase 1/2a Clinical Trial of its Janssen COVID-19 Vaccine Candidate. Immune responses were similar across the age groups studied, including older adults.

Clinical Trials

Clinical Trials Vaccine Trials Immune Response

semaglutide 2.4 mg achieved and maintained a significant amount of weight loss in a 68-week trial

The Pharma Data

MARCH 23, 2021

Data presented at ENDO 2021 demonstrate clinically relevant weight loss, without weight regain, in people treated with semaglutide 2.4 Bagsværd, Denmark, 23 March 2021 – New results from the STEP phase 3a clinical trial programme demonstrated weight loss with investigational treatment of once-weekly subcutaneous semaglutide 2.4

Trials

Trials Clinical Trials Treatment Disease

MindMed Adds Chief Development Officer with FDA Phase 2 Psilocybin Clinical Trial Experience

The Pharma Data

JANUARY 13, 2021

.” MindMed Chief Development Officer Rob Barrow said, “Psychedelic therapies offer a once in a lifetime opportunity to revolutionize the delivery of mental healthcare.

Clinical Trials

Clinical Trials FDA Trials Production

Sample Size Re-Estimation for Rare Disease Clinical Trials

Cytel

FEBRUARY 26, 2024

Adler, MPA, Director, Global Product Engagement, and Valeria Mazzanti, MPH, Associate Director, Customer Success In our previous post , we provided an overview of some of the challenges in studying rare disease therapies, as well as some of the statistical methods and design types that can be deployed to mitigate these challenges.

Clinical Trials

Clinical Trials Disease Trials Therapies

Keeping tabs on Covid-19: Cell and gene clinical trials advance despite Covid-19…

The Pharma Data

AUGUST 7, 2020

The governing body claims this proposal could have positive effects on mitigating burdensome requirements for gene therapy clinical trials in the future. In this light, Pharma IQ’s weekly round-up focuses on advancing therapies and clinical trials to combat Covid-19.

Clinical Trials

Clinical Trials Trials Packaging Therapies

Regulator and Funder? FDA’s Orphan Products Grants Program awards significant funding to help move promising treatments through clinical development

Coordinating the Cell Journey: Essentials in Early Clinical Development

Trending Sources

Deconstructing the Diligence Process: An Approach to Vetting New Product Theses

Hybrid FSP/FSO Solutions Enable Flexibility and Agility to Keep Biotech Trials On Time and On Budget

The Growing Role of CROs in Clinical Trials

How FSP Models Help Biotech Companies Augment Their Clinical Development Needs

Quantitative Strategies for Rare Disease Clinical Trials

How to Run DMC? It’s Tricky – FDA’s New Draft Guidance Provides Updated Recommendations on How to Best Use Data Monitoring Committees in Clinical Trials

How to Create and Optimize a Clinical Development Plan

Navigating CAPA in Clinical Development: Ensuring Compliance and Quality Assurance

AlgoTherapeutix Receives Regulatory Approval to Initiate Clinical Development of ATX01

The Evolution of Adaptive Protocols: Early Clinical Development

Novavax Announces COVID-19 Vaccine Clinical Development Progress

Scott Gaines on the Power of Simulation-Guided Design to Handle Increasingly Complex Clinical Trials

Clinical Trials Nexus 2023 Reflections: The Clinical Trial Industry and Vendors

Commercial and Open-Source Software Synergy for Clinical Trial Design

Bayer extends clinical development program for finerenone with Phase III study in patients with non-diabetic chronic kidney disease

Accelerated Approval and Confirmatory Trials: Timing is Everything

The science of ageing and restoring healthspan

Analytical Testing Considerations for Gene Therapy Products

Sandoz confirms late-stage clinical development plans for proposed biosimilar aflibercept, a key ophthalmology medicine

FSP Engagements Continue to Gain Popularity, Drive Success

How to Save Time and Limit Costs toward First-in-Human Clinical Trials

The Expansion of Cell Therapies in Asia-Pacific

How Many Clinical Trials Are Run by CROs?

Defining Probability of Success in Clinical Trial Design with Commercial Software and R Coding

Macrophage cell therapy: a new hope for chronic liver disease patients

The Competitive Edge of Biosimilars

EMA finalizes clinical anticancer therapeutic guidance update

6 Principles of Our Rare Disease Methodology That Drive Success for Our Sponsors

4 Unique Challenges of Oncology Trials

Supercharging Quantitative Decision-Making with Simulation-Guided Trial Design

Sentiment & Themes Emerging From JPM 2024

China National Medical Products Administration Approves Tislelizumab in Combination with Chemotherapy in First-Line Advanced Squamous Non-Small Cell Lung Cancer

Knopp Biosciences Announces Positive Top-Line Results from the Phase 2 EXHALE Trial of Oral Dexpramipexole in Moderate-to-Severe Eosinophilic Asthma

How Worldwide and Every Cure Are Using AI To Unlock New Rare Disease Treatments

Industry Voices: Yannis Jemiai on Simulation-Guided Design and the Changing Landscape of Clinical Trial Strategy

BioNTech announces first patient dosed in Phase 2 clinical trial of mRNA-based BNT111 in patients with advanced melanoma

Why Proactive AME Studies are Critical to Accelerating Your Approval Journey

Johnson & Johnson Posts Interim Results from Phase 1/2a Clinical Trial of its Janssen COVID-19 Vaccine Candidate

semaglutide 2.4 mg achieved and maintained a significant amount of weight loss in a 68-week trial

MindMed Adds Chief Development Officer with FDA Phase 2 Psilocybin Clinical Trial Experience

Sample Size Re-Estimation for Rare Disease Clinical Trials

Keeping tabs on Covid-19: Cell and gene clinical trials advance despite Covid-19…

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