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

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.

Clinical Development

Clinical Development Clinical Trials Trials Regulations

The AI model that is changing clinical trial design

Drug Target Review

JULY 1, 2025

Clinical trials are expensive, slow and often limited by outdated design constraints. They offer patient-specific outcome predictions, generated using machine learning models trained on real historical clinical data. Accelerating timelines and de-risking development decisions across the pipeline.”

Clinical Trials

Clinical Trials Trials FDA Disease

The rising impact of biomarkers in early clinical development

Drug Target Review

APRIL 7, 2025

Advanced strategies and tools are being implemented to establish the safety and efficacy of new therapeutic modalities, with the development of new biomarkers becoming one of the most relevant approaches for enhancing the precision and utility of early-stage studies.

Clinical Development

Clinical Development Clinical Trials Clinical Pharmacology Trials

Leveraging AI Solutions for Clinical Trial Efficiencies

PPD

JANUARY 15, 2025

As clinical trials become increasingly complex, particularly in decentralized trials and rare disease studies, sponsors experience increased challenges in site selection, forecasting and resourcing, and patient recruitment and enrollment.

Clinical Trials

Clinical Trials Trials Clinical Research Drug Development

Navigating Clinical Development Challenges to Advance Idiopathic Pulmonary Fibrosis Treatments

PPD

JUNE 20, 2025

The complexity of diagnosing IPF, understanding its root causes, and translating preclinical findings to clinical success make it a challenging disease for drug development. However, recent advancements and strategic approaches in clinical trials offer hope that additional treatments are on the way.

Clinical Development

Clinical Development Treatment Clinical Trials Trials

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

AI in drug discovery: faster, smarter, better

Drug Target Review

APRIL 30, 2025

It involves a dynamic and often unpredictable process where every stage, from target identification to clinical trials, generates vast amounts of data. Additionally, AI-driven predictive modelling can shorten the preclinical phase by simulating biological responses, leading to more targeted and efficient clinical trials.

Drugs

Drugs Therapies Clinical Trials Trials

Why radiopharmaceuticals are gaining ground in the fight against cancer

Drug Target Review

JUNE 6, 2025

“Protocol development can be a complex and meticulous process,” he explains, “as it’s crucial to ensure that trial candidates meet the necessary inclusion criteria.” Prior treatments and patient histories often introduce variables that can affect outcomes, making thoughtful trial design essential for generating reliable data.

Therapies

Therapies Treatment Drug Development Clinical Development

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

First Patient Dosed in Phase 3 REVEAL Trial of ION582 for Angelman Syndrome

The Pharma Data

JUNE 11, 2025

Ionis Begins Pivotal Phase 3 REVEAL Study of ION582 in Angelman Syndrome, Dosing First Patient in Global Trial Ionis Pharmaceuticals , Inc. This trial will build on previous positive data from the earlier Phase 1/2 HALOS study and is designed to rigorously assess the potential of ION582 as a disease-modifying therapy. “We

Trials

Trials Therapies Treatment Disease

CMC Content for Global Clinical Development of Pharmaceuticals

The Premier Consulting Blog

AUGUST 21, 2024

As clinical development of an investigational product proceeds, Sponsors often conduct global clinical trials which require preparation of content to support dossiers in multiple geographies, meeting the regulatory requirements of each region. P) is per the specific IMPD guidance.

Clinical Development

Clinical Development Pharmaceuticals Clinical Trials Trials

Leveraging Remote-Based Regional Hubs to Improve Efficiencies and Meet Timelines

PPD

JUNE 17, 2025

Biopharmaceutical and biotech drug developers have faced increasing challenges in recent years. In the past decade alone, the time needed to complete a clinical trial has increased by 20–30%, and the cost to bring a new drug to market has risen to an average of about $2.6

Clinical Development

Clinical Development International Drug Development Clinical Research

Continuous Innovation with WorldwideEdge™ — A World of a Difference in Bioanalytic Operations

Conversations in Drug Development Trends

JANUARY 21, 2025

Regulatory bodies such as the FDA oversee clinical trials to ensure that studies’ design, conduction, analysis, and reporting are per established guidelines and laws. Any delays or missteps in bioanalysis during a Phase I trial can derail the trajectory of a promising drug.

Clinical Trials

Clinical Trials Laboratories Trials Drug Development

New cell therapy model accelerates cancer treatment development

Drug Target Review

FEBRUARY 10, 2025

In just two years, CTMC has advanced eight therapies into clinical trials, harnessing genetic engineering to enhance T-cell effectiveness in the fight against cancer. Ive been involved in therapeutics development for over 25 years, working with small, medium, and large biotech companies.

Therapies

Therapies Treatment Engineering Clinical Trials

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 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

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

Navigating Varying Regulatory Feedback in Clinical Oncology Trials: A Guide for Clinical Research Professionals

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.

Clinical Research

Clinical Research Trials Clinical Trials Research

From siloed data to breakthroughs: multimodal AI in drug discovery

Drug Target Review

JUNE 11, 2025

Clinical genomics, powered by NGS, enables more precise target validation, improved patient stratification and optimised trial design, ultimately aiming to increase PoS. If it’s not mixable, the data value chain (from R&D phase to production phase) is neither interpretable or reproducible. Highlighting data integration.

Drugs

Drugs Clinical Trials Drug Development Pharmaceuticals

Overcoming Orphan Drug Development Challenges with Real-World Data and Evidence

PPD

MARCH 11, 2025

Real-world data (RWD) and real-world evidence (RWE) have emerged as transformational tools in addressing these hurdles, advancing rare disease drug development. Moreover, in some rare disease trials, it is unethical to design a control group of patients with a placebo.

Drug Development

Drug Development Clinical Trials Disease Trials

Pharmacy Considerations for Early Phase Trials

Fierce BioTech

DECEMBER 23, 2024

As Phase 1 clinical trials include several different design types with multiple objectives (e.g. png Listing Introduction Join this webinar to receive expert guidance and learn key considerations for successfully planning pharmacy activities in early phase trials. Listing Image DrVince_ListingLogo_250x190.png

Pharmacy

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The FDA’s REMS Removal: A Watershed Moment for CAR T Access

Conversations in Drug Development Trends

JULY 3, 2025

The CRO Perspective From a Contract Research Organization (CRO) specializing in the design and execution of studies for innovative therapies, the REMS requirements have significantly complicated the design and execution of CAR T clinical trials.

Therapies

Therapies Clinical Trials Regulations FDA

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

Novel Bispecific therapies in oncology

SugarCone Biotech

MARCH 2, 2025

Less clear is whether we can productively and safely inhibit TGF- activity at all given the toxicity issues associated with TGF- inhibition. We recently viewed quite positive updates from Scholar Rock who have a TGF--1 antagonist in the clinic. No chemotherapy regimen is used in the trial.

Therapies

Therapies Clinical Trials Trials Clinical Development

Actithera draws new investors to radiopharma drug pitch

BioPharma Drive: Drug Pricing

JULY 9, 2025

Deep Dive Library Events Press Releases Topics Sign up Search Sign up Search Pharma Biotech FDA Clinical Trials Deals Drug Pricing Gene Therapy An article from Dive Brief Actithera draws new investors to radiopharma drug pitch The four-year-old biotech raised about $75 million in a Series A round that involved nine venture capital firms.

Drugs

Drugs Clinical Development Therapies Clinical Trials

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

BeOne Medicines Presents Transformative Oncology Portfolio at Research & Development Day

The Pharma Data

JUNE 26, 2025

Its end-to-end R&D model integrates in-house discovery, development, and manufacturing capabilities—enabling rapid iteration, lower development costs, and sustained pipeline productivity. To date, the company has conducted more than 170 clinical trials across 40 countries, enrolling over 25,000 patients.

Research

Research Clinical Development Clinical Trials Engineering

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

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

J&J’s Dual-Target CAR T-Cell Therapy Shows Promise in Large B-Cell Lymphoma

The Pharma Data

JUNE 13, 2025

Director of Lymphoma Research at Sarah Cannon Research Institute and principal study investigator for the trial. Vice President of Early Clinical Development and Translational Research at Johnson & Johnson Innovative Medicine. “As formerly Cellular Biomedicine Group, Inc.).

Therapies

Therapies Treatment Clinical Development Trials

Revolution Medicines Secures $2B Funding from Royalty Pharma for RAS(ON) Cancer Drug Development

The Pharma Data

JUNE 24, 2025

Unlike traditional royalty deals, where investors purchase rights to a portion of revenue from marketed products, this synthetic royalty structure allows Royalty Pharma to provide upfront capital in exchange for a defined share of future revenue, subject to agreed terms and caps.

Drug Development

Drug Development Clinical Trials Therapies Drugs

POLB 001: tackling cytokine storms before they start

Drug Target Review

MAY 23, 2025

Our goals are to progress our pipeline and position each asset for clinical and commercial success. The tight-knit structure of the Poolbeg team allows Tremble to contribute not only to early-stage drug development but also to clinical trial design, patient stratification, and biomarker strategy.

Immune Response

Immune Response Therapies Treatment Clinical 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 Pharma Companies

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.

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.

Roche’s NXT007 Shows Early Promise for Normalising Clotting in Haemophilia A

The Pharma Data

JUNE 22, 2025

The results from the Phase I/II NXTAGE trial, announced during the 2025 International Society on Thrombosis and Haemostasis (ISTH) Congress in Washington, D.C., The trial is evaluating NXT007 in people with hemophilia A without factor VIII inhibitors who have not previously been treated with Hemlibra.

Clinical Trials

Clinical Trials Therapies Treatment Trials

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

How the FSP Model Augments Biotech Sponsors’ Internal Capabilities

PPD

OCTOBER 21, 2024

Meeting the never-ending challenges of drug development in this active environment — including pressure to identify drug prospects earlier and hire more conservatively — frequently leads biotech companies to outsource some portion of clinical development functions. With FSO, all tasks for a clinical trial are outsourced.

International

International Clinical Development Clinical Trials Clinical Research

PRITELIVIR MESYLATE

New Drug Approvals

JULY 8, 2025

Clinical Trials: Pritelivir is currently in phase II clinical trials, with ongoing research into its effectiveness and safety. Pritelivir (development codes AIC316 or BAY 57-1293 ) is a direct-acting antiviral drug in development for the treatment of herpes simplex virus infections (HSV).

Virus

Virus DNA Clinical Trials Treatment

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

How FSP Models Help Biotech Companies Augment Their Clinical Development Needs

Trending Sources

The AI model that is changing clinical trial design

The rising impact of biomarkers in early clinical development

Leveraging AI Solutions for Clinical Trial Efficiencies

Navigating Clinical Development Challenges to Advance Idiopathic Pulmonary Fibrosis Treatments

Coordinating the Cell Journey: Essentials in Early Clinical Development

AI in drug discovery: faster, smarter, better

Why radiopharmaceuticals are gaining ground in the fight against cancer

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

First Patient Dosed in Phase 3 REVEAL Trial of ION582 for Angelman Syndrome

CMC Content for Global Clinical Development of Pharmaceuticals

Leveraging Remote-Based Regional Hubs to Improve Efficiencies and Meet Timelines

Continuous Innovation with WorldwideEdge™ — A World of a Difference in Bioanalytic Operations

New cell therapy model accelerates cancer treatment development

Quantitative Strategies for Rare Disease 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

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

Navigating Varying Regulatory Feedback in Clinical Oncology Trials: A Guide for Clinical Research Professionals

From siloed data to breakthroughs: multimodal AI in drug discovery

Overcoming Orphan Drug Development Challenges with Real-World Data and Evidence

Pharmacy Considerations for Early Phase Trials

The FDA’s REMS Removal: A Watershed Moment for CAR T Access

The Evolution of Adaptive Protocols: Early Clinical Development

Novel Bispecific therapies in oncology

Actithera draws new investors to radiopharma drug pitch

Novavax Announces COVID-19 Vaccine Clinical Development Progress

BeOne Medicines Presents Transformative Oncology Portfolio at Research & Development Day

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

The Expansion of Cell Therapies in Asia-Pacific

J&J’s Dual-Target CAR T-Cell Therapy Shows Promise in Large B-Cell Lymphoma

Revolution Medicines Secures $2B Funding from Royalty Pharma for RAS(ON) Cancer Drug Development

POLB 001: tackling cytokine storms before they start

Accelerated Approval and Confirmatory Trials: Timing is Everything

The Competitive Edge of Biosimilars

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

Roche’s NXT007 Shows Early Promise for Normalising Clotting in Haemophilia A

Commercial and Open-Source Software Synergy for Clinical Trial Design

How the FSP Model Augments Biotech Sponsors’ Internal Capabilities

PRITELIVIR MESYLATE

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