Drug Development, Regulations and Trials

How AI will reshape pharma by 2025

Drug Target Review

DECEMBER 20, 2024

Artificial intelligence (AI) has revolutionised many industries, yet its adoption in pharmaceutical drug development has been notably slower. For years, AI and machine learning (ML) were often dismissed as little more than advanced statistics with little practical value in drug development.

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Understanding the Regulatory Environment in Japan for Generic Drug Development

Drug Patent Watch

DECEMBER 30, 2024

The regulatory environment in Japan for generic drug development is complex and has undergone significant changes in recent years. Types of Drug Applications The PMDA accepts three main types of drug applications: Investigational New Drug (IND) : Required for conducting clinical trials in Japan.

Drug Development

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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.

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Bridging Innovation & Patient Care: The Growing Role of AI

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When is a Confirmatory Trial “Underway” or Conducted with “Due Diligence” Enough for Accelerated Approval? FDA Explains Its New Authorities

FDA Law Blog: Drug Discovery

JANUARY 30, 2025

However, as we note in that post, the design, timing of initiation, and timely conduct of confirmatory trials are also important considerations in FDAs determination of whether accelerated approval is appropriate. This blog post focuses on interpreting these new authorities with respect to timely conduct of confirmatory trials.

FDA

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

PPD

MARCH 11, 2025

Developing treatments for individuals living with rare diseases is critical, but orphan drug development is laden with unique obstacles that necessitate innovative, multifaceted approaches. Moreover, in some rare disease trials, it is unethical to design a control group of patients with a placebo.

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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.

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The Real Cost of Complexity: What Every Drug Developer Needs to Know

PPD

DECEMBER 16, 2024

Our annual look at the state of the drug development industry highlights a dual set of challenges complicating progress. Pressure and requirements to engage diverse patient populations in trials have become more challenging and expensive, requiring tailored strategies that can stretch both resources and budgets.

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Regulatory Guidance for Oligonucleotide Bioanalysis in Drug Development

Alta Sciences

FEBRUARY 19, 2025

Regulatory Guidance for Oligonucleotide Bioanalysis in Drug Development pmjackson Wed, 02/19/2025 - 21:30 The unique physicochemical properties of oligonucleotides require the use of specialized bioanalytical approaches, with key considerations including selectivity and specificity, sensitivity, stability, and matrix effects.

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Including Patients with Borderline Personality Disorder in Clinical Trials

Conversations in Drug Development Trends

MAY 10, 2024

Over the past two decades, industry-sponsored clinical trials have targeted treatments for BPD, yielding some promising outcomes; however, broader psychiatric research often excludes BPD patients, a trend that extends to the emerging field of psychedelic studies.

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Why Including Patients in FDA Engagement Will Benefit Your Trial

Conversations in Drug Development Trends

AUGUST 23, 2023

Patients are the backbone of clinical trials, playing an essential role in the drug development process. This engagement is often less understood and is underutilized by sponsors, meaning a significant element of the trial and drug experience is missed during sponsor engagement with the FDA.

FDA

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Patient-Centric Strategies for Successful Oncology Trials

PPD

AUGUST 28, 2024

For pharmaceutical innovators and drug developers working to bring oncology therapies to market, patients are the “why” behind it all. For many patients, involvement in oncology clinical trials represents a last hope for an effective therapy. Many of these patients’ conditions are disabling.

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Establishing Effective Adaptive Trial Designs for Oncology in Multi-Regional Studies

Conversations in Drug Development Trends

AUGUST 9, 2024

Authors: Matt Cooper, PhD, Executive Director, Therapeutic Strategy Lead, Oncology; Megan Morrison, Vice President, Asia Pacific Strategy Lead Adaptive trial designs have become essential in oncology, offering a flexible and efficient approach for conducting clinical trials.

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Navigating the challenges and opportunities of AI in drug development and personalised medicine

Drug Target Review

JULY 18, 2023

Artificial Intelligence (AI) is poised to transform the field of target discovery in drug development, offering immense potential to enhance efficacy, personalised medicine, and accelerate the development of innovative compounds.

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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

Tobolowsky & Véronique Li, Senior Medical Device Regulation Expert & David B. Moreover, DMCs are being used in trials of modest size and in the context of increased globalization of medical product development.

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World Orphan Drug Congresses Europe vs. The US: Global Perspectives on Genetic Testing

Conversations in Drug Development Trends

DECEMBER 19, 2024

At the recent World Orphan Drug Congresses (WODC) in Europe and the United States, Worldwide Clinical Trials Derek Ansel , Vice President, Therapeutic Strategy Lead, Rare Disease, facilitated roundtable discussions to explore the operational challenges and ethical barriers surrounding genetic testing.

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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. Common challenges for certain patient groups include long-distance travel to clinical sites, time away from work and limited access to knowledge about CGT trials.

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Antibody Drug Conjugates: windows of opportunity

Drug Target Review

JULY 4, 2023

1 Regulators invest significant consideration balancing quality-of-life measures with overall survival when assessing novel oncology treatments. 2 However, when dosed at the MTD, ADCs display improved efficacy over small molecules in oncology trials. 3D rendering of Antibody Drug Conjugate Molecules.

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Shaping drug development success: how does artwork play a role?

Drug Target Review

AUGUST 7, 2024

The pharmaceutical industry is undergoing a major shift towards the development of breakthrough medicines and advanced therapies. This continued innovation highlights the complexity of the drug development process, particularly as the field is highly regulated by health authorities around the world.

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Logistics in Focus: Orchestrating the Challenges of Cell Therapy

Conversations in Drug Development Trends

NOVEMBER 12, 2024

Despite their exciting potential, the smooth operation of cell therapy development trials requires extraordinary orchestration, perfectly aligning the product and patient journeys. While clinical supply is essential to any successful trial, autologous cell therapy trials occupy the far end of the spectrum regarding risk tolerance.

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How FSP Solutions Support Risk-Based Methodologies and Tech Innovation in Clinical Operations

PPD

FEBRUARY 10, 2025

Sponsors find that an FSP solution is often the best choice to help advance their drug development projects, whether they need to fill small gaps in services or support large-scale programs with dedicated teams across functions. EMRs give clinical operations teams direct, remote access to an array of patient health information.

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Strategies for Successful Navigation of Complex Radiopharmaceutical Trials

Conversations in Drug Development Trends

APRIL 3, 2024

As with other targeted therapies, radiopharmaceutical development requires unique and specialized strategies to ensure successful execution. Are you aware of the challenges you must address for a successful radiopharmaceutical trial? These include ensuring informed consent, particularly around the risks and benefits of participation.

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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. Maintaining open lines of communication helps address issues as they arise, allowing for real-time clarification and adjustments.

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4 Essential Topics to Cover in Your First CRO Meeting

Alta Sciences

NOVEMBER 15, 2024

4 Essential Topics to Cover in Your First CRO Meeting pmjackson Fri, 11/15/2024 - 19:22 Starting a partnership with a contract research organization (CRO) is a sometimes intimidating—yet exciting—first step in your drug development journey. Let the CRO know any key outcomes from each.

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Advancements in hit identification for membrane protein drug discovery

Drug Target Review

APRIL 7, 2025

Involved in various physiological processes, such as vision, taste, smell, immune response and neurotransmission, GPCRs are activated by various molecules including hormones, neurotransmitters and environmental stimuli, which trigger a cascade of cellular events that help regulate bodily functions. References Sun D, et al. doi:10.1016/j.apsb.2022.02.002.

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Biotech leader champions targeted cancer treatments and diversity

Drug Target Review

NOVEMBER 25, 2024

I went on to complete my MBA and PhD at The Institute of Cancer Research (ICR) in drug development. We are developing a next-generation, highly selective inhibitor of PI3Kd, which is very exciting. Initially, we are developing roginolisib, a drug that inhibits PI3Kd signalling to treat uveal melanoma.

Treatment

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Navigating the Complex Regulatory CRO Landscape for Oncology Trials in the European Union

Conversations in Drug Development Trends

MAY 23, 2024

By: Sarah Bly, Regulatory Science and Innovation and Matt Cooper, Executive Director, Therapeutic Strategy Lead, Oncology The European Union (EU) presents a unique set of regulatory challenges and opportunities for clinical trials in oncology. Increased Transparency : Ensures that information on clinical trials is more accessible.

Trials

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Staying Connected and Compliant: Reimagining Clinical Trials with Longboat

Advarra

AUGUST 21, 2024

Nowhere is this more painful than in clinical trials, the backbone of drug development. These complex studies typically generate hundreds or thousands of documents to share across clinical trial sites, pharmaceutical sponsors, physicians, patients, and regulators. A better trial experience for everyone.

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Obesity care gets personalised: tailoring therapies with Phenomix

Drug Target Review

MARCH 4, 2025

The MyPhenome test has already gained traction among obesity specialists, particularly in Chicago and Florida, and Phenomix has formed strategic partnerships with multiple pharmaceutical companies to support clinical trials and further analyse obesity-related data. Obesity is rising worldwide, driven by lifestyle, diet, and genetics.

Therapies

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Next Generation Sequencing (NGS) in Clinical Trials: Challenges and Opportunities

Vial

DECEMBER 5, 2023

It has become a fundamental tool for researchers to explore the complexities of genetic information and conduct genetic-informed drug development. Sponsors have used NGS to screen patients for clinical trial eligibility and patient stratification , expanded CDx development, and comprehensive genomic profiling (CGP).

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Looking beyond traditional oncogenic pathways to break cancer resistance

Drug Target Review

SEPTEMBER 25, 2024

Classically, rational drug combinations are designed to target two nodes in the same oncogenic signalling pathway. BRAF and downstream mitogen-activated extracellular signal-regulated kinase (MEK) inhibitors are combined to treat BRAF-mutated melanoma, for example.

Small Molecule

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Revolutionizing Clinical Trial Data Management with AI-Powered Collaboration

Perficient: Drug Development

SEPTEMBER 25, 2024

Clinical trial data management is critical to pharmaceutical research, yet it remains a significant challenge for many organizations. Outdated systems: Many organizations rely on legacy data management tools that fail to meet the demands of modern clinical trials.

Clinical Trials

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Improving Oncology Clinical Trials for Accelerated Approval

H1 Blog

JULY 19, 2023

Streamlining recruitment and reporting to support advanced oncology drug development In March of 2023, the U.S. In order to qualify for accelerated approval, drug manufacturers must demonstrate that the medication provides meaningful clinical benefit to patients with life-threatening illnesses during their clinical trials.

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FDA Animal Rule: Overview & Impact on Drug Development

thought leadership

JULY 9, 2024

What is the Animal Rule?

FDA

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Advanced 3D cell-based technologies

Drug Target Review

SEPTEMBER 19, 2023

puts an end to the previous mandate that all drugs need to be tested on animals prior to human clinical trials. This major shift to the orthodox tradition of using animal experiments in drug testing dates back the Aristotle’s time and cemented 80 years ago with initial federal mandate of drug safety regulation of 1938.

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PTC Therapeutics Announces Initiation of a Registration-Directed Clinical Trial to Evaluate Vatiquinone in Mitochondrial Epilepsy

The Pharma Data

OCTOBER 25, 2020

NASDAQ: PTCT), today announced the initiation of a registration-directed Phase 2/3 clinical trial to evaluate vatiquinone (PTC743) in patients with mitochondrial epilepsy, the highly morbid condition of refractory seizures in children with inherited mitochondrial disease. . SOUTH PLAINFIELD, N.J. , Peltz , Ph.D.,

Clinical Trials

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8 Frequently Asked Questions About Genetic Testing in Clinical Trials

Conversations in Drug Development Trends

DECEMBER 12, 2022

Genetic testing provides patients with a diagnosis for their illness, helps patients and family members to understand risks of developing new diseases, and can be used to support clinical trial advancement. Can prior genetic testing results be utilized in a trial, or should a new genetic sample be taken before participating?

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Best CDMO Practices for Startups: Navigating the Complex World of Contract Development and Manufacturing

Drug Patent Watch

JANUARY 9, 2025

Navigating the complex landscape of drug development and manufacturing can be a daunting task. Enter the Contract Development and Manufacturing Organization (CDMO) a lifeline for many startups looking to bring their innovative therapies to market. Your timeline will significantly impact your CDMO requirements.

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FDA’s Draft Guidance on Externally Controlled Trials Answers Some Questions, Leaves Others Unanswered

FDA Law Blog: Drug Discovery

APRIL 23, 2023

This Draft Guidance focuses on the use of patient-level data from other clinical trials or from RWD sources. For example, objective response rate is often used as a single-arm trial endpoint in oncology given the established understanding that tumor shrinkage rarely occurs without an intervention.”

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What FDA’s Newest Gene Therapy Approval Tells Us About Durability: How Long is Long Enough?

FDA Law Blog: Drug Discovery

DECEMBER 7, 2022

However, with this most recent approval, FDA did not leave the question of durability as something to be answered postapproval, which signals to us that this issue looms large in FDA’s preapproval regulation of gene therapies. We can see this evolution for requiring longer-term follow-up play out with this most recent approval of Hemgenix.

Therapies

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CDER & CBER Real World Data and Real World Evidence

Pharmaceutical Development Group

DECEMBER 19, 2021

Effective use of Real World Data (RWD) and Real World Evidence (RWE) can reduce costs of Pharmaceutical Drug and Biologic Products and Process Development, Drug Development Services , expedite a FDA Pre-Submission Review, and lead to FDA Approval. Author Information William E. Spanogle, Ph.D.

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Distinguishing the Roles of Preclinical vs. Clinical CROs in Clinical Research

Vial

MAY 20, 2024

Distinguishing the Roles of Preclinical vs. Clinical CROs in Clinical Research Randomized clinical trials are the gold-standard of evidence-based medicine, representing the culmination of the long, arduous drug development pipeline. What is a Pre-Clinical CRO? The global preclinical CRO market was estimated to be valued at US$5.7

Clinical Research

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Analysis Life Sciences Thank You Meet C3TI, FDA’s new clinical trial innovation hub

Agency IQ

APRIL 19, 2024

Meet C3TI, FDA’s new clinical trial innovation hub On April 14, FDA announced that its Center for Drug Evaluation and Research (CDER) is launching a new clinical trial innovation hub, the CDER Center for Clinical Trial Innovation (C3TI). In particular, regulations in both the U.S.

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The Race for Ozempic Alternatives: Unlocking the Future of Weight Loss Medication

DrugBank

MARCH 7, 2024

Moreover, Novo Nordisk and Eli Lilly are expected to unveil new clinical trial data that could highlight additional health benefits of their drugs beyond weight loss and diabetes management, potentially paving the way for broader insurance coverage.

Pharmaceutical Companies

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Optimizing Clinical Data Registries: Opportunities for Diversity and Feasibility

H1 Blog

NOVEMBER 29, 2023

Breaking the Barriers: How Inaccurate Trial Records Are Delaying Drug Development and Limiting Diverse Patient Populations In an October 2023 report , BMC Medicine completed a follow-up on a 10-year effort to understand, report on and track data sharing challenges with big pharma, specifically for clinical trials.

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How AI will reshape pharma by 2025

Understanding the Regulatory Environment in Japan for Generic Drug Development

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

Webinars

When is a Confirmatory Trial “Underway” or Conducted with “Due Diligence” Enough for Accelerated Approval? FDA Explains Its New Authorities

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

The rising impact of biomarkers in early clinical development

The Real Cost of Complexity: What Every Drug Developer Needs to Know

Regulatory Guidance for Oligonucleotide Bioanalysis in Drug Development

Including Patients with Borderline Personality Disorder in Clinical Trials

Why Including Patients in FDA Engagement Will Benefit Your Trial

Patient-Centric Strategies for Successful Oncology Trials

Establishing Effective Adaptive Trial Designs for Oncology in Multi-Regional Studies

Navigating the challenges and opportunities of AI in drug development and personalised medicine

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

World Orphan Drug Congresses Europe vs. The US: Global Perspectives on Genetic Testing

The Expansion of Cell Therapies in Asia-Pacific

Antibody Drug Conjugates: windows of opportunity

Shaping drug development success: how does artwork play a role?

Logistics in Focus: Orchestrating the Challenges of Cell Therapy

How FSP Solutions Support Risk-Based Methodologies and Tech Innovation in Clinical Operations

Strategies for Successful Navigation of Complex Radiopharmaceutical Trials

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

4 Essential Topics to Cover in Your First CRO Meeting

Advancements in hit identification for membrane protein drug discovery

Biotech leader champions targeted cancer treatments and diversity

Navigating the Complex Regulatory CRO Landscape for Oncology Trials in the European Union

Staying Connected and Compliant: Reimagining Clinical Trials with Longboat

Obesity care gets personalised: tailoring therapies with Phenomix

Next Generation Sequencing (NGS) in Clinical Trials: Challenges and Opportunities

Looking beyond traditional oncogenic pathways to break cancer resistance

Revolutionizing Clinical Trial Data Management with AI-Powered Collaboration

Improving Oncology Clinical Trials for Accelerated Approval

FDA Animal Rule: Overview & Impact on Drug Development

Advanced 3D cell-based technologies

PTC Therapeutics Announces Initiation of a Registration-Directed Clinical Trial to Evaluate Vatiquinone in Mitochondrial Epilepsy

8 Frequently Asked Questions About Genetic Testing in Clinical Trials

Best CDMO Practices for Startups: Navigating the Complex World of Contract Development and Manufacturing

FDA’s Draft Guidance on Externally Controlled Trials Answers Some Questions, Leaves Others Unanswered

What FDA’s Newest Gene Therapy Approval Tells Us About Durability: How Long is Long Enough?

CDER & CBER Real World Data and Real World Evidence

Distinguishing the Roles of Preclinical vs. Clinical CROs in Clinical Research

Analysis Life Sciences Thank You Meet C3TI, FDA’s new clinical trial innovation hub

The Race for Ozempic Alternatives: Unlocking the Future of Weight Loss Medication

Optimizing Clinical Data Registries: Opportunities for Diversity and Feasibility

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