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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Conversations in Drug Development Trends

NOVEMBER 12, 2024

By Amy Raymond, PhD, PMP, Executive Director, Therapeutic Strategy Lead, Rare Disease Cell and gene therapies (CGTs) include cutting-edge approaches that offer the hope of a healthier, happier, and better tomorrow for a wide range of patient populations. Operationalizing these trials requires proactive and flawless management at every stage.

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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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Drug Target Review

APRIL 7, 2025

As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. A surrogate endpoint is a marker used in clinical trials as a substitute for a direct clinical outcome. There are several types of biomarkers to consider.

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Drug Target Review

APRIL 30, 2025

In the rapidly evolving field of cell and gene therapy (CGT), the ability to manage complex biological data, optimise manufacturing processes, and accelerate drug discovery is crucial. How AI is solving CGTs biggest challenges Cell and gene therapy is inherently complex. The result? Faster time-to-market and reduced costs.

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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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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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Drug Target Review

JUNE 9, 2025

A personal journey to Alltrna Michelle Werner’s career has spanned over 20 years in the pharmaceutical industry, where she developed her expertise in oncology drug development at leading companies such as Bristol Myers Squibb, AstraZeneca, and Novartis.

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DrugBank

OCTOBER 17, 2024

Data science has emerged as an innovative tool in the biopharmaceutical industry, leveraging the power of machine learning and artificial intelligence to drive innovation and efficiency across the entire drug development lifecycle. This was seen in the case of the BRAF V600E mutation test for melanoma patients receiving vemurafenib.

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PPD

OCTOBER 17, 2024

Cell and gene therapy (CGT) studies are rapidly gaining momentum in the Asia-Pacific region, fueled by growing patient demand and a thriving ecosystem of innovation. In China, the high incidence of solid tumors is driving an urgent need for advanced therapies, spurring the push for new treatment approaches.

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PPD

SEPTEMBER 25, 2023

As the clinical trial landscape evolves, drug developers are faced with novel challenges and changes in study recruitment, trial size and structure, and more. The adoption of new innovations, strategies and technologies offers opportunities to address persistent challenges and develop suitable approaches for the future.

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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.

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The Pharma Data

JUNE 24, 2025

The partnership represents a major financial and strategic milestone for Revolution Medicines, which has built a robust pipeline of targeted therapies aimed at cancers driven by mutations in the RAS family of genes—some of the most prevalent and historically difficult-to-drug oncogenes in human cancer.

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Drug Target Review

MARCH 4, 2025

Knowing a patients subtype or phenotype can be used to guide obesity treatments of all kinds, including drug therapy, devices, bariatric surgery and even diet and lifestyle interventions, Bagnall explains. Without understanding these differences, treatment becomes costly and a game of trial and error, Bagnall points out.

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SugarCone Biotech

JUNE 19, 2025

They have interesting patterns of expression in different cancer indications; thus, diverse therapies for attacking these targets have been developed. SCLC generally has a “hot” tumor microenvironment (TME) meaning that it is infiltrated by immune cells, including the T cells that are activated by immune checkpoint therapies.

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Drug Target Review

JUNE 11, 2025

Drug development is plagued by complex challenges, but multimodal AI is unlocking new opportunities. By integrating diverse data sources – from genomics to clinical insights – this approach is accelerating drug discovery, improving patient stratification and boosting success rates. Highlighting data integration.

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Drug Target Review

JULY 8, 2025

As Vice President of Oncology Early Development (OED) at AbbVie , she leads the charge on translating bold scientific ideas into real-world impact – and she’s doing it with a focus on some of the most tenacious cancers out there. Checkpoint inhibitors revolutionised cancer therapy; however, significant unmet clinical needs remain.

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DrugBank

JUNE 6, 2024

In the rapidly evolving landscape of oncology drug development, artificial intelligence (AI) has emerged as a revolutionary force, altering not just the pace but the very methodology of discovery and development.

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The Pharma Data

JUNE 27, 2025

The trial met both its primary and key secondary endpoints, signaling a potential breakthrough in a condition with few, if any, effective treatment options. The trial met both its primary and key secondary endpoints, signaling a potential breakthrough in a condition with few, if any, effective treatment options.

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Conversations in Drug Development Trends

OCTOBER 29, 2024

By: Juliane Mills, Senior Director, Therapeutic Strategy Lead, Rare Disease The rise of patient-led clinical research, particularly in rare disease, represents a significant shift in the clinical trial landscape. This proactive approach allows sponsors to address patient needs more effectively and streamline the development process.

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PPD

JUNE 7, 2023

The number of pediatric patients diagnosed with nonalcoholic fatty liver disease (NAFLD) and nonalcoholic steatohepatitis (NASH) is on the rise, yet there are currently no approved therapies to treat NAFLD and NASH in adult or pediatric populations. As therapies for the treatment of NASH in adult patients go this year to the U.S.

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PPD

SEPTEMBER 6, 2023

Approaches to outsourcing clinical trials have changed significantly in recent years. Mixing of service models — a strategy that drug developers are leveraging now more than ever — can bring life-changing therapies to market faster. Overseen by an insourced project manager.

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The Pharma Data

JUNE 9, 2025

These results further validate the potential of Ascendis’ proprietary TransCon technology to deliver long-acting therapies that improve both efficacy and patient experience. It provides sustained release of unmodified somatropin, supporting normal growth and development in children. mg/kg/week) during the trial.

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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

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FDA Law Blog: Drug Discovery

DECEMBER 7, 2022

Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

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Drug Target Review

FEBRUARY 1, 2024

But commitment to neuroscience drug development is critically important given the significant unmet medical needs and the ways in which patients, caregivers, and families suffer. This can present challenges when attempting to recruit an enriched patient population for clinical trials.

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Broad Institute

NOVEMBER 2, 2023

Bayer and Broad Institute extend cancer therapy research collaboration By Corie Lok November 2, 2023 Breadcrumb Home Bayer and Broad Institute extend cancer therapy research collaboration Long-standing industry-academia collaboration has already resulted in three clinical oncology candidates.

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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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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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SugarCone Biotech

JUNE 27, 2025

They have interesting patterns of expression in different cancer indications; thus, diverse therapies for attacking these targets have been developed. As we’ll see, ASCO 2025 was a breakout conference for B7-H3-targeted therapies. Advanced therapies targeting B7-H3 use different modalities, currently dominated by ADCs.

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Drug Patent Watch

DECEMBER 9, 2024

This approach has led to the discovery of numerous potential drug candidates. From Lab to Market: The Long Road of Drug Development Once a promising compound is identified, it enters the long and costly process of drug development. Q: How long does a drug patent last?

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PPD

AUGUST 14, 2024

Clinical trials have significantly increased in complexity over the last 20 years, creating new challenges. One of the top reported challenges facing drug developers today is recruiting, enrolling and retaining the right patients for studies. The increase in complexity isn’t just creating challenges for patients.

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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.

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Conversations in Drug Development Trends

JANUARY 24, 2024

Written By: Derek Ansel, MS, CCRA, Executive Director, Therapeutic Strategy Lead, Rare Disease Given that 80% of rare diseases have a genetic etiology, genetic implications should be addressed at the onset of a clinical program to support trial enrollment. One diagnostic example that I discussed in my presentation is autism.

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The Pharma Data

MARCH 20, 2021

Precision imaging analytics technology known as radiomics will see increased adoption in 2021 and will be incorporated into drug development strategies and clinical trials management, according to Rose Higgins, CEO of HealthMyne.

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PPD

DECEMBER 19, 2022

PPD, Thermo Fisher Scientific ’s clinical research business, surveyed more than 150 decision-making leaders at pharmaceutical and biotech companies around the globe to collect key insights on the state of the evolving drug development industry. The opportunity to leverage new technologies in drug development (e.g.,

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The Premier Consulting Blog

JANUARY 14, 2025

Established in 2017 under the 21st Century Cures Act, the OCE brings together multidisciplinary scientific expertise to accelerate the review and approval of drugs, biologics, and medical devices for cancer care. These reviews have provided life-saving cancer therapies a median of 3.1 Provides the Oncology Dosing Toolkit.

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