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

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.

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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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PPD

MARCH 11, 2025

While individual rare diseases affect populations that are small in numbers, collectively they impact millions globally, posing significant health and research challenges. Real-world data (RWD) and real-world evidence (RWE) have emerged as transformational tools in addressing these hurdles, advancing rare disease drug development.

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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. 2 This progress has implications for diagnosis, therapeutic efficacy, and potentially establishing clinically relevant endpoints.

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Conversations in Drug Development Trends

OCTOBER 14, 2024

Autoimmune diseases and bone health conditions are not just medical challenges – they’re personal battles that millions of people fight every day. Fortunately, advances in clinical research are providing hope for better treatments and outcomes.

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Advarra

NOVEMBER 14, 2024

Consider the differences between supporting (concomitant) medications used in different countries or the differences generally in how a disease state is considered or treated. For example, consider a global Phase III, double-blind study, in a novel new therapy.

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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.

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The Pharma Data

JUNE 12, 2025

The data, presented at the annual European Alliance of Associations for Rheumatology (EULAR 2025) congress in Barcelona, Spain, show that dapirolizumab pegol resulted in significant and clinically meaningful improvements in disease activity while addressing a major symptom that profoundly impacts patients’ daily lives — fatigue.

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Drug Target Review

JULY 3, 2025

Given macro healthcare influences (eg, economic uncertainty, environmental changes) and the numerous available treatments for major diseases, drug developers may need to reassess their therapeutic strategies. This is especially relevant with today’s heavier focus on enhancing personalised medicine via broader emerging scientific findings.

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Conversations in Drug Development Trends

JULY 29, 2024

Evolving sentiments recognize obesity as a medical condition and lifestyle choice as well as a chronic disease with genetic, psychological, cultural, and environmental causes (Figure 1). They should include a comprehensive set of outcomes and endpoints to capture multiple areas of potential improvement within obesity as a disease.

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PPD

JANUARY 2, 2025

FSP solutions are being more frequently utilized by biotech companies Biotech companies employ contract research organizations (CROs) to handle trial-related functions and services, often delivered through a full-service outsourcing (FSO) model, an FSP model or a mixed model combining both. FSP outsourcing is growing faster than FSO.

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PPD

JUNE 20, 2025

Patients diagnosed with idiopathic pulmonary fibrosis (IPF) — a progressive, rare lung disease marked by scarring and worsening lung function — often face a poor prognosis. While two approved treatments, pirfenidone and nintedanib, slow disease progression, there currently is no treatment that reverses the effects or offers a cure for IPF.

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Olympian Clinical Research

OCTOBER 30, 2020

The disease is more common than many may realize, with approximately 2.6 At Olympian Clinical Research we’ve gotten a chance to work with countless Schizophrenia patients over the years through our clinical trials, and we know just how untrue many of the common assumptions about Schizophrenia really are. .

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Drug Target Review

JUNE 9, 2023

This exclusive interview with Dr Sharon Benzeno, Chief Commercial Officer, Immune Medicine at Adaptive Biotechnologies, unveils some ground-breaking research on T- cell therapy for cancer , which has seen the first TCR-based therapeutic candidate progress to clinical development, offering promising advancements in innovative cancer treatments.

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Broad Institute

NOVEMBER 25, 2024

Ladders to Cures (L2C) Accelerator By Maria Nemchuk November 25, 2024 Breadcrumb Home Ladders to Cures (L2C) Accelerator The Ladders to Cures (L2C) Accelerator aims to catalyze progress across the research ecosystem and accelerates advances leading to treatments and cures for patients with rare genetic diseases.

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Clinical Trials Disease Trials Therapies 52

Conversations in Drug Development Trends

OCTOBER 14, 2024

Autoimmune diseases and bone health conditions are not just medical challenges – they’re personal battles that millions of people fight every day. Fortunately, advances in clinical research are providing hope for better treatments and outcomes.

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Research Clinical Trials Therapies Immune Response 59

PPD

OCTOBER 31, 2024

In response, the PPD clinical research business of Thermo Fisher Scientific conducted its third global survey of 150 drug developers to capture a comprehensive view of these evolving trends. This issue is particularly pronounced for rare diseases and trials requiring diverse patient populations.

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PPD

JUNE 7, 2023

Adults aren’t the only patients affected by the increasing prevalence of liver disease. 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.

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Vial

DECEMBER 4, 2023

Clinical research is an integral component of the healthcare ecosystem. It is pivotal in the development of cutting-edge treatments, therapies, and interventions for a wide array of diseases. Patient Payments in Clinical Research: Perception Over the Years In the U.S.,

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Drug Target Review

MAY 23, 2025

In the ever-evolving landscape of immuno-oncology, managing the side effects of advanced therapies has become just as important as enhancing their efficacy. I take responsibility for identifying pre-clinical research that can support our clinical programs, as well as designing and overseeing its execution, he explains.

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PPD

AUGUST 5, 2024

Pulmonary arterial hypertension (PAH) is a progressive and fatal lung disease that is caused or influenced by multiple factors. The clinical sites working on these trials are highly experienced and have a deep understanding of the complexities of successfully running PAH clinical trials. None offers a cure for PAH.

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PPD

MAY 18, 2023

For neurodegenerative disease research, the care partner, or caregiver, is equally important as the person with dementia. Even with stronger awareness of studies, drug developers must contend with the inherent lack of trust between the dementia community and clinical trials. Recognize and relieve the caregiver burden.

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Conversations in Drug Development Trends

JULY 9, 2024

The discussions at the conference shed light on innovative approaches to enhance long-term outcomes for patients, focusing on combination therapies that not only aid in weight loss but also help preserve muscle mass, an essential component of healthy body composition.

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Drug Target Review

FEBRUARY 29, 2024

During my academic career, I had the opportunity to examine the underlying causes of neurodegenerative diseases like Parkinson’s disease and develop novel animal models to investigate potential disease-modifying therapeutics. AskBio) I continue to work to bring innovative gene therapies to patients in need.

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Advarra

AUGUST 8, 2023

Gene therapy research is booming in the clinical setting. In this blog, we summarize the growth, risks, and regulatory requirements for gene therapy research. We also discuss how a centralized biosafety review process can benefit this type of research.

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Drug Target Review

DECEMBER 16, 2024

In this interview, she discusses her path to becoming a physician and clinical researcher, the obstacles she faced, and her dedication to improving outcomes for cancer patients. Dr Hingorani also shares insights into the strengths women bring to STEM, the importance of mentorship, and her vision for the future of oncology research.

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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. billion in 2023 and is expected to grow at a CAGR of 7.0% between 2024 and 2030.

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Drug Target Review

OCTOBER 25, 2024

How does precision neuroscience differ from traditional approaches in the treatment of neurodegenerative diseases, and what advantages does it offer? Traditionally, Alzheimer’s disease is diagnosed by clinical examination, medical history and cognitive testing.

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Alta Sciences

JUNE 26, 2025

1] It’s a privilege to participate in such a prestigious gathering of vision science professionals, and to gain insight into the latest innovations and treatments for complex ocular diseases. These technologies are helping pave the way toward more objective, reliable tools for clinical research.

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Drug Target Review

MAY 7, 2024

Why are biomarkers particularly critical in the context of rare diseases? Developing novel therapeutic approaches for rare diseases poses inherent challenges due to the limited populations available for testing. How do AI-enhanced approaches accelerate the drug development process for rare diseases?

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PPD

SEPTEMBER 16, 2024

In a recent panel discussion at DIA Global , our experts explored how these elements are reshaping clinical research and drug discovery. This can be converted to a clinical trial simulator, which can model a clinical trial before the trial design is finalized and initiated.

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PPD

JANUARY 5, 2023

In an industry survey led by PPD, Thermo Fisher Scientific’s clinical research business, rare diseases emerged as a top therapeutic area in drug developers’ pipelines, with 39% of global biotech and biopharma leaders pursuing therapies in the broad rare disease space.

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Advarra

NOVEMBER 29, 2022

The field of cell and gene therapies (CGT) is constantly evolving, and there has been significant progress in this area of research. However, despite the promise of these therapies, the regulations governing them lag the science, which in turn hinders the clinical translation of these novel medicines.

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PPD

MARCH 13, 2024

In combination, they provide confidence in dose escalation or protocol adaptation decisions, which ultimately benefit patients who often have critical and life-limiting diseases. Within cell therapy clinical trials, apheresis collections contribute to the specific constituents of the given therapy.

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Drug Target Review

APRIL 11, 2024

Targeting pathways involved in SMC phenotypic modulation holds promise for developing novel therapeutic approaches for cardiovascular diseases. Can you explain the methodology used to investigate the genetic variants regulating extracellular matrix (ECM) secretion in SMCs, and predict proteins associated with vascular disease?

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PPD

DECEMBER 16, 2024

The drug development industry faces rising costs and increasingly complex protocols as top challenges, with 49% of developers citing costs as their primary concern and 39% pointing to protocol complexity as a key hurdle, according to a recent survey of 150 drug developers worldwide by the PPD clinical research business of Thermo Fisher Scientific.

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