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.

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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

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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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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Drug Target Review

JUNE 6, 2025

At the forefront of this transformation is Dr Ebrahim Delpassand, a nuclear medicine physician and the driving force behind RadioMedix (RMX), a radiopharmaceutical company focused on developing targeted diagnostics and therapies. The founding of RadioMedix was born from a recognition of unmet needs in cancer treatment.

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Quanticate

AUGUST 2, 2024

Real-world evidence (RWE) is changing clinical drug development, bridging the gap between controlled clinical trial environments and the complexities of everyday patient experiences.

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Quanticate

SEPTEMBER 13, 2024

With the implementation of technology rapidly increasing, stricter regulatory standards, and growing demand for innovative treatments, the trends within the industry continue to evolve, impacting the future of clinical trials both positively and negatively.

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PPD

JUNE 20, 2025

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. However, recent advancements and strategic approaches in clinical trials offer hope that additional treatments are on the way.

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Broad Institute

DECEMBER 18, 2023

Brain disorders are difficult to study and many drug candidates have failed in clinical trials, causing pharmaceutical companies to reduce their investments or even exit the field entirely. But a new path for bringing treatments to patients is starting to emerge. We all recognize the vast, unmet medical need.

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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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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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Drug Target Review

NOVEMBER 25, 2024

As the CEO of iOnctura, an innovative oncology biopharmaceutical company she co-founded in 2017, Catherine has played a key role in advancing the development of highly targeted small molecules aimed at revolutionising cancer treatment. I went on to complete my MBA and PhD at The Institute of Cancer Research (ICR) in drug development.

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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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Conversations in Drug Development Trends

JANUARY 6, 2025

With 20-25% of the global population affected by metabolic problems, finding effective treatments is crucial for cardiovascular and metabolic (CVM) conditions. Traditional study designs feature predetermined sample sizes, assessments, and analyses that must remain in place over long treatment durations.

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Drug Target Review

DECEMBER 23, 2024

Antibody-drugconjugates (ADCs) represent a significant advancement in drug discovery, combining the precision of monoclonal antibodies with the cancer-killing power of cytotoxic drugs. Properly managing these toxicities is crucial to enhancing the safety and therapeutic effectiveness of ADC treatments.

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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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BioPharma Drive: Drug Pricing

JUNE 23, 2025

Supply chain and ethical considerations The podcast discussion highlighted how soaring demand has strained manufacturing capacity, with contract manufacturing organizations dedicating large portions of their output to GLP-1 drugs. You can unsubscribe at anytime. The balance is complex, as obesity carries its own serious health risks.

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Drug Target Review

MARCH 17, 2025

Neuropsychiatric treatment is on the verge of a major transformation. Historically, treatment options have been limited, with patients relying on daily medications that have minimal efficacy and troublesome side effects. “Zelquistinel is a positive modulator of NMDA receptors,” Donello explains.

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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. Currently, treatment options are limited to supportive care aimed at managing symptoms rather than targeting the underlying cause.

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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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SugarCone Biotech

JUNE 19, 2025

Typical treatment has been chemotherapy and radiotherapy with an initially high overall response rate (ORR) but then rapid recurrence followed by poor prognosis. Tarlatamab triggered an ORR of ~40% in refractory (second-line or later) SCLC patients in the DeLLphi-301 clinical trial published in 2023. months (versus 20% ORR and 8.3

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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.   What sets RADR ® apart is its robust data-driven approach.

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The Pharma Data

JUNE 24, 2025

The lead asset in this portfolio, daraxonrasib (RMC-6236) , is a multi-selective RAS(ON) inhibitor currently being evaluated in clinical trials for patients with non-small cell lung cancer (NSCLC) and colorectal cancer harboring KRAS mutations. Source link

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Conversations in Drug Development Trends

MAY 10, 2024

By: Christine Moore, PhD, Vice President, Neuroscience, Scientific Solutions Patients with borderline personality disorder (BPD) face a heightened risk of substance abuse and suicide, as well as substantial delays in receiving treatment.

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PPD

JUNE 7, 2023

As therapies for the treatment of NASH in adult patients go this year to the U.S. Food and Drug Administration (FDA) for approval , much work remains for drug developers aiming to advance treatments for pediatric populations. Partnership with an experienced CRO is essential to your trial’s success.

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Conversations in Drug Development Trends

JANUARY 21, 2025

Rigorous procedures to ensure that drugs are effective and safe. Regulatory bodies such as the FDA oversee clinical trials to ensure that studies’ design, conduction, analysis, and reporting are per established guidelines and laws. No matter how small, any lack of efficiency can snowball into costly delays in drug approval.

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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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The Pharma Data

JUNE 9, 2025

The COACH study is the first clinical investigation to assess the combined effects of two once-weekly treatments—TransCon® CNP (navepegritide) and TransCon® hGH (lonapegsomatropin)—in children with achondroplasia. TransCon CNP as a monotherapy has demonstrated the potential to transform the treatment of achondroplasia,” Dr. Shu stated.

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Drug Target Review

OCTOBER 30, 2024

Now take a step further: envision testing drugs in these organoids to identify the ones that can treat disease safely and effectively without needing to run expensive clinical trials first. A large body of evidence demonstrates that these platforms can accurately predict tumour response to drugs.

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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. Her team sits right at the edge of innovation, where discovery science meets clinical execution.

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Conversations in Drug Development Trends

MARCH 1, 2024

These designs facilitate streamlined trial logistics and centralized governance and create higher-quality data. Master protocols allow a trial to perform multiple tests on diverse patient populations or diseases under a unified design. Do you have various stakeholders invested in the success of your development programs?

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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. Source link: [link].

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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.

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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. The result?

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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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Drug Target Review

JULY 4, 2023

These therapies have broadened treatment options for patients to expand beyond the more traditional small molecule drug alternatives. ADCs have the potential to redress the poor balance between safety and efficacy seen with traditional cancer treatment options. 3D rendering of Antibody Drug Conjugate Molecules.

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Conversations in Drug Development Trends

JANUARY 6, 2025

Tom Zhang, PhD, Chief Scientific Officer, Large Molecule Bioanalysis Revolutionizing Therapeutic Development with Cutting-Edge Bioanalysis Automation Cutting-edge advancements in bioanalysis (BioA) and drug R&D are reshaping the future of preventative and interventional care, but how do we harness these to their fullest potential?

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