Drug Development, FDA and Trials - Drug Discovery Digest

Real-World Evidence in Clinical Drug Development

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.

Drug Development

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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. However, patients also play a vital role in engaging directly with the FDA. It also means sponsors are less likely to waste time and resources developing programs that don’t work for patients.”

FDA

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FDA Moves Beyond COVID-19, But Impacts on COVID-19 Era Clinical Trials Remain

FDA Law Blog: Drug Discovery

OCTOBER 11, 2023

Snow — On September 18, 2023, FDA published an updated, final iteration of guidance for immediate implementation entitled, “ Considerations for the Conduct of Clinical Trials of Medical Products During Major Disruptions Due to Disasters and Public Health Emergencies.” By James E. Valentine & Charles D.

Clinical Trials

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Webinars

From Diagnosis to Delivery: How AI is Revolutionizing the Patient Experience

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FDA Animal Rule: Overview & Impact on Drug Development

thought leadership

JULY 9, 2024

The Animal Rule , a regulation set by the US Food and Drug Administration (FDA), applies to the development and testing of drugs and biological products intended to reduce or prevent serious or life-threatening conditions caused by exposure to lethal or permanently disabling toxic agents such as chemical, biological, radiological, or nuclear substances, (..)

FDA

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What Do Patients Have to Say about Gene Therapy Trials? An Upcoming FDA Public Meeting to Hear from Patients and Caregivers

FDA Law Blog: Drug Discovery

MARCH 13, 2023

Valentine — Incorporating patient and caregiver experiences into every phase of drug development has become increasingly prioritized during both development and review ( see, e.g. , previous coverage here ). At least some (if not all) of the panelists will have had experience participating in gene therapy trials.

Therapies

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The Collaboration Between Industry and Academia in Drug Development

DrugBank

AUGUST 15, 2024

Drug development is challenging, including not only the complexities of biomolecular drug mechanisms but also the convolutions of regulatory pathways and commercialization strategies. This includes funding large-scale clinical trials and establishing robust manufacturing/distribution networks.

Drug Development

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At Long Last, FDA Unveils Plan for Rare Disease Innovation Hub

FDA Law Blog: Drug Discovery

JULY 17, 2024

Importantly, the Hub is intended to establish a new model within FDA, which leverages cross-Agency expertise in providing guidance and conducting reviews for products for rare disease populations. By Sarah Wicks & James E. Valentine & Frank J. those reviewed by the CDER Division of Rare Diseases and Medical Genetics).

FDA

FDA Disease Clinical Pharmacology Science

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

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FDA Poised to Make Impact on Underrepresented Populations in Clinical Studies

H1 Blog

JULY 30, 2024

Diversity Action Plans (DAPs) aim to improve data that the FDA receives by increasing the clinical enrollment of historically underrepresented participants. The recent draft by the FDA introduces additional guidelines for Diversity Action Plans aimed at improving diversity in clinical research.

FDA

FDA Clinical Trials Compliance Pharma Companies

Sarah Wicks Returns to HPM to Elevate Drug Development and Regulatory Practice

FDA Law Blog: Drug Discovery

MAY 1, 2024

proudly announces the return of attorney Sarah Wicks to its drug development and compliance group. Sarah brings a wealth of experience and a proven track record of advising innovative drug and biologics companies through the intricate landscape of product development and commercialization.

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FDA Issues Draft Guidance on Psychedelic Drug Development

thought leadership

JULY 26, 2023

Food and Drug Administration (FDA) recently issued a draft guidance on the development of psychedelic drugs. There has been a growing interest in using psychedelics for the treatment of medical conditions in recent years, and this guidance by the FDA is timely to support researchers investigating this use.

FDA

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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

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

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Cognitive and Pharmacodynamic Testing in CNS First-In-Human Trials

Alta Sciences

JULY 22, 2024

Recently, we have seen increasing numbers of complex FIH studies in CNS-active drugs to gather comprehensive data with a wider range of doses as early as possible. jpg Tags Clinical Trials Weight 1

Trials

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How to Prioritize Pediatric Populations in NASH Trials

PPD

JUNE 7, 2023

Food and Drug Administration (FDA) for approval , much work remains for drug developers aiming to advance treatments for pediatric populations. Reflecting Patient Diversity in NASH Trials Data from NASH clinical trials within adult populations may support clinical research into treatment for pediatric populations.

Trials

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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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In Silico Modeling Unveils a New Era in Rare Disease Drug Development

The Premier Consulting Blog

JUNE 18, 2023

This has opened new opportunities in pharmaceutical drug development, such as the ability to evaluate large complex databases and to integrate information in useful ways. One exciting application of these technologies is the use of in silico trials in the development of novel therapies for rare diseases.

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Predicting Drug Development in 2023

EG Life Sciences

FEBRUARY 15, 2023

The year 2022 reflected a transformative path for the drug development industry. It is without a doubt that 2022 predicted change and opportunity in biopharma and biotech clinical trials in 2023. The US Food and Drug Administration (FDA) approved around 26 novel drugs in 2022.

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Clinical Trials Join the Remote Work Revolution: FDA’s New Draft Guidance on Decentralized Clinical Trials

FDA Law Blog: Drug Discovery

MAY 4, 2023

Cato — On May 2nd, FDA released a new draft guidance with recommendations for decentralized clinical trials (DCTs) for drugs, biologics, and devices. In a DCT, trial-related activities may occur in trial participants’ homes, at local health care providers’ offices, or in local clinical laboratories. By McKenzie E.

Clinical Trials

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Five Promising Treatment Areas in Early-Phase Drug Development in 2024

Alta Sciences

APRIL 17, 2024

Five Promising Treatment Areas in Early-Phase Drug Development in 2024 aasimakopoulos Wed, 04/17/2024 - 15:52 Early-phase drug development is an ever-changing landscape, as emerging science leads to new promising areas of research for the treatment of human health issues.

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News from ESMO: Challenging the Status Quo of Early Phase Clinical Trial Design — Moving from “Why” to “How”

Cytel

OCTOBER 27, 2023

Speakers discussed investigator, regulatory (FDA), industry, and patient perspectives during the special symposium “Challenging the Status Quo of Early Phase Clinical Trial Design: Project Optimus.” To address this challenge, the FDA Oncology Center of Excellence initiated Project Optimus. The key paradigm shift includes:

Clinical Trials

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FDA Approval of New Therapy, Duvyzat, for Duchenne Muscular Dystrophy Represents Several Meaningful Firsts

FDA Law Blog: Drug Discovery

MARCH 26, 2024

Sasinowski — On March 21st, FDA announced the approval of the first nonsteroidal therapy for the treatment of Duchenne Muscular Dystrophy (DMD) (FDA press release available here ). Duvyzat (givinostat), a histone deacetylase (HDAC) inhibitor developed by Italfarmaco, S.p.A., By Charles G. Raver & James E. Lancet Neurol.

FDA Approval

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The Evolution of Oncology Trials: Project Optimus and Study Design Trends

Conversations in Drug Development Trends

JUNE 13, 2024

The FDA Oncology Center of Excellence (OCE) is committed to further enhancing oncology research and as a result, has recently implemented several projects aimed to revolutionize oncology clinical trials. Find key highlights from the webinar below, and check out the webinar for a full overview!

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Decoding Clinical Trial Costs and Efficiency Roadblocks

Vial

MAY 13, 2024

Introduction The biopharma industry is poised to make revolutionary advances in and redefine drug development, and the current climate for innovation appears ideal. only 5% of molecules in oncology Phase I trials reach the market taking, on average, 7.5 An estimated 80% of clinical trials do not finish on time.

Clinical Trials

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Top 5 Scientific Resources in CNS Clinical Trials

Alta Sciences

MARCH 21, 2024

Top 5 Scientific Resources in CNS Clinical Trials blussier Thu, 03/21/2024 - 18:09 HTML CNS Center of Excellence Resource Library Altasciences has an extensive understanding of psychiatric and neurologic disorders, stemming from our decades of conducting complex studies in various therapeutic indications impacting the central nervous system.

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FDA Creates a New Advisory Committee for Genetic Metabolic Diseases – Could This Be an Opportunity to Support Rare Disease Product Development More Broadly?

FDA Law Blog: Drug Discovery

DECEMBER 13, 2023

Sasinowski — On December 12, 2023, FDA announced the creation of a new advisory committee specifically for treatments for genetic metabolic diseases, the Genetic Metabolic Diseases Advisory Committee, or “GeMDAC.” Drug development for these conditions has unique and complex challenges, therefore few treatments are available to patients.”

Disease

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The Growing Trend of Patient-Led Clinical Research in Rare Disease

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. Why Is There an Increase in Patient-Led Rare Disease Research?

Clinical Research

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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. The FDA has established accelerated approval pathways for certain treatments for cancer and other serious diseases. Recent news showed the FDA setting a higher bar for accelerated approvals for cancer drugs.

Clinical Trials

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COVID-19 Master Protocol Trial Participants Should Be Randomized, FDA Says | 2021-05-17

The Pharma Data

MAY 17, 2021

Sponsors using master protocols to test new drugs and therapies for treating or preventing COVID-19 should base their analyses on comparisons between control arm participants who were concurrently randomized, the FDA advised in a final guidance released yesterday. Read the guidance here: www.fdanews.com/05-17-21-COVID-19.pdf.

FDA

FDA Trials Therapies Treatment

Webinar: Assessing Cognition Impairment Using Driving Simulators in Clinical Trials

Alta Sciences

FEBRUARY 8, 2024

The FDA guidance, Evaluating Drug Effects on the Ability to Operate a Motor Vehicle , indicates that testing in early-phase clinical development should emphasize sensitivity over specificity in CNS effects. Tags Clinical Trials Weight 12 View the Driving Simulation Fact Sheet for more information.

Clinical Trials

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FDA Guidance Outlines a Framework for the Evaluation of Long-Term Neurodevelopmental Safety Studies in Neonatal Product Development

FDA Law Blog: Drug Discovery

OCTOBER 28, 2024

To this end, FDA recently announced the publication of a guidance titled “Considerations for Long-Term Clinical Neurodevelopmental Safety Studies in Neonatal Product Development,” (the “Guidance”). What factors should be considered when developing a long-term safety study plan?

Production

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Artificial Intelligence (AI), Machine Learning (ML) and the FDA

The Premier Consulting Blog

NOVEMBER 12, 2023

In 2019, the FDA published a discussion paper on a proposed regulatory framework for modification to AI/ML-based software as a medical device (SaMD), which received a significant amount of feedback from stakeholders. FDA Discussions As a result of the feedback received in 2019, the FDA outlined the five-pillar approach to regulating AI/ML.

FDA

FDA Drug Development Regulations FDA Approval

FDA Approves Duvystat, New Oral Treatment for Duchenne Muscular Dystrophy (DMD)

PLOS: DNA Science

MARCH 28, 2024

A new drug has entered the arsenal against Duchenne muscular dystrophy (DMD), a genetic disease that affects boys and is challenging to treat. FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. In 2023, two gene-based treatments became available.

FDA Approval

FDA Approval Treatment FDA DNA

Rare Oncology and the FDA: Taking the Guesswork Out of Expedited Pathways

The Premier Consulting Blog

JUNE 26, 2023

1 With their poorly understood natural histories, phenotypic heterogeneity, and diverse clinical manifestations, rare cancers pose challenges to drug development and represent a significant unmet need in oncology. Rare cancers account for 25-30 percent of all new cancer diagnoses and 25 percent of cancer deaths.

FDA

FDA Clinical Trials Therapies Trials

Analysis Life Sciences Thank You Halfway there: Novel drug approvals and their supportive clinical trials so far in 2024

Agency IQ

JULY 22, 2024

Halfway there: Novel drug approvals and their supportive clinical trials so far in 2024 In the first half of 2024, the FDA’s Center for Drug Evaluation and Research (CDER) approved 21 novel drug products. CDER is the FDA office in charge of reviewing pharmaceuticals and therapeutic biologics.

Clinical Trials

Clinical Trials Trials Science FDA

Article FDA Thank You FDA updates guidance on developing drugs for Covid-19, replacing pandemic-era version

Agency IQ

DECEMBER 1, 2023

FDA updates guidance on developing drugs for Covid-19, replacing pandemic-era version Last week, FDA published the third update to its guidance on the development of products to prevent or treat Covid-19. Read Agency IQ’s complete breakdown of the FDA’s PHE guidance documents here.]

FDA

FDA Clinical Trials Trials Vaccine

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.

Clinical Trials

Clinical Trials Trials Science FDA

Four Strategies for Preparing FDA Diversity Action Plans

H1 Blog

MAY 25, 2023

Bridging the Gap Between Pharmaceutical Development and Advancement of Therapeutics In the race to bring new treatments and cures to market, clinical trial operations and feasibility teams often face a formidable challenge: ensuring that patient populations are accurately represented in their drug development studies.

FDA

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Top Life Science Resources for Fall 2024

Alta Sciences

OCTOBER 28, 2024

TOP CLINICAL SCIENTIFIC RESOURCES Video: The True Meaning of Moving in Unison Watch how collaboration and precision ensure seamless clinical trial implementation. The study used a double-blind, crossover design with recreational drug users to evaluate subjective effects and likability. Read the white paper. Read the blog.

Science

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FDA Flexes its New FDORA Muscles in Withdrawing an Accelerated Approval

FDA Law Blog: Drug Discovery

FEBRUARY 28, 2024

This is the third withdrawal of an accelerated approval FDA has performed, following Avastin in 2011 and Makena in 2023. Notably, this decision marks the first use of the new expedited procedures for withdrawal of an accelerated approval that were enacted in the Food and Drug Omnibus Reform Act of 2022 (FDORA).

FDA

FDA Trials Therapies Drugs

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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Operation Warp Speed for Rare Diseases: Expected Boom in Drug Development and Approval

Advarra

AUGUST 10, 2023

Rapid growth in gene therapy is expected to receive additional support as the Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER) prepares to launch Operation Warp Speed for Rare Diseases. Operation Warp Speed for Rare Diseases looks to accelerate both drug development and approval.

Drug Development

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ACI’s 40th FDA Boot Camp – March 22-23, 2023 (Virtual)

FDA Law Blog: Drug Discovery

JANUARY 2, 2023

Hosted by American Conference Institute, the FDA Boot Camp returns for its 40th iteration with the continued intent of providing an essential working knowledge of core FDA concepts, and real-world examples that will help you to excel in your everyday practices. The essentials of the approval process for drugs and biologics.

FDA

FDA Clinical Trials Trials Production

What is the role of a CRO in a clinical trial?

Vial

FEBRUARY 8, 2024

To advance new therapies, pharmaceutical, biotech, and medical device companies engage contract research organizations (CROs) for their know-how in navigating the complex landscape of drug development and regulatory pathways and to run clinical trials.

Clinical Trials

Clinical Trials Trials Clinical Research Research

Applying Artificial Intelligence to Transform the Patient Enrollment Paradigm in Clinical Research

PPD

JULY 29, 2024

In today’s data-driven world, AI has become valuable and indispensable, enabling organizations to extract valuable insights from vast amounts of data, make informed decisions and drive innovation across different sectors — including drug development.

Clinical Research

Clinical Research Clinical Trials Research Trials

Real-World Evidence in Clinical Drug Development

Why Including Patients in FDA Engagement Will Benefit Your Trial

FDA Moves Beyond COVID-19, But Impacts on COVID-19 Era Clinical Trials Remain

Webinars

FDA Animal Rule: Overview & Impact on Drug Development

What Do Patients Have to Say about Gene Therapy Trials? An Upcoming FDA Public Meeting to Hear from Patients and Caregivers

The Collaboration Between Industry and Academia in Drug Development

At Long Last, FDA Unveils Plan for Rare Disease Innovation Hub

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

FDA Poised to Make Impact on Underrepresented Populations in Clinical Studies

Sarah Wicks Returns to HPM to Elevate Drug Development and Regulatory Practice

FDA Issues Draft Guidance on Psychedelic Drug Development

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

Cognitive and Pharmacodynamic Testing in CNS First-In-Human Trials

How to Prioritize Pediatric Populations in NASH Trials

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

In Silico Modeling Unveils a New Era in Rare Disease Drug Development

Predicting Drug Development in 2023

Clinical Trials Join the Remote Work Revolution: FDA’s New Draft Guidance on Decentralized Clinical Trials

Five Promising Treatment Areas in Early-Phase Drug Development in 2024

News from ESMO: Challenging the Status Quo of Early Phase Clinical Trial Design — Moving from “Why” to “How”

FDA Approval of New Therapy, Duvyzat, for Duchenne Muscular Dystrophy Represents Several Meaningful Firsts

The Evolution of Oncology Trials: Project Optimus and Study Design Trends

Decoding Clinical Trial Costs and Efficiency Roadblocks

Top 5 Scientific Resources in CNS Clinical Trials

FDA Creates a New Advisory Committee for Genetic Metabolic Diseases – Could This Be an Opportunity to Support Rare Disease Product Development More Broadly?

The Growing Trend of Patient-Led Clinical Research in Rare Disease

Improving Oncology Clinical Trials for Accelerated Approval

COVID-19 Master Protocol Trial Participants Should Be Randomized, FDA Says | 2021-05-17

Webinar: Assessing Cognition Impairment Using Driving Simulators in Clinical Trials

FDA Guidance Outlines a Framework for the Evaluation of Long-Term Neurodevelopmental Safety Studies in Neonatal Product Development

Artificial Intelligence (AI), Machine Learning (ML) and the FDA

FDA Approves Duvystat, New Oral Treatment for Duchenne Muscular Dystrophy (DMD)

Rare Oncology and the FDA: Taking the Guesswork Out of Expedited Pathways

Analysis Life Sciences Thank You Halfway there: Novel drug approvals and their supportive clinical trials so far in 2024

Article FDA Thank You FDA updates guidance on developing drugs for Covid-19, replacing pandemic-era version

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

Four Strategies for Preparing FDA Diversity Action Plans

Top Life Science Resources for Fall 2024

FDA Flexes its New FDORA Muscles in Withdrawing an Accelerated Approval

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

Operation Warp Speed for Rare Diseases: Expected Boom in Drug Development and Approval

ACI’s 40th FDA Boot Camp – March 22-23, 2023 (Virtual)

What is the role of a CRO in a clinical trial?

Applying Artificial Intelligence to Transform the Patient Enrollment Paradigm in Clinical Research

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