Pharmaceutical Development Group

JANUARY 7, 2022

Effective and consistent use and application of Data Standards can reduce costs of Pharmaceutical Drug and Biologic Products and Process Development, Drug Development Services, 505(b) NDA, IND Consulting , NDA Consulting, BLA Consulting , and effective FDA Pre-Submission collectively resulting in FDA Approval.

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FDA FDA Approval Biosimilars Science 52

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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FDA Law Blog: Drug Discovery

OCTOBER 11, 2023

In the case of trials for orphan drugs, this is particularly concerning because there is often already difficulty in interpreting clinical trial results to establish effectiveness due to the common challenges inherent in rare disease drug development.

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Clinical Trials Trials FDA Drug Development 64

PerkinElmer

JANUARY 7, 2022

The pharmaceutical industry is under huge pressure to address the high attrition rates in drug development. With around 90% of candidates failing during clinical development, 1 the process is not only long and risky, but also expensive for those involved. Sci Rep 9, 18911 (2019). 2021 [cited 16 December 2021].

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Drugs FDA Approval Pharmacokinetics Drug Development 52

Alta Sciences

FEBRUARY 8, 2024

Image Blog-Thumbnail_Driving-sim-webinar.jpg Synopsis Learn more about how Altasciences can test cognition, and the impact of driving under the influence of medication, using our state-of-the-art driving simulators. View the Driving Simulation Fact Sheet for more information.

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Conversations in Drug Development Trends

MAY 30, 2024

This trend is on the rise despite recent disappointments with clinical trial outcomes, which have the potential to destabilize the industry in the short term regarding drug development strategy and optimal study designs. The shared placebo design is another approach to accelerating drug development in ALS research.

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Treatment Clinical Trials Small Molecule Therapies 81

The Premier Consulting Blog

APRIL 15, 2024

In this blog, we will go into more detail about the unwritten nonclinical requirements for the PIND meeting. Embrace Alternatives The combination of experience and technical ability is great for determining a potential drug development path.

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FDA Pharmacokinetics Laboratories FDA Approval 52

PerkinElmer

APRIL 21, 2020

Using the isolated ZIKV MEX_I_7 strain, researchers got a head start on potential leads by interrogating a library of existing, FDA-approved drugs that can be used off-label for treatment of Zika. High-content imaging was used in a recent study aiming to identify novel ZIKV inhibitors from a pool of 774 compounds.

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Virus FDA Approval Laboratories Vaccine 52

The Premier Consulting Blog

NOVEMBER 12, 2023

The FDA is actively supporting this increased AI/ML integration in a variety of ways, including: Establishing the CDER AI Steering Committee (AISC) to provide guidance and oversight on AI/ML in drug development. Developing a framework for AI/ML-based devices to ensure that these devices are safe and effective.

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FDA Drug Development Regulations Science 52

NIH Director's Blog: Drug Development

JUNE 14, 2016

Several years ago, the Food and Drug Administration (FDA) recommended that drug developers take special care to show that potential drugs to treat diabetes don’t adversely affect the cardiovascular system [1].

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Drugs Disease Drug Development Clinical Trials 52

The Premier Consulting Blog

OCTOBER 11, 2022

Case Study 1: Using RWD and RWE to support an ultra-rare orphan drug program Recently, Premier Consulting was engaged by a sponsor who had developed a potential treatment for an ultra-rare, life-threatening congenital disease.

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The Premier Consulting Blog

SEPTEMBER 11, 2024

However, the success of a 505(b)(2) application hinges on a tailored development strategy that carefully considers the specific characteristics of the newly proposed drug product, and the nature of the changes made in comparison to a prior US FDA-approved listed drug (LD) or the drug reported in literature.

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NIH Director's Blog: Drug Development

JULY 20, 2017

These results, along with data from another clinical trial expected to begin later this year, will be used by Vertex to select which next-generation targeted drug or combination of drugs will move forward into larger scale Phase III studies. About 30,000 Americans have CF.

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Clinical Trials Small Molecule Therapies Disease 52

NIH Director's Blog: Drug Development

FEBRUARY 16, 2021

It is testing medicines previously developed for other conditions that might be beneficial in treatment of COVID-19. In addition to trials conducted under the ACTIV partnership, NIH has prioritized and tested additional therapeutics in “ACTIV-associated trials.”

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Molecular Design

OCTOBER 14, 2018

One criticism of PAINS filters that I have made, both in my article and the Molecular Design blog , is that some of the frequent-hitter behavior in the PAINS assay panel may be due to quenching or scavenging of singlet oxygen which is an essential component of the the AlphaScreen readout.

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FDA Law Blog: Biosimilars

MARCH 13, 2024

4,631,286, where the PTO considered “whether Hoechst-Roussel is eligible to file an application for [PTE] based on a regulatory review conducted by its competitor, the marketing applicant Warner-Lambert, wherein Hoechst-Roussel was not associated with the regulatory review that led to FDA approval for commercial marketing of the approved product.”

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The Premier Consulting Blog

MARCH 11, 2025

The FDAs draft guidance, Early Alzheimers Disease: Developing Drugs for Treatment, released in March 2024, aims to help sponsors by providing a clear framework for developing and evaluating new treatments, particularly in the early stages of the disease that occur before the onset of overt dementia (i.e.,

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Drug Development Treatment Clinical Trials FDA 52

The Premier Consulting Blog

NOVEMBER 12, 2024

For drug developers the 505(b)(2) pathway presents an expedited pathway to FDA approval. In our experience, the conversation on 505(b)(2) pathways commonly focuses on predicate data or bridging strategies to accelerate FDA approval but lacks dialogue on the needs of US payers. Non-preferred coverage.

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Marketing FDA Approval FDA Drugs 52

The Premier Consulting Blog

JANUARY 14, 2025

In this blog, we outline how the OCE gets involved in product reviews and offer a high-level summary of the programs and resources available for guiding oncology product development. Determining a new products pathway The OCE does not receive regulatory applications directly from sponsors who are developing new cancer therapies.

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Clinical Trials Therapies FDA Trials 52

LifeSciVC

MARCH 12, 2025

Truth be told, I considered making this the subject of my blog. This circumventing the wheel requires navigating around the roadblocks that hampered earlier drug development efforts to bring new treatment options forward for patients. Today there are 15 FDA-approved ADCs for oncology.

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FDA Law Blog: Biosimilars

OCTOBER 13, 2024

A recent analysis from NORD showed that among the 39 rare pediatric diseases for which vouchers were awarded, only three had any FDA-approved products on the market before the program’s enactment. We heard and felt the anxiety from many of our clients about the uncertainty this caused. After all, this is what incentives are for.

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Disease FDA FDA Approval Government 64

Drug & Device Law

OCTOBER 10, 2022

The drug at issue is the only FDA-approved pharmaceutical treatment for ATTR-CM (although off-label use of other drugs, and organ transplant, are sometimes used as treatments). 2370 (2022), which the Blog recently wrote about here. ATTR-CM only affects about 150,000 Americans, who are mostly elderly.

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