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Clinical Studies, Phases I-IV Articles and Insights

FAQS: In Vitro Diagnostic Medical Device Regulation

On May 26, 2017, the EU Regulation 2017/746 of the European Parliament and the Council on in vitro diagnostic medical devices (IVDR) became active under the EU Regulation on Medical Devices (MDR). Recertification by May 26, 2022, became required for all previously approved products. The

Biologic Therapeutics Development, Part 2: Regulatory Pathways and Pharmacometric Analysis

Sponsors developing biologics must manage numerous scientific considerations specific to large-molecule products, including biochemical characterization studies to confirm structural identity, biological activity studies to confirm potency, and mechanism of action maintenance. Clinical trials of biologics are designed to determine pharmacokinetics (PK), pharmacodynamics (PD), safety, and efficacy.

Biologic Therapeutics Development, Part 1: Definition and Distinct Characteristics

The development of biologics represents a major advancement, enabling the treatment of patients with many illnesses for which no other therapeutics were previously available. When developing biologics, sponsors must manage several scientific considerations specific to large-molecule products, including biochemical characterization studies to confirm structural identity,

Advancing from Research to Development: What Can Go Wrong?

The drug development process is a long journey, beginning with drug discovery, moving through nonclinical and clinical studies, and ultimately culminating in regulatory approval. With many steps in between, each as important as the next, there are multiple factors regarding development strategy and approach that

Gene Therapy and Pharmacokinetics

How and When to Incorporate PK Design into Your Gene Therapy Development Plan Gene therapy, in its infancy around 30 years ago, is fast gaining prominence in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. When developing a clinical development program for

GxP Quality Guidelines and Regulations: Frequently Asked Questions

GxP refers collectively to several types of “good practice” quality guidelines and regulations, each serving a specific purpose. In pharmaceutical product development, these include, but are not limited to: GCP (good clinical practice) GLP (good laboratory practice) GMP (good manufacturing practice) GxP standards broadly cover

In the News: Regulatory and Development Updates

Rare Disease Clinical Trials Most Often Terminated Due to Regulatory and Recruitment Issues The difficulty associated with successfully completing rare disease clinical trials is well-known. Most people believe that recruitment is the primary reason because, by definition, the number of potential trial participants is extremely

Multidisciplinary Team Advances Immuno-Oncology Development

Developing a product from concept to commercialization is a multi-faceted and risky process. Particularly for complex programs, development plans must be customized based on real-world patient experience and optimized for a product’s post-approval success. A cross-disciplinary team is essential for crafting and assessing complex development

FDA Issues Guidance on Clinical Trials During COVID-19

In March 2020, at the start of the COVID-19 pandemic, the FDA issued guidance on the conduct of clinical trials. “The FDA released this guidance to emphasize that at all times, patients’ safety should continue to be at the forefront of considerations,” said Anand Shah, M.D., FDA

Streamline Early Clinical Development With an INTERACT Meeting

In October 2018, the Food and Drug Administration (FDA) issued Standard Operating Policy and Procedure (SOPP) – SOPP 8214 Version 1.0: INTERACT Meetings with Sponsors for Drugs and Biological Products. This document details recommendations for the Center for Biologics Evaluation and Research (CBER)’s engagement with

The Missing Parts of the Inactive Ingredient Database (IID)

Yesterday, FDA released draft guidance on the use of the Inactive Ingredient Database (IID) in drug development. The guidance is one step FDA is taking to address feedback from the generics industry that IID enhancements are needed to help sponsors make the right formulation decisions. What is the IID?

Seamless Clinical Trials: Why Didn’t We Think of That?

Seamless clinical trials have become the new buzz word in drug development since FDA Commissioner Scott Gottlieb promoted their use this month. But are they new, and which products are best suited to this style of clinical trial? Oncology drugs are the obvious examples of

Alkermes Prodrug for Treatment of Multiple Sclerosis: NCE?

The Food and Drug Administration (FDA) began requiring drug efficacy, in addition to safety, for approval in 1962 based on the Kefauver-Harris Amendment. Despite this requirement, many drugs that have been approved by FDA have limited efficacy (eg, drugs that treat cancer or Alzheimer’s disease).

Drug Development Planned Like the Titanic

How many drug development companies leave it up to the CMO to design or execute their formulation and manufacturing without oversight? Like those who boarded the Titantic 100 years ago, they seem to trust the mantra that their contractor’s work is unsinkable. MAP Pharmaceuticals seemed

One vs. Two Batches for Single-Dose and Multiple-Dose Studies

Today’s posting stems from a client question.  The client’s product candidate is an oral product that requires both single- and multiple dose pharmacokinetic studies. Question:  Do companies ever use one pivotal batch for single-dose (SD) study and another batch for the multi-dose (MD) study?  What

505(b)(2) Patent & Marketing Exclusivity

IP attorney Stephen Albainy-Jenai and I just concluded a webinar hosted by DIA entitled 505(b)(2) Patent & Exclusivity.  23 different companies attended, showing the increasing interest in 505(b)(2) issues.  Earlier this year, DIA hosted my overview of the 505(b)(2) drug development process where the attendees had many questions asking