An investigational device exemption (IDE) allows an investigational device to be used in a clinical study to collect data on its safety and effectiveness.[1] This exemption applies to the conduct of trials necessary for developing new medical devices. Clinical studies are often conducted to support
On September 29, 2023, the U.S. Food and Drug Administration (FDA) made public a potentially game-changing proposal concerning the regulatory framework for laboratory-developed tests (LDTs). Understanding the nuances and implications of these changes is paramount for specialists in regulatory affairs and the clinical development arena.
Progress in advanced computational methods using modeling and simulation has been enabled by advancements in computing technologies and the rise of artificial intelligence and machine learning. This has opened new opportunities in pharmaceutical drug development, such as the ability to evaluate large complex databases and
Computer modeling and simulation of humans, both healthy and with diseases, is a powerful tool. It can augment preclinical and clinical research through mechanistic and predictive investigations that would otherwise be impossible. In recent years, regulatory agencies have begun to accept evidence obtained through modeling
A quality electronic Trial Master File (eTMF) is essential to the product development and approval process, as it is the foundation for clinical trial inspections by competent authorities. eTMFs for drug, biologic, and medical device trials should be actively managed as studies progress and should
One of the specialized capabilities in Premier Consulting’s toolbox is coordinating the Active Substance Master File (ASMF) submission process on behalf of sponsors. The ASMF procedure (formerly known as the European Drug Master File [EDMF] procedure) has in recent years become an increasingly utilized instrument
If you develop or manufacture in-vitro diagnostic products, you know the May 2022 roll-out date for the In Vitro Diagnostic Medical Devices Regulation (EU) 2017/746 (IVDR) is just around the corner. But what, exactly, does that mean? Many of our sponsors have been approaching us
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
Regulatory approval is the final hurdle sponsors face when launching an in vitro diagnostic (IVD). After spending many years and millions of dollars on development, it can prove frustrating when the finish is in sight, but there are barriers to success. Fortunately, many of those
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.
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,
“There are only four kinds of people in the world: Those who have been caregivers, those who are currently caregivers, those who will be caregivers, and those who will need caregivers.” — Roslyn Carter Each November, National Family Caregivers Month recognizes and honors all those
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, multiple factors regarding development strategy and approach must be considered
Barry Mangum, Senior Vice President, Pediatrics at Premier Research, talks about the importance of pediatric research, its evolution over the years, and the obstacles and opportunities the industry faces. Q: How long have you been in pediatric clinical research, and how did you enter the
How and When to Incorporate PK Design into Your Gene Therapy Development Plan Gene therapy, which was 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
Incorporating patient-reported outcomes (PROs) into clinical trials can help sponsors better understand patients’ symptoms and how a therapy will affect their quality of life, and these insights can be particularly valuable in oncology research. However, there has been a lack of guidance to help sponsors
While the COVID-19 pandemic has accelerated innovation in many areas, including clinical trial design, many recent advances were already in motion before 2020 as sponsors and CROs sought to improve the patient journey and encourage trial participation. Traditional trials, especially those for pediatric populations, often
Conducting a clinical study in a pediatric patient population requires careful planning, as providing treatment to a child without causing harm is a weighty responsibility. The first tasks in striking this balance are dose selection and dosing interval determination, and this blog post describes how PK modeling
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
In February, the European Medicines Agency (EMA) released the fourth version of its Guidance on the Management of Clinical Trials During the COVID-19 (Coronavirus) Pandemic. As the pandemic continues to impact clinical research worldwide, these updated guidelines reflect the EMA’s evolving stance, clarifying questions raised by
In 2019, only 1.2 percent of global clinical studies included India, even though the country is estimated to bear 20 percent of the global disease burden. One reason for the slow growth of clinical research in India has been its stringent regulatory requirements. The Ministry
Drug development is an extremely cumbersome process, requiring the testing of an agent from in vitro studies to in vivo studies to in silico modeling. Given that it can take up to 20 years for a final product to be approved, it is unsurprising that
As a sponsor designs clinical studies, the respective comparison control groups become a critical factor to consider. Often, to gain clinical trial design insights, a sponsor reviews the physician package inserts from approved New Drug Applications (NDAs) and Biologics License Applications (BLAs) with similar indications
Sponsors and regulators use surrogate endpoints to predict the long-term benefits of drug products that treat serious or life-threatening conditions for which clinical outcomes cannot be rapidly determined. For many conditions, particularly cancers, proving survival or decreased morbidity could require years, so sponsors can use
In an era of pandemic urgency, repurposed drugs may be our first, best hope to finding effective treatments for COVID-19. Dozens of sponsors are now seeking efficient, streamlined processes to test and establish the safety and efficacy of existing products for the treatment of the novel
In an effort to maintain the continuity of our clinical trials during the COVID-19 pandemic, Premier Consulting is helping sponsors take steps to safeguard the well-being of patients who were previously expected to go to a medical facility to receive treatment. A Direct-to-Patient (DTP) model
In response to the newly released U.S. Food and Drug Administration (FDA) guidance on COVID-19, the Statistics Department at Premier Research has committed to highlight specific and necessary actionable considerations that directly address key FDA recommendations in the guidance. These considerations call attention to some general actions required
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
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
With drug discovery completed and your lead asset identified, it’s time to initiate IND-enabling activities, moving one step closer to clinical deployment. According to the Tufts Center for the Study of Drug Development, fewer than 10 percent of nonclinical lead assets move into the clinic.
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 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
The overarching goal of the Special Protocol Assessment Draft Guidance for Industry May 2016 (HHS, FDA, CDER, & CBER) is to improve the quality of new drug applications (NDAs) and biologic license applications (BLAs) by providing more certainty in the clinical protocol design process. Among
Randomized controlled Phase 3 (and some Phase 2) studies are clinical trials that are designed to answer specific questions, such as whether the proposed drug is effective in treatment or prevention of a particular disease. As such, the primary endpoint is the most important one
With the signing of the 21st Century Cures Act, the US Congress tasked the FDA with developing a framework to evaluate how the use of data from sources other than traditional clinical trials may be used to support drug approvals. This framework will apply to
As part of the 505(b)(2) drug development and registration process, the applicant of the new drug product can reference the literature and the FDA’s findings of safety and/or effectiveness (e.g., as listed on the Listed Drug product’s label) to fulfill various registration requirements.[1] Since many
The Food and Drug Administration released the revised draft guidance entitled ‘‘Leveraging Existing Clinical Data for Extrapolation to Pediatric Uses of Medical Devices” on June 21, 2016 (Docket No. FDA–2015–D–1376). This guidance explains the circumstances in which appropriate extrapolation of medical device data (either from
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).
The goal of drug product development is commercial success. If this statement wasn’t true, how would patients access the life-saving or life-enhancing drugs we are developing? Yet, all too many companies focus just on FDA approval, which in our view should be just a very
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
At Premier Consulting, we believe that the 505(b)(2) drug development pathway is best used when we can improve the safety and/or efficacy of an existing drug product. We see many opportunities to improve clinical effectiveness, but before trying to prove this in a clinical setting
On September 29, 2010 FDA published a Final Rule revising the requirements for safety reporting for INDs and other BE/BA studies. At the same time FDA issued an accompanying draft guidance to assist in interpreting the new rule. The Final Rule, revising 21 CFR §§
Sponsors and their investors continue to seek cost efficient ways to meet their financial milestones. Modeling can be used as a cost effective way to estimate the effect of changing an oral product from immediate release to extended release. Modeling avoids the cost of making
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
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
Well, freedom from generic competition for a while at least. Generally, most companies’ business plans specify some means to keep the competition at bay until the product can make a profit – when revenues start to exceed investments. There are two ways for 505(b)(2) applications
