Case Study: Developing a Complex Dosing Drug Schedule for a DART Pompe Disease Study

Amics Therapeutics, based in Cranbury, NJ (USA), focuses on finding treatments for a DART Pompe disease studyrange of devastating and rare and orphan diseases. Its lead biologics program is ATB200/AT2221, a uniquely engineered Pompe disease enzyme replacement therapy that will be administered in combination with a pharmacological chaperone and tested in multiple human genetic diseases.

A Short-Half Life Investigational Drug Suggests an Every-Other-Day Complex Dosing Regime

The Amicus and Covance dialogue began at the 2017 Society of Toxicology conference and resulted in a large series of DART studies being awarded to Covance at its center for biotech excellence in Greenfield, IN. Early in the engagement, Covance’s most experienced US-based DART team member provided consultation on study design to make it more consistent with FDA guidelines and expectations. Continue reading

Empowering CRAs with Mobile Technology to Improve Site Quality through Monitoring Efficiency

Clinical research associates (CRAs) need immediate access to relevant and accurate site performance metrics, but information is often isolated within multiple databases and spread across different tracking systems. To deliver key site performance data to CRAs via mobile and web applications – quickly and conveniently – Covance has created a specialized clinical analytics suite of products, Covance Xcellerate® Informatics. This powerful solution integrates data from all sources and allows a CRA to gain a clear and concise view of all clinical trial data, in one place, for all the sites that a CRA is monitoring.

Xcellerate Informatics CRA Blog Continue reading

When to Assess a Drug’s Potential for Abuse: Series Introduction

Drug abuse and potential drug abuse are critical issues for today’s pharmaceutical White Pills Drug Abuse industry and the health of patients. Understanding the latest FDA regulatory requirements, different study types, relevant timelines and logistics can be challenging. Conducted at a critical stage of drug development, the assessment for drug abuse potential is complex and must be acceptable to regulators.

If a drug or its major metabolite penetrates the brain, then an abuse potential assessment is required, regardless of the therapeutic indication. 

Developing a strategy around relevant timelines

The doses for drug abuse testing are selected based on the exposure/plasma levels produced in humans by the highest therapeutic dose. Therefore, drug abuse studies are not recommended until the human therapeutic dose range has been determined; however, your testing strategy should begin much earlier. Continue reading

Non-Compartmental Analysis of Pharmacokinetic Data: Considerations to Gain Efficiencies and Meet SEND Requirements

Pharmacokinetic (PK) data gathered in the early phases of drug discovery program can provide insights on a compound’s mechanism of action, identify specific attributes of interest and guide decision points to optimize downstream development. Selecting the most appropriate analysis technique is essential to computing PK parameters.

This article discusses how non-compartmental analysis (NCA) of pharmacokinetic data can help support regulatory filings, create predictive simulations and help researchers select lead molecules or formulations. We also explore the topic of data handling as differing approaches and anomalous results can cause delays through investigations and inconsistencies across a program. Finally, we’ll cover unique considerations when working with biologics and the challenges involved with submitting regulatory filings formatted to the Standard for Exchange of Nonclinical Data (SEND) specifications. Continue reading

The Promise of Vaccines for Fighting C. difficile

Over the last decade, Clostridium difficile (C. difficile) infection has rapidly become more prevalent. C. difficile, often abbreviated as C. diff, usually spreads through hospitals and other healthcare facilities, and the elderly are particularly vulnerable. Our society’s overuse of antibiotics has been eliminating normal microbes, allowing C. difficile to take over. Infected patients then release bacterial spores and spread the pathogen to others.

Vaccines are a promising strategy to address this critical public health issue. They are a well-established form of medicine that could be utilized to prevent illness rather than treating an existing infection. While fecal transplants also are being explored, these treatments are very new, and clinicians do not yet know the long-term effects of such procedures. Continue reading

Exploring New Opportunities for Biomarkers in Immuno-Oncology

Pharmaceutical companies are increasingly relying on biomarkers to deliver precisionOpportunities for Biomarkers in Immuno-Oncology medicine in immuno-oncology. Biomarkers can accelerate drug development and reduce the overall cost; they also allow sponsors to identify failed treatments sooner so that resources are not wasted on expensive, late-stage trials with unsafe or inactive compounds. Finally, these tests lead to better outcomes for patients, which help companies make a stronger case for reimbursement.

However, biomarker discovery requires substantial time and resources. While expenses will likely be outweighed by increased development efficiency, companies must ensure that drug and diagnostic timelines are closely aligned so that the treatment and test can launch simultaneously. Technical, workflow and commercial factors are critical to the successful use of immuno-oncology biomarkers. Continue reading

Dr. Barbara Gillespie to participate in a scientific workshop collaboration with the National Kidney Foundation, EMA and FDA

Kidney disease is often called a “silent killer” as it often develops unrecognized and DrGillespie_Headshot
gradually progresses into chronic kidney disease. Earlier detection to identify kidney disease and slow its progression has relied on measuring changes in two key biomarkers – glomerular filtration rate (GFR) and albuminuria.

Dr. Barbara Gillespie, vice president and therapeutic head of nephrology at Covance, was recently asked to attend an invite-only workshop on March 15-16, 2018 sponsored by the National Kidney Foundation (NFK), U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA).

As the only representative from a CRO invited to this unique meeting, Dr. Gillespie will offer valuable insights from the perspective of clinical research. She also serves on the NFK regional medical advisory board and is the only CRO member of the NFK scientific advisory board for chronic kidney disease (CKD) registry.

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Moving the Needle for Rare Diseases

Each year, we designate the last day in February as Rare Disease Day to raise awareness about the potential impact we can make for this important segment of the world’s population. From policy makers to researchers to health authorities, we all play a part in making a difference for more than an estimated 300 million people in the world suffering from a rare disease.

To mark the event, I would like to reflect on my role to move the needle and make a difference to patients that are awaiting life-changing treatment. My journey started as a clinician where I had the privilege in caring for patients with a variety of rare diseases. At a personal level, I am also the aunt of a very special boy who suffered for 8 years with a rare cardiopulmonary disease. To me, rare diseases are not so rare.  Continue reading

Establishing Control Over the Manufacturing Process and the Quality of your Biologic

101679_Large-Molecule_1575263331When a patient reads the label on their medicine bottle, he or she naturally relies on the medicine to contain the correct drug, be safe, work as intended and list the correct dosage. The pharmaceutical companies that produce these medicines similarly must rely on their internal manufacturing processes and quality control testing to generate the medicine responsible for this patient trust.

For the development of biologic medicines, the process of generating a quality product is less straightforward than that of a small-molecule medicine, like pain relievers such as aspirin. Selecting the right partner, such as Covance and its ‘Central GMP Testing Laboratory’ model, can smooth the path to validation and consistent manufacturing quality for your biologic.    Continue reading

Achieving Compliance with E6: Exploring the Workflow Challenges

Most sponsors are well aware of the recent revision (R2) of ICH GCP E6, which outlines recommendations to unify standards across the EU, Japan and the U.S. with defined requirements for sponsors regarding the role of monitoring and risk management, there are many factors to consider for a successful implementation.

This article discusses a variety of challenges sponsors may face when preparing to address these recommendations and also examines opportunities to drive greater efficiencies in today’s complex trial ecosystem.

Addressing varying levels of adoption

According to the latest revision, sponsors are tasked with a requirement to employ an adaptable quality management system and are expected to maintain oversight of CROs. But with various tracking systems, vendor logs, CTMS and monitoring platforms, seeing the complete picture can be an overwhelming, error-prone effort. Continue reading