Clinical Research: Phase 1 - Phase 4

Applying Sound Research Practices in the Development of Medical Devices

Medical device and diagnostic companies face greater demand to provide clinical evidence of product efficacy than ever before. With increased scrutiny from regulators, healthcare systems, and even patients, more device companies are performing clinical trials to improve the likelihood of commercial success. In today’s competitive landscape, device companies are facing increased demand for clinical evidence...

Clinical Trials in Rare Diseases: Every Patient Counts

In many rare and ultra-rare disease studies, sponsors do not have the luxury of choosing the countries or sites where the study will be conducted. Instead, they must use the sites where those patients are already being treated, regardless of geography. Additional protocol criteria – such as treatment-naïve patients or general prevalence of a disease...

Proven Strategies for Rare Disease and Orphan Drug Development in the U.S.

Orphan drugs are medicinal products intended to treat diseases so rare that sponsors are reluctant to develop them under usual marketing conditions. Orphan drugs are a growing market, due in large part to tax incentives, longer periods of market exclusivity, and shorter, smaller clinical trials, as well as the premium pricing associated with many orphan...

The Science of Hope

Despite the inherently small market for orphan drugs, orphan drug development has recently undergone significant growth, with global sales of over $100 billion in 2015. In 2015, more than 20 new drugs were approved by the U.S. Food and Drug Administration (FDA) and 18 new drugs were approved by the European Medicines Agency (EMA) for...

Fit-For-Purpose: A Strategic Approach to Biomarker Method Validation for Rare Disease

Ensuring the integrity of your surrogate endpoints Rare disease studies present unique obstacles to drug developers, including small patient populations, slow disease progression, lack of prior clinical research, and uncertainty around validating biomarker measurements intended for use as surrogate endpoints. Our white paper describes a fit-for-purpose approach to validating bioanalytical methods in orphan drug trials....

Maximizing Success in Early Stage Oncology Trials

The success rate of investigational compounds eventually approved for clinical use in cancer remains the lowest among all diseases. Of the more than 750 drugs currently under development for the treatment of cancer, it is predicted based on past performance that only a few will ultimately demonstrate sufficient efficacy and safety for regulatory approval and...

The Science and Art of Conducting Clinical Trial Feasibility in Rare Disease and Pediatric Studies

Rare disease and pediatric studies present significant challenges, including country selection, site selection, and patient recruitment. A thoughtfully-designed, well-executed feasibility study can help sponsors obtain a realistic assessment of the best path forward for developing and conducting successful global clinical trials. Performing a thorough assessment of clinical trial feasibility is an important early step in...

Biomarker Method Validation in Rare Disease Drug Development

While drug discovery has seen major advances over the last few years — especially in the domain of rare disease research — the number of new drug approvals has not kept pace with increasing development costs. However, the innovative use of biomarkers has the potential to change that trend. Biomarkers in Rare Disease Research Biomarkers are objectively measurable characteristics...

3 Innovations Improving Neuropsychological Testing in CNS Drug Development

Although current limitations in neuropsychological testing may discourage development of treatments for central nervous system (CNS) disorders, some researchers see these unmet needs as an opportunity for innovation. Take a look at three strategies that are improving the validity and viability of neuropsychological testing. Rater Training Success or failure of a CNS trial can come down to...

The Why, What, and How of the Current State of CNS Drug Development

Central nervous system (CNS) disorders are a diverse group of conditions that include psychiatric, neurological and substance abuse disorders. Unfortunately for patients, treatment options for CNS disorders are often limited (or non-existent). To make matters worse, comparatively few CNS drugs are in the development pipeline. What aspects of CNS disorders contribute to lagging drug development? Read on to...