Both registry studies and natural history studies play important roles in rare disease research. Understanding the differences between the two types of studies and how they can be used to inform clinical development can help sponsors plan for success. Sound drug development requires a comprehensive understanding of the disease being treated. To design reliable clinical...
The study of rare cancers poses special challenges for drug developers, who often must draw on their experience in both oncology and rare disease. Careful consideration of clinical trial design and regulatory pathways can help increase the likelihood of success in rare oncology clinical trials. Researchers studying rare cancers must call on expertise in both...
Proper preparation of an Investigational New Drug (IND) application is a carefully orchestrated process that requires many disciplines to collaborate in delivering a consistent message regarding the safety of an investigative compound. Proactively identifying and avoiding common IND submission pitfalls can help ensure that your application is clear, concise, and error-free. Preparation of an Investigational...
A systemic disease with a variety of comorbidities, psoriasis is a common condition with several clinical subtypes. In this white paper, we discuss the pathogenetic and clinical aspects of psoriasis, as well as the nuances of designing and conducting psoriasis clinical trials. Psoriasis is a chronic skin disorder and the most prevalent autoimmune disease in...
Acne is the most common skin condition in the U.S., affecting up to 50 million Americans annually. In this white paper, we explore the history and future of acne treatment, with a focus on emerging therapies that may help patients with moderate to severe acne gain and maintain control of their acne. Acne is a...
Introduction Atopic dermatitis (AD), often called eczema, is a complex disorder involving skin barrier function abnormalities and skin inflammation. It is the most common skin condition in children under the age of 11 years – about 50 percent of patients develop the disease by the age of one year and up to 85 percent by...
Neurodegenerative disorders present some of the biggest challenges in planning and conducting clinical drug trials. Identifying strategies to proactively address or mitigate these challenges can help to ensure a successful trial. Since the first drugs for neurodegenerative diseases (NDDs) were approved in the 1990s, an increasing number of disease- modifying therapies have been approved for...
While adaptive design is associated with many potential benefits, it may also present challenges to observing the basic ethical principles of research in human subjects. In this white paper, we review the features of particular clinical trial design adaptations and discuss the ethical obstacles they can present and those they can potentially resolve. The value...
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...
As our understanding of the genetic and molecular basis of cancer advances, rare oncology research is accelerating at an unprecedented pace. In fact, in 2014, more than 40 percent of U.S. Food and Drug Administration’s orphan drug designations were for rare cancers. Moreover, the trend toward increasing international cooperation among big pharma, biotech, and academia...