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...
While there is currently no cure for atopic dermatitis (AD) — commonly called eczema — this prevalent condition can often be well managed through conventional drug therapies. Current AD strategies used by dermatologists include symptom relief, patient/parent education, and the prevention of secondary complications. The “Gold Standard” for Mild to Moderate Atopic Dermatitis The most frequently used therapies...
Biologics have revolutionized psoriasis treatment over the last two decades. These targeted immunomodulatory therapies act directly on the cytokine pathways that are upregulated in psoriasis and other autoimmune diseases. Two major classes make up biologics in psoriasis treatment: tumor necrosis factor (TNF) inhibitors and interleukin (IL) inhibitors. TNF Inhibitors TNF inhibitors became the first available biologic...
A little less than five percent of the world’s population are estimated to suffer from more than 7,000 different types of known rare diseases. There are even more medical conditions where those who have them are too embarrassed to talk about what’s wrong or simply don’t know exactly what ails them. Yet, these individuals are...
The Initiative on Methods, Measurement, and Pain Assessment in Clinical Trials (IMMPACT) is a major group providing guidance for design considerations of acute pain clinical trials. IMMPACT is made up of a team consisting of some of the most respected researchers and clinicians in the field of pain medicine. As of 2016, IMMPACT recommends these ten best practices...
Atopic dermatitis (AD), also known as atopic eczema, is a common skin disorder affecting millions of people worldwide. While many cases are mild, AD can take a significant financial and emotional toll on patients and their loved ones. What are the symptoms of atopic dermatitis? AD is characterized by skin barrier function abnormalities and skin...
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...
Psoriasis is an autoimmune disease that affects 7.5 million people in the United States and roughly 100 million worldwide. While its main manifestation is in unsightly, often irritating skin lesions, many patients also experience painful psoriatic arthritis. Additionally, psoriasis often comes with a wide range of other chronic conditions, including Type 2 diabetes, cardiovascular disease and...
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...