Do Patients Care About What Your Drug Does?

Do Patients Care About What Your Drug Does? By Sara Ray, MA Be sure the trial outcomes echo the patient voice, article says In most randomized pharmaceutical clinical trials, researchers are looking for a statistically significant performance difference between the two courses of therapy. But which is more important to the patient: Statistically significant improvement of a variable, or clinically significant improvement? “The operation was a success, but the patient died” is a succinct example of a misplaced sense of priorities. The patient was probably hoping for a better result than academic satisfaction. Here’s another example. [...]

Clinical Trials and PROs: What Patients with Rare Diseases Say

Clinical Trials and PROs: What Patients with Rare Diseases Say By Kathleen Hoffman, PhD MSPH Challenges to developing clinical trials for rare diseases begin with their rarity – each condition affects fewer than 200,000 Americans. Approximately seventy-five percent of those affected by rare diseases are children. It is estimated that 80 percent of rare diseases are caused by genetic changes. Many rare diseases are progressive and debilitating. One third of the children affected by these conditions do not reach 5 years of age.1 Getting to a rare disease diagnosis can take years. In a way, it [...]

Video Vignettes: Through Their Own Eyes: Rare Autoimmune Liver Diseases

Video Vignettes: Through Their Own Eyes: Rare Autoimmune Liver Diseases By Kathleen Hoffman, PhD, MSPH The liver has been called a forgiving organ because of its capacity to regenerate. However, chronic unrelenting damage over years can overwhelm its regenerative abilities, resulting in permanent scarring. Well-known risk factors-being overweight, alcohol abuse, viral infections like Hepatits-C--make cirrhosis of the liver the seventh leading cause of death in the US, affecting an estimated 1 in 400 adults. 1 Yet, a small number of cirrhosis cases are caused by three rare autoimmune diseases,  autoimmune hepatitis (AIH), primary biliary cholangitis (PBC) and [...]

Finding Patients With Rare Cancers Quickly

Finding Patients with Rare Cancers Quickly Almost one-third of all cancer deaths are caused by rare cancers but rare cancers represent only between 23.4% and 27% of all cancer cases.1 Between 25 and 30 million Americans and around 30 million people in the European Union live with rare diseases.2 Rare cancers are classified under rare diseases and are defined by the EU Society of Medical Oncology as having an incidence of less than six occurring per 100,000 persons per year.3 The National Cancer Institutes define the incidence of rare cancers as 15 per 100,000 persons per year.4 [...]

Tackling the Challenges of Rare Diseases: Introducing RareXcel

Tackling the Challenges of Rare Diseases: Introducing RareXcel Neurofibromatosis is a rare, genetic condition causing tumors to form on nerve tissue all over the body. The masses can grow both internally and externally, often appearing as lumps under the skin surface. People can experience deformities of skeletal structures as well, making this a visible disease. As a member of Inspire’s Neurofibromatosis Network support community relates, living with this condition can test self-esteem: “I'm disappointed. Life had been going pretty smoothly for me these past couple weeks….Then today happened...the depression and darkness comes out of nowhere stealing [...]