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 [...]

What Members Tell Each Other About Clinical Trials

What Members Tell Each Other About Clinical Trials By Kathleen Hoffman, PhD MSPH "Failing to enroll a sufficient number of subjects in a trial is a long-standing problem"1 but understanding the factors impeding enrollment can be difficult. Public posts on social media are an effective resource for understanding how patients feel and what they understand about clinical trials. On Inspire, “clinical trials” are a popular topic. Close to 58,000 posts mention clinical trials. A cursory search of Inspire postings show the diversity of opinions and understandings: some member posts provide solid information, some members post incorrect [...]

Are You Listening to Patients? Patient Preferences and Clinical Trial Design

Are You Listening to Patients? Patient Preferences and Clinical Trial Design By Kathleen Hoffman, PhD, MSPH Last fall, over 1500 Inspire members living with sarcoidosis, scleroderma, ovarian cancer, prostate cancer, colorectal cancer, arthritis and psoriasis, completed a  survey on exploring patient preferences and insights on clinical trials. Our recent webinar, "A Hopeful Mindset," described preliminary results. Thirty-nine percent of respondents had cancer, 32% had a chronic condition - arthritis or psoriasis - and 29% had a rare disease. Over the next few months we will be sharing more details from the data.  Last week in "5 Benefits [...]

5 Benefits of Patient Focused Drug Development Sponsors Need to Know

The Patient Is Speaking: 5 Benefits of Patient Focused Drug Development Sponsors and CROs Need to Know By Sara Ray, MA Since the 2012 FDASIA reauthorization of the Prescription Drug User Fee Act (PDUFA) and the 21st Century Cures Act ‐‐ Directing and Enabling Patient Focused Drug Development in 2016, integrating the patient perspective has been a priority. Our research team has conducted studies with patient and caregiver Inspire members for a variety of sponsors. I'd like to describe how these research projects produce important patient insights that inform and can improve clinical trials. Patient focused drug [...]