rare disease

Getting the Most from Online Surveys

Getting the Most from Online Surveys By Kathleen Hoffman, PhD, MSPH Do you wonder about online surveys? Are they reliable? Is there bias? A recent article by the Pew Research Center For Weighting Online Opt-In Samples, What Matters Most? 1provides guidance to allay those concerns. Pew experimented with different procedures for [...]

Getting the Most from Clinical Trial Protocol Feasibility Studies

Getting the Most from Clinical Trial Protocol Feasibility Studies By Monica St Claire Webinar provides expert tips for getting real feasibility data, optimizing trial design When the drug development process is at the point of moving to clinical trial, the next step is conducting a feasibility study. Stakeholders are all looking [...]

Do You Really Know How Patients Talk About Their Condition?

Do You Really Know How Patients Talk About Their Condition? By Kathleen Hoffman, PhD MSPH There are classic examples of advertising messages that ended up offending the target customer, but the Utah Department of Transportation provided the most recent. The Utah DOT’s lighthearted attempt to adopt a specific culture’s way of [...]

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 [...]

By |Oct 2019October 22nd, 2019|clinical trials|0 Comments

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 [...]

Hacking clinical trials: Two awareness and protocol strategies invented by patients and caregivers that work

Hacking clinical trials: Two awareness and protocol strategies invented by patients and caregivers that work By Kathleen Hoffman, PhD Patients with life-threatening diseases aren’t waiting for someone to make participating in clinical trials easier for them. They are breaking down the barriers themselves. Two of these innovative methods were described at a [...]

What I told researchers trying to find a cure for my rare disease

What I told researchers trying to find a cure for my rare disease By Seth Rotberg When I was about 12, I started noticing that something was wrong with my mom. She wobbled from poor balance, often tripping and falling over, as if she were drunk. She had severe mood swings; one [...]

By |Jan 2019January 29th, 2019|Patient Stories|0 Comments

Social Listening to Understand the Unmet Needs of Patients

Social Listening to Understand the Unmet Needs of Patients By Marina Shayevich and Sara Ray Across healthcare, patients lament the imperfection of pharmaceutical treatment. Although grateful to have life saving and life improving medications, patients are often met with frustration at the limitations of their medications. Nowhere is that more evident than [...]

What Matters Most to People Impacted by Rare Disease: An Inspire-FDA Collaboration

What Matters Most to People Impacted by Rare Disease: An Inspire-FDA Collaboration By Sara Ray What happened when we compared research strategies used to obtain patient insights? We discovered that using multiple strategies is key to gaining usable understandings. In a recent collaboration between Inspire and the FDA, we compared and contrasted [...]

By |Nov 2018November 13th, 2018|Patient Insights|0 Comments