Next Generation Online Health Communities

Next Generation Online Health Communities: Data empowering patients and caregivers improves care and accelerates research By Richard Tsai The World Orphan Drug Congress USA 2020 (WODC 2020) provided four days of powerful panels and keynotes about collaborations, successes and failures in creating what rare disease patients and families need and desire most – effective treatments for their conditions. In one failure turned to success, Kim Stephens shared her story involving inappropriate clinical trial endpoints. Stephen’s son has Hunter Syndrome, an extremely rare inherited condition where children lack an essential enzyme in their cells that eliminates waste . [...]

Accelerating Rare Disease Research

Accelerating Rare Disease Research By Richard Tsai COVID-19 has challenged the non-profit arena’s fundraising arm, which is largely based on face-to-face events like galas, runs and walk-a-thons. This limit on the number and amount of donations has occurred just as rare disease patient organizations have reached a tipping point in their influence on government and industry. According to Malcolm Gladwell, the “tipping point” is “the moment of critical mass, the threshold, the boiling point,” where “ideas...spread like viruses do.” 1In this case, the tipping point refers to the efforts made by rare disease patient organizations to provide [...]

Getting the Real Story from Patients

Getting the Real Story from Patients By Kathleen Hoffman, PhD, MSPH Humans evolved in a storytelling milieu where stories taught and explained the world around us. Our brains carry this legacy, research shows that the brain can store more information and retrieve it more easily when it is in story form. Narrative is easier to understand, is more engaging, and is an effective tool to communicate science to lay individuals.1,2 Personal narratives make science relatable. But mistakes made in the process of capturing a patient narrative can be cumulative. When writing native advertising (sponsored content), copy [...]

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 weighting results from surveys with online opt-in samples to discover which techniques best reduced bias on estimates. They compared online results with the results given for 24 benchmark questions drawn from “high-quality federal surveys,” public surveys that were conducted using more traditional methods. This was [...]

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 to hit the jackpot: figuring out the best place to conduct the clinical study, and identifying the design factors that result in maximum patient retention. Recently, the topic of the WCG Institute’s podcast “Stump the Experts” was feasibility. It featured Diane Carozza and Danya Kaye, [...]

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 speaking was predictably ridiculed when “Hey Teens, Buckling Up is Totes Yeet Yo” (meaning “the use of seatbelts is excellent”) appeared on the state’s electronic highway signs.1 honestly pic.twitter.com/axXJwjbJrH — Nico Di Angelo Fan Blog (@Laney_Brynn) October 23, 2019 The story is funny, but illustrates [...]

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 [...]

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 March 18 Clinical Trials Transformation Initiative (CITI)-FDA event, “Enhancing the incorporation of the patient perspective in clinical trials.”1 Breaking barriers to clinical trial awareness In 2012, Tom Marsilje and his team from Novartis presented a cancer drug he had co-invented at the annual American Society [...]

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 minute she was fine and the next, she was angry, frustrated, or depressed. Multiple physicians’ visits resulted in a diagnosis of bipolar disorder and major depression, but our family knew there was something else going on. Lacking an adequate diagnosis, my mother was becoming a [...]