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 [...]

Why peer-to-peer online patient communities are important to patients, caregivers and pharma

Why peer-to-peer online patient communities are important to patients, caregivers and pharma By Kathleen Hoffman, PhD What do patients and caregivers say about online patient communities? Inspire posts, podcasts, and patient advocates concur on the powerful and varied benefits, especially in research collaborations and as a trusted source of treatment and clinical trial information. Opportunity for research collaborations As early as 2005, Inspire member, Katherine K. Leon searched for other people with Spontaneous Coronary Artery Dissection (SCAD). Slowly she found members in Inspire’s WomenHeart community. Motivated by a lack of information explaining why her coronary artery dissected, [...]

1.6 Million People Accelerate Medical Progress

1.6 Million People Accelerate Medical Progress When Brian Loew founded Inspire in 2005, he never dreamed of its impact on members, on himself and on the people who worked with him. “When someone tells a story about how, by connecting with others, they learned that a certain treatment was possible. And they credit being alive because of that -- it makes you realize that this is not just a matter of 1.6 million people -- but a matter of a ton of individual lives that are affected by what you created.”1 On April 3, Loew shared this [...]

Referring patients to clinical trials is personal, study shows

Referring patients to clinical trials is personal, study shows By Kathleen Hoffman, PhD MSPH Why is it so hard to get physicians to refer patients to clinical trials?  One cynical assumption is that physicians are afraid they’ll lose that patient’s business.  Not so, according to a Tufts Center for the Study of Drug Development survey.1 Doctors are confident about and familiar with the clinical trial process. The survey of over 750 physicians found a high degree of comfort with clinical trials; almost 90 percent said they were comfortable providing and discussing clinical trial opportunities with patients. Yet [...]

Looking for Guidance? FDA and PFDD at “Patients as Partners” 2019

Looking for Guidance? FDA and PFDD at "Patients as Partners" 2019 By Hannah Eccard Pujita Vaidya, Senior Advisor, Patient Focused Drug Development (PFDD) program at the Center for Drug Development and Research (CDER) at the FDA, told an audience at the Patients as Partners Conference last week that patients are the experts on their conditions and on day-to-day life with their disease. A patient outcome—where improvement with treatment is meaningful in daily life—can only be understood in a patient’s own words. Vaidya noted that, “patients ‘chief complaints’ may not be factored explicitly into drug development plans.” 1 [...]

Patient and caregiver perceptions of clinical trials: Preliminary findings

Patient and caregiver perceptions of clinical trials: Preliminary findings By Kathleen Hoffman, PhD, MSPH Many of you have been in Philadelphia for the “Patients as Partners Conference.” Yesterday, Hannah Eccard, Research manager at Inspire, participated in a panel discussion titled, “Mapping Out the Patient Decision Journey.” The panel discussed the link between understanding patients --their lives, lifestyle, experiences-- and trial protocol design. Recently, Inspire’s research team completed the preliminary analysis of a survey to understand patient and caregiver perceptions and attitudes toward clinical trials. Over 1500 (1644), patient and caregiver members  experiencing either ovarian, prostate or colorectal [...]

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 patient and caregiver generated online communication with the results of one of FDA’s Voice of the Patient meetings. The Data Sets Inspire’s membership includes over 350,000 patients and caregivers living and coping with rare diseases. In the last three years, there has been 203% growth [...]

Listen to People Impacted by Rare Disease

Listen to People Impacted by Rare Disease By Kathleen D. Hoffman, PhD “The success (or failure) of the majority of rare disease drug development programmes rests on surrogate outcomes (e.g. laboratory measures, organ size) that may not reflect treatment benefits that patients value.”1 ~Morel and Canto Thomas Morel and Stefan Canto’s position paper from 2017 clearly describes the dilemma faced in rare disease research. Lab measures and organ size don’t speak to what patients with rare diseases experience, need or value. This focus on measures that are not meaningful impacts the development of rare disease treatments [...]

Rethinking patient engagement in clinical trials: Be part of the solution

Rethinking patient engagement in clinical trials: Be part of the solution By Kathleen D. Hoffman, PhD Seated in the waiting room of a large medical center, I overheard the bright eyed, young man behind the reception desk ask a new patient, “Would you like to learn more about participating in our research?” I smiled and thought, this is part of the solution. But it is a small part. I’ve been going to the same clinic for five years and today is the first time I’ve heard this question. I wondered if anyone would ask me - [...]

Patient Engagement Leadership Series Part 1: Clinical Trial Participants Are Medical Heroes

Patient Engagement Leadership Series Part 1:  Clinical Trial Participants Are Medical Heroes “I think a hero is any person really intent on making this a better place for all people.”  ~ Maya Angelou In Embracing the Patient Centric Culture, I discussed the paradigm shift facing healthcare. “Instead of doing things for or to the patient, now the goal must be empowering and working in partnership with the patient...to elevate the perspectives of patients and caregivers, incorporating their needs and priorities…. Building active listening to, or obtaining a continuous stream of insights from patients and caregivers into [...]